Personalized Medicine Partnering Deals in 2011 - The Burrill Report

The Burrill Report 

Vol.2, Issue 9 

October 2011 

In This Issue 

FOCUS: 

Personalized 

Medicine 

• Medco Drives 

Integration of 

Personalized Medicine 

• Evolution of the DTC 

genetics field 

• Making Good on a 

Promise 

• Personalized Medicine 

Deals 

• Letter From Europe 

• August Gainers and 

Losers 

• Venture Financings 

• Public Financings 

• Clinical Trials 

• Patents 

• PDUFA Dates and more 

2011* 2010* Change 

Total Global Venture Capital 6,532 6,676 -2.2% 

U.S. VC 4,881 5,339 -8.6% 

Total IPOs 3,250 1,458 122.9% 

(34 in 2011 v. 24 in 2010) 

U.S. IPOs 1,298 820 58.3% 

(13 in 2011 v. 11 in 2010) 

Total Global PIPEs 2,659 2,620 1.5% 

U.S. PIPES 1,148 1,415 -18.9% 

Total Global Follow-ons 4,668 2,482 88.1% 

U.S. Follow-ons 4,047 1,995 102.9% 

Medco Drives Integration of 

Personalized Medicine 

With 2010 acquisition of DNA Direct, the pharmacy benefits manager 

is an important force behind putting new approaches into practice 

By Daniel S. Levine 

The integration of personalized 

medicine into medical 

practice faces a number of challenges, 

including physicians’ lack 

of understanding of genetic tests, 

the difficulties payers have separating 

molecular diagnostics that 

are valuable and ones that are 

not, and insurers, and hospitals, 

need to know when a test is appropriate 

to use. It is this last gap 

that the pharmacy benefits manager 

Medco Health Solutions is 

seeking to fill. 

In February 2010, Medco 

reached an agreement to acquire 

Market volatility in August 

battered life sciences companies 

that went public this year, 

wiping away nearly $1.3 billion in 

market capitalization as tumultuous 

trading activity hit them harder 

than the broader market, IPOs 

in general, and biotech stocks as a 

whole, a Burrill & Company analysis 

shows. 

The market’s gyrations, triggered 

by the United States’ debt 

ceiling fight, worries about the 

nation’s creditworthiness, the 

debt crisis in Europe, and the 

stalled economic recovery, threw 

U.S. life sciences IPOs into a dramatic 

reversal. As a group, the return 

from the 13 U.S. life sciences 

San Francisco-based DNA Direct, 

one of the pioneers of consumer 

genetics. Already the company 

had been expanding its business 

model to work with physicians 

and payers. But its integration 

into Medco has been transfor- 

Volatile Markets Sap Life Sciences IPOs 

Month In Review 

Life Sciences Capital Scorecard, August 2011, In USD M 

2011* 2010* Change 

Global Debt Offerings 32,105 23,156 38.6% 

U.S. Debt 18,584 17,595 5.6% 

Global Other Financings 9,160 7,318 25.2% 

U.S. Other Financings 3,820 5,597 -31.7% 

Total Global Public Financings 55,292 37,034 49.3% 

Total U.S. Public financings 28,817 27,522 4.7% 

Global Partnering 24,269 42,645 -43.1% 

U.S. Partnering 20,845 24,580 -15.2% 

Global M&A 129,844 107,128 21.2% 

U.S. M&A 108,914 51,780 110.3% 

(continued on next page) ❱❱ 

IPOs completed in 2011 on U.S. 

markets had moved into negative 

territory by the end of August, 

down an average 7.4 percent 

from their IPO price compared 

to the 17.2 percent gain they had 

realized by the end of July. The 

group in August fell 18.9 percent, 

with 3 advancers and 10 decliners 

compared to 9 advancers and 

4 decliners at the end of July. 

In all, the life sciences IPOs had 

actually outperformed broader 

market indices by the end of July, 

but now are significantly underperforming 

the Dow Jones Industrial 

Average, the Nasdaq Composite 

Index, the Burrill Biotech 

Select Index, and the AMEX Biotech 

Index. As a class, they have 

also failed to match the performance 

of U.S. IPOs overall, which 

as a whole were down 3 percent 

at end of August, according to Renaissance 

Capital. 

Investors took flight from risk 

in the recent market turmoil and 

that does not bode well for life 

sciences companies hoping to 

*Year to date as of August 31 

(continued on page 4) ❱❱


Medco 

❱❱ (continued from page 1) 


▼ 

PUBLISHER 

G. Steven Burrill 

EDITOR 

Daniel S. Levine 

ASSOCIATE EDITOR 

Marie Daghlian 

ASSOCIATE EDITOR 

Michael Fitzhugh 

GRAPHIC DESIGNER 

Carol Collier 

SALES 

Nicole Boice 

(949) 680-7088 

nboice@b-c.com 

Ken Sorensen 

(415) 591-5471 

ksorensen@b-c.com 

▼ 

ISSN:1943-7617 

PUBLISHED MONTHLY BY: 

BURRILL & COMPANY 

ONE EMBARCADERO CENTER 

SUITE 2700 

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T: 415-591-5400 

EMAIL: dlevine@b-c.com 

mative as Medco has leveraged 

its Fortune 50 muscle to turn its 

DNA Direct subsidiary into a significant 

force advancing personalized 

medicine into everyday 

clinical practice. 

Ryan Phelan, president and 

founder of DNA Direct, says she 

saw the deal as a way not to just 

help provide these services, but to 

get them into mainstream healthcare 

systems. “Before Medco, it 

was really challenging having 

anyone with real clout at the table,” 

she says. “As a start-up company, 

whether it was providers, 

big medical centers, or healthcare 

payers, I think they would look at 

a company like DNA Direct as a 

small, entrepreneurial, venture 

backed company and say, ‘This 

is great, but before we could run 

this kind of program out to our 

millions of members we need to 

know it could scale.’ ” 

Today Medco has integrated 

all of its personalized medicine 

programs under the umbrella of 

DNA Direct. Through the subsidiary, 

Medco provides a list of offerings 

that is helping bring personalized 

medicine into the here 

and now. 

Through more than 300 midand 

large-sized employers who 

have enrolled their employee 

bases into Medco’s clinical testing 

programs, more than 10 million 

members can have the choice 

of pharmacogenetic testing when 

they are prescribed any of a growing 

list of drugs with a known 

safety or efficacy issue related to 

a patient’s genetics. 

The company also provides 

members an extensive electronic 

database with information on 800 

genetic tests. In addition, its genetic 

counseling call center provides 

information to physicians 

on more than 2,000 genetic tests 

and provides pre-authorization 

services for the use of these tests 

to clients such as the health insurance 

giant Humana. Through 

DNA Direct, Humana’s coverage 

management system targets high 

cost tests that are at risk of misuse. 

They found that about 25 percent 

of tests were ordered inappropriately. 

At the same time, about 35 

percent of tests were being done 

at labs outside their network and 

could instead be steered to labs 

within their network. 

DNA Direct is also building 

out a Genomic Medicine Network 

to work with hospitals to help 

their physicians develop insight 


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October 2011 2


Medco 

❱❱ (continued from previous page) 

into new diagnostics and provide 

continuing medical education 

classes and webinars to improve 

their understanding of genomic 

medicine. 

El Camino Hospital in Mountain 

View, California is one of 

six hospitals currently in DNA 

Direct’s Genomic Medicine Network. 

Lynn Dowling, executive 

director of the hospital’s Genomic 

Medicine Institute says she sees 

plenty of doctors who return 

from scientific conferences excited 

about using new genomic 

tools in their practice only to become 

stymied when seeking answers 

to very practical questions. 

“They come back to their local 

hospital in their local communities 

and none of their colleagues 

are ordering these tests, and they 

are a little reluctant. Besides, 

they don’t know where to order 

them, how much they cost, or 

whether their patients are going 

to be reimbursed. They don’t how 

to counsel their patients on the 

privacy issues either. So they are 

very, very, very interested and excited 

about the science, but they 

are totally not confident about 

how to implement it,” says Dowling. 

“That’s what we’re trying to 

do—to give them those tools.” 

As an example of how El 

Camino Hospital is integrating 

personalized medicine into everyday 

practice, Dowling points 

to a new tool it is rolling out on 

its website to allow patients to 

screen their family history for the 

risk of breast cancer. Though not 

developed by DNA Direct, the 

program is integrated into the 

work with the company. Should 

the family history warrant it, a 

counselor will refer the patient 

for genetic testing. If the patient 

is found to have a genetic predisposition 

to breast cancer, a plan 

for monitoring and preventive 

measures is created. Should the 

patient eventually develop the 

disease, the approach should allow 

for early intervention and 

treatment. The breast cancer tool 

is the first of several the hospital 

plans to roll out. 

Jane Barlow, vice president of 

clinical innovation for Medco, 

says personalized medicine is 

still in its early stages, but that 

adoption is accelerating. Pointing 

to the FDA approval of two 

new cancer therapies with companion 

diagnostics in August— 

Roche’s melanoma drug Zelboraf 

and Pfizer’s non-small cell 

lung cancer drug Xalkori—she 

says the big wave of the future 

is in drugs with companion diagnostics. 

Ultimately, with 40 million 

members who have expressed 

a willingness to participate in 

research, Medco is in a unique 

position to work with research 

institutes and diagnostic companies 

to both help identify genes 

at play in specific disease and 

speed the translation of research 

findings into clinical benefits. 

Express Scripts in July reached 

an agreement to purchase Medco 

for $29.1 billion. That deal must 

still pass muster with the Federal 

Trade Commission, but should it 

go through, it could greatly expand 

the reach of Medco’s personalized 

medicine efforts. 

■ 

REGISTRATION UNDERWAY 

The 21st-century healthcare paradigm—which begins 

with prevention and wellness and continues to 

personalized medicine—has come of age. Two megatrends, 

a rapidly aging global population and the 

emergence of new technologies, are converging to 

answer consumer demand for quality of life and value. 

The 7th Annual Burrill Personalized Medicine Meeting 

provides attendees with a detailed “window” into this 

personalized medicine world. 

The agenda features leading authorities across the 

spectrum of personalized medicine and the meeting 

encourages participation, focusing on intimate 

discussions with small groups of panelists and expert 

moderators, and encourages participation from the 

expert audience. As such the meeting will appeal to: 

October 3 – 4, 2011 

The San Francisco Airport Marriott 

Burlingame, CA 

- Researchers 

- Drug/Diagnostics/Tools Developers 

- Payors 

- Investors/Analysts 

- Public Policy/Regulators 

For More Information Email: events@b-c.com 

or visit burrillandco.com 



Month In Review ❱❱ (continued from page 1) 

complete public offerings. If the volatile 

market activity that characterized August 

persists, it could cause private life 

sciences companies to turn away from 

the IPO market and seek financing elsewhere. 

Life sciences companies demonstrated 

a willingness to seek funding 

outside the United States, going where 

they felt they had the best chance of 

raising money in difficult markets. Two 

U.S. companies did manage to go public 

in August, but on exchanges outside the 

United States. EcoSynthetix, a renewable 

chemicals company, completed a 

$101.6 million offering on the Toronto 

Stock Exchange on August 4. GI Dynamics, 

a medical device company commercializing 

non-surgical treatments 

for diabetes and obesity, closed an $85 

million offering on the Australian Securities 

Exchange on August 30, planning 

to list on September 7. That’s a trend 

that’s likely to continue. Genomatica, a 

renewable chemical company, added itself 

to the IPO queue, raising the total 

to 36 life sciences companies in registration 

globally. 

IPOs were not alone in being punished 

within the life sciences sector. Dendreon 

had the misfortune of reporting 

disappointing sales for its groundbreaking 

cancer vaccine Provenge as markets 

tumbled in response to the Congressional 

fight over raising the debt ceiling. 

The company saw nearly two-thirds of 

its value wiped away as its shares closed 

at $11.69 on August 4, down from $35.84 

the previous day. Dendreon’s problem 

in ramping up sales spilled over to other 

companies as investors grew concerned 

about the pace at which other recently 

approved drugs will be able to realize 

their market potential. 

Overall, the life sciences sector in August 

saw nearly 6 decliners for each advancer 

for stocks trading over $1 at the 

end of month. With values depressed, 

it could fuel increased M&A activity 

among cash rich pharmas looking to expand 

their pipelines. 

Big Pharma’s problems have not gone 

away. The need to find new sources of 

revenue to replace income from sales of 

drugs going off patent continues to fuel 

dealmaking. With values depressed, acquirers 

will likely find more bargains 

today when they go shopping. We could 

see a pick-up in activity between now 

and year-end. 

The stock market’s wild swings 

dampened activity in the capital markets 

for life sciences companies across 

the board. In August, the immunotherapeutics 

company Agenus was the only 

life sciences company to complete a 

follow-on offering. It raised just $7 million. 

Only four companies in the United 

States completed PIPEs raising a total of 

$83.6 million. 

Personalized medicine emerged in 

August as a bright spot for the sector 

with the U.S. Food and Drug Administration’s 

approval of Roche’s melanoma 

drug Zelboraf and Pfizer’s non-small 

cell lung cancer drug Xalkori. Both 

drugs were approved for use with companion 

diagnostics to determine which 

patients would benefit from their use. 

The FDA also approved Seattle Genetics’ 

lymphoma drug Adcetris, a drug 

that marries an antibody to a toxic chemotherapeutic 

payload to deliver a targeted 

therapy to a certain subgroup of 

lymphoma patients. 

The approvals follow the announcement 

in July that pharmacy benefits 

manager Express Scripts reached an 

agreement to acquire its competitor 

Medco Health Solutions, a leader in integrating 

personalized medicine into 

healthcare. Although the deal must pass 

regulatory scrutiny, and will likely face 

opposition from drugmakers concerned 

about a concentration of buying power, 

it provides Express Scripts with a strategic 

position in personalized medicine 

that it had been lacking. 

There has been criticism of late about 

the slow progress in realizing the promise 

of personalized medicine. The recent 

activity, though, points to the very real 

changes that are underway to incorporate 

genomic medicine into standard 

practice of healthcare today. 

■ 

Performance of 2011 U.S. IPOs In August 

COMPANY TICKER IPO PRICE (USD) AMOUNT 

RAISED (USD M) 

RETURN FROM IPO 

7/29/11 

RETURN FROM IPO 

8/31/11 

Endocyte ECYT 6 86.3 122.2% 82.7% 

Sagent Pharmaceuticals SGNT 16 105.8 74.2% 43.7% 

Pacira Pharmaceuticals PCRX 7 42 43.1% 9.4% 

Fluidigm Corporation FLDM 13.5 86.3 25.6% -0.1% 

KiOR KIOR 15 150 -3.7% -8.0% 

Horizon Pharma HZNP 9 50 0.4% -11.1% 

BG Medicine BGMD 7 40.3 7.6% -13.7% 

Solazyme SZYM 18 227.2 26.9% -22.2% 

Tranzyme TZYM 4 54 -1.3% -22.5% 

Gevo GEVO 15 123 10.2% -28.9% 

AcelRx Pharmaceuticals ACRX 5 40 -18.0% -32.0% 

Epocrates EPOC 16 85.8 2.8% -33.6% 

Kips Bay Medical KIPS 8 17 -65.90 -60.00 

■ 



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DTC Genetic Companies Evolve 

Business models diverge as companies seek path to profits 

“At the end of the 

day, that testing 

that is done outside 

of the healthcare 

system becomes 

suspect by the 

system and that 

becomes part of 

the problem of 

becoming a real, 

viable company 

today.” 

Ryan Phelan 

President and founder, 

DNA Direct 

By Daniel S. Levine 

Business models in the directto-consumer 

genetics field 

are diverging as companies face 

regulatory uncertainty, consumer 

resistance, and difficult capital 

markets. Though some continue 

to provide information to individuals 

about the substance and 

meaning of their DNA, they are 

betting that their best opportunity 

for success lies elsewhere. 

DNA Direct is one example 

of how these companies have 

evolved. It was acquired in 2010 

by Medco Health Solutions and 

now operates as a subsidiary of 

the pharmacy benefits manager. 

In 2005, DNA Direct’s tailored 

messages on the home page of 

its web site primarily to consumers. 

Today, the company provides 

a list of services to help patients, 

physicians, hospitals, and payers 

understand and navigate the 

world of genetic testing. 

“The realization that I had 

early on was that the more significant 

the genetic test is in terms 

of the medical implications of 

that test, the more the consumer 

wanted it to be part of their medical 

record, they wanted it to be 

covered by their medical insurer. 

They didn’t really want to go 

outside the system,” says Ryan 

Phelan, president and founder of 

DNA Direct. “At the end of the 

day, that testing that is done outside 

of the healthcare system becomes 

suspect by the system and 

that becomes part of the problem 

of becoming a real, viable company 

today.” 

Industry watchers say while 

most companies haven’t given 

up on their consumer focus, it 

has shifted. Some companies are 

focusing more on working with 

physicians and employer groups. 

Others are emphasizing supporting 

researchers. And still others 

are looking to additional models 

to leverage their customer base to 

conduct research. 

