Corporate Presentation - InSite Vision
Corporate Presentation - InSite Vision
Corporate Presentation - InSite Vision
Create successful ePaper yourself
Turn your PDF publications into a flip-book with our unique Google optimized e-Paper software.
<strong>Corporate</strong> <strong>Corporate</strong> <strong>Presentation</strong>:<br />
<strong>Presentation</strong>:<br />
Annual Annual Shareholder Shareholder Meeting<br />
Meeting<br />
Tim Ruane, CEO<br />
<strong>InSite</strong> <strong>Vision</strong><br />
May 31, 2012
Our Our Approach: Approach: Novel Novel Products, Products, Reduced<br />
Reduced<br />
Risk, Risk, Efficient Efficient Development<br />
Development<br />
DuraSite ® : Polymer drug delivery technology<br />
• Increases efficacy<br />
• Improves dosing and compliance<br />
Low safety and regulatory risks<br />
• FDA approved technology<br />
• Combine with known agents<br />
Shorter development timelines<br />
• Target indications with high unmet need<br />
• Leverage existing data on platform and products<br />
Reduced development costs<br />
• Streamline clinical trial programs<br />
• Work closely with FDA to define expectations and program<br />
2
Undervalued Undervalued Company Company with with Strong Strong Focus<br />
Focus<br />
and and Investment Investment Fundamentals<br />
Fundamentals<br />
Significant royalties from two partnered commercial products<br />
• Merck<br />
• Bausch + Lomb<br />
Three Phase 3 product candidates advancing<br />
• AzaSite Plus & DexaSite<br />
• BromSite (ISV‐303)<br />
Proven platform technology<br />
Multiple partnering opportunities<br />
• Own global rights to the three Phase 3 products & Phase I/2 product<br />
Strong cash position and oversight of resources<br />
Management team focus on execution<br />
3
<strong>InSite</strong> <strong>InSite</strong> <strong>Vision</strong>’s <strong>Vision</strong> s Pipeline<br />
Pipeline<br />
New IND<br />
Planned<br />
Phase 1/2<br />
On-hold<br />
Phase 3/SPA<br />
Enrolling<br />
Phase 3<br />
Starting Soon<br />
4
AzaSite®<br />
AzaSite in in North North America America Opportunity<br />
Opportunity<br />
Partner: Merck (via 2011 acquisition of Inspire Pharmaceuticals)<br />
• Approved in U.S. & Canada for bacterial conjunctivitis<br />
<strong>InSite</strong> <strong>Vision</strong> receives 25% royalty<br />
• 2012 minimum royalty guarantee: $17M<br />
• Escalating 2013 minimum royalty guarantee<br />
• Includes AzaSite Xtra (ISV‐405; 2% azithromycin<br />
in DuraSite) as “life cycle management” opportunity<br />
2011 Royalties of $13.9M<br />
• Merck intends to grow in bacterial conjunctivitis<br />
Patent protection<br />
• AzaSite: Issued IP to March 2019<br />
• AzaSite Xtra: Issued IP to October 2027<br />
5
Besivance®<br />
Besivance Opportunity<br />
Opportunity<br />
Partner: Bausch + Lomb<br />
Approved in U.S. for bacterial conjunctivitis<br />
• Launched in mid‐2009 in U.S.<br />
• Asia/Latin America/ROW launches in 2011<br />
• Additional filings end‐2011/early 2012<br />
<strong>InSite</strong> <strong>Vision</strong> receives middle single‐digit<br />
royalty on global sales<br />
• 2011 Royalties: $1.2M (vs. $0.5M in 2010)<br />
“Besivance Global Commercialization” ongoing<br />
Patent protection to 2021<br />
6
Blepharitis: Blepharitis:<br />
Acute Acute and/or and/or Chronic<br />
