InVivo Therapeutics

InVivo Therapeutics 

Developing Innovative Products for 

Spinal Cord Injury 


1

Forward-Looking Statements 

Before we begin, we would like to remind everyone that during our 

presentation, we will be making forward-looking statements about our 

business, plans, and objectives. These statements are based on how we 

see things today. These statements can be identified by words such as 

believes, estimates, expects, or similar references to the future, and 

include statements we may make regarding our product development 

strategy, business prospects, and clinical and operational milestones. We wish 

to caution you that actual events or results may differ materially from 

those expressed in forward-looking statements made by us or on our 

behalf. For more information on the many factors that can result in 

actual performance differing from our forward-looking statements, 

please see our filings made with the SEC, including our 2014 Annual 

Report on Form 10-K filed on March 11, 2015 and our Quarterly Reports on 

Form 10-Q filed on May 7, 2015, August 5, 2015, and November 4, 2015. 


2

Company Overview 

• Publicly traded company (NVIV) based in Cambridge, MA 

– Dedicated solely to developing effective therapies for spinal cord injury (SCI) 

• Co-founded by Dr. Robert Langer in 2005 with focus on innovative 

biomaterials for treating spinal cord injury (SCI) 

• Programs in acute and chronic SCI 

– Acute SCI: The Neuro-Spinal Scaffold pilot study converted to INSPIRE study – a 

pivotal study for marketing application 

– Chronic SCI: Bioengineered Neural Trails - neural stem cells delivered in 

biomaterials by a novel, minimally invasive delivery system in pre-clinical research 

• World-class advisory team including 

– Robert Langer, Sc.D. (co-founder, SAB): Holds largest number of medical patents with 

>1,050 patents issued and pending worldwide 

– Richard Roberts, Ph.D. (BOD, SAB): Nobel Laureate in medicine & physiology 

– V. Reggie Edgerton, Ph.D. (SAB): Author of 2014 Brain article showing recovery of 

voluntary motor function in paraplegic patients after electrostimulation 

– James Guest, M.D., Ph.D. (SAB): Professor of neurosurgery pioneering spinal cord cell 

transplantation for the treatment of paralysis 


3

Financial Information 

Trading Symbol 

NVIV 

Stock Price 1 $7.00 

Exchange 

Market Cap 1 

Primary Shares Outstanding 2 

Fully Diluted Shares 2 

NASDAQ 

$191.5 M 

27.0 M 

30.7 M 

Avg. Daily Trading Volume (3 mo) 1 290,365 

Cash on Hand 2 

$22.1 M 

• Financings in May 2014 and Jan 2015 resulted in net proceeds of 

$14.6 M and $11.0 M, respectively 

• Established $50 M ATM with Cowen 

• Q1-Q3 2015 warrant and stock option exercises resulted in net proceeds of $8.8 M 

• Monthly cash burn of about $1.2 M 


1 

As of 1/6/16 

2 

As of 9/30/15 

4

2014 – 2015 Corporate Accomplishments 

1. Experienced leadership team in place 

2. Raised cash to fund 2015-2016 operations 

3. Accelerated Neuro-Spinal Scaffold development program 

4. Implanted 5 patients with the Neuro-Spinal Scaffold in the pilot study 

5. Announced promising clinical results 

6. Converted pilot study to INSPIRE study – a pivotal study for marketing application 

7. Announced Bioengineered Neural Trails program for chronic SCI 

8. Dramatically strengthened and broadened intellectual property portfolio 

9. Major increase in publications and presentations at scientific meetings 

10. Uplisted to NASDAQ 


5

Experienced Leadership Team 

Mark Perrin 

Chief Executive Officer 

and Chairman 

Lorianne Masuoka, M.D. 

Chief Medical Officer 

Tom Ulich, M.D. 

