PAYING FOR BREAKTHROUGH EYE THERAPIES (BIOLOGICS, BIOSIMILARS & GENE THERAPIES) - Pfenex

Innovative Solutions for Global Health

PF582: Biosimilar Product to Lucentis

Hubert C. Chen, M.D.8 August 2016

2Pfenex Confidential- Do Not Share Without Permission

Pfenex Corporate Overview

• Located in San Diego, California

• Founded as a platform technology company with expertise in protein production and analysis

• Now focusing on biosimilars product development

• Capabilities in molecular biology, analytical chemistry, manufacturing, clinical research and commercialization

• IPO in 2014


Mechanism of Action for PF582: Biosimilar to Ranibizumab (Lucentis®)

Wet AMD arises from abnormal growth of blood vessels, resulting in retinal

damage

Ranibizumab inhibits choroidal neovascularization by blocking VEGF-A

RPE: Retinal pigment epitheliumCNV: Choroidal neovascularization


4

Generics and Biosimilars: Contrasting Impact on the Healthcare System

Generic drugs now account for >80% of all prescriptions in the US.

In contrast, the first biosimilar was just approved by the FDA in 2015.

Why is there such a large discrepancy?


5

Creating Equivalent Copies of Aspirin vs. Humira

Monoclonal AntibodyAspirin


6

Aspirin

X-Ray Doppler Ultrasound Laser Measuring Device

Monoclonal Antibody



7

Monoclonal AntibodyAspirin


Mass Spec CD SDS-PAGE


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How Data Are Prioritized in Drug DevelopmentAnd Regulatory Approval

Phase 3

Phase 1/2

BiologicalCharacterization

StructuralCharacterization

New Drugs

Incr

easi

ng Im

porta

nce

Modern analytical techniques provide the foundation for biosimilars development and approval.

Structural Characterization

BiologicalCharacterization

PK/PD

Generics/Biosimilars

Decr

easin

g Em

phas

is

EfficacySafety


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Science

PAYING FOR BREAKTHROUGH EYE THERAPIES (BIOLOGICS, BIOSIMILARS & GENE THERAPIES) - Pfenex