Chronic<br />
Inflammation Inflammation of of the the Eyelids<br />
Eyelids<br />
Signs &<br />
Symptoms<br />
Possible<br />
Causes<br />
Prevalence<br />
Image Source: WebMD.com<br />
• Redness<br />
• Flaking skin<br />
• Crusting<br />
• Cysts<br />
• Irritation<br />
• Bacteria<br />
• Viruses<br />
• Allergy<br />
• Environmental conditions<br />
• Systemic disease<br />
• Gritty sensation<br />
• Itching<br />
• <strong>Vision</strong> impairment<br />
• Discomfort<br />
• Estimated 34 million people in the<br />
U.S. alone<br />
• Widely considered both<br />
under-diagnosed and misdiagnosed<br />
Blepharitis<br />
(also known as Lid Margin Disease)<br />
7
Blepharitis: Blepharitis:<br />
Global Global Landscape Landscape Overview<br />
Overview<br />
No approved agents<br />
Clear market need<br />
• Ophthalmologists see worst patients; many “silent sufferers”<br />
Off‐label prescribing by ophthalmologists<br />
• Scant reimbursement; patients pay out‐of‐pocket<br />
High FDA hurdle: traditional endpoint<br />
• Onerous in chronic settings of inflammation/infection (blepharitis)<br />
Industry “Holy Grail”: front‐of‐eye focus<br />
Classic first‐to‐market opportunity<br />
• Only company in Phase 3 development with SPA in place<br />
8
AzaSite AzaSite Plus<br />
Plus (ISV‐502) (ISV 502) for for Blepharitis<br />
Blepharitis<br />
Intended to rapidly reduce signs and symptoms of acute<br />
and chronic blepharitis<br />
Combines low doses of azithromycin and dexamethasone<br />
with DuraSite to address both infection and inflammation<br />
Administered twice‐daily for 14 days<br />
Safety and superior efficacy established in 2008 Phase 3<br />
clinical trial versus AzaSite<br />
Issued patents to 2019; potential exclusivity to 2031<br />
based on 2011 intellectual property filings<br />
9
DexaSite<br />
DexaSite (ISV‐305) (ISV 305) for for Blepharitis<br />
Blepharitis<br />
Low‐dose dexamethasone in DuraSite<br />
Administered twice‐daily for 14 days<br />
Originally intended to rapidly reduce acute inflammation<br />
in the treatment of non‐microbial blepharitis<br />
Safety and efficacy established in 2008 Phase 3 clinical<br />
trial versus AzaSite Plus<br />
Phase 3 results indicate unanticipated high activity of BID<br />
dexamethasone in DuraSite<br />
• Basis for 2009 intellectual property filings<br />
• Potential exclusivity to 2029<br />
10
Special Special Protocol Protocol Assessment Assessment (SPA) (SPA) Approval:<br />
Approval:<br />
DOUBle<br />
DOUBle Phase Phase 3 3 for for AzaSite AzaSite Plus Plus & & DexaSite<br />
DexaSite<br />
DOUBle<br />
(Dual Ophthalmic agents Used in Blepharitis)<br />
Simultaneous evaluations of AzaSite Plus, AzaSite, DexaSite and<br />
DuraSite (vehicle)<br />
• Valuable data on our two agents ‐ and AzaSite ‐ in one Phase 3 study<br />
Four‐arm Study of 900 Patients Design and Statistical Analysis Plan<br />
AzaSite Plus<br />
N= 300<br />
AzaSite<br />
N= 150<br />
DexaSite<br />
N= 300<br />
DuraSite<br />
N= 150<br />
N= 900<br />
Randomized<br />
Blinded<br />
Powered at 80%<br />
2‐sided Fisher’s Exact Test<br />
11
Special Special Protocol Protocol Assessment Assessment (SPA) (SPA) Approval:<br />
Approval:<br />
DOUBle<br />
DOUBle Phase Phase 3 3 for for AzaSite AzaSite Plus Plus & & DexaSite<br />
DexaSite<br />
DOUBle<br />
