Ed Schnipper, M.D

Cancer Still a Major Problem

• 600,000 deaths per year in US

• 1,500 per day

Progress has been made

• People living longer with cancer

• Five year survival overall is 67%

• Better understanding of the science of

cancer has led to better diagnosis and

treatment

Death rates declining for some tumors

• Prostate

• Breast

• Lung

• Colorectal

Death rates rising for others

• Pancreas

• Liver

New ways of thinking about cancer

Old

by site and type of tissue ie

adenocarcinoma of the lung

New

by cellular abnormality ie EGRF mutation

Understanding pathways and interactions

• Leading to targeted therapies

• Companion diagnostics

• Better understanding of resistance

mechanisms

• Combination approaches

New understanding of factors external to tumor

• Tumor vasculature and stromal cells

• Immune tolerance

Able to target specific abnormalities

• More individualized approach

• Companion diagnostics to screen patients

likely to respond

• Better understanding of resistance

mechanisms

Many drugs in trials

• More than a thousand cancer trials

• 47 new cancer drugs approved in last 10

years

Few patients enter trials

• Less than 5% of eligible patients

Many trials /drugs fail

• 5% of drugs that enter trials are approved

• 60% of successful Phase II drugs fail in

Phase III

Causes of trial failure

• Too few patients

• Poor choice of endpoint

Health economics

• Need for differentiation/advantage

• Puts pressure on companies to do larger

more expensive trials

Biotech engine for inovation

• Pharma pipeline cannot keep up with

demand

Biotech model broken

• Over last 10 years 0.8X return to investors

Less money available to startups

• VC’s more cautious

• Less money invested in biotech

Significant problem for oncology drugs

Traditionally poor prediction from

animal models

some improvement with

knockout models and patient derived

samples

Trials long and expensive

Pressure for early less expensive answers

• Biomarkers

• Clever trial design

• Patient enrichment

• Need for “Phase III results from Phase I trial”

Opportunity for Ockham

• Partner of choice to derisk programs early