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