Pullan Consulting

Biotech Business Development Consulting

www.pullanconsulting.com and www.lindapullan.com

email:  lpullan@msn.com  805-558-0361

Issue #22

Pullan's Pieces

Commentary on Science & Business of Drug Development

For Business Development & Others

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“Patients Like Me” – creating evidence-based medicine as a community of patients

A group of patients suffering from multiple sclerosis (MS) began a web site www.patientslikeme.com, to share their experiences with the treatments for their diseases and to better manage their diseases.  It has now expanded to cover ALS, HIV, Parkinson’s and mood disorders.  In the MS section, more than 4500 patients share their medical information, tracking doses of medicines and using a custom scoring system to track their responses to the treatments, and the average duration and severity of MS symptoms.  Patients can select other patients like themselves and see what those patients are taking and see their responses.  All the data gets aggregated into bar graphs.  The for-profit business behind PeopleLikeMe is aiming to sell the aggregated data to researchers and pharma companies, providing a rare look at experiences outside clinical trials (FierceHealthIT).       

According to the NY Times Magazine (March 23, 2008, pp32-37), some patients have found that the experiences of others have really helped, encouraging them to try higher doses leading to better results, for instance.   But some physicians are concerned that patients are acting on unpublished, un-reviewed findings to change their own treatments, creating their own clinical trials as a community.  There are certainly risks to making decisions on incomplete information, but if I were seriously ill, I would seek this kind of community-created evidence-based medicine. 

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Biotech has a higher failure rate than Pharma in Phase III to Approval

An analysis (Nature Reviews Drug Discovery 7: 197- 198) of FDA approvals from January 2006 to December 2007, finds that biotech, while being the origin for 65% of the approvals and 56% of the novel drugs (new chemical or biological entities), has a higher failure rate than pharma companies.  For all novel drugs, there were 1.6 Phase III failures for every approval.  Novel drugs developed by biotech companies fared worse, on average, with 4.7 failures for every approval (9 approvals, 42 failures).  Biotech drugs developed in alliances with pharma companies did not do worse than those originated by pharma themselves.   Developing novel drugs is, understandably, riskier than developing me-toos or line-extensions of existing drugs.  Biotech companies had better Phase III track records with me-toos (20 approvals, 17 failures) and even better with line-extensions (18 approvals, 9 failures) than with novel drugs.  The authors conclude that the higher failure rate of biotech drugs is due to underfunding of the clinical programs.  There may also be selection of the less risky programs from biotech for pharma partnering. 

Certainly great innovation often means huge risk and even pharma can suffer failures,  as seen, for example,  with the Pfizer failure in a Phase III melanoma trial with the CTLA4 antibody (Wall St. Journal (April 1, 2008).   However, particularly for novel drugs, the extra resources of big pharma should reduce the risks. 

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2 RNAi drugs may have effects through immune system rather than specific gene silencing

Big bets have been placed on RNAi.  Merck paid $1.1 billion in 2006 to acquire Sirna Therapeutics, the RNA interference company.  Now researchers at the U. Kentucky have now reported (Kleinman ME et al, Nature, epub Mar 26, 2008) that the RNA interference drugs for macular degeneration from Opko and Allergan were not working via gene silencing.   Virtually any sequence of RNA worked equally well in stopping blood vessel formation in the eyes of mice.  The RNAi pieces were actually binding to the toll-like receptor 3, part of the innate immune system that evolved to defend against invasive microorganisms.  In mice without the toll-like receptor 3, the drugs did not stop the blood vessel formation.  While the findings may not apply to all RNAi sequences being developed, to be specific to their gene targets, RNAi drugs will need to avoid the innate immune system.  (NY Times, April 2, 2008).   

This challenge to the specificity of RNAi is in addition to the challenges of delivering RNAi to the desired intracellular targets.  Roche and the big RNAi player Alnylam just bought a total of $10 million in equity in the new merger of an RNAi therapeutics company, Tekmira, and a liposomal delivery company, Protiva Biotherapeutics.  The new merged company is to be called Tekmira.  Alnylam gave Tekmira a license to its RNAi patents for 7 drugs.  Roche and Alnylam will get access to the delivery technology.  (In Vivo Blog, March 31, 2008). 

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I hope to see many of you at BioEurope Spring or at AACR!   I’m always happy to have discussions about Pullan’s Pieces, consulting work or your thoughts on science and business. 

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Pullan Consulting

Linda M. Pullan, Ph.D.

Biotech Business Development

www.pullanconsulting.com and www.lindapullan.com

e-mail: lpullan@msn.com

805-558-0361

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