“They’ve definitely morphed. 

If you go back to the beginning of 

what most people generally think 

of as the DTC era, there was this 

thought that people were going 

to be interested and going to pay 

to access their genomes directly,” 

says Dan Vorhaus, editor of Genomics 

Law Report and an attorney 

with Robinson, Bradshaw & 

Hinson. “It’s changed a lot since 

them. I’m not sure people have 

given up on the consumer focus, 

it’s just shifted.” 

The pivotal shift for another 

DTC company, Navigenics, came 

more than two years ago, when 

it began to focus on the use of 

genomics to motivate behavior 

change, such as diet, exercise and 

compliance. “If you look at who 

those sorts of behavior changes 

are most important to, it is health 

plans, employers, and physicians,” 

says Vance Vanier, CEO of 

Navigenics. “For the last couple 

of years, we’ve focused our efforts 

on partnering with those organizations 

where our genomic 

service is part of a broader prevention 

package.” 

In April, Navigenics announced 

a partnership with 

Highmark, one of the nation’s 

largest Blue Cross and Blue 

Shield plans. Under the partnership, 

Highmark offers a personalized 

wellness and prevention 

program to the employers it covers 

that starts with Navigenics 

identifying genetic risk factors of 

an individual covered employee 

through DNA analysis using a 

saliva sample. Navigenics selects 

only health conditions where it 

says genetic insight can guide an 

individual to an informed plan of 

action. 

Results are coupled with access 

to a board-certified genetic 

counselor, the ability to coordinate 

with personal physicians, 

and the tools and resources to 

understand steps to address the 

identified health risks in conjunction 

with an individual’s overall 

health profile. Highmark then 

offers its personalized wellness 

program through its group customer 

relationships in order for 

employers to provide a personalized 

option of health and wellness 

to their employees. 

For 23andMe, its consumer 

business remains an important 

focus, but it is seeking to build 

out a community of customers. It 

has dropped the price of its test 

to $99, but it also requires cutomers 

to subscribe for one year 

to its service at $9 a month. This 

provides access to interpretative 

tools and information on an ongoing 

basis. The company is also 

entering into partnerships with 

institutions conducting research 

using customers who volunteer 

to participate. 

To that end, the critical task 

is to build a broad base of participants 

willing to share not only 

their genetic information, but 

information about their health 

and behavior. Akin to the kind 

of social network research model 

of PatientsLikeMe, the inducement 

is not only to participate in 

research that could improve understanding 

of diseases, but also 

to get access to greater levels of 

information about themselves as 

it becomes available. 

“What we’re doing is based on 

the success of the business we’ve 

already been growing on the consumer 

side and we will continue 

increasing the B2B side of the 

business,” says Ashley Ledbetter 

Dombkowski, chief business 

officer for 23 and Me. “We’re not 

walking away from the consumer 

side. It’s critical. They end up fitting 

together.” 

■ 



Making Good on a Promise 

NIH, FDA make progress toward pledge to realize the benefits of personalized medicine 

By Michael Fitzhugh 

In July 2010, about ten years after 

researchers completed the draft 

map of the human genome, National 

Institutes of Health Director Francis 

Collins and U.S. Food and Drug Administration 

Commissioner Margaret 

Hamburg took to the pages of the New 

England Journal of Medicine with 

a pledge: together they would work 

to bring the benefits of personalized 

medicine to patients. 

“With all of these advances it’s 

breathtaking to consider how fast 

we are moving.” 

Francis Collins, 

Director, National Institutes of Health 

Critics of the Human Genome 

Project complained that its impact to 

date had been small. An editorial in 

The New York Times called it “a sobering 

realization” that despite scientific 

advances linked to the project, it had 

done “relatively little to improve medical 

treatments or human health.” 

The pessimism was not wholly 

unfounded. Although the molecular 

causes of disease are better understood 

today than ever before, scientific 

and policy challenges have slowed the 

genomic revolution. However, much 

has been done since Collins and Hamburg 

laid out their path to realizing the 

potential of personalized medicine. 

The NIH, long at the forefront of supporting 

genetic research, has continued 

to spearhead scientific progress. The organization 

has focused on some of the 

most pressing scientific problems, such 

as determining which genetic markers 

have the most clinical significance, 

how to limit off-target effects of genebased 

therapies, and conducted clinical 

studies to identify genetic variants that 

correlate with a drug response. 

“It’s a pretty exciting time right 

now,” says Collins. “With all of these 

advances it’s breathtaking to consider 

how fast we are moving.” 

NIH-supported tissue banks holding 

thousands of tumor samples 

alongside information linking them to 

clinical outcomes represent one prong 

of the NIH strategy for progress. Such 

data will be essential in supporting 

broad assessments of the clinical importance 

of genetic variation across a 

range of conditions, yielding insights 

that can only come from large and 

long-term studies. That effort—already 

underway when Collins and Hamburg 

wrote their commentary— is advancing. 

Already it is amassing what Collins 

anticipates will, by the end of 2012, 

be 73,000 genomes, as well as their exomes, 

the part of the genome that contains 

the coding portions of genes. 

“The amount of information that 

is going to be pouring out of this is 

pretty phenomenal and will certainly 

extend what is already a pretty amazing 

database of information on genetic 

risk factors to an even longer list,” says 

Collins. 

In particular, he said the Cancer 

Genome Atlas is now making it possible 

to derive information about what 

causes a good cell to go bad and turn 

malignant. Collins expects this understanding 

to accelerate with the rapid 

decline in the cost of sequencing. 

The NIH has also built out the Therapeutics 

for Rare and Neglected Diseases 

program, or TRND, a program 

created to accelerate the development 

of new drugs for rare and neglected 

disease, many of which are singlegene 

disorders. Under the program, 

researchers are able to take promising 

compounds through preclinical development 

and ultimately to a stage where 

a pharmaceutical comapny would be 

willing to license the drug and fund 

further clinical development. 

While that program is still in its 

early days, it has already advanced 

five projects to a pilot stage, including 

a project initiated in May to focus 

on further development of an existing 

small molecule drug used to treat arthritis 

called auranofin as a treatment 

for the rare disease relapsed chronic 

lymphocytic leukemia. 

TRND’s mission is to get such projects 

through “the valley of death” between 

idea and clinical testing. But it is 

also designed to address other barriers 

that continue to stymie the creation of 

new personalized medicines. TRND 

is really an experimental lab for drug 

development, says its founder, Chris 

Austin, a scientific director for the NIH 

Center for Translational Therapeutics. 

The Genetic Testing Registry, another 

key part of NIH’s plans for advancing 

personalized medicine, will 

aggregate information about the availability, 

validity, and usefulness of the 

more than 2,000 genetic tests currently 

available through clinical laboratories. 

While it is still in development, it is 

expected to launch by year-end. Once 

operational, the registry will provide 

access to information about genetic 

tests for inherited and somatic genetic 

variations, including newer types of 

tests, such as arrays and multiplex panels. 

That information primarily comes 

from voluntary data submissions by 

test developers and manufacturers. 

NIH is also making progress on its 

plans to launch the National Center 

for Advancing Translational Sciences. 

That represents an effort to reengineer 

the process of developing diagnostics, 

devices, and therapeutics. “We’re 

hopeful that could stand up this fall,” 

says Collins. He cautions it’s not yet 

finalized because it is dependent upon 

securing a Congressional appropriation. 

While genetics researchers supported 

by NIH uncover new data and information 

about the genomic and molecular 

basis of disease, the FDA has 

sought to establish a level of regulation 

for genetic tests that both protects patients 

and clears the way for companies 

to develop new personalized therapies 

by working more closely with industry 

and providing new formal guidance 

documents. 




Making Good on a Promise 

❱❱ (continued from previous poge) 

A year ago, about 10 percent of labels 

for FDA-approved drugs contained 

pharmacogenomic information. 

Since then, two more important new 

drugs were added to that list: Roche’s 

melanoma drug Zelboraf and Pfizer’s 

non-small cell lung cancer drug Xalkori. 

Both drugs were approved for use 

with companion diagnostics to determine 

which patients harbor genetic 

mutations that mark them as likely to 

benefit from their use. 

The FDA has been forging new policies 

intended to ease the way for more 

approvals of targeted therapies and to 

help ensure the safety of patients as the 

field develops further. The creation of 

draft guidance for the development of 

companion diagnostics in July reflects 

one key step. The draft guidance offers 

clarity about how the FDA intends to review 

diagnostics paired with drugs and 

the process for producing sufficient data 

to establish the safety and effectiveness 

of the diagnostic-product pairings. 

The FDA offered additional guidance 

in August to help clarify the process 

companies will need to follow to 

substantiate that the biomarkers they 

pick to guide more personalized therapeutic 

use and dosing reflect the biological 

process, response, or event, that 

the drugmakers claim. That guidance 

should also help support the agency’s 

goal of establishing the reliability of 

diagnostic tests to support major medical 

decisions. 

The FDA and NIH continue to collaborate 

on regulatory and translational 

science to accelerate the development 

of new therapies. Through the 

FDA-NIH Joint Leadership Council, a 

group co-founded and co-led by Collins 

and Hamburg, the two agencies 

have already sparked new ideas about 

how to tackle some of the fundamental 

steps in drug development in smarter 

and more personalized ways. That will 

continue to be important as the success 

of personalized medicine will depend 

on the efforts of many actors in both 

institutions. 

■ 

Biggest Market Movers in August 2011 

TICKER COMPANY CLOSING 

PRICE 

7/29/2011 

CLOSING 

PRICE 


PRICE 

CHANGE 

PERCENT 

CHANGE 

REASON 

ADVANCERS 

VRNM Verenium 1.6 3.13 1.53 95.60% Reports improved first half financial results and 

repurchases $8.2 million in principal amount of 

its outstanding convertible notes in a private 

transaction for $7.7 million cash. 

ECTE Echo Therapeutics 3.19 4.03 0.84 26.30% Regains ground lost during July and early August 

GNMK GenMark 

4.97 6.25 1.28 25.80% Regains ground lost during July and early August 

Diagnostics 

AMPE Ampio 

Pharmaceuticals 

6.32 7.82 1.5 23.70% Company says late stage clinical results for 

Zertane, a repositioned pain medication, as 

a treatment for premature ejaculation will be 

published in European Urology. 

ROSG Rosetta Genomics 1.3 1.53 0.23 17.70% Shares rise after company reports preliminary 

data on discovery of blood-based microRNA 

signature in blood for congestive heart failure 

■ 

DECLINERS 

JGBO 

Jiangbo 


3.08 0.27 -2.81 -91.20% Stops trading and moves to pink sheets after 

lawsuit filed by shareholders alleging false and 

misleading statements of its financial condition 

DNDN Dendreon 36.9 12.28 -24.62 -66.70% Shares tank after company reports poor quarter 

sales of its prostate cancer vaccine Provenge 

ANX 

Adventrx 


2.95 1.15 -1.8 -61.00% FDA does not approve company’s new drug 

application for Exelbine for the treatment of nonsmall 

cell lung cancer 

PATH NuPathe 6.9 2.74 -4.16 -60.30% Shares fall after three key events: a $30 million 

common stock purchase agreement with Aspire 

Capital; increased net loss in 2011 half year 

financial results over 2010; and Wedbush analyst’s 

lowered outlook for the company 

INSM Insmed 11.49 4.75 -6.74 -58.70% Downgrade to Sector Perform from Outperform 

by RBC Capital analysts 

Includes life sciences stocks with closing price of $1 or more on July 29, 2011 



Individualized Care for the Masses 

Government, industry, and research institutes band together 

to advance personalized medicine in Europe 

By Lucy Clarke 

Europe has lagged the United States 

in the development of personalized 

medicine, but a careful look at the individual 

countries shows that there are an 

increasing number of programs underway 

that are harnessing the potential of 

tailored drugs. 

A United Kingdom program aimed 

at creating personalized cancer care has 

captured headlines most recently. The 

program is being led by Cancer Research 

U.K. and is also backed by the government’s 

Technology Strategy Board, AstraZeneca, 

and Pfizer, along with PA 

Consulting, which will provide managerial 

and I.T. expertise. The collaboration 

will begin collecting up to 9,000 tumor 

samples later this year for molecular diagnosis 

to identify gene faults specifically 

linked to cancer. Molecular diagnosis of 

tumors is not yet available for all patients 

from the National Health Service and 

is currently only possible using a single 

test for each mutation. The new program 

aims to develop a multi-gene panel that 

can test for genetic markers for drugs already 

used in the clinic as well as those 

for new drugs in late-stage trials. Bespoke 

cancer care, it is hoped, will both 

improve patient outcomes and help the 

cash strapped health service save money. 

While the new combined-diagnostic 

project has the highest profile of any 

personalized medicine program backed 

by the Technology Strategy Board, it is 

certainly not the board’s first. Earlier in 

the year, it announced that it was backing 

two other programs in the fields of 

inflammation. In one program, the board 

is working with the Medical Research 

Council and the Association of the British 

Pharmaceutical Industry on chronic 

obstructive pulmonary disease and 

arthritis. In another project the board 

is working to address the mismatch between 

the work being carried out by the 

pharmaceutical and diagnostics industries, 

with the goal of making their work 

complementary and allowing for more 

drug stratification. 

Letter From Europe 

The Technology Strategy Board’s work 

in what the United Kingdom now refers 

to as stratified medicines began in October 

2010 and aims to invest $79.7 million 

(£50 million) over five years to push forward 

the concept of tailor-made drugs 

and healthcare. Programs are selected 

through a competitive process and funding 

from the board is generally matched 

by partners. The next competition for 

stratified medicines will be launched 

around October. 

In the rest of Europe, the picture is 

fragmented. But several personalized 

medicine programs are underway. 

In France, the National Cancer Institute 

set up a network of 28 regional centers 

in 2006, linked with both public and 

private hospitals, where cancer patients’ 

tumors can be rapidly analyzed to establish 

their suitability for drug treatment. 

Between 2004 and 2010, 31 cancer drugs 

were approved in France for use in 49 indications, 

of which almost half are targeted 

therapies. Furthermore, the institute 

estimates that targeted cancer therapies 

now account for around 57 percent of 

cancer treatments used in France’s public 

hospitals sector. 

Once a new cancer drug is approved 

in France, the National Cancer Institute 

moves quickly to allocate funding to the 

testing centers and to put the appropriate 

molecular diagnostics in place. To improve 

the time-to-access for new drugs, 

the institute is now planning to anticipate 

new drug launches and this year has 

earmarked $4.9 million (€3.5 million) to 

detect biomarkers in lung and colorectal 

cancers and melanoma. 

The National Cancer Institute concludes 

that “targeted cancer treatment in 

France shows that innovation can be successfully 

integrated into the healthcare 

system, that molecular stratification is 

cost effective, and that this organization 

could be easily expanded in other European 

settings”. 

In the Netherlands, Rene Bernards is 

at the helm of many of the current initiatives 

in personalized medicine. He is head 

of the division of molecular carcinogenesis 

of the Netherlands Cancer Institute, 

professor of molecular carcinogenesis at 

Utrecht University, as well as co-founder 

and chief scientific officer of the molecular 

cancer diagnostic company Agendia. 

Agendia’s work has been attracting a 

lot of attention from the EU and industry, 

as well as at the public level in the Netherlands. 

In July the company announced 

that it was in line to receive $1.8 million 

(€1.3 million) in funding from the EU under 

the 7th Framework Program over a 

five-year term for the development of new 

molecular diagnostics to help guide individualized 

treatment of cancer patients. 

EuropaBio, the pan-European biotechnology 

association, has been raising the 

profile of personalized medicine since 

2008, when it set up a 20-company, expert 

task force. Several workshops have been 

held to give the European Commission a 

comprehensive overview of the impact of 

personalized medicine across the biotech 

industry as well as the future challenges 

and potential solutions. In parallel with 

the workshops, EuropaBio is in dialogue 

with the Commission’s Directorate General 

for Enterprises and Industry. Together 

they are considering the implications of 

a shift towards a personalized medicine 

paradigm on current health technology 

assessment practices and pricing and reimbursement 

to ensure that the value of 

personalized medicine is recognized and 

that it effectively delivers on its promises 

to patients, healthcare systems, and society 

as a whole. 

Europe is fast getting on board with 

personalized medicine as it realizes that it 

helps address two of the major problems 

facing society: the increasing incidence of 

chronic diseases and the need to spend 

scarce resources effectively. But the challenges 

are many—research, regulatory 

and implementation—and will need to 

be agreed to across the board at European, 

national, regional, and local levels. 

■ 



Personalized Medicine Makes Headway 

Dealmaking and financing activity is strong in 2011 

By Marie Daghlian 

Although there has been criticism 

about the slow progress in realizing 

the promise of personalized medicine, 

dealmaking and financing activity in the 

sector have been robust so far this year. 

The August approval of three personalized 

medicine therapies – Roche’s melanoma 

drug Zelboraf, Pfizer’s non-small 

cell lung cancer drug Xalkori, both approved 

in conjunction with a companion 

diagnostic to identify patients likely 

to benefit from treatment, and Seattle 

Genetics’ lymphoma drug Adcetris – is 

further validation that the promise of 

personalized medicine is near at hand. 