(Dual Ophthalmic agents Used in Blepharitis)<br />
SPA Agreed Primary Endpoints: May 2011<br />
Traditional Endpoint<br />
Complete (100%) resolution of all<br />
clinical signs & symptoms (cure)<br />
vs.<br />
vs.<br />
AzaSite Plus<br />
AzaSite<br />
DexaSite<br />
DuraSite (Vehicle)<br />
New Endpoint #1<br />
Time to recurrence:<br />
Patients with complete (100%)<br />
resolution of clinical signs & symptoms<br />
(cure)<br />
vs.<br />
AzaSite Plus<br />
DexaSite<br />
vs.<br />
vs.<br />
Other Endpoints<br />
Improvement in clinical signs &<br />
symptoms: All other patients<br />
Exacerbation of clinical signs &<br />
symptoms: All other patients<br />
AzaSite Plus<br />
AzaSite<br />
DexaSite<br />
DuraSite (Vehicle)<br />
12
DOUBle DOUBle Study Study Execution Execution Update<br />
Update<br />
Update as of May 24 th<br />
• All 45 sites up and running<br />
• 44/45 sites have enrolled at least 1 patient<br />
626 patients (~70%) enrolled since November 16 th FPO<br />
• 95 patients have now completed the study<br />
• 26 patients dropped out early<br />
13
The The <strong>InSite</strong> <strong>InSite</strong> <strong>Vision</strong> <strong>Vision</strong> Opportunity<br />
Opportunity<br />
Classic first‐to‐market advantage<br />
Potential for broad payor strategy, reimbursement, promotion<br />
and use upon approval<br />
SPA discussions/agreement reveal FDA interest in an approved<br />
blepharitis drug<br />
• Well aware of acute vs. chronic disease endpoint issue<br />
• Clearly understand reality of ophthalmology clinical practice<br />
Two <strong>InSite</strong> “pipeline shots on goal”<br />
• AzaSite Plus needs superiority over AzaSite and DexaSite<br />
• DexaSite needs only be superior to DuraSite (vehicle)<br />
Third potential “BD shot on goal” with AzaSite vs. Vehicle<br />
• Potential 1st positive Phase 3 to support AzaSite blepharitis NDA path<br />
• Merck owns no rights to these data<br />
14
AzaSite AzaSite Plus Plus and and DexaSite:<br />
DexaSite<br />
Substantial Substantial Market Market Opportunities<br />
Opportunities<br />
Assuming moderate market penetration and current<br />
pricing of drugs prescribed for blepharitis, possible sales<br />
ranges are:<br />
• AzaSite Plus<br />
• DexaSite<br />
• AzaSite<br />
$500M to $1B<br />
[Based on 10% at $120/prescription]<br />
$200M to $400M<br />
[Based on 5% at $90/prescription]<br />
Reiterated 2011 Guidance for 2012<br />
• Fully accrued in 2H 2012<br />
• Results end‐2012/early 2013<br />
We haven’t examined possible $ effects of a<br />
positive outcome for AzaSite via this study<br />
We believe our blepharitis program will allow us to file<br />
one and, hopefully, two NDAs in the U.S. in 2013<br />
15
BromSite<br />
BromSite (ISV‐303) (ISV 303) for for Reduction Reduction of<br />
of<br />
Inflammation Inflammation and and Pain Pain Post‐Cataract Post Cataract Surgery<br />
Surgery<br />
Lower dose formulation of bromfenac (0.075%) in<br />
DuraSite<br />
• Preclinical data indicate superior performance to bromfenac<br />
IND filed July 2010<br />
Phase 1/2 clinical study completed January 2011<br />
• Four‐arm study evaluating safety and efficacy<br />
Top‐line results reported March 2011<br />
• Final/full results to be presented and/or published in future<br />
Superior PK performance reported October 2011<br />
