FoldRx Announces Progress with Development Program for Lead Candidate

FoldRx Pharmaceuticals, Inc. (FoldRx) has announced that enrollment is underway in an open-label Phase II clinical study with its lead drug candidate, Fx-1006A, for patients suffering from TTR Amyloid Cardiomyopathy (ATTR-CM). The company also announced progress in several other ongoing studies that are part of a larger development program evaluating its lead candidate.

Fx-1006A is designed to stop the progression of TTR amyloidosis caused by the ‘misfolding’ of a protein called transthyretin (TTR) and the subsequent accumulation of amyloid fibrils in various tissues, such as the heart and peripheral nerve tissue, with resultant cardiomyopathy and neuropathy, respectively.

Enrollment is complete in a previously announced pivotal multinational Phase II/III clinical trial of Fx-1006A for TTR amyloid polyneuropathy. Patients having completed this 18-month Phase II/III pivotal study are being enrolled in a one-year open-label follow-up study with Fx-1006A.

Additionally, recruitment is underway in an open-label Phase II study for ATTR polyneuropathy patients with various TTR mutations not included in our Phase II/III trial.

“The launch of this latest study constitutes our first interventional study for cardiomyopathy stemming from TTR misfolding and the fourth study with our lead drug candidate, Fx-1006A, since initiating the development program only two years ago,” noted FoldRx President and CEO Richard Labaudiniere, Ph.D.

“Together, these studies will serve as the pivotal efficacy and safety data supporting registration of Fx-1006A for the treatment of TTR amyloid polyneuropathy and we anticipate results from the fully enrolled pivotal multinational Phase II/III clinical for TTR amyloid polyneuropathy by July 2009.”

In ATTR-CM, a fatal, under-diagnosed disorder, amyloid fibrils are deposited in the myocardium, resulting in diastolic dysfunction that may progress to restrictive cardiomyopathy and symptomatic heart failure. ATTR-CM is caused by either specific point mutations in the TTR gene or age-associated TTR deposition.

“TTR amyloid cardiomyopathy is emerging as a significant cause of heart failure in the elderly population and in carriers of TTR mutations,” said Mathew Maurer, M.D., associate professor of clinical medicine, Columbia University Medical Center and director of the Clinical Cardiovascular Research Laboratory for the Elderly at the Allen Pavilion of NewYork-Presbyterian Hospital, who is the principal investigator for the CUMC trial site.

“The ability to halt the deposition of TTR amyloid in ATTR-CM, for which there is currently no treatment, would represent a significant clinical advance in the care of patients with heart failure. As a clinical trial site, we at Columbia have administered the first-in-the-world trial dose of this novel drug candidate and we look forward to evaluating its efficacy as a potential benefit to patients with this rare, but serious disease.”