Disease Foundations Get New Clout, But Face New Risks as Well

Disease foundations have been taking a larger role in drug discovery and development. The financial crisis has made it easier for such groups to get partnerships with biotech firms, who have seen venture capital and big pharma deals decline drastically. But this new role also brings more risk to the foundations.

A recent article in Mass High Tech describes how the Cystic Fibrosis Foundation (CFF) ended up having to find a new partner and put an additional $3 million into a project after Altus Pharmaceuticals stopped working on a drug candidate CFF was helping to fund. The drug was being tested in a phase III clinical trial, which is usually the last step before a company can ask the Food and Drug Administration to approve the medication for sale. But Altus was facing a financial crisis and could not afford to keep developing all the drugs in its pipeline. Altus abandoned the potential cystic fibrosis treatment because that disease is relatively rare — affecting just 30,000 people nationwide. The company’s attention is now focused on a drug that treats growth deficiency, which has a much larger potential market.Eventually, the CFF did find a new partner — Alnara Pharmaceuticals, and the phase III trial back on track. Liprotamase, as the drug is now being called, is a new type of pancreatic enzyme that could make life much easier and more comfortable for the 90% of cystic fibrosis patients who suffer from low levels of such enzymes.

Other foundations that have major collaborations with biotech companies include the Michael J. Fox Foundation for Parkinson’s Disease Resarch and the Juvenile Diabetes Research Foundation. The MHT article quotes Peter Lomedico, the jead of the JDRF, as saying that his groups is looking much more closely at companies’ financial health before doing deals with them: “There have been times we’ve had to walk away from some really good science, because the company is just too vulnerable financially.”