Certainly drugmakers and investors 

seemed to think so as evidenced by the 

number of deals in the space so far this 

year. 

Perhaps the most significant deal 

was Daiichi Sankyo’s $935 million bet 

on Plexxikon, the Bay Area biotech that 

originally discovered the antibody behind 

the Roche drug. 

Plexxikon had retained U.S. co-promotion 

rights for Zelboraf. It also had a 

pipeline of other compounds in development, 

including multiple agents targeting 

cancer. 

Another significant potential acquisition 

in the space, slightly outside the 

realm of life sciences companies but 

critical to personalizing healthcare, is 

Express Scripts pending deal to acquire 

Medco Health Solutions for $29.1 billion. 

Although it must still pass muster with 

regulators, it would combine two of the 

largest pharmacy benefits managers 

in the United States and give Express 

Scripts a foothold in personalized healthcare. 

Medco has been a leader in integrating 

personalized health solutions into its 

practice, mainly through the genetic services 

of its DNA Direct division. 

Large diagnostics players have also 

moved aggressively to strengthen their 

personalized medicine offerings. Quest 

Diagnostics made two significant purchases 

to increase its gene-based testing 

capabilities, acquiring the Athena Diagnostics 

division of Thermo Fisher Scientific 

for $740 million and buying Celera 

Corporation, one of the pioneers in genetic 

diagnostics discovery and development, 

for $657 million. 

Qiagen, a leading player in personalized 

medicine, also beefed up its capabilities 

by taking a major stake in French 

molecular diagnostics company Ipsogen 

and a strategic stake in German biotech 

Alacris Theranostics. 

Japanese pharma Eisai pledged $200 

million to newly formed U.S. biotech H3 

Biomedicine for research and support in 

the use of biomarkers to guide the development 

of cancer drugs for personalized 

treatment regimens. 

Venture investors pumped more than 

$600 million into companies in the sector 

in the first 8 months of 2011. CardioDx, 

which makes a non-invasive, finger prick 

genetic test that measures a person’s risk 

of developing cardiovascular disease, 

raised $57.5 million in a $78.3 million series 

E round of equity and options. The 

beauty of CardioDx’ test for predicting 

cardiovascular risk, says the company, is 

that it is non-invasive and accurate. Current 

standards for diagnosing risk, including 

catheter-based angiography and 

non-invasive methods that expose the 

patient to radiation and contrast agents, 

carry the chance of misdiagnosis. 

And French firm Advanced Accelerator 

Applications raised $56.8 million to 

fund development of innovative diagnostic 

and therapeutic applications and 

products for the personalized treatment 

of serious diseases. 

Two biotechs focused on personalized 

medicine have also gone public this 

year. Cardiovascular diagnostics firm 

BG Medicine raised $40.3 million in an 

initial offering in January pricing at half 

its target range. But the real star has been 

Endocyte, a biotech with a late stage 

ovarian cancer treatment that is being 

paired with a companion diagnostic. Although 

Endocye had to slash its offering 

price in half to get its deal done in February, 

it was one of only three companies 

in the 2011 U.S. Life Sciences IPO class to 

stay in positive territory after the violent 

market swings in August with a return 

on its IPO price of 82.7 percent. 

Burrill & Company, the publisher of 

The Burrill Report, is an investor in Endocyte. 

■ 

Personalized Medicine Financings in 2011 

COMPANY TICKER RAISED (USD M) CATEGORY PRINCIPAL ACTIVITY 

VENTURE 

CardioDx 60.0 Diagnostics Genomic tests 

Advanced Accelerator 

56.8 Tools/Technology Personalized medicine 

Applications (France) 

Verinata Health 46.5 Diagnostics Prenatal 

Oxford Nanopore Technologies 

(United Kingdom) 

41.0 Tools/Technology Genomics 

(continued) ❱❱ 



❱❱ (continued) 



Blueprint Medicines 40.0 Tools/Technology Personalized cancer 

therapeutics 

Atlas Genetics (United 

27.3 Diagnostics Point of care testing 

Kingdom) 

T2 Biosystems 23.0 Diagnostics 

Biodesix 20.0 Diagnostics 

Cleveland HeartLab 18.4 Diagnostics Cardiovascular 

HTG Molecular Diagnostics 16.2 Diagnostics Cancer Dx 

High Throughput Genomics 15.7 Tools/Technology Genomics 

Metabolon 13.1 Diagnostics Metabolic profiling 

Astute Medical 13.0 Diagnostics 

AssureRx 11.0 Tools/Technology Personalized medicine 

Advanced Animal Diagnostics 11.0 Diagnostics Animal health 

Wuhan Kindstar Diagnostics 

11.0 Diagnostics 

(China) 

Diagnoplex (Switzerland) 10.4 Diagnostics Cancer 

Foundation Medicine 10.0 Diagnostics Genomic tests for cancer 

Microvisk (United Kingdom) 9.5 Diagnostics Blood tests 

23andMe 9.0 Tools/Technology Personal genomics 

BioOptix Diagnostics 9.0 Diagnostics 

OctreoPharm Sciences 

7.0 Diagnostics 

(Germany) 

glcare Pharma (Canada) 7.0 Diagnostics Colonoscopy 

Atossa Genetics 6.6 Diagnostics Cancer 

Curetis (Germany) 6.4 Diagnostics Infectious disease 

Quanterix 6.0 Diagnostics Neurology, oncology 

Allegro Diagnostics 5.4 Diagnostics 

Knome 5.0 Tools/Technology Personal genomics 

Second Genome 5.0 Tools/Technology Personalized medicine 

Fluidigm 5.0 Tools/Technology Genomics 

VeraLight 5.0 Diagnostics 

Asurgen 5.0 Diagnostics Companion diagnostics 

OrganOx (United Kingdom) 4.4 Diagnostics 

Neuroptix 4.0 Diagnostics Alzheimer’s 

Cleveland HeartLab 4.0 Diagnostics Heart attack risk test 

Multiplicom (Belgium) 3.0 Diagnostics Molecular 

Stat Diagnostica (Spain) 2.9 Diagnostics 

One Way Liver Genomics 

2.8 Tools/Technology Personalized medicine 

(Spain) 

Chronix Biomedical 2.8 Diagnostics Genetic tests 

Lumicell Diagnostics 2.7 Diagnostics Cancer 

HistoRx 2.6 Tools/Technology Personalized medicine 







iNTELOMED 2.6 Diagnostics Cardiovascular 

NanoString Technologies 2.5 Tools/Technology Genomics 

KellBenx 2.5 Diagnostics Prenatal 

Precision Biopsy 2.5 Diagnostics Prostate cancer 

Lumora (United Kingdom) 2.4 Diagnostics 

NanoDetection Technology 2.3 Diagnostics 

Crescent Diagnostics (Ireland) 2.2 Diagnostics Predictive tests 

Stratos Genomics 2.1 Tools/Technology Genomics 

Lophius Biosciences (Germany) 2.0 Diagnostics Transplantation 

Oxford Medical Diagnostics 

1.9 Diagnostics Drug delivery, ophthalmic 


OncoHealth 1.6 Diagnostics 

Abcodia (United Kingdom) 1.6 Diagnostics Biomarkers 

Radisens Diagnostics (Ireland) 1.6 Diagnostics Point of care testing 

Oxford Medical Diagnostics 

1.6 Diagnostics Drug delivery, ophthalmic 

(united Kingdom) 

Sera Prognostics 1.4 Diagnostics Women’s health 

BerGenBio (Norway) 1.2 Tools/Technology Genomics 

VentriPoint Diagnostics 1.2 Diagnostics Cardiovascular 

BioMarker Strategies 1.1 Diagnostics Cancer Dx 

Predictus BioSciences 0.7 Diagnostics 

ECI Biotech 0.6 Diagnostics Infection sensors 

BioBehavioral Diagnostics 0.3 Diagnostics ADHD 

Phthisis 0.2 Diagnostics Infectious 

Computable Genomix N/A Tools/Technology Pharmacogenetics 

TOTAL 600.6 

IPO 

PIPE 

Endocyte ECYT 86.4 Therapeutics Cancer 

BG Medicine BGMD 40.3 Diagnostics Cardiovascular 

MEDIAN Technologies (France) Alternext: ALMDT 14.3 Diagnostics Oncology imaging 

Glycominds (Israel) TASE:GLCM 8.4 Diagnostics Blood tests 

TOTAL 149.4 

WaferGen Biosystems OTC:WGBS 30.4 Tools/Technology Genomics 

Genetic Technologies 

GENE 25.4 Diagnostics Genetic tests 

(Australia) 

MDxHealth (Belgium) Euronext MDXH 11.6 Diagnostics 

Genfi t (France) Euronext: ALGFT 7.5 Diagnostics Cardiovascular disease 

biomarkers 

CombiMatrix CBMX 6.8 Tools/Technology Genomics 

Rosetta Genomics (Israel) ROSG 6.0 Diagnostics Molecular 







Verisante Technology (Canada) TSX-V:VRS 6.1 Diagnostics 

Exiqon (Denmark) CSE:EXQ 3.6 Tools/Technology Pharmacogenetics 

ARCA Biopharma ABIO 3.0 Therapeutics Cardiovascular 

Biohit Oyj (Finland) HSE:BIOBV 2.9 Diagnostics 

Response Genetics RGDX 2.3 Diagnostics Cancer 

Radient Pharmaceuticals RPC 0.9 Diagnostics 

Innovotech (Canada) TSX:IOT 0.9 Diagnostics 

Med BioGene (Canada) TSX-V:MBI 0.4 Diagnostics Pharmacogenetics 

Biomagnetics Diagnostics BMGP:Pk 0.3 Diagnostics Infectious 

Amorfix Life Sciences (Canada) TSX:AMF 0.1 Diagnostics Neurology 

TOTAL 108.2 

FOLLOW ON 

Complete Genomics GNOM 79.1 Tools/Technology Genomic sequencing 

Endocyte ECYT 71.6 Therapeutics Cancer 

Quidel QDEL 52.6 Diagnostics 

Nanosphere NSPH 34.5 Diagnostics 

Genmark Diagnostics GNMK 30.0 Diagnostics 

Vermillion VRML 21.8 Diagnostics Molecular 

TOTAL 289.6 

DEBT 

Quest Diagnostics DGX 1,250.0 Diagnostics 

Illumina ILMN 800.0 Tools/Technology Genomics 

Radient Pharmaeuticals RPC 8.4 Diagnostics Cancer 

TOTAL 2,058.4 

■ 

Personalized Medicine M&A in 2011 

ACQUIRER COUNTRY TARGET COUNTRY DEAL VALUE 

(USD M) 

Thermo Fisher 

Scientific 

PRINCIPAL 

FOCUS 

RATIONALE/PRINCIPAL ASSET 

United States Phadia AB Sweden 3,500.0 Diagnostics Thermo Fisher Scientific acquires Phadia, 

a maker of blood-test systems for clinical 

diagnosis and monitoring of allergy and 

autoimmune diseases, from Cinven, its private 

equity owner. 

Daiichi Sankyo Japan Plexxikon United States 935.0 Cancer Daiichi Sankyo acquires privately-held Plexxikon 

for its lead program partnered with Roche, a 

drug that targets the BRAF mutation for the 

treatment of melanoma and other solid tumors. 

The deal includes $805 million upfront and up to 

$130 million in near-term milestones. 






(USD M) 

Quest 

Diagnostics 

Quest 

Diagnostics 

United States 

United States 

Athena 

Diagnostics 

(TMO) 

Celera 

Corporation 

PRINCIPAL 

FOCUS 


United States 740.0 Diagnostics Thermo Fisher Scientific sells its Athena 

Diagnostics division, a reference laboratory 

that provides gene-based diagnsotic testing 

for neurological and other diseases, to Quest 

Diagnostics for $740 million in cash. 

United States 657.0 Diagnostics Quest Diagnostics acquires Celera, one of 

the world’s pioneers in genetic diagnostics 

discovery and development, for $8 per share. 

Novartis Switzerland Genoptix United States 470.0 Diagnostics Novartis acquires Genoptix, a specialized 

laboratory providing personalized diagnostic 

services to community-based hematologists and 

oncologists that increase their ability to define 

and monitor individualized treatment programs. 

Qiagen Netherlands Cellestis Australia 380.0 Diagnostics Qiagen acquires Cellestis for $380 million in 

cash, providing Qiagen with access to a novel 

pre-molecular technology that offers a new 

dimension in disease detection not currently 

possible with other diagnostic methods. 

Cellestis has commercialized a test for latent TB 

and is working on a test for monitoring disease 

risk from cytomegalovirus. 

Ventana Medical 

(Roche) 

Switzerland 

mtm 

laboratories 

Germany 260.0 In vitro 

diagnostics 

Qiagen Netherlands Ipsogen France 131.0 Molecular 

diagnostics 

LabCorp United States Orchid 

Cellmark 

Myriad Genetics United States Rules-Based 


BioMerieux France A.B. Services France 60.0 Molecular 

diagnostics 

Mingyuan 

Medicare 

Development 

China 

Shanghai 

Yuanqi 

Ventana Medical Systems, part of the Roche 

Group, will acquire mtm laboratories, a 

privately-held developer of in vitro diagnostics 

with a focus on early detection and diagnosis of 

cervical cancer. 

Qiagen offers to purchase a 47% initial stake in 

French molecular diagnostics company Ipsogen 

and subsequently make a public offer to fully 

acquire the global maker of molecular profiling 

and personalized healthcare diagnostics for a 

broad range of hematological cancers. 

United States 85.4 Diagnostics Laboratory Corporation of America acquires 

Orchid Cellmark, an international provider of 

DNA testing services primarily for forensic and 

family relationship applications. 

United States 80.0 Diagnostics Myriad Genetics acquires molecular diagnostics 

developer Rules-Based Medicine for $80 million 

in a deal that it expects will help build its 

companion diagnostics franchise. 

bioMerieux acquires Argene, which specializes 

in molecular diagnostics for infectious diseases. 

China 55.0 Diagnostics Mingyuan Medicare Development buys a 70% 

stake in 18-month-old Shanghai Yuanqi for 

354 million RMB ($55 million). Both companies 

provide cancer screening kits to the PRC. 

Shanghai Yuanqi has developed molecular 

diagnostic kits for leukemia, lymphoma, and 

individualized cancer therapy. Minyuan makes 

a protein chip that tests blood samples for 12 

tumor markers that detect 12 types of cancer. 







(USD M) 

OraSure 

Technologies 

PRINCIPAL 

FOCUS 

United States DNA Genotek Canada 53.0 Molecular 

diagnostics 

Luminex United States EraGen 

Biosciences 

Lipoxen 

United 

Kingdom 

Qiagen Germany Alacris 

Theranostics 

Rules-Based 


DxTerity 

Diagnostics 

Lab21 Limited 

United States 34.0 Molecular 

diagnostics 


OraSure Technologies, a maker of oral fluid 

diagnostic devices, expands into the molecular 

diagnostics market with the acquisition of DNA 

Genotek, giving it a portfolio of products that 

enable collection, stabilization, transportation, 

and storage of DNA and RNA samples. 

Luminex will acquire privately-held EraGen 

Biosciences, an innovator in molecular 

diagnostic testing technologies for infectious 

disease and genetic applications. 

SymbioTec Germany 14.5 Cancer Lipoxen acquires cancer company SymbioTec 

in a stock deal valued at GBP 8.8 million ($14.5 

million), issuing 80 million shares to cover 

the acquisition. SymbioTec’s lead candidate 

OncoHist is a recombinant human histone in a 

phase 2b trial in Russia. 

Germany N/A Biomarkers Qiagen acquires a strategic stake in Alacris 

Theranostics, a German start-up developing 

individualized cancer treatment strategies based 

upon a patient’s genomic profile, and for the 

acquisition of an exclusive option to access all 

biomarkers emerging from this discovery program. 

United States Satoris United States N/A Biomarkers Rules-Based Medicine acquires the intellectual 

property assets from Satoris that are based on 

extensive research to discover biomarkers and 

biomarker panels for clinical and research use in 

Alzheimer’s disease, frontal temporal dementia, 

Parkinson’s disease, and other conditions that 

affect the central nervous system. 

United States Source MDx IP United States N/A Cancer 

diagnostics 

United 


Myconostica 

United 


Genetic testing firm DxTerity Diagnostics 

acquires intellectual property rights to several 

blood-based cancer diagnostic tests from 

SourceMDx. The IP covers gene expression 

assays for early diagnosis of breast and lung 

cancer and a test for determining survival and 

therapy response in melanoma. 

N/A Diagnostics Lab21 Limited, the global specialist in 

personalised medicine and clinical diagnostics, 

acquires Myconostica, a molecular diagnostic 

company focusing on the diagnosis of fungal 

infections of clinical importance. The deal gives 

Lab21 its first set of nucleic acid-based assays. 