Chief Scientific Officer 

Steven McAllister 

Chief Financial Officer 

William D’Agostino 

SVP, Operations 

Tamara Joseph, JD 

SVP, General Counsel & 

Chief Compliance Officer 

Christopher McNulty 

SVP, Business Development 

& Investor Relations 

Lisa Crockett 

VP, Regulatory Affairs & 

Reimbursement Planning 

Kristin Neff 

VP, Clinical Operations & 

Project Management 

James Blackington 

VP, Head of Human 

Resources 


6

Spinal Cord Injury: 

A Huge Unmet Clinical Need 

• No products today treat SCI 

Causes of Spinal Cord Injury 1 

• Large patient population 

– 12,500 new cases of acute SCI per year in US 1 

– 276,000 currently live with chronic SCI in US 1 

– Only small percentage of patients ever regain 

function 2 

• Direct cost of spinal cord injury 

– Cost of care for the first year post-SCI: 

$340K - $1.0M+ 1 

– Net present value of a quadriplegic injured at 

25 for life: $4.6M+ 1 1. National Spinal Cord Injury Statistical Center, Facts and 

Figures at a Glance. Birmingham, AL: University of 

Alabama at Birmingham, February 2015. 

2. Guidelines for the conduct of clinical trials for spinal 

cord injury as developed by the ICCP panel: 

spontaneous recovery after spinal cord injury and 

statistical power needed for therapeutic clinical trials. 


9.2% 11.4% Automobile 

Falls 

36.5% 

Violence 

14.3% 

28.5% 

Spinal cord (2007). 

Sports 

Other/unknown 

7

InVivo’s Mission 

To Redefine the Life of the SCI Patient 

• Motor → recover muscle control, movement, strength 

• Sensory → avoid injuries, such as bed sores 

• Autonomic → recover bowel/bladder control and sexual 

function 

• Quality of life → reduce disabling pain, including neuropathic 

pain, and improve self-care 


8

Neuro-Spinal Scaffold 

for Acute SCI 

Designed to Promote Healing in 

Spinal Cord Injury 


9

Spinal Cord Injury Includes Both Primary 

Traumatic Injury and Secondary Injury 

Primary Spinal Cord Contusion Injury 

Sequelae of the Injury 

time 

Hemorrhage 

Fractured 

Vertebral Body 

Spinal Cord 

Injury 

Spinal cord 

swelling 

Reduced 

blood flow 

Appearance of 

tissue void 

(acute cavity) 

Ischemic 

necrosis 

Cyst 

development 

White matter 

reduction 

Chronic injury 

and mature 

cavity formation 


10

Progression of Acute SCI to Post-Traumatic 

Cysts in Rat Contusion Model 

Normal 2 hours after SCI 24 hours after SCI 

Highly vascularized gray matter 

White matter 

Acute hemorrhage & necrosis 

Liquefactive necrosis 

1 week after SCI 4 weeks after SCI 12 weeks after SCI 

Microcystic degeneration 

Cystic cavitation 


Mature cystic cavity 

Poster D8-06; National Neurotrauma 

Society 2015 Symposium; Santa Fe, NM. 

11

InVivo’s Pioneering Clinical Approach for Acute SCI: 

The Neuro-Spinal Scaffold 

1) Innovative surgical approach for Neuro-Spinal Scaffold 

implantation 

– Decompressive myelotomy relieves spinal cord tissue pressure with 

release of liquefied, necrotic tissue resulting in a cavity 

– Mild irrigation and debridement further defines the acute cavity 

2) Neuro-Spinal Scaffold 

– Fills tissue void (cavity) 

– Provides structural support to surrounding viable tissue 

– Serves as a locus for appositional healing 

– Physical substrate for neural regeneration 

– Preserves macroscopic spinal cord architecture 


12

Acute Cavitation Within the Spinal Cord Allows 

For Neuro-Spinal Scaffold Implantation 

Porcine Acute Spinal Cord Contusion Injury Shows 

Presence of Cavity Following Mild Irrigation 

4 hrs post-injury 6 hrs post-injury 24 hrs post-injury 

Neuro-Spinal Scaffold implantation procedure reduces elevated spinal cord tissue pressure 

following release of liquefied, necrotic tissue. 


Guest et al., manuscript submitted 

13

First-in-Human Surgery Demonstrates Ease of 

Neuro-Spinal Scaffold Implantation Within Acute Cavity 

Intraoperative Image 

Showing Acute Cavity 

Intraoperative Ultrasound 

Showing Neuro-Spinal Scaffold 

Implantation performed by Dr. Nicholas Theodore at the 

Barrow Neurological Institute (Phoenix, AZ) in October 2014 


14

Neuro-Spinal Scaffold Provides Structural 

Support to Surrounding Viable Tissue 

A window of opportunity exists when the white matter is 

still viable and can be preserved 

Irrigation Pipette 


Midline Dorsal Pial Incision 

& Gentle Irrigation 

Placement of Neuro-Spinal Scaffold in Rat Contusion 


Poster D8-06; National Neurotrauma 

Society 2015 Symposium; Santa Fe, NM. 