• Phase 2 PK study vs. Ista’s Bromday – the current market leader<br />
Anticipate exclusivity into 2029 based on 2009 filings<br />
16
R<br />
N=169<br />
Confirm Confirm BromSite<br />
BromSite Regulatory Regulatory Pathway Pathway with with FDA<br />
FDA<br />
Following Following Surprisingly Surprisingly Strong Strong Phase Phase 1/2 1/2 Data<br />
Data<br />
Phase 1/2 design evaluated BromSite administered once‐<br />
and twice‐daily against ISTA’s Xibrom and DuraSite<br />
Primary Endpoint Secondary Endpoints<br />
Absence of cells in anterior chamber of<br />
the eye at Day 15<br />
BromSite (BID)<br />
BromSite (QD)<br />
XiBrom (BID)<br />
DuraSite (BID)<br />
53.3%<br />
19.0%<br />
p=0.0016<br />
Reduction of:<br />
• Flare<br />
• Pain & discomfort from inflammation<br />
• Others<br />
53.3%<br />
42.2%<br />
p=NS<br />
All statistically<br />
significant<br />
superiority @<br />
Days 8, 15 & 29<br />
17
BromSite<br />
BromSite vs. vs. Bromday<br />
Bromday Phase Phase 2 2 PK PK Study<br />
Study<br />
Phase 2 pharmacokinetic study designed to evaluated<br />
BromSite against ISTA’s Bromday (market leader)<br />
R<br />
N=58<br />
• Statistically compared mean aqueous humor concentrations of<br />
bromfenac for the 2 treatment arms<br />
BromSite<br />
(0.075% bromfenac in in DuraSite)<br />
Bromday<br />
(0.09% bromfenac)<br />
Study Design & Analysis<br />
Randomized<br />
Double‐blind<br />
Multi‐center (N=3)<br />
Patients dosed QD<br />
• Day ‐2 & ‐1 pre‐surgery<br />
• Day 0 of surgery<br />
100‐200 µl fluid extracted<br />
HPLC‐MS analysis<br />
18
BromSite<br />
BromSite vs. vs. Bromday<br />
Bromday Phase Phase 2 2 PK PK Study<br />
Study<br />
BromSite achieves >2x the tissue concentration in<br />
the eye vs. Bromday<br />
• Potential benefits in avoiding cystoid macular edema<br />
(CME), a potentially serious adverse event post‐surgery<br />
• Demonstrated superiority over current market leader<br />
Mean concentrations of bromfenac in the aqueous humor<br />
Measured ~3 hours after last dose<br />
R<br />
N=58<br />
BromSite<br />
(0.075% bromfenac in in DuraSite)<br />
Bromday<br />
(0.09% bromfenac)<br />
P=0.0032<br />
19
BromSite<br />
BromSite Summary<br />
Summary<br />
February 2012 pre‐Phase 3 FDA meeting<br />
• Finalized Phase 3 protocols<br />
• Standard endpoints (same as 2010 Phase 1/2 Study)<br />
• BromSite<br />
need only show superiority vs. DuraSite<br />
• 2 Phase 3 Studies (n=240 each)<br />
• Clinical trial material now manufactured<br />
Reiterated 2011 Guidance<br />
• Fully accrued in 2H 2012<br />
• Results end‐2012/early 2013<br />
(vehicle)<br />
We believe our BromSite program will give us an<br />
opportunity to file an additional NDA in 2013<br />
• IND to potential NDA filing: 3 years<br />
• Ideal model for future <strong>InSite</strong> development plans<br />
20
Europe: Europe: Expanding Expanding our our Commercial<br />
Commercial<br />
Footprint<br />
Footprint<br />
February 2012 EU Regulatory Meetings (2 Countries)<br />
• <strong>InSite</strong> blepharitis programs: AzaSite Plus & DexaSite<br />
• Covered 2008 Phase 3 data & ongoing DOUBle<br />
Key Results<br />
study<br />
• U.S. efficacy/safety data sufficient to support EU filings; there is<br />
NO de facto need for EU patient data<br />
• EU/North American formulation differences (preservative levels)<br />
