PubMed Drug Discovery Method

Martin Shkreli explains how he used PubMed's database of biomedical literature to identify drug opportunities and build billion-dollar companies.

Core Strategy

• PubMed contains 36M biomedical research papers documenting every medical innovation
• Success requires patience and willpower to continuously sift through papers
• Focus on understanding protein/drug mechanisms and their relationships to diseases

Research Process

• Study where proteins are expressed in the body
• Analyze how proteins function and their mechanisms
• Evaluate what happens when you inhibit specific proteins
• Research historical attempts by other companies
• Look for diseases where the drug could be beneficial
• Create comprehensive lists of potential disease applications
• Identify which diseases have the highest probability of success

Case Study: Kidney Disease Drug Development

• Found drug asset originally developed by Bristol Myers for hypertension
• Bristol Myers spent $50M developing it but quit due to market saturation
• Through PubMed research, identified potential for rare kidney disease (FSGS)
• Acquired rights to drug for $2M
• Developed new clinical trial approach for kidney disease
• Drug eventually received FDA approval
• Company now worth ~$1.5B

Keys to Success

• Ability to deeply understand pharmaceutical mechanisms through research
• Patience to thoroughly analyze historical research attempts
• Identifying overlooked opportunities in existing drug compounds
• Understanding both the science and business aspects
• Willingness to spend extensive time reading and analyzing papers
• Focus on finding clear evidence of potential efficacy

Value Creation Process

• Start with thorough literature review
• Identify undervalued drug assets
• Develop new applications for existing compounds
• Run focused clinical trials
• Build company around promising assets
• Take company public to fund development
• Shepherd drug through FDA approval process

The key insight is that all the information needed to build billion-dollar drug companies exists freely in PubMed - it just requires the dedication to thoroughly analyze it.