15

Neuro-Spinal Scaffold – Designed to Act as a 

Physical Substrate to Promote Neural Repair 

• Highly porous biopolymer Neuro-Spinal Scaffold 

• Composition: 

– PLGA is the inert biodegradable skeleton along which cells can grow 

– Poly-L-Lysine promotes cellular adhesion 


16

The Neuro-Spinal Scaffold Facilitates 

Appositional Healing and Nerve Sprouting 

+ 

+ 

- - - - 

+ 

+ 

Cell 

Poly-Lysine 

- - - - - - - - - - 

+ 

+ + 

PLGA 

Neuro-Spinal Scaffold designed to promote: 

3D appositional healing, similar to 

a butterfly bandage 

A permissive environment for 

neuronal sprouting and regeneration 

Butterfly Bandage 


2D Wound Healing 

Internal 3D Wound Healing 


17

Primate Hemisection Model Used to Demonstrate 

Appositional Healing and Nerve Sprouting 

Primate Injury Model 

Hemisection 

Winner of 2011 Apple Award 

granted by American Spinal Injury 

Association for the best published 

paper in the SCI rehabilitation 

literature 


Pritchard CD et al., Neuroscience Methods (2010) 

18

Neuro-Spinal Scaffold Increases Functional 

Tissue Volume by Appositional Healing 

Remodeled tissue 

volume (mm 3 ) 

25 

20 

* 

15 

10 

5 

Remodeled tissue 

by appositional 

healing 

0 

Control 

Scaffold 

12 weeks after primate 

hemisection injury 


Slotkin JR et al., manuscript submitted 

19

Neuropermissive Neuro-Spinal Scaffold Supports 

Creation of Detour Circuits By Collateral Sprouting 

Myelin Basic Protein 

(MBP) stained axons in 

remodeled tissue 

MBP positive fibers appear as dots (in 

cross section) and fibers (arrows) 



20

The Neuro-Spinal Scaffold Improved 

Functional Recovery in Primates 

Kinematics score 

4 

Normal gait score 

0 

(n=8) 

-4 

-8 

(n=6) 

(n=6) 

* 

-12 

Control 

Scaffold 

Kinematics score is an objective, video-based composite of hindlimb 

stepping patterns that eliminates subjective observer scoring of gait. 

Based on results of primates with large (complete hemisection) lesions 



21

The Neuro-Spinal Scaffold Acts as a Physical Substrate 

to Preserve Macroscopic Spinal Cord Architecture 

Remodeled Tissue 

Volume (mm 3 ) 

Rat Acute Spinal Cord Contusion Injury 

Control 

Scaffold-Treated 

Control 

Cyst 

Scaffold 

Cavity Volume (mm 3 ) 

6 

4 

2 

0 

Cyst Reduction White Matter Sparing Remodeled Tissue 

* 

Control Scaffold 

White Matter Width (mm) 

0.6 

* 

* 

2.0 

1.5 

0.4 

1.0 

0.2 

0.5 

0.0 

0.0 



*P

Neuro-Spinal Scaffold for 

Acute SCI 

Clinical Translation 


23

Humanitarian Device Exemption (HDE) is an 

Accelerated Approval Path 

• HDE Path to approval 

– Simpler and faster path to approval (safety and probable benefit) 

– Indicated population must be fewer than 4,000 patients in the US 

• Thoracic and cervical SCI patients with complete paralysis (AIS A)* 

• Probable benefit 

– “The device will not expose patients to an unreasonable or significant risk 

of illness or injury and the probable benefit to health from the use of the 

device outweighs the risk of injury or illness from its use, taking into 

account the probable risks and benefits of currently available devices or 

alternative forms of treatment.” -FD&C Act Section 520(m) 

*Excludes penetrating injuries such as gunshot or knife injuries 


24

The INSPIRE Study 

InVivo Study of Probable Benefit of the Neuro-Spinal Scaffold for Safety and 

Neurologic Recovery in Subjects with Complete Thoracic AIS A Spinal Cord Injury 

• Designed as a single 20-patient clinical study to be used for HDE application 

– FDA approved protocol amendment to convert pilot study to a pivotal probable benefit study 

