FoldRx Gets $29M Venture Round, to Seek First Approval
June 16, 2010
By Trista Morrison
Staff Writer
FoldRx Pharmaceuticals Inc. raised $29 million in venture funding as it prepares to file for approval of tafamidis, a molecular chaperone drug designed to stabilize the mutated protein associated with transthyretin amyloid polyneuropathy, a rare genetic disease.
The Cambridge, Mass.-based firm previously closed a $16 million Series A round in 2004 and a $43 million Series B round in 2006. The current round - the series of which FoldRx declined to disclose - brings the biotech's cumulative venture backing to about $88 million. (See BioWorld Today, Dec. 14, 2004, and May 31, 2006.)
New investors in the current round included Novo Ventures and Morgenthaler Ventures, which joined existing investors HealthCare Ventures, Fidelity Biosciences, TPG Biotechnology, Alta Partners, and Novartis Venture Funds.
FoldRx Chief Business Officer Chris Adams noted that the biotech had several venture firms in due diligence, despite the challenging financing environment. "The orphan drug space is a very good space to be in right now," he told BioWorld Today.
In fact, as FoldRx was announcing its financing, Pfizer Inc. was announcing the creation of new research unit focused on rare diseases. GlaxoSmithKline plc launched a similar effort earlier this year.
But while many existing drugs for rare diseases involve replacing an enzyme, FoldRx's approach is to create small molecules that prevent protein misfolding. Taking a similar approach are Amicus Therapeutics Inc., which is in Phase III for Fabry disease with Amigal (migalastat hydrochloride), and CytRx Corp., in Phase II/III with arimoclomol for amyotrophic lateral sclerosis.
FoldRx completed a pivotal Phase II/III trial last year evaluating tafamidis for transthyretin amyloid polyneuropathy, a rare genetic disease in which misfolding of the transthyretin (TTR) protein in the liver causes potentially fatal amyloid buildup. The only existing treatment option is a liver transplant. (See BioWorld Today, July 22, 2009.)
In the trial, 91 of 128 patients completed the study, with the majority that dropped out doing so to get a liver transplant. A subset analysis of the patients completing the trial demonstrated that tafamidis significantly improved outcomes compared to placebo according to the trial's two primary endpoints: disease progression as measured by the Neuropathy Impairment Score - Lower Limb (p = 0.041) and quality of life as measured by the Norfolk Quality Of Life (p = 0.045). Adams declined to discuss outcomes in the overall trial population but noted that the completer subset analysis was prespecified.
FoldRx intends to use the data from its pivotal trial, along with additional data gathered from an open-label extension study, to file for approval. Adams said the company plans to file in Europe in the third quarter but did not specify when a U.S. filing can be expected.
The money from the current venture round is being used primarily to prepare for commercialization of tafamidis in key markets. Partnership discussions are ongoing, but Adams said FoldRx is "making sure we are ready to commercialize this drug if we go it alone."
Some of the funding will also support development of tafamidis for amyloid cardiomyopathy, the condition that occurs when transthyretin amyloid accumulates in the heart. An open-label Phase II trial has been completed, although the results have not been announced. Adams said they will be presented at an upcoming medical meeting.
Earlier in its pipeline, FoldRx has a cystic fibrosis program being funded under a $22 million milestone-based collaboration with the Cystic Fibrosis Foundation. FoldRx expects to select a clinical candidate for the program by the end of next year. (See BioWorld Today, Aug. 14, 2007.)
FoldRx also has done some research in Parkinson's disease under a small grant from the Michael J. Fox Foundation.