Nestle Switzerland Prometheus 

Laboratories 

United States N/A Diagnostics Prometheus Laboratories, a specialty 

pharmaceutical and diagnostic company, is 

acquired by Nestle Health Science, a whollyowned 

subsidiary of Nestle, to help it pioneer 

science-based nutritional solutions for improved 

personalized healthcare for medical conditions. 

IntegenX United States GenVault United States N/A Genomic tools Privately-held IntegenX secures its leading 

position in sample preparation for genetic analysis 

with the acquisition of substantially all the stock 

of GenVault. With a concurrent venture financing, 

IntegenX will commercialize a system for nextgeneration 

sequencing library preparation and a 

system for DNA-based human identification. 







(USD M) 

PRINCIPAL 

FOCUS 


Pharmascience Canada Aegera 

Therapeutics 

Canada N/A Cancer Pharmascience, the largest generic 

pharmaceutical company in Quebec, acquires 

Aegera Therapeutics, a Montreal-based clinicalstage 

biotech focused on developing targeted 

therapeutics to address unmet major medical 

needs, primarily in oncology. 

Synthon Netherlands Syntarga Netherlands N/A Cancer Synthon acquires Syntarga BV, a 

biopharmaceutical company focused on the 

development of innovative chemistries that arm 

antibodies with a cytotoxic payload. 

■ 

Personalized Medicine Partnering Deals in 2011 

DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 

PRINCIPAL ASSET/RATIONALE 

Partnership Epizyme United 

States 

Eisai Japan 206.0 Cancer Epizyme and Eisai partner to 

discover, develop and commercialize 

therapeutics targeting EZH2, an 

epigenetic enzyme, for the treatment 

of lymphoma and other cancers in 

genetically-defined patients. 

Collaboration H3 Biomedicine United 

States 

Eisai Japan 200.0 Personalized 

medicine 

Eisai pledges $200 million to newco 

H3 Biomedicine for research and 

clinical development support. H3 

Biomedicine will also have access to 

Eisai’s drug development capabilities. 

H3 Biomedicine will use biomarkers 

to guide the development of cancer 

drugs for personalized treatment 

regimens. 

License 4SC AG Germany Yakult Honsha Japan 192.3 Cancer Yakult Honsha granted exclusive 

rights in Japan to develop and 

commercialized cancer candidate 

resminostat, an HDAC inhibitor in 

phase 2 testing to treat hepatocellular 

carcinoma, Hodgkin’s lymphoma and 

colorectal cancer in patients with 

mutat K-Ras. 4SC will get $8.7 million 

upfront and is eligible for up to $183.6 

million in milestones, plus doubledigit 

royalties. Yakult Honsha will be 

responsible for all development in 

Japan. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


License 

Zinfandel 


United 

States 

Takeda 

Pharmaceutical 

Japan 87.0 Alzheimer’s 

diagnostic 

Takeda and Zinfandel 

Pharmaceuticals enter into an 

exclusive, worldwide licensing 

agreement regarding Zinfandel’s 

TOMM40 assay as a biomarker for the 

risk of Alzheimer’s disease in high-risk 

older adults with normal cognition. 

The companies will study pioglitazone 

in connection with the TOMM40 

biomarker and Alzheimer’s disease. 

License Adimab United 

States 

Merrimack 


United 

States 

57 Cancer 

antibodies 

Merrimack exercises option to license 

global rights to cancer candidate 

MM-151,a mixture of three mAbs 

against non-overlapping epitopes 

of human epidermal growth factor 

receptor I, discovered under a 

2009 deal. Adimab gets $1million 

option exercise fee, and is eligible 

for up to $13.5 million in milestones 

per therapeutic area for the first 

four areas plus up to $500,000 in 

milestones per diagnostic developed 

for MM-151, plus mid-single-digit 

royalties. 

License DiagnoCure Canada Signal Genetics United 

States 

13.3 Cancer 

diagnostics 

Signal Genetics, a predictive genetic 

testing company focusing on 

oncology, and DiagnoCure, a firm 

that develops and commercializes 

high-value cancer diagnostic tests, 

will collaborate commercialize the 

Previstage GCC Colorectal Cancer 

Staging Test, and further develop 

novel genomic cancer tests in the 

field of Personalized Medicine. Signal 

Genetics is granted a worldwide 

exclusive license to the Previstage 

GCC Colorectal Cancer Staging 

Test developed by DiagnoCure, and 

acquires DiagnoCure’s U.S. CLIA 

service laboratory. 

Agreement EpiStem United 


License Oxford BioMedica United 


Sanofi-Aventis France 4.0 Cancer EpiStem will provide its biomarker 

gene expression profining and 

immunohistochemistry technologies 

to support Sanofi’s cancer programs 

in 3-year deal worth up to $4 million 

in research support, including 

reimbursement costs. 

Pfizer 

United 

States 

4.0 Diagnostics Extension of 2001 deal with Wyeth 

adds non-exclusive rights to Oxford 

BioMedica’s 5T4 tumor antibodies 

for diagnostic use and an option to 

commercialize 5T4-based diagnostics 

for cancer indications. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


License Med BioGene Canada Precision 


United 

States 

3.5 Diagnostics Med BioGene grants Precision 

exclusive, worldwide rights to 

develop and commercialize its 

LungExpress Dx technology for 

$2.3 million upfront and high-single 

digit royalties. The test is a 15-gene 

expression-based assay that classifies 

cancer patients into high- and lowrisk 

prognostic groups based on the 

molecular profile of a patient’s tumor, 

and is in registration. 

Agreement Transgene France Beckman Coulter United 

States 

N/A 

Companion 

diagnostic 

Beckman Coulter will develop a 

companion diagnostic to select 

patients for Transgene’s cancer 

candidate TG4010. The diagnostic 

will measure levels of triple-positive 

activated natural killer (NK) cells. 

Agreement BG Medicine United 

States 

Siemens 

Healthcare 

Germany N/A Diagnostics Companies will develop galectin-3 

test for Siemens automated 

immunoassay platforms for clinical 

laboratories. 

Partnership Qiagen Netherlands EyeSense Switzerland N/A Diagnostics Partnership to develop an optical 

measurement technology for 

blood glucose monitoring in 

diabetes patients using Qiagen’s 

ESE fluorescense-based optical 

technology and EyeSense’s glucose 

sensing technology. Qiagen acquired 

a minority stake in EyeSense and is 

eligible for royalties. 

Partnership Seegene Korea Caliper Life 

Sciences 

United 

States 

N/A 

Molecular 

diagnostics 

Strategic partnership to enable 

Seegene’s SeePlex multiplex 

diagnostic assays to run on Caliper’s 

innovative LabChip Dx platform. 

License Biocartis Switzerland Johnson & 

Johnson 

United 

States 

N/A Diagnostics Biocartis granted J&J’s Janssen 

Pharmaceutica exclusive, worldwide 

rights to develop and commercialize 

assays on Biocartis’ molecular 

diagnostics system for undisclosed 

neurological and infectious diseases. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


License Merck KGaA Germany Apeiron 

Biologics 

Agreement XDx United 

States 

Collaboration 

Collaboration 

InVivoScribe 

Technologies 

Partners 

HealthCare 

United 

States 

United 

States 

LabCorp 

Vienna N/A Cancer Apeiron Biologics granted rights to 

further develop and commercialize 

the investigational immunocytokine 

hu14.18-IL2, against the GD2 antigen, 

expressed on neuroblastoma, 

melanoma and other malignant 

tissues, that is fused to the cytokine 

interleukin-2. hu14.18-IL2 has 

shown preliminary activity in a 

subset of children suffering from 

neuroblastoma in a mid-stage study. 

Apeiron will continue developing 

the protein’s therapeutic potential 

for pediatric neuroblastoma, an 

indication with high unmet medical 

need, and will evaluate strategies to 

advance it in other cancer indications, 

such as melanoma. 

United 

States 

Novartis Switzerland N/A Companion 

diagnostic 

Luminex 

United 

States 

N/A Diagnostics XDx, a molecular diagnostics firm 

focused on noninvasive gene 

expression tests to monitor immunemediated 

conditions, enters into a 

multi-year agreement with Labcorp 

to provide heart transplant patients 

eligible for XDx’s AlloMap test with 

the convenience of having their blood 

drawn at participating LabCorp sites 

in the United States. The expanded 

agreement follows a successful oneyear 

pilot between the companies at 

select LabCorp blood draw sites. 

N/A 



Invivoscribe Technologies and Novartis 

will collaborate to develop and 

commercialize a companion diagnostic 

test to identify FLT3 positive acute 

myeloid leukemia patients for specific 

use in connection with the Novartis 

development compound, midostaurin, 

a targeted small molecule inhibitor of 

FLT3 tyrosine kinase currently in phase 

3 trials for newly diagnosed patients 

with FLT3 mutated AML who are 

receiving midostaurin or a placebo in 

combination with chemotherapy. FLT3 

is mutated in approximately one-third 

of all AML patients and FLT3 mutations 

are associated with poor prognosis. 

Luminex has teamed with Partners 

HealthCare toward the discovery of 

novel biomarkers and development 

of clinical assays to enable Partners 

HealthCare Center for Personalized 

Genetic Medicine to develop novel 

molecular diagnostic assays based 

on biomarkers discovered within 

Partners HealthCare entities.The tests 

will operate on Luminex’s proprietary 

platform. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Collaboration Ingenuity Systems United 

States 

Partnership 

Cell Signaling 

Technology 

United 

States 

Covance 

United 

States 

N/A 

Next-gen 

sequencing 

Astellas Pharma Japan N/A Personalized 


Partnership bioMerieux France Ipsen France N/A Personalized 


Ingenuity¨ Systems, a leading provider 

of information and analytics solutions 

for life science researchers, enters 

into a research collaboration with 

Covance Genomics Laboratory for 

next-generation sequencing data 

analysis. 

Cell Signaling Technology and 

Astellas Pharma enter into a joint 

commercialization partnership 

focused on the delivery of 

personalized cancer therapeutics 

and diagnostics. The companies pool 

their respective global IP relating 

to the fusion kinase, EML4-ALK, to 

enable the commercialization of 

diagnostic and therapeutic products 

targeting this cancer enzyme. CST 

has sole global rights to exploit the 

joint IP in the field of human medical 

diagnostics while Astellas has sole 

global rights in the field of human 

therapeutics. 

Ipsen and bioMerieux partner 

to create a global collaboration 

in theranostics, with a focus on 

hormone-dependent cancers, signing 

a framework agreement to leverage 

their expertise and resources to 

develop a personalized approach 

to medicine based on Ipsen’s broad 

portfolio of innovative compounds 

and bioMerieux’s diagnostic tests. 

They will jointly identify programs 

that would benefit from the codevelopment 

of a therapeutic and a 

companion diagnostic test, notably 

in the prevention and treatment of 

prostate and breast cancers, neuroendocrine 

tumors and pituitary 

tumors. 

License Epigenomics Germany Qiagen Germany N/A Diagnostics Epigenomcs and Qiagen announce 

an option agreement allowing Qiagen 

to develop a colorectal cancer 

blood test based on Epigenomics’ 

mSept9 biomarker and certain DNA 

methylation analysis technologies. 

Qiagen has two years to exercise its 

option. 

Collaboration 

RainDance 

Technologies 

United 

States 

Roche Switzerland N/A Next-gen 


RainDance Technologies and 

454 Life Sciences, a division of 

Roche,will collaborate to develop 

and commercialize a targeted nextgeneration 

sequencing solution 

for simple and cost-effective 

investigation of drug absorption, 

distribution, metabolism, and 

excretion. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Collaboration 

License 

Collaboration 

Institute for 

Systems Biology 

Cell Signaling 

Technology 

Shenzhen Kang 

Sheng Bao Bio- 

Technology 

United 

States 

United 

States 


Collaboration MolecularMD United 

States 

Covance 

Ventana Medical 

(Roche) 

SuperNova 

Diagnostics 

Ariad 


Partnership Definiens Germany Advanced Cell 

Diagnostics 

United 

States 

N/A 

Central 

nervous 

system 

Switzerland N/A Personalized 

cancer Dx 

United 

States 

United 

States 

United 

States 

Covance’s genomics laboratory 

in Seattle will collaborate with the 

Institute for Systems Biology to 

study brain tumors, according to 

GenomeWeb. Researchers will study 

the genetic differences within tumors 

and find molecular targets that would 

enable personalized medicine. 

Cell Signaling Technology grants 

Ventana Medical Systems a 

worldwide non-exclusive license to 

CST patent rights and proprietary 

antibody reagents relating to the 

detection of epidermal growth factor 

receptor mutations for the field of 

tissue-based in vitro diagnostic 

testing. Mutant forms of EGFR are 

important determinants in the clinical 

management of lung cancer. 

N/A Diagnostics Collaboration to expand 

R&D, manufacturing, and 

commercialization of diagnostics in 

China and affiliated markets. 

N/A 

N/A 

Companion 

diagnostic 



Exclusive collaboration agreement 

in which MolecularMD will develop 

and commercialize a companion 

diagnostic test to identify the T315I 

mutation of the BCR-ABL gene 

in patients with chronic myeloid 

leukemia and Philadelphia positive 

acute lymphoblastic leukemia. Ariad 

is advancing its investigational, pan- 

BCR-ABL inhibitor, ponatinib, in the 

pivotal PACE trial of patients with 

resistant or intolerant CML and Ph+ 

ALL, or those with the T315I mutation. 

Ariad had used MolecularMD’s test 

in a phase 1 trial. The companies 

plan to submit marketing approval 

applications for ponatinib and its 

companion diagnostic in 2012, at 

approximately the same time. 

Strategic partnership to develop 

image analysis applications that will 

enable researchers to accurately 

quantify specific RNA molecules 

within individual cells in routine 

clinical specimen. Definiens will 

develop a unique and proprietary 

image analysis application for ACD’s 

RNAscope platform and add new 

specialized image analysis solutions 

that will automatically process and 

analyze images from RNAscope 

assays, enhancing the utility of 

RNAscope-based diagnostic tests for 

personalized medicine. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Partnership Definiens Germany Cernostics 

Pathology 

Partnership Seegene Korea Samsung 

Medical Center 

Partnership CombiMatrix United 

States 

Partnership Orasi Medical United 

States 

Collaboration 

Bar Harbor 

Biotechnology 

United 

States 

Clarient (GE 

Healthcare) 

Biogen Idec 

Clinical 

Reference 

Laboratory 

United 

States 

N/A Diagnostics Cernostics will apply Definiens’ image 

analysis technology to develop new 

molecular diagnostic tests for the 

treatment of a variety of cancer types. 

Korea N/A Diagnostics Seegene partners with Samsung 

Medical Center to co-develop 

perconalized medicine cancer tests, 

which will detect genetic mutations 

indicative of certain cancers. The 

initial tests to be developed will 

be for non-small cell lung cancer, 

colon cancer, pancreatic cancer, and 

cholangiocarcinoma. 

United 

States 

United 

States 

United 

States 

N/A Diagnostics CombiMatrix partners with GE 

Healthcare’s Clarient, giving Clarient 

exclusive rights to commercialize 

oncology-related tests among 

commercial laboratories using 

CombiMatrix DNAarray tests, which 

are based on aCGH technology and 

provide physicians a comprehensive 

genomic profile of solid cancer 

tumors and blood-based cancers, 

helping to better guide patient 

treatment decisions. The DNAarray 

tests are utilized for both prognostic 

and predictive purposes, and can 

help identify therapeutic targets 

that are directly associated with 

FDA-approved chemotherapeutic 

agents, as well as therapies that 

are being utilized in large scale 

biopharmaceutical clinical trials. 

N/A Diagnostics Orasi Medical and Biogen Idec 

partner to test novel biomarkers in 

the development of treatments for 

central nervous system disorders. The 

companies will use Orasi’s technology 

to identify and test biomarkers 

based on magnetoencephalography, 

non-invasive and patient-friendly 

scans that will be used to assess the 

integrity of brain communication 

networks in real time and measure 

brain function in patients. 

N/A Diagnostics Bar Harbor BioTechnology, a 

developer of genetic risk assessment 

testing, and Clinical Reference 

Laboratory, a provider of laboratory 

testing services in molecular 

diagnostics and clinical trials, will 

work together in the development 

of new genetic diagnostic tests, 

starting with development of BHB’s 

new genetic-based risk assessment 

test for colorectal cancer with clinical 

validation support provided by CRL’s 

CLIA-certified laboratory services. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Partnership Atonomics Denmark Beckman Coulter United 

States 

Collaboration 

Partnership 

Codevelopment 

Foundation 


Exagon 

Diagnostics 

Clovis Oncology 

United 

States 

United 

States 

United 

States 

Partnership MolecularMD United 

States 

Celgene 

Medco Health 

Solutions 

United 

States 

United 

States 

N/A Diagnostics Partnership will develop diagnostics 

for Atonomics’ Atolyzer immunodiagnostic 

system, in early 

development for use in a near-patient 

setting for indications including 

cardiovascular diseases, prostate 

cancer, and maternal health. Beckman 

makes an undisclosed equity 

investment in Atonomics. 

