Restoring Sight

Treatment Options

Currently available: Vitamin A

Has not shown consistent benefits in clinical evaluation

In clinical trials:Retinal Prosthesis

Vision limited to few pixels

Light-sensitive genesare extracted

Genes are added to patient’s DNA

1. Algae

Light is shone directly into eyes

Re-grow rods + cones in retina

2. Gene therapy injection

3. Light mediation

5. A Better Life

OptogeneticTreatment for RP

Competitive AnalysisVitamin A Retinal

ProsthesisOptogenetics

Invasiveness Non-invasive Invasive surgery Simple outpatient injection

Efficacy No consistently proven efficacy

Patients see only fuzzy shapes

Genes repaired, photoreceptors regrown

Patient Population Size

Target market size: 10,000 x $60,000 = $600 Million

Pricing Benchmarks

Retinal Prosthesis for RP

$60K-$100K

Ranibizumab for macular degeneration

$48K

Bottom Up Market Size Analysis

US target market size: $540 million

Purchaser PayerUserPatient

$$$Therapeutic Package

Revenue Model

Intellectual Property

Manufacturers

Clinical Trials Core Competency: Research + Development

IP

Data, FDA Approval

Gene TherapySerum

Optical Device

Venture Capital

$$$

Grants

$$$

Partners & Allies

$$$

$$$

$$$

Equity

Key StakeholdersRETINAL SPECIALISTS

Value proposition: increased clinical efficiency

Key activities: •publish papers•sales team•Retinal centers & conferences

PAYERS

Value proposition: increased cost effectiveness

Key activities: •Clinical trial design addresses cost effectiveness outcomes•Hire experience reimbursement VP

RP PATIENTS

Value proposition: Effective treatment

Key activities: •RP communities & foundations•eye clinics & centers•internet & social media

Timeline

Animal Research Preclinical Research

Clinical Trials

Pre-Animal Research

today year 1 year 2 year 3 year 4 year 5

Safety & Tolerability Research

Phase I Phase II Phase III

Market launch

Major Risks

Development Pre-Clinical Clinical Market

TechnologyRegulatory

RegulatoryMarket

Market

Milestone Major Expenses Amount Source

Proven animal efficacy

Licensing / Hiring/ R&D

$3.5MM Philanthropic grantsSBIRAngel fundingVenture funding

Preclinical safety demonstrated

Pre-Clinical studies $4MM Venture FundingPhilanthropic grantsSBIR

Human trials completed

Phase I,II,III trials, regulatory submission

$15MM Venture FundingNIH grants

Positive cashflow Establish sales and distribution force

$3MM Venture Funding

Funding

Major ExpensesPatent and Licensing Costs5 patents * ~40K = ~$200K

Hiring the Initial Team3 yrs * (CEO+CSO) = ~1MM3 yrs * (key hires) = ~1.5MM

Research Lab Upkeep3 yrs * $250K = $750K

Trial Contractors$400K (pre-clinical) + $50K (animal)

Clinical Trials$4000/patient * 200 = $800K

Sales / Marketing50 reps * $50K = 2.5MM

Development

Pre-clinical

Clinical

Initial Core TeamFounders

Scientific Advisory BoardExperts in: Example

Gene therapy Eg. Dr. Richard Mulligan, Harvard Gene Therapy Initiative

Optogenetics Eg. Dr. Ed Boyden, Chief, MIT Synthetic Neurobiology Group

Retinal ophthalmology Eg. Dr. John Pach, Mayo Clinic

CEO

Qualification: Proven biotech leader, led multiple companies from inception to exit

Example: Ralph Kauten, MBA

Chief Scientific Officer

Qualification: Pioneer in field of optogenetics

Example: Dr. Karl Deisseroth, MD, PhD

Key Early HiresVP of Regulatory Affairs

Qualification: Track record of success with FDA PMA and RAC applications

Example: Barbara Niksch, MBA

VP of R&D

Qualification: Expert in development of protein biology systems

Example: Jennie Lill, PhD

VP of Reimbursement

Qualification: Track record of success in reimbursement of ophthalmologic products

Example: Stuart Raetzman

Legal consultant

Qualification: Leading biotech law firm

Example: Wilson Sonsini Goodrich Rosati

Conclusion

Mission: Restoring vision to RP patients who currently lack a solution

Market:US market $700-800MWorld market $2.1-2.4B

Risk:Significant technical and regulatory risk; partially mitigated by risk benefit profile for RP

Future:Platform technology with wide IP coverage, has spectrum of promising therapeutic applications

AcknowledgementsTeaching Team

Professor Tom ByersThomas HaymoreKurt Sandholtz

MentorDr. Jason Davies, UCSF Neuroscience

OphthalmologistsDr. Mark Blumenkranz, Chairman of Stanford OphthalmologyDr. Joyce Liao, Stanford OphthalmologyDr. Theodore Leng, Stanford Ophthalmology

Optogenetic ResearchersDr. Raag Airan, Johns Hopkins RadiologyDr. Polina Anikeeva, Stanford BioengineeringRemy Durand, Stanford Bioengineering

Funding and Business Strategy ExpertsDana Mead, Partner, KPCBDr. Beth Seidenberg, Partner, KPCBOleg Nodelman, Portfolio Manager, Biotechnology Value Fund

Intellectual Property ExpertsGregg Kyle, Senior Associate, Office of Technology LicensingTrevor Roubison, Associate, Office of Technology Licensing

NeurosurgeonsDr. Jamie Henderson, Stanford NeuroscienceDr. Paul Larson, UCSF Neuroscience

Drug Discovery ExpertsDr. Dr. Solow-Cordero, Manager of Stanford High Throughput Bioscience CenterDr. Kevin Grimes, Director of SPARK

Other PhysiciansDr. Homero Rivas, Stanford Bariatric SurgeonDr. Graham Creasey, Stanford Professor of Spinal Cord Medicine

Appendix

Global Market Size

New patient market size

US target market size: $14 million