While cancer death rates have declined for some cancers, cancer remains a major public health problem, causing over 600,000 deaths per year in the US alone. Recent progress includes better understanding of cancer at the molecular level, leading to more targeted and personalized therapies. However, fewer patients enroll in clinical trials and many drug candidates fail in late-stage trials due to challenges including a lack of predictive animal models and the high cost of large trials. There is pressure to find cheaper and earlier answers on drug efficacy through biomarkers and trial design innovations to improve the challenging process of bringing new cancer treatments to patients.

1. Ed Schnipper, M.D

2. Cancer Still a Major Problem
• 600,000 deaths per year in US
• 1,500 per day

3. 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

4. Death rates declining for some
tumors
• Prostate
• Breast
• Lung
• Colorectal

5. Death rates rising for others
• Pancreas
• Liver

6. 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

7. Understanding pathways and
interactions
• Leading to targeted therapies
• Companion diagnostics
• Better understanding of resistance
mechanisms
• Combination approaches

8. New understanding of factors
external to tumor
• Tumor vasculature and stromal cells
• Immune tolerance

9. Able to target specific
abnormalities
• More individualized approach
• Companion diagnostics to screen patients
likely to respond
• Better understanding of resistance
mechanisms

10. Many drugs in trials
• More than a thousand cancer trials
• 47 new cancer drugs approved in last 10
years

11. Few patients enter trials
• Less than 5% of eligible patients

12. Many trials /drugs fail
• 5% of drugs that enter trials are approved
• 60% of successful Phase II drugs fail in
Phase III

13. Causes of trial failure
• Too few patients
• Poor choice of endpoint

14. Health economics
• Need for differentiation/advantage
• Puts pressure on companies to do larger
more expensive trials

15. Biotech engine for inovation
• Pharma pipeline cannot keep up with
demand

16. Biotech model broken
• Over last 10 years 0.8X return to investors

17. Less money available to
startups
• VC’s more cautious
• Less money invested in biotech

18. Significant problem for oncology
drugs
Traditionally poor prediction from
animal models
some improvement with
knockout models and patient derived
samples
Trials long and expensive

19. Pressure for early less expensive
answers
• Biomarkers
• Clever trial design
• Patient enrichment
• Need for “Phase III results from Phase I trial”

20. Opportunity for Ockham
• Partner of choice to derisk programs early