N/A 

Cancer 

diagnostics 

Roche Switzerland N/A Companion 

diagnostic 

Medco Health 

Solutions 

United 

States 

Celgene will use Foundation 

Medicine’s clinical cancer genomics 

test in ongoing clinical trials of 

Celgene drug candidates. The test in 

development will be used to facilitate 

faster recruitment of target patient 

populations and for broad genetic 

characterization of all trial enrollees 

which may help to identify those 

patients most likely to respond to 

Celgene drug candidates. 

N/A Diagnostics Exagen Diagnostics and Medco 

Research Institute start a joint pilot 

program called NIMBLE designed 

to help physicians optimize 

the effectiveness of low-dose 

methotrexate therapy for patients 

who suffer from rheumatoid arthritis. 

The NIMBLE pilot program will use 

Exagen’s proprietary Avise PG lab 

test to measure whether patients 

are receiving an appropriate 

dose of MTX. Avise PG measures 

MTX polyglutamate levels, the 

active metabolites of MTX. These 

measurements can help establish 

how MTX is being absorbed, retained 

and metabolized by the patient. 

With this information, a physician can 

more accurately adjust the dose of 

MTX, making it more effective for the 

patient. 

N/A 



Clovis Oncology and Roche will 

co-develop an in vitro PCR-based 

companion diagnostic test. The goal 

is to identify activating epidermal 

growth factor receptor (EGFR) 

mutations in patients with non-small 

cell lung cancer, including the EGFR 

T790M mutation. 

Medco Health Solutions and 

MolecularMD launch a personalized 

medicine program for chronic 

myeloid leukemia patients through 

which Medco will offer patients 

diagnosed with CML a molecular 

test known as qRT-PCR BCR-ABL to 

monitor the disease. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Collaboration Merck United 

States 

Collaboration Life Technologies United 

States 

Partnership 

Neogenix 

Oncology 

United 

States 

Partnership ResearchDx United 

States 

Partnership 

Medco, United 

BioSource 

United 

States 

Roche Switzerland N/A Cancer 

diagnostics 

Fox Chase 

Cancer Center 

Cambridge 

Biomedical 

CompanDX 

United 

States 

United 

States 

United 


Merck and Roche will collaborate on 

the application and development of 

diagnostic assays for use with Merck’s 

investigational cancer therapy portfolio. 

N/A Genomics Fox Chase Cancer Center and Life 

Technologies will collaborate under 

a new program to provide nextgeneration 

sequencing analysis of 

solid tumors, a foundational element in 

the planned Cancer Genome Institute 

at Fox Chase, a facility that will foster 

new discoveries and treatments and 

establish the institution as a leader 

in developing personalized medicine 

strategies for cancer care. 

N/A 

N/A 

Cancer 

diagnostics 

Molecular 

diagnostics 

Sanofi France N/A Personalized 


Neogenix Oncology and Cambridge 

Biomedical partner to develop 

Neogenix’ pancreatic and colorectal 

cancer diagnostic serum ELISA 

assays. Neogenix has developed 

tumor specific biomarkers for 

the detection of pancreatic and 

colorectal cancer in tissue and 

serum. These diagnostic assays will 

serve as the companion products 

for the Neogenix anti-cancer mAb 

therapeutic pipeline and hold 

additional hope for early detection of 

pancreatic and colorectal cancer. 

ResearchDx partners with CompanDX 

to offer a full capability to discover 

and deliver novel molecular diagnostic 

tests that offer turnkey solutions for 

companion diagnostic delivery. 

Medco Health Solutions subsidiary 

United BioSource and Sanofi enter 

into a global, multi-year agreement 

to improve patient care in realworld 

settings by leveraging several 

capabilities, including personalized 

medicine and pharmacogenomics, 

health economics, and safety 

research, as well as clinical/adherence 

support.The goal of the collaboration 

is to enable Sanofi to precisely 

identify patient populations with the 

greatest unmet medical needs, and 

determine those populations in which 

drugs are most effective; generate 

real-world comparative effectiveness 

data to support product value 

that meets stakeholder evidence 

requirements; and facilitate the 

development and implementation 

of novel care models to improve 

practice of care, adherence, and 

patient outcomes. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


Alliance Evotec Germany Roche Switzerland N/A Cancer 

Biomarkers 

Partnership 

Metamark 

Genetics 

United 

States 

Alliance Unilabs Denmark Biogen Idec United 

States 

Collaboration 

Skyline 

Diagnostics 

Netherlands 

Collaboration GNS Healthcare United 

States 

Definiens Germany N/A Cancer 

diagnostics 

Janssen 

Pharmaceutica 

(J&J) 

Bristol-Myers 

Squibb 

United 

States 

United 

States 

N/A 

Companion 

diagnostic 

Evotec and Roche will collaborate 

in novel protein-activity based 

biomarkers for Roche’s oncology drugs 

under development. Evotec will use 

its technology to discover proteinphosphorylations 

that predict favorable 

dosage and efficacy of targeted 

cancer drugs in patients. Roche will be 

responsible for conducting clinical trials 

as well as assessing the development 

of companion diagnostics for patient 

stratification. 

Metamark Genetics and Definiens 

partner to develop and commercialize 

cancer diagnostics. Metamark will 

combine its proprietary multiplex 

protein detection platform with 

image analysis technology developed 

jointly with Definiens to deliver 

innovative prognostic and predictive 

assays for early-stage cancers. 

Unilabs will develop a companion 

diagnostic anti-JCV antibody assay 

test, that Biogen Idec will use to help 

it calculate the risk of Tysabri-treated 

patients developing progressive 

multifocal leukoencephalopathy. 

N/A Diagnostics Skyline Diagnostics, a developer and 

marketer of gene signature-based 

diagnostic assays will work with 

Janssen to generate and analyze 

diagnostic information related to 

the use of an undisclosed, marketed 

compound. Skyline will receive R&D 

funding and could receive payments 

on the achievement of certain 

research milestones. 

N/A 



Healthcare analytics firm GNS 

Healthcare will collaborate with 

Bristol-Myers Squibb to support 

personalized medicine. GNS will use 

clinical and molecular information 

from a BMS clinical trial to build 

computer models simulated to 

identify key molecular mechanisms. 

Using patient data comprising genetic 

information, gene expression levels, 

specific blood markers and clinical 

outcomes, researchers will use 

REFS to construct a comprehensive 

inflammation disease model. The 

resulting disease model is intended to 

support virtual clinical trials that will 

simulate the clinical effect of inhibiting 

various drug targets and help predict 

novel molecular targets to combat 

immuno-inflammation that would be 

effective for specific types of patients. 






DEAL TYPE 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY 

DEAL 

VALUE 

(USD M) 

PRINCIPAL 

FOCUS 


License VitaPath Genetics United 

States 

Alere 

United 

States 

Partnership Qiagen Germany Pfizer United 

States 

Partnership 

Collaboration 

Prometheus Labs 

(Nestle) 

Beijing Genomics 

Institute 

United 

States 

Medco Research 

Institute (Medco 

Health Solutions) 

United 

States 

N/A Diagnostics VitaPath Genetics grants Alere 

worldwide marketing rights to 

VitaPath’s spina bifida risk assessment 

assay, which is expected to be 

commercially launched in 2012. 

The assay is a genetic test that 

identifies elevated risk in women of 

childbearing age for the common 

birth defect spina bifida. 

N/A 

N/A 

Companion 

diagnostic 

Companion 

diagnostic 

China Diagenode Belgium N/A Next-gen 


Collaboration Biomonitor Denmark Phadia Sweden N/A Personalized 


Partnership Biocept United 

States 

Clarient (GE 

Healthcare) 

United 

States 

Partnership Qiagen Germany Eli Lilly United 

States 

Qiagen and Pfizer partner to develop 

a companion molecular diagnostic 

test for use with Pfizer’s dacomitinib, 

an oral inhibitor of HER-1, HER-2, 

and HER-4 tyrosine kinases, in global 

clinical development for treatment 

of non-small cell lung cancer. The 

HER (human epidermal growth factor 

receptor) signaling pathway plays a 

role in the complex process of cell 

growth and metastasis. 

Prometheus Laboratories and Medco 

Research Institute announced a deal 

to evaluate Prometheus’ test for 

identifying patients who may benefit 

from a therapy for inflammatory 

bowel disease. 

BGI and Diagenode will collaborate 

in the implementation of standard 

methods in epigenetics and next 

generation sequencing sample 

preparation. Diagenode will provide 

BGI with early access to its new 

developments in chromatin or DNA 

methylation analysis methods and 

tools. 

Phadia and Biomonitor will 

collaborate in the field of 

immunogenicity testing and 

monitoring of patients treated with 

biopharmaceutical drugs, expanding 

Phadia’s personalized medicine 

offerings. 

N/A Diagnostics GE Healthcare’s Clarient will 

commercialize Biocept’s OncoCEE- 

Br blood test for circulating tumor 

cells. Biocept will run the test in its 

CLIA lab and Clarient will analyze the 

results. The companies say the test 

will be the first commercially available 

that includes analysis of a specific 

treatment-associated biomarker. 

N/A 

Companion 

diagnostic 

Qiagen will develop, manufacture, 

and co-commercialize a test to 

identify patients most likel to benefit 

from treatment with Lilly’s JAK2 

inhibitor in development to treat 

blood cancers. 

■ 



AUGUST STATISTICS 

Financings: Secondary Financings Dry Up 

Public market deals slow to a trickle 

Not all was 

gloom and 

doom, however. 

Venture investors 

continued to fund 

the innovative 

companies in the 

sector. During 

a normally slow 

month, U.S. VCbacked 

companies 

raised $551 

million, with 

medical device, 

digital health, 

and diagnostics 

firms scoring the 

majority of the 

funding. 


Life science companies looking 

to raise money in public 

markets found few opportunities 

in August. Just one follow-on 

offering, a $7 million financing 

by immunotherapeutics developer 

Agenus, took place in the 

month. The dearth of offerings 

is not surprising. August will be 

remembered for the wild swings 

in the capital markets as investors 

hung on every political and economic 

move, hoping for the best 

one day and fearing the worst the 

next. Many life sciences stocks 

fell along with every major index 

during the month. Political fights 

in the United States exacerbated 

an already fragile economy where 

businesses, many sitting on cash, 

are waiting to see events play out 

before committing to new hiring 

and expansion. 

Many small- and mid-size life 

sciences companies, however, 

don’t have the cash cushion of 

bigger firms. And while venture 

capital continues to back private 

companies at a fairly steady pace, 

financing venues for public companies 

during volatile markets 

has slowed considerably. 

The Burrill Report data shows 

that total U.S. follow-on offerings 

averaged $575 million per month 

through the first 7 months of 2011. 

That average drops to $504 million 

per month when August is 

included, a 12.3 percent decline. 

PIPES have faired slightly better 

but only $84 million was raised 

through private placements in 

the United States in August, compared 

to an average $142 million 

per month in 2011, 41 percent less 

than the average. 

Life science IPOs have also 

slowed to a trickle with no completed 

offerings in the United 

States in August. Two U.S. companies, 

renewable chemicals developer 

EcoSynthetix and obesity 

control device maker GI Dynamics, 

completed offerings outside 

the country. EcoSynthetix raised 

$101.6 million on the Toronto 

Stock Exchange while GI Dynamics 

went to Australia to raise 

$85 million in an initial offering. 

Although companies continue to 

add themselves to the IPO queue, 

that has also slowed, and the 

prospect for more companies going 

public this year has dimmed. 

Not all was gloom and doom, 

however, with venture investors 

continuing to fund the innovative 

companies in the sector. 

During a normally slow month 

when many people go on holiday, 

U.S. VC-backed companies 

raised $551 million. Unlike most 

months, however, medical device, 

digital health, and diagnostics 

firms scored the majority of 

the funding with $187 million 

raised by medical device firms, 

$138 million by diagnostics developers, 

and $94.5 million by 

digital health firms. Therapeutics 

developers scored $101 million, 

with $31 million going to tools 

and technology companies. 

Prenatal diagnostics startup 

Verinata Health raised $46.5 million 

in a series C financing round 

and expected to add another $2 

million by the end of the month. 

Based in San Carlos, California, 

the diagnostics firm develops 

non-invasive prenatal tests that 

identify fetal chromosomal abnormalities. 

Existing investors 

Mohr Davidow Ventures, Sutter 

Hill Ventures, and Alloy Ventures 

led the financing. Verinata plans 

to use the funding to continue 

the development and subsequent 

commercialization of its prenatal 

test, which it hopes to launch in 

the first quarter of 2012. 

New York City-based ZocDoc 

offers a web-based service that 

allows patients to find nearby 

doctors who accept their insurance, 

and book appointments instantly 

online by tapping into the 

hidden supply of availabilities— 

the 10 to 20 percent of medical 

appointments that are cancelled 

or rescheduled at the last minute. 

The service, used by about 

700,000 people each month to 

find a doctor or dentist, is free for 

patients but charges doctors $250 

per month. ZocDoc raised $50 

million in a series C investment 

from DST Global, an investment 

firm focused exclusively on internet 

companies, which will allow 

the digital health company to 

continue its expansion into new 

markets and grow its base of doctors 

and patients. 

Restoration Robotics raised $41 

million in a series C financing led 

by Clarus Ventures with participation 

by Sutter Hill Ventures, Alloy 

Ventures and Interwest Partners. 

The Mountain View, Californiabased 

medical device firm makes 

a hair restoration system. The 

company will use the funding to 

commercialize its technology and 

launch it in the United States. 

■ 




August 2011 Venture Financings 

COMPANY 

RAISED 

(USD M) 

PRINCIPAL FOCUS FINANCING ROUND INVESTORS 

ZocDoc 50.0 Digital Health Series C DST Global 

Verinata Health 46.5 Diagnostics Series C; add’l $2 million Mohr Davidow Ventures; Sutter Hill Ventures; Alloy Ventures 

due 8/31 

Restoration Robotics 41.0 Medical devices Series C Clarus Ventures; Sutter Hill Ventures; Alloy Ventures; 

Interwest Partners 

Entellus Medical 35.0 Medical devices Coviedien Ventures; SV Life Sciences; Essex Woodlands; Split 

Rock Partners; Greenspring Associates 

Awarepoint 27.0 Digital Health Series F Kleiner Perkins Caufield & Byers; Top tier Capital Partners; 

Cardinal Partners; Venrock; Jafco Ventures 

Vessix Vascular 23.0 Medical devices Series B Edmond de Rothschild Investment Partners; OrbiMed 

Advisors; CDF Dinova Venture Partners; NeoMed 

Management; Chrristopher Weil & Company; Fjord Invest 

T2 Biosystems 23.0 Diagnostics Series D Aisling Capital; Flagship Ventures; Polaris Venture Partners; 

Flybridge Capital Partners; Physic Ventures; Partners 

Healthcare; Arcus Ventures; RA Capital; Camros Capital; WS 

Investments 

Fluidnet 19.8 Medical devices Part of $25 million round 19 investors 

Cleveland HeartLab 18.4 Diagnostics Series B Excel Venture Management; Helath; HealthCare Ventures 

Ellipse Technologies 17.6 Medical devices Series C HBM BioVentures; BioStar Ventures; Wexford Capital; 

MedFocus Funds 

Proteon Therapeutics 15.2 Therapeutics 19 unnamed investors 

Yaupon Therapeutics 14.4 Therapeutics Series D Vivo Ventures; Palo Alto Investors; Burrill & Company; 

Aperture Venture Investors 

Pulmatrix 14.0 Therapeutics Series B1 Polaris Venture Partners; 5AM Ventures; Arch Venture 

Partners; Novartis Venture Fund 

Metabolon 13.1 Diagnostics Series D Keating Capital; Sevin Rosen Funds; Aurora Funds; Harris & 

Harris Group; Syngenta Ventures; Fletcher Spaght; Fulcrum 

Financial Partners 

Ardelyx 12.4 Therapeutics Series B, close of $30 million Amgen Ventures; individual investors 

round 

Intuity Medical 12.0 Medical devices Series D, second tranche Accuitive Medical; Investor Growth Capital; Thomas 

McNerney and Partners; Venrock; Versant Ventures; U.S. 

