Participants:

Trex Topping, MDOphtahlmic Consultants of Boston

John Thompson, MDRetina Specialists

Jay Duker, MDHemera Biosciences

Joshua Schimmer, MD Senior Research Analyst

Piper Jaffray

Co-Moderated By:

Tarek Hassan, MDAssociated Retinal Consultants

Gilbert H. Kliman, MD Managing Director InterWest Partners

Paying for Breakthrough Eye Therapies (Biologics, Biosimilars & Gene Therapies)

Panel Discussion

1. Which drug therapy would you use on your own eye for

wet AMD if all the drugs costs the same?

PIPER JAFFRAY | 5

Trends in Drug Pricing: Pressure Across the Board

• Factors driving pricing pressure:

Industry growth ($450B/yr+)

Increasing competition

Consolidation of payors

Aging population straining budgets

• Source:

The industry is steadily shifting to ‘value-based’ pricing and most new drugs will have to fit into this paradigm

• Ophthalmology focus:

Only 2-3% of drug spend

Wet AMD biggest driver by far

Innovation in front/back of eye

Increasing # Orphan eye drugs

PIPER JAFFRAY | 6

Pricing Trends: Power Shifting To Payors

Price increases are being mostly offset by discounts/rebates. Payors have greater control over drug utilization than ever before

Source: IMS Health report

2. Payor dynamics seem to be driving patient care. Are AMD drug prices too high, or is the willingness to pay too

low?

PIPER JAFFRAY | 8

Biosimilar Pipelines Getting Full

• Source: biopharma.com

Long-list of biopharma players ranging from large biopharma companies down to small/mid global biosimilar companies in U.S., South Korea, India and beyond

Most advanced biosimilar Lucentis

program (Pfenex/PFE) is starting pivotal trials this

year

3. Assuming biosimilar anti-VEGFs are approved, where

will pricing come out and what will be the impact on

clinical practice?

PIPER JAFFRAY | 10

Innovation in Ophtho: Gene Therapy and BeyondCONTENT

• Source: Company reports

PUBLICLY TRADED COMPANIES PRIVATE COMPANIES

Company Clinical Preclinal Company Clinical Preclinical

Spark LCAChoroideremia RP, LHON NightstaRx Choroideremia TBD

Oxford Biomed AMD, Stargardt, Usher

Corneal graft Retro-Sense Optogenics/ RP, AMD Optogenics

AGTC XLRS, achromatopsia XLRP, AMD Gensight LHON RP, dry AMD

Adverum AMD XLRS, color vision MeiraGTx N/A LCA, XLRP, AMD,

achromatopsia

REGENX-BIO AMD 4D Molecular Choroideremia

Editas LCA10

4. If one-time gene therapy proves to be a successful therapy for orphan retina diseases, how should it be

priced?

Can this pricing model work for common conditions such

as AMD?