FoldRx: Unfolding the Money
June 21, 2010 - BioCentury
By Michael Flanagan & Stacy Lawrence
Senior Writers
Sometimes intent-to-treat analyses don't tell the full story. Though tafamidis missed the co-primary endpoints of a Phase II/III trial last July, FoldRx Pharmaceuticals Inc. argued the results would support approval of the small molecule to treat familial amyloid polyneuropathy (FAP).
Eleven months later, a group of VCs led by new investors Novo Ventures and Morgenthaler Ventures has agreed. Last week, they ponied up $29 million in a round designed to carry the program to commercialization. Existing investors Healthcare Ventures, Fidelity Biosciences, TPG Biotechnology, Alta Partners, and Novartis Venture Funds also participated.
FAP is a progressive neurodegen-erative disorder in which genetic mutations or denaturation stress cause transthyretin (TTR) to unfold and refold improperly. The protein, which is synthesized in the liver and transports proteins in the blood and cerebrospinal fluid, ends up aggregating into neurotoxic plaques.
"We are talking about a fatal disease where patients have little alternatives," said CBO Chris Adams. The only option is a liver transplant.
Tafamidis binds to and stabilizes TTR before it can unfold.
As it turned out, 26 of the 128 patients enrolled in FoldRx's Phase II/III trial dropped out to get transplants, which meant they were all treatment failures according to the protocol. "If not for this, the results would have been highly significant," said Adams.
In the per-protocol population, tafamidis significantly improved both disease progression (p=0.041) and quality of life (p=0.045) vs. placebo. In addition, a prospectively defined analysis that adjusted for the impact of liver transplantation showed a significant improvement in disease progression for patients on tafamidis (p=0.039).
Alta's Ed Hurwitz told BioCentury that the full dataset clearly demonstrates that tafamidis is having a disease-modifying impact.
We have not disclosed the follow-up or crossover data from the study, but the sophisticated investors who came on board after having seen the results should signal that there is an overwhelming consensus that this really works,"he said.
FoldRx plans to submit an MAA and NDA for tafamidis next half.
Hurwitz said the financing is designed to begin commercializing the product. Though "the money may not be enough to reach profitability; we now have the resources sitting around the table to be certain we can fund the company all the way," he added.