Venture Partners 

Tandem Diabetes 12.0 Digital Health Part of $13.7 million round Not disclosed 

Care 

NextDocs 10.3 Tools/Technology Series A OpenView Venture Partners 

Foundation Medicine 10.0 Diagnostics Part of planned $20.5 million Third Rock Ventures; private investors 

round 

OncoCyte 10.0 Therapeutics BioTime (majority shareholder); outside investor 

ViewRay 10.0 Medical devices Convertible notes Kearny Venture Partners; OrbiMed Advisors; Fidelity 

Bioscience; Aisling Capital; Siemens; 10 other investors 

Jennerex 8.6 Therapeutics Existing investors 

Cardiox 8.0 Medical devices Series C Early Stage Partners; other investors 

Metabolic Solutions 

Development 

6.8 Therapeutics Existing investors Hopen Life Sciences and Southwest 

Michigan First Life Science Fund plus 21 undisclosed exisitng 

investors 

Quanterix 6.0 Diagnostics Nine investors; previous investors include Bain Capital 

Ventures; Arch Venture Partners; Flagship Ventures 







COMPANY 

RAISED 

(USD M) 


Beacon Endoscopic 5.0 Medical devices Series B MVM Life Science Partners; existing angel investors 

Knome 5.0 Tools/Technology Part of $20 million round Not disclosed 

NeoChord 5.0 Medical devices Part of $11 million round Investors include Clarian Health Ventures; Heron Capital; 

Tgap Ventures 

VeraLight 5.0 Diagnostics Psilos’ CMEA Capital; vSpring Capital; InLight Solutions 

Second Genome 5.0 Tools/Technology Series A Advanced Technology Ventures; Morgenthaler Ventures; 

Wavepoint Ventures; Seraph Group; individual Series Seed 


Asurgen 5.0 Diagnostics Series C, first tranche Not disclosed 

LaserGen 5.0 Tools/Technology Series A Aspen MedTech Ventures III Limited; LBS Technologies; San 

Luis Developments; Houston area angel investors 

Nuclea 

4.7 Diagnostics Cash and equity Not disclosed 

Biotechnologies 

BiO2 Medical 4.6 Medical devices 66 investors 

Embera 

NeuroTherapeutics 

4.5 Therapeutics Series A Louisiana Ventures; Louisiana Fund I; Themelios Ventures; 

private investors 

Lantos Technologies 4.1 Diagnostics Series B Catalyst Health Ventures; Excel Venture Management; Mass 

Medical Angels 

PathoGenetiX 4.0 Tools/Technology Series B, first close of $9.5M 

round 

Excel Venture Management; CB Health Ventures; HealthCare 

Ventures; existing investors 

SironRX Therapeutics 3.4 Therapeutics Series A North Coast Angel Fund; Cleveland Clinic; JumpStart; 

Fletcher Spaght Investments; Glengary; Ohio Tech Angel 

Fund III; Early Stage Partners; X Gen; individual investors 

AliveCor 3.0 Digital Health Series A Burrill & Company; Qualcomm Ventures; Oklahoma Life 

Science Fund 

iScience 

Interventional 

3.0 Medical devices Not disclosed; Investors include Affinity Capital; Asset 

Management; Clarian Health Ventures; De Novo Ventures; 

Johnson & Johnson Development; L Capital Partners; Pacific 

Horizon Ventures; Prism VentureWorks; Three Arch Partners 

Aura Biosciences 3.0 Therapeutics Series B 23 investors; including OXO Capital 

Azumio 2.5 Digital Health Series A Founders Fund; Accel Partners; Felicis Ventures 

Foresight 

2.0 Therapeutics Not disclosed 

Biotherapeutics 

Presage Biosciences 1.6 Tools/Technology Part of $10.5 million round Not disclosed 

Janus 

1.6 Therapeutics Part of $3 million round Six undisclosed investors 


Hygieia 1.5 Medical devices Oakwood Medical Investors 

Mirna Therapeutics 1.5 Therapeutics Existing investors 

Thetis 


1.5 Therapeutics Connecticut Innovations; Stonehenge Partner; angel 


Daktari Diagnostics 1.2 Diagnostics Debt financing Investors include Bill & Melinda Gates Foundation; Hub 

Angels; Launchpad Venture Group; Mass Medical Angels; 

Norwich Ventures; Partners Innovation Fund 

BioMarker Strategies 1.1 Diagnostics Part of $4 million round Not disclosed 

BrainCells 1.0 Therapeutics Part of $14 million round Not disclosed 

Acutus Medical 1.0 Medical devices Seed stage Index Ventures; other investors 







COMPANY 

RAISED 

(USD M) 


Armgo Pharma 1.0 Therapeutics Not disclosed 

Nyx Devices 0.5 Medical devices Not disclosed 

Aciex Therapeutics N/A Therapeutics Minority equity stake Akorn 

Cancer Prevention N/A Therapeutics Bridge funding Not disclosed 


TOTAL U.S. VENTURE FINANCINGS 551.4 

Enobia (Canada) 40.0 Therapeutics Series D Undisclosed pharmaceutical and venture investors 

Asymchem 

Laboratories (China) 

BoneSupport 

(Sweden) 

39.0 Tools/Technology Infinity Group; Tianjin Venture Capital 

26.0 Medical devices Lundbeckfond Ventures; Industrifonden; HealthCap 

Occlutech (Germany) 21.6 Medical devices Interlink Investments (Hong Kong); current investors and 

distribution partners from Japan, Switzerland, Ireland, 

Austria, and Turkey 

Vaxxas (Australia) 16.6 Therapeutics Series A OneVentures; Brandon Capital; Medical Research 

Commercialization Fund; HealthCare Ventures 

Autifony (United 

Kingdom) 

Aleva 

Neurotherapeutics 

(Switzerland) 

KalVista 



Mission Therapeutics 


Biosceptre 

International 

(Australia) 

Spinifex 


(Australia) 

16.5 Therapeutics Series A Imperial Innovations; SV Life Sciences; GlaxoSmithKline 

(company is a spinout of GSK) 

13.8 Medical devices Series A BioMedInvest; BB Biotech Ventures III; Initiative Capital 

Romandie; private invstors 

13.2 Therapeutics Series A Novo A/S; SV Life Sciences 

9.8 Therapeutics Series A Sofinnova Partners; SR One; Roche Venture Fund; (University 

of Cambridge spin-out) 

8.4 Therapeutics Silvercrest Investment Holdings 

7.4 Therapeutics Series B-2, close GBS Venture Partners; Brandon Capital; Uniseed; UniQuest 

UgiChem (Austria) 2.0 Therapeutics BioScience Ventures Group; V+ GmbH & Co; Fonds 3 KG; 

private investors 

Alchemy Healthcare 


0.1 Therapeutics Seed stage North West Fund for Biomedical 

TheraSolv (Belgium) N/A Digital Health Series A Thuja Capital; LRM 

TOTAL NON-U.S. VENTURE FINANCINGS 214.4 

TOTAL AUGUST VENTURE FINANCINGS 765.8 





August 2011 Public Financings 

COMPANY TICKER AMOUNT RAISED 

(USD M) 

PRINCIPAL FOCUS 

IPOS 

EcoSynthetix TSX:ECO 101.6 Renewable chemicals 

GI Dynamics ASX:GID 85.0 Medical devices 

TOTAL U.S. IPOS 186.6 

TOTAL AUGUST IPOS 186.6 

PIPES 

Vivus VVUS 45.8 Obesity; diabetes; ED 

Fibrocell Science OTC:FCSC 22.8 Regenerative medicine 

PositiveID PSID 13.8 Diabetes management 

Vicor Medical OTC.VICO 1.2 Surgical devices 

TOTAL U.S. PIPES 83.6 

Carmat (France) Euronext:ALCAR 41.7 Artificial heart 

Syngis Pharma (Germany) Xetra:LIOK 8.8 Neurology 

Neovasc (Canada) TSX-V:NVC 4.8 Medical devices 

Biohit Oyj (Finland) HSE:BIOBV 2.9 Diagnostics 

Hamilton Thorne (Canada) TSX-V:HTL 2.6 Regenerative medicine 

Lorus Therapeutics (Canada) TSX:LOR 2.2 Cancer 

Innovotech (Canada) TSX:IOT 0.9 Diagnostics 

PharmaGap (Canada) TSX-V:GAP 0.5 Tools/Technology 

Microbix Biosystems (Canada) TSX:MBX 0.4 Virology 

Medifocus (Canada) TSX-V:MFS 0.3 Cancer 

ProMetic Life Sciences (Canada) TSX:PLI 0.2 Cancer 

TOTAL NON-U.S. PIPES 65.3 

TOTAL AUGUST PIPES 148.9 

FOLLOW ONS 

Agenus AGEN 7.0 Cancer; infectious 

TOTAL U.S. FOLLOW ONS 7.0 

TOTAL AUGUST FOLLOW-ONS 7.0 

DEBT 

Thermo Fisher Scientific TMO 2,100.0 Tools/Technology 

Dentsply International XRAY 1,000.0 Medical devices 

TOTAL U.S. DEBT 3,100.0 

TOTAL AUGUST DEBT 3,100.0 






August 2011 Public Financings 

COMPANY TICKER AMOUNT RAISED 

(USD M) 


OTHER FINANCINGS 

Conceptus CPTS 50.0 Medical devices 

NeurogesX NGSX 20.0 Pain management 

Somaxon Pharmaceuticals SOMX 15.0 Neurology 

Furiex Pharmaceuticals FURX 15.0 Drug development 

Unilife UNIS 10.0 Drug delivery systems 

NuPathe PATH 0.5 CNS therapeutics 

HDS International Private 0.5 Algae-to-biofuel 

Uluru ULU 0.1 Specialty pharma 

TOTAL U.S. OTHER FINANCINGS 111.1 

Genfi t (France) Euronext:ALGFT 7.6 Diagnostics 

Evolva (Switzerland) SIX:EVE 0.6 Renal; infectious 

IntelGenx (Canada) TSX-V:IGX 0.5 Drug delivery 

TOTAL NON-U.S. OTHER FINANCINGS 8.7 

TOTAL AUGUST OTHER FINANCINGS 119.8 

■ 




August 2011 Grants and Contracts 

COMPANY 

AMOUNT 

RAISED 

(USD M) 

PRINICPAL FOCUS 

FUNDING AGENCY 

GRANTS 

Siga Technologies 7.7 Antivirals for arenaviruses National Institutes of Health 

HemoShear 4.3 Predictive drug technologies NIH Phase II SBIR 

GeoVax Labs 4.4 HIV/AIDS vaccine program NIAID Integrated Preclinical/Clinical AIDS Vaccine 

Development Grant 

Kineta 2.8 Antivirals NIAID SBIR 

NeoStem 1.7 VSEL Technology to treat osteoporosis DOD Peer Reviewed Medical Research Program 

Inviragen 1.5 Chikungunya virus vaccine Part of NIAID grant awarded to UTMB 

Eureka Genomics 0.8 Anthrax detection Department of Homeland Security 

Stratos Genomics 0.8 Genome sequencing NIH NHGRI 

RxGen; miRagen Therapeutics 0.7 microRNA-based cardiovascular NIH NHLBI SBIR 

Guided Therapeutics 0.5 Cervical scan National Cancer Institute 

Akonni Biosystems 0.3 Biological testing National Institutes of Health 

Madera Biosciences 0.3 Alzheimer’s disease Alzheimer’s Drug Discovery Foundation 

NeoProteomics 0.3 Personalized medicine software National Institutes of Health 

GMS Biotech 0.1 Blood-transfusion chip National Institutes of Health 

Melior Discovery N/A Parkinson’s disease Michael J. Fox Foundation 

Trevena N/A Depression NIH Blueprint Neurotherapeutics Network 

TOTAL U.S. GRANTS 26.2 

Takeda Pharmaceutical (Japan) 310.7 Flu vaccine manufacture Japanese gov’t for vero cell culture vaccine 

EDAP TMS (France) 3.5 Therapeutic ultrasound Fonds Unique Interministerial 

Crossbeta Biosciences 

1.1 Alzheimer’s disease Eurotrans-Bio 

(Netherlands) 

Prolor Biotech (Israel) 1.7 Biobetter proteins Israeli Office of the Chief Scientist 

Medgenics (Israel) 0.9 Drug delivery Israeli Office of the Chief Scientist 

Prana Biotechnology (Australia) N/A Parkinson’s disease Michael J. Fox Foundation 

TOTAL NON-U.S. GRANTS 317.9 

TOTAL AUGUST GRANTS 344.1 

CONTRACTS 

Albany Molecular Research 43.0 Neuromuscular NIH Blueprint Neurotherapeutics Network 

PharmAthene 5.7 Medical countermeasures Department of Defense 

Avaxia Biologics 2.9 GI/radiation exposure HHS BARDA 

ApoCell 2.9 Tumor detection system National Cancer Institute 

Apogee Biotechnology 2.0 GI/radiation exposure HHS BARDA 

Firefly BioWorks 2.0 Microassay validation National Cancer Institute 

Selecta Biosciences N/A Malaria vaccine NIAID/SAIC subcontract 

TOTAL AUGUST CONTRACTS 58.5 

■ 



PIPELINE 

M&A: Two Shots Are Better Than One 

Partners combine development programs to enhance their chance of success 

Genentech and 

Array BioPharma, 

longtime partners 

in oncology drug 

development, feel 

that combining 

their programs can 

maximize their 

chance of success. 

Both biotechs 

are developing 

drugs with similar 

mechanisms 

of action. 


Genentech and Array BioPharma 

teamed up to develop 

their cancer drugs in concert as 

partnering took the lead during a 

slow August for dealmaking activity. 

The companies, longtime 

partners in oncology drug development, 

feel that combining 

their programs can maximize 

their chance of success. Both biotechs 

are developing drugs with 

similar mechanisms of action designed 

to enhance the efficacy of 

some chemotherapeutic agents. 

Although full financial terms 

were not disclosed, the collaboration 

could earn Array as much 

as $713 million, with $25 million 

upfront and as much as $685 million 

in clinical and commercial 

milestone payments, plus up to 

double-digit royalties on sales of 

any resulting drugs. Genentech 

will take responsibility for all 

clinical development and commercialization 

activities. 

The drugs are both ChK-1 inhibitors. 

ChK-1 is a protein kinase 

that regulates the tumor cell’s 

response to DNA damage often 

caused by treatment with chemotherapy 

by blocking cell cycle 

progression in order to allow for 

repair of damaged DNA. This 

limits the efficacy of chemotherapeutic 

agents. The companies 

believe that inhibiting ChK-1 in 

combination with chemotherapy 

can enhance tumor cell death by 

preventing these cells from recovering 

from DNA damage. 

Genentech’s compound, GDC- 

0425, is currently in phase 1 trials. 

Array’s compound, ARRY- 

575, is being prepared for an 

investigational new drug application 

to initiate a phase 1 trial in 

cancer patients. The companies 

have worked together since 2004 

in preclinical cancer drug development 

and currently have one 

drug, GDC-0068, an AKT inhibitor 

in early stage development. 

Two other partners, Celgene 

and Acceleron, are broadening 

their ongoing collaboration with 

a new agreement to develop and 

commercialize ACE-536, a novel 

protein therapeutic to treat anemia 

that is ready for human clinical 

studies. The new agreement 

delivers an upfront payment of 

$25 million to Acceleron and 

makes it eligible for up to an additional 

$217 million in milestone 

payments. 

The companies were already 

working together under a 2008 

drug development agreement to 

develop ACE-011, or sotatercept, 

as a treatment for blood cancers. 

Acceleron received $50 million 

upfront and an equity investment 

by Celgene as part of that 

deal. Sotatercept is currently 

in mid-stage clinical trials in 

patients with chemotherapyinduced 

anemia and in patients 

with end-stage renal disease 

on hemodialysis. The two investigational 

biologics are biochemically 

distinct but both 

work to inhibit members of the 

TGF-beta superfamily involved 

in erythropoiesis, for the treatment 

of anemia. 

Under the new pact, the companies 

will collaborate to develop 

both products and potentially 

others for treating anemia across 

a wide range of indications. They 

will jointly develop and commercialize 

ACE-536, with Acceleron 

responsible for conducting initial 

clinical trials and Celgene taking 

over for later stage clinical 

studies. Acceleron, which will 

pay a share of the development 

expenses through the end of 2012 

after which Celgene will finance 

development, is keeping North 

American co-promotion rights 

and is eligible for tiered doubledigit 

royalties on worldwide net 

sales. As part of their deal, Celgene 

will have an option to future 

Acceleron programs developed 

for anemia. 