– Safety and neurologic outcome data from pilot study patients included in the 20 

– Currently approved to enroll up to 12 patients 

• Approval for 20 patients expected following submission of 6-month safety data for first five patients 

• Primary Objective – to evaluate whether the Neuro-Spinal Scaffold is safe and 

demonstrates probable benefit for the treatment of patients with complete T2-T12/L1 SCI 

• Primary Endpoint – proportion of patients that improve by at least one ASIA Impairment 

Scale (AIS) grade at 6 months post-implant 

• Additional Endpoints: ISNCSCI sensory and motor scores, bladder and bowel function, 

Spinal Cord Independence Measure (SCIM III), pain, Quality of Life and MRI measures 

• Total number of allowed US clinical sites increased to 40 

– Plan to also include Canada and United Kingdom clinical sites in 2016 


25

Objective Performance Criterion 

• An objective performance criterion (OPC) to support probable benefit 

based on AIS conversion rates is under discussion with the FDA 

• Large natural history databases provide historical benchmarks for AIS 

conversion rates in patients with complete (AIS A) thoracic injury 

– European Multicenter Study about Spinal Cord Injury (EMSCI) 

– Spinal Cord Injury Model System (US database) 

– Sygen clinical trial in spinal cord injury 

• An additional protocol amendment may be required to establish the OPC 

Historical Benchmarks for Complete (AIS A) Thoracic SCI AIS Conversions 

EMSCI 1 

15.6% 

(6 months) 

Spinal Cord Injury Model 

Systems 2 Sygen 3 

15.5% 

(12 months) 

12.9% 

(6 months) 


1 

Zariffa et al., Spinal Cord (2011) 

2 

Lee et al., J. Spinal Cord Med. (2014) 

3 

Fawcett et al., Spinal Cord (2007) 

26

Clinical Sites 

• Banner University Medical Center Tucson (Dr. T. Dumont) 

• Barnes-Jewish Hospital at Washington University Medical Center (Dr. P. Santiago) 

• Barrow Neurological Institute (Dr. N. Theodore)* 

• Carolina Neurosurgery & Spine Associates (Dr. D. Coric & Dr. B. Bockenek)* 

• Cooper Neurological Institute (Dr. S. Yocom) 

• Goodman Campbell Brain and Spine / Indiana University Neuroscience Center (Dr. E. Horn) 

• Keck Hospital of University of Southern California (Dr. P. Hsieh)* 

• Medical College of Wisconsin (Dr. S. Kurpad) 

• Mount Sinai Hospital, New York, NY (Dr. A. Jenkins) 

• Oregon Health & Science University (Dr. A. Raslan) 

• Rutgers New Jersey Medical School (Dr. R. Heary) 

• University of California, Davis (Dr. K. Kim)* 

• University of Kansas Medical Center (Dr. P. Arnold) 

• University of Pittsburgh Medical Center Presbyterian (Dr. D. Okonkwo) 


* Sites that have implanted Neuro-Spinal Scaffold 

27

Promising Neurologic Outcomes and Favorable Safety 

Profile in Ongoing Neuro-Spinal Scaffold Pilot Study 

Patient 

Date of 

Implantation 

Neurologic Level 

of Injury 

Neurologic Outcome to Date 

1 Oct. 2014 T11 

2 Jan. 2015 T7 

3 June 2015 T4 

Converted to AIS C at Month 1 with substantial 

ongoing lower limb motor and sensory 

improvement through Month 12 

Remains AIS A but with marked bowel and 

bladder improvement through Month 12 

Converted to AIS B at Month 1 with additional 

sensory improvement (from mid-chest to midabdomen) 

through Month 6 

4 Aug. 2015 T3 Remains AIS A at Month 3 

5 Sept. 2015 T8 Remains AIS A at Month 3 

As previously communicated, with the exception of dramatically positive or negative results, the Company 

expects to communicate any interim information according to industry standards. The Company does not 

consider a patient’s unchanged AIS classification or a medically insignificant adverse event to be material. 


