FoldRx Reports Positive Results From Phase II/III Clinical Study Of Tafamidis

FoldRx has reported positive results from its pivotal phase II/III clinical study of ttafamidis (Fx-1006A), in patients suffering from TTR amyloid polyneuropathy (ATTR-PN). Its a fatal orphan disease also known as Familial Amyloid Polyneuropathy (FAP).

The international, randomized, double-blind, placebo controlled pivotal study enrolled 128 patients in eight sites suffering from TTR amyloidosis polyneuropathy, with confirmed V30M TTR mutation. Participants underwent an 18-month treatment regimen with once-a-day dosing of tafamidis or placebo. The co-primary endpoints measured response to treatment at 18 months via the Neuropathy Impairment Score – Lower Limb (NIS-LL), and quality of life, as measured by the Norfolk QOL-DN. These two endpoints correlate with disease severity and are sensitive and relevant endpoints in demonstrating neuropathy disease progression.

Richard Labaudiniere, president and CEO of FoldRx, said: “The consistency of the results across the various endpoints and analyses clearly demonstrate the robust effect of tafamidis in halting disease progression in ATTR-PN.”

“If approved, tafamidis would be the first disease modifying agent targeting protein misfolding. We are very excited by the results of the trial and look forward to bringing this innovative therapy to patients worldwide. We plan discussions with the US and European regulatory agencies later this year and we anticipate filing marketing applications in 2010,” he added.