■ 




August 2011 M&A 

ACQUIRER COUNTRY TARGET COUNTRY DEAL VALUE (USD M) PRINCIPAL FOCUS 

Fresenius Medical Care Germany Liberty Dialysis Holding United States 1,700.0 Dialysis clinics 

Catalent Pharma 

Solutions 

United States 

Aptuit’s Clinical Trial 

Supplies 

United States 410.0 Supply/Service 

Par Pharmaceuticals United States Anchen Pharmaceuticals United States 410.0 Generics 

Fresenius Medical Care Germany American Access Care 

Holdings 

United States 385.0 Dialysis clinics 

Baxter International United States Baxa Corporation United States 380.0 Pharmacy technology 

Shionogi Japan C&O Pharmaceutical 

Technology 

China 182.0 Pharmaceuticals 

Thoratec United States Levitronix Medical United States 150.0 Medical devices 

Medrad (Bayer 

Healthcare) 

Germany 

Pathway Medical 

Technologies 

United States 125.0 Medical devices 

Sanofi France Universal Medicare unit India 109.0 Nutraceuticals 

Cantel Medical United States Byrne Medical United States 100.0 Medical supplies 

Valeant Pharmaceuticals Canada Afexa Life Sciences Canada 76.0 Respiratory OTC products 

Mingyuan Medicare 

Development 

China Shanghai Yuanqi China 55.0 Diagnostics 

Shanghai Pharmaceutical China Shanhe Pharmaceutical China 53.8 Drug distributor 

Bohai Pharmaceuticals China Yantai Tianzheng 


China 35.0 Traditional Chinese medicine 

Hi-Tech Pharmacal United States Mallinckrodt’s TussiCaps United States 24.1 Respiratory 

Paladin Labs Canada Labopharm Canada 20.5 Specialty pharma 

Lipoxen United Kingdom SymbioTec Germany 14.5 Cancer 

Bluefish Pharmaceuticals Sweden BioPhausia (Medivir) Sweden 6.0 Generics 

Vasomedical United States Life Enhancement 

Technology; Biox 

Instruments 

China 3.2 Medical devices 

Advanced Accelerator 

Applications 

France Dompe’s FabOvar Italy N/A Ovarian cancer 

VWR International United States LabPartner China N/A Supply/Service 

Symmetry Medical United States Olsen Medical United States N/A Surgical devices 

GE Healthcare United States PAA Laboratories Austria N/A Cell culture media 

Sinopharm China Wuhan Zhongliang 


China N/A Pharmaceuticals 

Advent International United States Laboratorio LKM Argentina N/A Specialty pharma 

Vention Medical United States Ansamed Ireland N/A Medical devices 

ConvaTec United States Kinetic Concepts United States N/A Wound care 

EMD Millipore (Merck 

KGaA) 

Germany Amnis Corporation United States N/A Tools/Technology 

Fonterra New Zealand Brahmar Cellulose 

Private 

India N/A Drug ingredients 

■ 




August 2011 Partnering 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

Array BioPharma United States Genentech 

(Roche) 

COUNTRY DEAL TYPE POTENTIAL 

DEAL VALUE 

(USD M) 

Acceleron Pharma United States Celgene United States Joint 

development 

UPFRONT 

PAYMENT 

(USD M) 

Switzerland License 713.0 28 Cancer 

242.0 25 Anemia 


Zymeworks Canada Merck United States License 187.0 N/A Bi-specific antibodies 

Intercept 

United States Servier France Collaboration 163.0 Type-2 diabetes 


NPS Pharmaceuticals United States Amgen United States Agreement 145.0 Endocrine 

Vectura Group United Kingdom Not disclosed United States License 45.0 10 Generic asthma drug 

Galapagos Belgium Genentech 

(Roche) 

Switzerland Collaboration 35.0 Drug discovery 

Vectura Group United Kingdom Sandoz 

(Novartis) 

Switzerland License 11.6 Generic asthma drug 

MolMed Italy GlaxoSmithKline United Kingdom Agreement 7.8 Drug development 

Unigene United States GlaxoSmithKline United Kingdom Agreement 2.2 Drug development 

Vanda 


United States 

Biotoscana 

Farma 

Colombia License N/A Central nervous 

system 

Aciex Therapeutics United States Akorn United States License N/A OTC eye care 

Serum Institute of 

India 

Amarantus 

BioSciences 

India Merck United States Collaboration N/A Pneumococcal 

vaccine 

United States 

Generex 

Biotechnology 

Canada Collaboration N/A Diabetes 

Orion Finland Nycomed Switzerland Collaboration N/A Inhaler for respiratory 

Lab21 United Kingdom Becton, 

United States Collaboration N/A Diagnostic assays 

Dickinson 

Vicor Technologies United States Zoll Medical United States License N/A Medical Technology 

Amyris United States Kuraray Japan Partnership N/A Renewable chemicals 

Amyris United States Total Gas & 

Power USA SAS 

France Joint Venture N/A Renewable fuels and 

chemicals 

NPS Pharmaceuticals United States GlaxoSmithKline United Kingdom Collaboration N/A Calcilytics 

Ariadne Genomics United States Hadasit Israel Partnership N/A Pharmacogenetics 

Lipoxen United Kingdom SynBio Russia Codevelopment 

N/A 


China Medical 

Technologies 


Leica 

Microsystems 

GNS Healthcare United States Bristol-Myers 

Squibb 

Germany Collaboration N/A Diagnostics 

United States Collaboration N/A Personalized 


Enigma Diagnostics United Kingdom GlaxoSmithKline United Kingdom Collaboration N/A N/A Diagnostics 

SG Biofuels United Kingdom Bharat 

Renewable 

Energy 

India Alliance N/A Biofuels 

Solazyme United States Bunge United States Joint Venture N/A Renewable chemicals 

VitaPath Genetics United States Alere United States License N/A Diagnostics 






August 2011 Partnering 

COMPANY/ 

LICENSER 

COUNTRY 

COMPANY/ 

LICENSEE 

COUNTRY DEAL TYPE POTENTIAL 

DEAL VALUE 

(USD M) 

UPFRONT 

PAYMENT 

(USD M) 


LifeCodexx Germany Sequenom United States License N/A N/A Prenatal Diagnostics 

4-Antibody Germany Human Genome United States Collaboration N/A Antibodies 

Sciences 

PnuVax Canada Beijing SL 


China Joint Venture N/A Vaccines 

STRATEC Germany Quanterix United States Partnership N/A Diagnostics 

Qiagen Germany Pfizer United States Partnership N/A Companion 

diagnostic 

Prometheus Labs 

(Nestle) 

United States 

Medco Research 

Institute 

(Medco Health 

Solutions) 

United States Partnership N/A Companion 

diagnostic 

ZeaChem United States Chrysler United States Agreement N/A Biofuels 

Genomatica United States Novamont Italy Joint Venture N/A Bioplastics 

Isconova Sweden Genocea 

Biosciences 

Clinigene 

International (Biocon) 

Beijing Genomics 

Institute 

India 

Spaulding 

Clinical 

Research 

United States Collaboration N/A Infectious disease 

vaccines 

United States Partnership N/A Pharmacology 

services 

China Diagenode Belgium Collaboration N/A Next-gen sequencing 

Biomonitor Denmark Phadia Sweden Collaboration N/A Personalized 

medicine Dx 

Amyris United States Albemarle United States Supply 

agreement 

N/A 

Renewable chemicals 

Biodiversity Italy Becton, 

Dickinson 

Chugai 


United States Partnership N/A Diagnostics 

Japan Roche Switzerland License N/A Cancer 

Cel-Sci Corp United States IDC-GP Pharm South America Marketing 

rights 

Biocept United States Clarient (GE 

Healthcare) 

N/A 

Cancer 

United States Partnership N/A Diagnostics 

Adimab United States Biogen Idec United States Collaboration N/A Antibody technology 

Adimab United States Novo Nordisk Denmark Collaboration N/A Antibody technology 

NovAliX France Galapagos Belgium Collaboration N/A Osteoarthritis 

Bezwada Biomedical United States Orthocon United States License N/A Medical devices 

Lumora United Kingdom 3M United States License N/A Diagnostics 

■ 


PIPELINE 


August 2011 Clinical Trial Results 

DATE COMPANY TICKER DRUG INDICATION RESULT NOTES 

PHASE 3 

8/31/11 Eisai TYO: 4523 Zonegran epilepsy Positive The placebo-controlled study included 207 patients aged 

6-17 years with partial-onset seizures, who were already taking 

1 or 2 anti-epileptic drugs. Participants were randomized to 

receive either Zonegran or placebo in addition to their existing 

medications. The primary endpoint was the proportion of 

patients who responded to therapy by a 50 percent or more 

reduction in seizure frequency after 12 weeks of maintenance 

therapy. The results showed that 50.5 percent of patients 

responded positively to the addition of Zongran, compared with 

a response rate of 31 percent among placebo patients. 

8/29/11 Bristol-Myers 

Squibb, Pfizer 

BMS, PFE Eliquis blood thinner Positive The phase 3 trial evaluated Eliquis compared to warfarin 

for the prevention of stroke or systemic embolism in 18,201 

patients with atrial fibrillation and at least 1 risk factor for stroke. 

Results demonstrated that Eliquis as compared with warfarin 

significantly reduced the risk of stroke or systemic embolism by 

21 percent, major bleeding by 31 percent, and mortality by 11 

percent. 

8/24/11 United 


UTHR 

oral 

treprostinil 

pulmonary 

arterial 

hypertension 

Failed 

Preliminary analysis of the data from the 310-patient, late-stage 

trial demonstrated that the trial did not achieve statistical 

significance for the primary endpoint of a six-minute walk 

distance at week 16. 

8/17/11 Zogenix ZGNX Zohydro chronic pain Positive The phase 3 trial successfully met the primary efficacy endpoint 

of the study in demonstrating Zohydro resulted in significantly 

improved chronic pain relief compared to placebo. The 2 

key secondary endpoints were also met, specifically, the 

proportion of patients with at least 30 percent improvement 

in pain intensity and the improvement of overall satisfaction 

of medication. Additional study endpoints were supportive 

of the efficacy of Zohydro compared to placebo. The study 

demonstrated that Zohydro was safe and well tolerated. 

8/15/11 GlaxoSmithKline, 

Impax 


GSK, IPXL IPX066 Parkinson’s 

disease 

Positive 

The primary endpoint of the double blind crossover study was 

the percentage of off time during waking hours as measured 

by patient diary. Off time is the functional state when patients’ 

medication effect has worn off and there is a return of the motor 

symptoms of Parkinson’s disease. Patients entered the study 

with a baseline off time of 36.1 percent, and at the end of the 

randomized IPX066 treatment phase patients had off time of 

24 percent during waking hours compared to 32.5 percent for 

carbidopa-levodopa plus entacapone. This represents a 33.5 

percent decrease in percent off time for IPX066 from baseline 

versus a 10 percent decrease for carbidopa-levodopa plus 

entacapone. 

8/15/11 Gilead Sciences GILD Quad HIV Positive The late-stage trial of the fi xed-dose, single-tablet Quad 

regimen of elvitegravir, cobicistat, emtricitabine and tenofovir 

disoproxil fumarate, being evaluated for HIV-1 infection in 

treatment-naïve patients, met its primary objective, which was 

non-inferiority at week 48 as compared to Atripla. The primary 

endpoint analysis indicated that 88 percent of patients in the 

Quad arm compared to 84 percent in the Atripla achieved viral 

load of less than 50 copies/mL through week 48. 

8/1/11 Teva 

Pharmaceuticals, 

Active Biotech 

TEVA, 

ACTI 

laquinimod 

multiple 

sclerosis 

Failed 

Results showed that the late-stage study did not achieve its 

primary endpoint of reducing the annualized relapse rate in 

multiple sclerosis. The study was designed to evaluate the 

efficacy, safety and tolerability of oral laquinimod compared to 

placebo and to provide a benefit-risk assessment comparing 

oral laquinimod and a reference arm of injectable Avonex. 



PIPELINE 





PHASE 2 

8/31/11 Pearl 


private 

formoterol 

fumarate 

metered 

dose inhaler 

chronic 

obstructive 

pulmonary 

disease 

Positive 

In the phase 2b, dose-ranging study, the formoterol fumarate 

metered dose inhaler, a long-acting beta-2 agonist, was 

compared to placebo and Foradil Aerolizer in patients with 

moderate-to-severe chronic obstructive pulmonary disease. 

All doses of formoterol fumarate metered dose inhaler tested 

produced highly statistically significant improvements in lung 

function compared to placebo. Dose ordering (incremental 

increase in efficacy with increasing doses) was observed 

across the 3 formoterol fumarate metered dose inhaler doses 

evaluated, and the 2 lower doses tested were comparable to 12 

mcg Foradil, the currently approved dose. 

8/30/11 BioNovo BNVI Menerba menopausal 

hot flashes 

8/29/11 Actelion ATLN macitentan idiopathic 

pulmonary 

fibrosis 

Positive 

Failed 

A total of 35 postmenopausal women with an average age of 

53, who at baseline had on average 73 hot flashes per week, 

were enrolled to the mid-stage trial. The primary endpoint of 

the study was to test the safety of 2 higher doses of Menerba. 

There were no reported cases of serious adverse events, no 

abnormal findings on endometrial biopsies, no abnormal lab 

results, no changes to blood pressure, heart rate or weight and 

no concerning side effects from the treatments. 

Data from the mid-stage study with macitentan in patients 

with idiopathic pulmonary fibrosis did not meet the primary 

endpoint of forced vital capacity. Macitentan did demonstrate 

a promising safety and tolerability profile, with no difference 

being observed between placebo and macitentan with regard 

to liver enzyme elevations. 

8/17/11 Verona Pharma VRP RPL554 asthma Positive The mid-stage trial successfully demonstrated that RPL554 had 

sustained bronchodilator actions throughout the treatment 

period. There was no accumulation of the drug in plasma and 

no safety issues were observed. There was a minor increase in 

heart rate at day 6 only, being the last day of treatment. 

8/10/11 Adolor 

Corporation 

ADLR ADL5945 opioidinduced 

constipation 

Positive 

Statistical significance was achieved for the primary endpoint 

in the 0.25 mg twice daily dose group. The primary endpoint 

of both studies was the change from baseline in the weekly 

average number of spontaneous bowel movements. Statistical 

significance was also achieved in the 0.25 mg twice daily dose 

group for a key secondary endpoint, a responders analysis, with 

a 56 percent response rate for the active arm and a 26 percent 

response rate for the placebo arm of the study. 

8/9/11 Biogen Idec, 

Abbott 

Laboratories 

BIIB, ABT 

daclizumab 

hyp 

relapsingremitting 

multiple 

sclerosis 

Positive 

Results showed that daclizumab HYP, administered 

subcutaneously once every 4 weeks, significantly reduced 

annualized relapse rate by 54 percent in the 150 mg dose arm 

and 50 percent in the 300 mg dose arm compared to placebo 

at 1 year. Daclizumab HYP met key secondary endpoints for 

the 150 mg and 300 mg arms, respectively, providing a highly 

statistically significant reduction in the cumulative number of 

new gadolinium-enhancing lesions between weeks 8 and 24; in 

the number of new or newly enlarging T2 hyperintense lesions 

at 1 year; and in the reduction in the proportion of patients 

who relapsed. Daclizumab HYP also showed a trend toward 

improvement in quality of life measures at 1 year. 



PIPELINE 





8/9/11 Lexicon 


LXRX 

telotristat 

etiprate 

carcinoid 

syndrome 

Positive 

Telotristat etiprate was well tolerated with mild to moderate 

adverse events. 5 telotristat etiprate patients achieved clinical 

responses characterized by reductions of at least 30 percent 

in the number of bowel movements per day for 2 weeks or 

more during the study. 6 telotristat etiprate patients reported 

adequate relief of carcinoid symptoms at the end of the study. 

There were 9 telotristat etiprate patients with a complete 

biochemical response defined as a reduction of at least 50 

percent in urinary 5-HIAA, a biomarker of serotonin synthesis. 

No patients on placebo experienced a clinical response, 

adequate relief of symptoms, or biochemical response during 

the study. 

8/5/11 Genmab GEN ofatumumab relapsed/ 

refractory 

aggressive 

lymphoma 

Positive 

A total of 61 patients with aggressive lymphoma, who had 

persistent or progressive disease after first-line treatment with 

rituximab combined with chemotherapy, were treated in the 

study. The overall response rate was 61 percent. There were 

no unexpected safety findings. The most common grade 3 

or higher adverse events were thrombocytopenia, anemia, 

neutropenia, lymphopenia, leukopenia, febrile neutropenia, and 

hypokalemia. 

8/4/11 Healthpoint 


private HP802-247 venous leg 

ulcers 

Positive 

HP802-247 achieved statistical significance, as compared to 

control plus standard care, in both the primary and secondary 

endpoints. Statistical significance was also achieved for percent 

change from baseline in the target wound area at 10 of the 12 

double-blind evaluation visits, and proportion of complete 

wound closure at 9 of the 12 double-blind evaluation visits. 

HP802-247 appeared to be generally safe and well tolerated, 

with the safety profile of the active groups being similar to 

placebo. 

8/4/11 Novavax NVAX A/H1N1 influenza Positive The mid-stage study data showed that hemagglutinationinhibiting 

antibody responses to the vaccine fulfilled the 

immunogenicity criteria that are required to be considered 

for accelerated approval of seasonal and pandemic influenza 

vaccines by the FDA Center for Biologics Evaluation and 

Research. These criteria were met at all dose levels after a single 

injection, including the lowest dose of 5 micrograms. Single 

administrations of the virus-like particle vaccine induced high 

levels of hemagglutination-inhibiting titers in subjects without 

pre-existing detectable immunity to the pandemic strain. 

Overall, the data indicated that the vaccine was well-tolerated 

and immunogenic. 

8/4/11 Roche RHHBY lebrikizumab asthma Positive In the study, lebrikizumab, a humanized monoclonal antibody 

designed to block interleukin-13 cytokine, resulted in a 

statistically significant increase in FEV1 in adults with asthma 

whose symptoms were inadequately controlled with inhaled 

corticosteriods. The overall frequency of adverse events was 

similar in both the placebo and the treatment group. 

8/4/11 Prolor Biotech PBTH hGH-CTP human growth 

hormone 

Positive 

The mid-stage data show that a single weekly injection of hGH- 

CTP has the potential to replace 7 consecutive daily injections 

of currently marketed human growth hormone. Additionally, 

hGH-CTP demonstrated excellent safety and tolerability in all 

patients across all trial cohorts, with no apparent issues. There 

were no indications that hGH-CTP can induce excessive levels 

of IGF-1 in patients above the normal range when used in high 

doses. 