28

Marked Functional Improvement in First Patient 

Implanted with Neuro-Spinal Scaffold 

• Prior to surgery, the patient had a complete AIS A spinal cord injury at 

T11/T12 with no motor or sensory function below the level of injury 

• Improved from AIS A to AIS C at one-month exam 

– Fewer than 5% of AIS A patients with a T10-T12 injury progress to AIS C or D at 

one month and fewer than 14% at 48 weeks 1 

• Return of motor function in the hip and palpable contraction for knee 

extensor muscles at three-month exam 

• Return of sensation to two dermatomes extending down to top of legs and 

sacral region at three-month exam 

• Knee motor function continued to improve at six month exam 

• Bilateral contractions at ankles at one year exam 

• Regained bowel function and improved bladder function 


1 

Zariffa, J., et al., "Characterization of neurological recovery following 

traumatic sensorimotor complete thoracic spinal cord injury." Spinal 

Cord 49.3 (2010): 463-471 

ASIA = American Spinal Injury Association; AIS = ASIA Impairment Scale 

29

Significant Neurological Improvement in Third 

Patient Implanted with Neuro-Spinal Scaffold 

• Prior to surgery, the patient had a high (T4/mid-chest) thoracic complete 

AIS A injury with no motor or sensory function below the level of injury. 

• At one-month exam, improved from a complete AIS A SCI to an 

incomplete AIS B SCI having regained sacral sensation; bladder function 

also improved 

– Fewer than 4% of patients with a high thoracic neurological level of injury convert 

from AIS A to AIS B in one month and fewer than 10% at 48 weeks. 1 

• At three-month exam, regained partial sensation from mid-chest level to 

mid-abdominal level 


1 

Zariffa, J., et al., "Characterization of neurological recovery 

following traumatic sensorimotor complete thoracic spinal cord 

injury." Spinal Cord 49.3 (2010): 463-471 

30

Future Clinical Development Strategy 

• Path to Commercialization for Acute Thoracic SCI 

– Target completion of study (enrollment and follow-up) and submission 

of HDE application in 2017 

• New Clinical Programs in Expanded Populations 

– Acute Cervical Spinal Cord Injuries (projected study initiation mid-2016) 

• InVivo can pursue rapid, streamlined program via HDE approval 

– Premarket Approval (PMA) path 

• Ability to expand to entire acute SCI population (complete and incomplete 

thoracic and cervical injuries) 

• Expedited Access Pathway (EAP) may be an option for devices to treat SCI 

» Similar pathway to Breakthrough Therapy Designation for drugs to reduce time to 

approval 

» Two-phase study allows for PMA approval based on pre-specified criterion and 

remaining confirmatory information to be obtained post-approval 


31

InVivo’s Chronic SCI Product: 

Bioengineered Neural Trails 

Neural Stem Cells Incorporated into an 

Injectable Scaffold for Minimallyinvasive 

Delivery 


32

Chronic Spinal Cord Injury: 

An Enormous Unmet Clinical Need 

Transforming Neural Stem Cell Transplant to 

Implanting Bioengineered Neural Trails 

• 276,000 currently live with SCI in US 1 

• Therapeutic options are limited 

• Neural stem cell transplantation 

offers therapeutic promise 


1. National Spinal Cord Injury Statistical Center, Facts 

and Figures at a Glance. Birmingham, AL: University of 

Alabama at Birmingham, February 2015. 

33

Bioengineered Neural Trails: InVivo’s Novel 

Neural Stem Cell Product for Chronic SCI 

I. Surgery 

– A minimally invasive surgical approach using an innovative, patented 

method and device to create Bioengineered Neural Trails 

II. Biomaterial 

– Injectable scaffold composed of a soft gel improves delivery, viability 

and differentiation of transplanted Neural Stem Cells (NSCs) 

III. Neural Stem Cells 

– Injection of NSCs with an in vivo differentiation profile that supports 

the therapeutic mechanism of action 

IV. Therapeutic Goal 

– Bioengineered Neural Trails bridge the site of spinal cord injury to 

create neuronal detour circuits 

– Activation of the lumbar central pattern generator (CPG) 


34

I. Novel Surgical Approach: Minimally Invasive Injectable 

Scaffold to Create Bioengineered Neural Trails 

Lacks initial connectivity 

across lesion 

Establishes immediate 

connectivity across 

the lesion 


35

InVivo’s Intellectual Property Portfolio Supports 

the Creation of Bioengineered Neural Trails 

• Method and System for Cellular Transplantation in the Spinal 

Cord (US 7,666,177) 