are a non‐issue; we simply need appropriate EU CMC package<br />
• Centralized filing/review procedure should be available to us<br />
We are evaluating opportunities to file in EU first<br />
We believe these principles should apply to AzaSite &<br />
BromSite<br />
21
European European Regulatory Regulatory Pathways: Pathways: Continued<br />
Continued<br />
Q2 2012 EU Regulatory Meetings<br />
• BromSite Phase 3 program: Package submitted; meetings<br />
scheduled<br />
• AzaSite NDA filing pathway: Package submitted; awaiting<br />
scheduled<br />
Key Questions<br />
• Seeking agreement in BromSite<br />
Phase 3 clinical program<br />
• Seeking agreement that AzaSite NDA data sufficient to support EU<br />
filings; no need for additional EU patient data<br />
Informal inputs: AzaSite Xtra as sNDA to AzaSite or full NDA<br />
• CMC issues on EU/North American formulation differences; seek<br />
agreement for appropriate EU CMC package<br />
• Is centralized filing/review procedure available for AzaSite and<br />
BromSite?<br />
22
R&D R&D Pipeline Pipeline –<br />
What’s What s Next?<br />
Next?<br />
ISV‐101: Bromfenac solution in DuraSite<br />
• IND filed and Phase 1/2 study approved; on hold due to pipeline prioritization<br />
AzaSite Xtra (ISV‐405): Azithromycin (2.0%) solution in DuraSite<br />
• “Double strength” AzaSite with issued global IP through end‐2027<br />
• Completed all formulation/stability data and all GLP toxicology work to support<br />
global IND and/or IND‐equivalent filings<br />
ISV‐102: Tetracycline‐based solution in DuraSite<br />
• Completed formulation/stability studies<br />
• Potential therapy for the treatment of ocular infections<br />
ISV‐620: Prostaglandin analog solution in DuraSite<br />
• Completed formulation/stability studies<br />
• Potential therapy for cosmetic eyelash enhancement<br />
ISV‐215: Prostaglandin analog solution in DuraSite<br />
• Completed formulation/stability studies<br />
• Potential therapy for treatment and/or prevention of Glaucoma<br />
23
IP IP Update<br />
Update<br />
November 2011: USPTO definitively rules against UCSF in<br />
AzaSite patent interference<br />
• USCF files appeal end‐2011; we just received basis of appeal filing<br />
• Our case; Merck in monitoring role<br />
Merck continues its prosecution of paragraph IV/ANDA<br />
case vs. Sandoz<br />
• Merck’s case; <strong>InSite</strong> monitoring role<br />
• Likely Q1 2013 trial date<br />
Continued close coordination of AzaSite IP/legal issues by<br />
Merck/<strong>InSite</strong><br />
24
<strong>InSite</strong> <strong>InSite</strong> <strong>Vision</strong>: <strong>Vision</strong>: Strong Strong Fundamentals,<br />
Fundamentals,<br />
Near‐Term Near Term Catalysts, Catalysts, Undervalued Undervalued Opportunity<br />
Opportunity<br />
Two partnered commercial products<br />
• Merck<br />
• Bausch & Lomb<br />
Three Phase 3 product candidates advancing<br />
• AzaSite Plus <br />
• DexaSite <br />
• BromSite <br />
(ISV‐303)<br />
Multiple NDA/registration plans<br />
• North America: AzaSite Plus & DexaSite, and BromSite<br />
• Europe now fully on our radar screen<br />
AzaSite, AzaSite Plus, DexaSite, BromSite<br />
Potential for multiple 2013 NDA filings in U.S. and/or Europe<br />
25
Thank Thank you<br />
www.insitevision.com
Change language