PIPELINE 





PHASE 1 

8/26/11 Emergent 

BioSolutions 

EBS CPG 7909 anthrax Positive Data from the trial show that BioThrax plus CPG 7909 generated 

peak antibody responses that were 6-fold higher than those 

generated by BioThrax alone. BioThrax plus CPG 7909 also 

accelerated the time to reach the peak immune response 

seen following BioThrax vaccination by 3 weeks. Both of these 

findings were statistically significant. 

8/5/11 Genentech RHHBY GDC-0941 breast cancer Positive In an early-stage study of GDC-0941, focusing on the gene 

P13K, which is abnormal in 20 to 30 percent of advanced breast 

cancer patients, 2 of 97 individuals initially tested experienced 

significant shrinkage of tumors. 

8/1/11 Alnylam 


ALNY ALN-VSP liver cancer Positive In the early-stage study with ALN-VSP, 41 patients were enrolled 

and treated at doses ranging from 0.1 to 1.5 mg/kg. A total of 

209 doses were administered, with a range of 1 to 28 doses 

per patient. Disease control was observed in 13 of 31 evaluable 

patients treated at doses of 0.4 to 1.5 mg/kg. The average 

duration of disease control is approximately 5 months, with 

a range of 2 to 14 months. Currently, 5 patients with disease 

control continue to receive ALN-VSP under an extension 

protocol. 

■ 

Patents Awarded in August 2011 

COMPANY TICKER COMPANY DESCRIPTION GRANTING 

AGENCY 

Cortex 


OTCBB: CORX. 

OB 

A clinical-stage neuroscience 

company focused on the 

discovery, development and 

commercialization of a novel 

class of glutamate modulators 

called Ampakine compounds, 

which have been shown to 

enhance impaired breathing 

by stimulation of AMPA-type 

glutamate receptors in neurons 

in the brain stem 

Angle AIM: AGL Angle is an 83 percent owner of 

Parsortix, a medical diagnostics 

company, which obtained the 

patent. 

U.S. Patent and 

Trademark Office 



PATENT 

NUMBER 

Notice of 

Allowance 

PATENT COVERS 

Patent protects the use of Ampakine 

molecules for the treatment or 

prevention of respiratory depression. 

It broadly covers these molecules, 

including competitor compounds, the 

company said. The patent is licensed 

exclusively to Cortex from the University 

of Alberta. 

7,993,908 The patent covers Parsortix’s proprietary 

devices and methods for isolating 

cells from blood and other mixed cell 

suspensions. The proprietary devices 

and methods can be used for a wide 

range of medical diagnostic applications 

including, for example, the isolation 

of disseminated or circulating tumor 

cells from cancer patient samples and 

the capture of a developing fetus’ cells 

from a sample of its mother’s peripheral 

blood. 



PIPELINE 





AGENCY 

PATENT 

NUMBER 

PATENT COVERS 

Trophos Private A clinical stage pharmaceutical 

company developing 

therapeutics to clinical 

validation for indications 

with under-served needs in 

neurology and cardiology 



7,985,774 The patent provides coverage for the 

use of Trophos’ TRO40303 to reduce 

the cardiac reperfusion injury that 

contributes significantly to the morbidity 

and mortality seen after a heart attack. 

Ohr 


OTCBB: OHRP 

A development-stage 

pharmaceutical company 

focused on drugs for 

underserved therapeutic areas 

in large markets 



7,981,876 The patent includes claims related to 

composition of matter for the lactate 

salt form of Squalamine and its delivery 

using any pharmaceutically acceptable 

carrier. Squalamine is a small molecule 

anti-angiogenic with a novel intracellular 

mechanism of action, that counteracts 

not only Vascular Endothelial Growth 

Factor (VEGF) but also other angiogenic 

growth factors such as Platelet Derived 

Growth Factor (PDGF). 

Suven Life 

Sciences 

NSE: SUVEN 

A biopharmaceutical company 

focused on discovering, 

developing and commercializing 

novel pharmaceutical products, 

which are first in class or best in 

class CNS therapies through the 

use of GPCR targets 

Patent offices 

in Europe, Sri 

Lanka, and Korea 

Europe: 

2155674; Sri 

Lanka: 14612 

and 14833; 

and Korea: 

049868 

The granted claims of the patents 

include the class of selective 5-HT 

compounds discovered by Suven and 

are being developed as therapeutic 

agents and are useful in the treatment 

of cognitive impairment associated 

with neurodegenerative disorders like 

Alzheimer’s disease, Attention deficient 

hyperactivity disorder, Huntington’s 

disease, Parkinson’s disease, and 

Schizophrenia. 

Syndax 


Private 

A clinical-stage epigenetics 

oncology company 

European Patent 

Office 

Notice of 

Allowance 

The patent covers the novel polymorph 

form B of the oral histone deacetylase 

inhibitor, entinostat, which is the specific 

polymorph being developed by Syndax 

for combination therapy with aromatase 

inhibitors for metastatic breast cancer 

and epidermal growth factor receptor 

tyrosine kinase inhibitors for advanced 

non-small cell lung cancer. 

Aegis 


Private 

A drug delivery technology 

company commercializing its 

patented drug delivery and 

drug formulation technologies 

through product-specific 

licenses 



7,998,927 The patent provides broad protection 

for stabilized formulations of GLP-1 

peptide analogs suitable for all routes 

of administration including the noninvasive 

oral or metered nasal spray 

delivery routes, or injection. 

Apricus 

Biosciences 

Nasdaq: APRI 


leveraging the flexibility of 

its drug delivery technology 

to enable multi-route 

administration of new and 

improved compounds across 

numerous therapeutic classes 

Candian Patent 

Office 

2,512,015 The patent covers a form of the 

company’s room temperature 

technology for its Vitaros product for the 

treatment of erectile dysfunction. 



PIPELINE 





AGENCY 

PATENT 

NUMBER 

PATENT COVERS 

MAP 


Nasdaq: MAPP 


focused on developing and 

commercializing new therapies 

to address undermet patient 

needs in neurology 



7,994,197 The patent results from the company’s 

discovery that dihydroergotamine 

(DHE) can be administered to achieve 

pharmacokinetic profiles that result in 

both rapid efficacy and minimal side 

effects. Levadex, which contains the 

company’s proprietary formulation of 

DHE, is an orally inhaled investigational 

drug for the potential acute treatment 

of migraine in adults that targets the 

pharmacokinetic profiles described in 

this patent. 

Agennix 

FRANKFURT: 

AGX; XETRA: 

AGX 


that is focused on the 

development of novel therapies 

that have the potential to 

substantially improve the length 

and quality of life of critically ill 

patients in areas of major unmet 

medical need 

European Patent 

Office 

1,507,554 The patent covers the use of oral human 

lactoferrins, including the company’s 

oral immunotherapy talactoferrin, to 

treat cancer. It also covers the use of 

talactoferrin in combination with other 

therapies, including chemotherapy, 

immunotherapy, radiation therapy and 

other treatments. 

Acorda 


Nasdaq: ACOR 

A biotechnology company 

developing therapies for 

multiple sclerosis, spinal cord 

injury and related nervous 

system disorders 



Notice of 

Allowance 

The claims of the patent application 

relate to methods to improve walking, 

walking speed, lower extremity muscle 

tone and lower extremity muscle 

strength in patients with multiple 

sclerosis (MS) by administering 10 mg 

of sustained release 4-aminopyridine 

(dalfampridine) twice daily. 

Silence 


AIM: SLN 

An RNA interference 

therapeutics company 



Notice of 

Allowance 

The patent covers fundamental 

technology involved in AtuPLEX, one of 

Silence’s proprietary small interfering 

RNA delivery systems. The allowable 

subject matter is directed to AtuFect, 

the specific proprietary lipid that serves 

as the basis for the AtuPLEX delivery 

system. 

Sunesis 


NASDAQ: SNSS 


focused on the development 

and commercialization of 

new oncology therapeutics 

for the treatment of solid and 

hematologic cancers 



7,989,468 The patent covers methods of use for 

the company’s lead drug candidate, 

vosaroxin, at clinically relevant dose 

ranges and schedules, for the treatment 

of leukemia. 

Vermillion NASDAQ: VRML A molecular diagnostics 

company 



Notice of 

Allowance 

The patent claims cover the biomarker 

alpha1beta glycoprotein and biomarker 

combinations that include alpha1beta 

glycoprotein for the diagnosis of 

Peripheral Artery Disease. 

■ 


PIPELINE 


Upcoming PDUFA Dates 

COMPANY TICKER PROPRIETARY NAME ESTABLISHED NAME INDICATION PDUFA DATE 

EUSA Pharma Private Erwinaze L-asparaginase Acute lymphoblastic 

leukemia 

Pacira Pharmaceuticals Nasdaq: PCRX Exparel depobupivacaine Post-surgical pain 

management 



pSivida 

Alimera Sciences 

Nasdaq: PSDV 

Nasdaq: ALIM 

Iluvien fluocinolone acetonide Diabetic macular edema 11/12/2011 

IntelGenX TSX: IGX CPI-300 bupropion 

hydrochloride 

Major depressive disorder 11/13/2011 

Teva Pharmaceuticals 

BioSante Pharmaceuticals 

Nasdaq: TEVA 

Nasdaq: BPAX 

Bio-T-Gel testosterone Male low testosterone levels 11/14/2011 

Regeneron 


Bayer 

Nasdaq: REGN 

OTC: BAYRY 

Eylea VEGF Trap-Eye Wet age-related macular 

degeneration 


EffRx Pharmaceuticals 

Merck 

Private 

NYSE: MRK 

N/A EX101 Osteoporosis 12/5/2011 

Antares Pharma AMEX: AIS Anturol oxybutynin Overactive bladder 12/8/2011 

Amylin; Eli Lilly; Alkermes 

Nasdaq:AMLN; NYSE:LLY: 

Nasdaq:ALKS 

Bydureon 

exenatide extendedrelease 

Type 2 diabetes 1/28/2012 

Protalix BioTherapeutics 

Pfizer 

AMEX: PLX 

NYSE: PFE 

Uplyso taliglucerase alfa Gaucher disease 2/1/2012 

Alexza Nasdaq: ALXA Adasuve loxapine Schizophrenia 

Bipolar disorder 

2/4/2012 

MAP Pharmaceuticals 

Allergan 

Nasdaq: MAPP 

NYSE: AGN 

Levadex dihydroergotamine Migraine 3/26/2012 

Affymax Nasdaq:AFFY Hematide peginesatide Anemia associated with 

chronic kidney disease 

3/27/2012 

■ 

New Drug Approvals in August 2011 

REGION OF APPROVAL COMPANY PROPRIETARY NAME ESTABLISHED NAME INDICATION 

UNITED STATES Pfizer Xalkori crizotinib Non-small cell lung cancer 

Shire Firazyr icatibant Acute Type I and II attacks of hereditary 

angioedema 

Seattle Genetics Adcetris brentuximab vedotin Hodgkin lymphoma and systemic 

anaplastic large cell lymphoma 

Roche, Daiichi Sankyo Zelboraf vemurafenib Untreated BRAF V600 mutation-positive 

metastatic melanoma 

EUROPE Amgen Xgeva denosumab Solid tumor that has spread to the bone 

GSK Benlysta belimumab Lupus 

Biogen Idec Fampyra fampridine Multiple sclerosis 

■ 


INDICES 


AUGUST INDICES 

Burrill Small-, Medium-, and Large-Cap Indices, August 2011 

50 

40 

Burrill Large Cap Index 

Burrill Mid Cap Index 

Burrill Small Cap Index 

30 

20 

10 

Percent change 

0 

-10 

-20 

12/31/10 

1/14/11 

1/28/11 

2/11/11 

2/2511 

3/11/11 

3/2511 

4/22/11 

4/29/11 

5/6/11 

5/13/11 

5/20/11 

5/27/11 

6/03/11 

6/10/11 

6/17/11 

6/24/11 

6/29/11 

7/4/11 

7/9/11 

7/14/11 

7/19/11 

7/24/11 

7/29/11 

8/5/11 

8/12/11 

8/19/11 

8/26/11 

Date 

PERFORMANCE OF INDEX COMPONENTS 

LARGE CAP 

Percent change August 2011 

Index -10.1% 

CBST 1.9% 

AMGN 1.4% 

ISIS -12.7% 

ALR -15.3% 

ILMN -16.4% 

SVNT -38.3% 

HGSI -38.7% 

DNDN -66.6% 

MID-CAP 


Index -3.0% 

VRUS 7.9% 

RGDX -16.4% 

LXRX -18.5% 

ARNA -18.6% 

IMGN -19.6% 

TRGT -20.3% 

SIGA -24.3% 

OSIR -24.8% 

SMALL-CAP 


Index -10.1% 

AZZ 6.1% 

CRTX 3.4% 

AVNR -24.3% 

VICL -24.7% 

AMRI -26.6% 

BIOD -29.9% 

STEM -32.0% 

FOLD -33.6% 


INDICES 


Burrill Biotech Select Index, August 2011 

Percent Change 

25 

20 

15 

10 

5 

0 

-5 

Burrill Select Index 

DJIA 

NASDAQ 

BURRILL BIOTECH 

SELECT INDEX 


Index -4.1% 

ONXX 3.3% 

ALXN 2.1% 

BIIB -7.5% 

EBS -12.1% 

VRTX -12.4% 

ILMN -16.4% 

OSIR -24.8% 

ALNY -25.3% 

-10 

-15 

12/31/10 

1/14/11 

1/28/11 

2/11/11 

2/2511 

3/11/11 

3/2511 

4/22/11 

4/29/11 

5/6/11 

5/13/11 

5/20/11 

5/27/11 

6/03/11 

6/10/11 

6/17/11 

6/24/11 

6/29/11 

7/4/11 

7/9/11 

7/14/11 

7/19/11 

7/24/11 

7/29/11 

8/5/11 

8/10/11 

8/16/11 

8/22/11 

8/28/11 

Burrill Personalized Medicine Index, August 2011 

Date 

20 


15 

10 

5 

0 

-5 

Burrill Personalized Medicine Index 

DJIA 

NASDAQ 

BURRILL PERSONALIZED 

MEDICINE INDEX 


Index -7.2% 

LMNX 13.4% 

APPY 10.6% 

HOLX -10.2% 

VIVO -14.4% 

ALR -15.3% 

RGDX -16.4% 

ILMN -16.4% 

CBMX -20.6% 

-10 

12/31/10 

1/14/11 

1/28/11 

2/11/11 

2/2511 

3/11/11 

3/2511 

4/22/11 

4/29/11 

5/6/11 

5/13/11 

5/20/11 

5/27/11 

6/03/11 

6/10/11 

6/17/11 

6/24/11 

6/29/11 

7/4/11 

7/9/11 

7/14/11 

7/19/11 

7/24/11 

7/29/11 

8/5/11 

8/10/11 

8/16/11 

8/22/11 

8/28/11 

-15 

Date 


INDICES 


Burrill BioGreenTech Index, August 2011 


15 

10 

5 

0 

-5 

Burrill BioGreenTech 

DJIA 

Nasdaq 

BURRILL BIOGREENTECH 

INDEX 


Index -4.1% 

VRNM 93.8% 

PLL 3.2% 

ADM -6.3% 

GRH -6.7% 

RDS.A -8.7% 

DOW -18.4% 

MBLX -20.8% 

SEED -30.0% 

-10 

6/10/11 

6/17/11 

6/24/11 

6/29/11 

7/4/11 

7/9/11 

7/14/11 

7/19/11 

7/24/11 

7/29/11 

8/5/11 

8/10/11 

8/16/11 

8/22/11 

8/28/11 

-15 

12/31/10 

1/14/11 

1/28/11 

2/11/11 

2/2511 

3/11/11 

3/2511 

4/22/11 

4/29/11 

5/6/11 

5/13/11 

5/20/11 

5/27/11 

6/03/11 

Date 

Burrill Diagnostics Index, August 2011 


25 

20 

15 

10 

5 

0 

-5 

Burrill Diagnostics Index 

DJIA 

NASDAQ 

BURRILL DIAGNOSTICS 

INDEX 


Index -4.6% 

ABT 2.4% 

QDEL 2.3% 

DGX -7.3% 

BIO -7.8% 

GHDX -9.0% 

EXAS -9.3% 

SQNM -13.2% 

ALR -15.3% 

-10 

12/31/10 

1/14/11 

1/28/11 

2/11/11 

2/2511 

3/11/11 

3/2511 

4/22/11 

4/29/11 

5/6/11 

5/13/11 

5/20/11 

5/27/11 

6/03/11 

6/10/11 

6/17/11 

6/24/11 

6/29/11 

7/4/11 

7/9/11 

7/14/11 

7/19/11 

7/24/11 

7/29/11 

8/5/11 

8/10/11 

8/16/11 

8/22/11 

8/28/11 

-15 

Date

Personalized Medicine Partnering Deals in 2011 - The Burrill Report

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