– Dr. James Guest, University of Miami, Miller School of Medicine, and the 

Miami Project to Cure Paralysis 

• Spinal Multisegmental Cell and Drug Delivery System 

(US 9,011,410) 

– Dr. Martin Marsala, University of California, San Diego 

• Methods and Systems for Delivery of a Trail of a Therapeutic 

Substance (Provisional Patent Application) 

– InVivo Therapeutics 


36

Trail Injection Provides Many Advantages Over 

Bolus Injection 

Bolus approach with multiple injection sites prone to reflux and sub-optimal cell 

distribution without longitudinal connectivity 


12X Speed 

Trail approach employs a single injection site, results in homogeneous cellular 

suspension, no reflux, and immediate longitudinal connectivity 

37

A Novel Patented Surgical Device for Creation of 


Instrumentation 

Syringe 

Pump 

Disposable 

tubing, guide 

needle and 

injection 

needle 

Tubing 

Pre-filled syringe 

with NSCs in soft gel 

Complete product 

Stepper Motor 

Guide Needle 

y z y z 

x 

x 

Injection 

Needle 

Stereotaxic Positioning 

Mechanism 


38

Neurosurgeons Successfully Test the Injection 

Device in the Clinically Relevant Porcine Model 

C-arm fluoroscope 

Dr. James Guest and Dr. Christoph Hofstetter perform the first ever porcine 

surgery to create a longitudinal trail of neural stem cells 


39

Fluoroscopy and Ultrasound Guide the Safe 

Extension and Retraction of the Injection Needle 

CSF 

Central canal 

Curved 

guide 

needle 

Intrathecal contrast 

Myelogram (contrast agent in thecal 

space) makes cord easy to visualize. 

Ultrasound does not show any pathology after trail 

injection. Doppler mode shows blood vessels. 


40

MRI Demonstrates a Longitudinal Trail 

Traversing White and Grey Matter 

Rostral 

Caudal 

~1 cm 

~1 mm 

• The depicted 4 cm trail is a clinically 

relevant length to traverse a typical 

spinal cord injury 

• The NSC trail begins in the dorsal white 

matter (~T10), traverses the central grey 

matter, and terminates in ventral white 

matter (~T12) 

• Trail created in 1.3 minutes 


41

II. A Biomaterial Formulation Serves as the 

Injectable Scaffold to Deliver NSCs 

• The injectable scaffold is a soft biomaterial to improve the 

delivery, viability and differentiation of transplanted Neural 

Stem Cells (NSCs) 

• The biomaterial can serve as a scaffold for NSC attachment 

• The biomaterial serves as a spacer for cell maturation and 

axonal extension 

• The biomaterial serves to maintain homogeneous cell 

suspension during shipment, during the injection process, and 

within the cell trail 

• The biomaterial is easily injectable through a small gauge 

needle, but exhibits a gel-like property once within the trail 


42

III. Neural Stem Cells Create a Bioengineered 

Neural Trail In Vitro 

Neural plexus 

Axonal outgrowth from trail into matrix 

Immunofluorescence for a neuronal marker (MAP2) 


43

IV. Therapeutic Goal: a Defined Mechanism of 

Action to Improve Neurological Function 

• Creation of neuronal detour circuits across the site of 

spinal cord injury enables cerebrally-driven functional 

recovery 

• Re-activation of distal spinal circuits and central pattern 

generators (CPG) 

Endogenous Neurons 

Chronic spinal cord injury showing 

loss of connection with intact 

spinal circuits of the CPG below 

the level of the injury. 


Transplanted Neurons 


enable the creation of neuronal 

detour circuits and activate caudal 

spinal circuits and the CPG 

44

Next Steps for Bioengineered Neural Trails 

• Optimize all aspects of product profile in preparation for IND: 

instrumentation, biomaterial, and NSCs 

• Strengthen and broaden intellectual property portfolio 

• Aim to partner with a stem cell company to accelerate project 

timelines 

• Target pre-IND meeting with the FDA by the end of 2016 


45

Neuro-Spinal Scaffold and 


Commercial Opportunities, IP, and 

Summary 


46

Neuro-Spinal Scaffold and Bioengineered Neural Trails 

Commercial Opportunities 

• Cost of spinal cord injury 

– Cost of care for the first year post-SCI: $340 k - $1.0 M+ 1 

– Net present value to maintain a quadriplegic injured at 25 for life: $4.7 M+ 1 

• US commercial opportunity 

– Majority of acute SCIs treated in Level I trauma centers (203 in US) 

– Feasible to vertically integrate with a focused commercial organization 

Spinal Cord Injury Type 

US Incidence/ 

Prevalence 1,2 US Market 2 

Complete (AIS A); non-penetrating 3 3,750/yr $200 - $600 M/yr 

All acute SCI 12,500/yr $600 M - $1.8 B/yr 

Chronic SCI 276,000 $>10 B 


1. National Spinal Cord Injury Statistical 

Center, Facts and Figures at a Glance. 

Birmingham, AL: University of Alabama at 

Birmingham, February 2015. 

2. Company estimates 

3. Subset of SCI patients in HUD application 

47

Dramatically Strengthened and Broadened 

Intellectual Property Portfolio 

• Core technology covering Neuro-Spinal Scaffold licensed from MIT 

and Boston Children’s Hospital 

– US composition and methods patent 8,858,966, expires 2027 

• Japanese patent issued; European patent application in prosecution 

– Broader US composition patent 9,101,695, expires 2027 

– Co-inventors: Drs. Robert Langer, Rajiv Saigal, and Yang Teng 

• Exclusive agreements with UC San Diego and Dr. James Guest cover 

delivery methods and devices for Bioengineered Neural Trails 

– UC San Diego exclusive license – US device and methods patent 9,011,410, 

expires 2031 

– Dr. Guest assignment – US methods patent 7,666,177, expires 2024 

• Additional patents and applications held by InVivo 

– Packaging, Instrumentation and Manufacturing patents are pending for 


– Additional intellectual property filed for Bioengineered Neural Trails 


48

Increased Publications and Presentations at 

Major Scientific Meetings 

• Presentations to date 

– 83rd American Association of Neurological Surgeons (AANS) Annual 

Scientific Meeting 

• James Guest, M.D., Ph.D et al., presented Reduction of Intraparenchymal 

Pressure after Porcine Acute Spinal Cord Contusion Injury by Neuro-Spinal 

Scaffold Implantation 

• Alexander Ropper, M.D. et al., presented First Human Implantation of a 

Polymer Scaffold for Traumatic Spinal Cord Injury: A Clinical Pilot Study for 

Safety and Feasibility 

– 2015 Annual Symposium of the National Neurotrauma Society (NNS) 

– 17th Spinal Research Network Meeting 

– 2015 Tissue Engineering & Regenerative Medicine International Society 

(TERMIS) World Congress 

– 2015 Annual Meeting of the Congress of Neurological Surgeons (CNS) 

• Numerous publications in process 


49

Major 2015-2016 Achievements 

• Achievements 

– Organizational 

• Senior leadership team in place to drive maximum value of company’s assets 

– Scientific & Clinical 

• The INSPIRE Study – received FDA approval to convert pilot study into pivotal 

probable benefit study 

• First three of five patients implanted with Neuro-Spinal Scaffold have shown 

significant improvement 

» Patient 1: improved from AIS A to C in one month 

» Patient 2: marked improvement in bowel and bladder function 

» Patient 3: improved from AIS A to B in one month 

• Major increase in publications and presentations at major scientific meetings 

– Intellectual Property 

• Granted two major US patents covering Neuro-Spinal Scaffold 

• Entered into agreements with UCSD and Dr. James Guest to establish intellectual 

property foundation for Bioengineered Neural Trails 

– Financial 

• Raised $34.4M (net) in two financings, warrant and stock option exercises 

• Established $50 M ATM with Cowen & Company 


50

Upcoming Anticipated Milestones 

• Continue enrollment of patients into INSPIRE pivotal probable 

benefit study and provide patient updates as appropriate 

• Establish Objective Performance Criteria (OPC) for INSPIRE 

• Expand INSPIRE to United Kingdom and Canada 

• Complete enrollment in INSPIRE 

– Targeting HDE submission in 2017 

• Initiate acute cervical spinal cord injury study 

• Target pre-IND meeting on Bioengineered Neural Trails by 

end of 2016 


51

InVivo Therapeutics

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