RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 Spinal Muscular Atrophy

نوشته شده توسط PTC Therapeutics, Inc در .

Data presented at the 22nd International World Muscle Society (WMS) Congress -

NEWS PROVIDED BY PTC Therapeutics, Inc

Oct 03, 2017, 10:12 ET



SOUTH PLAINFIELD, N.J., Oct. 3, 2017/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from Part 1 of the SUNFISH trial of RG7916 in Type 2/3 spinal muscular atrophy ( SMA ) patients.  RG7916 is part of PTC's joint development program in  SMA  with Roche and the  SMA  Foundation ( SMA F). The data were presented at the 22nd International World Muscle (WMS) Congress in St. Malo, France. An interim analysis of the five cohorts treated with RG7916 for 28 days or longer demonstrated an exposure-dependent increase in  SMN  protein.  SMA  is characterized by reduced levels of  SMN  protein, motor neuron loss, and muscle atrophy. It is the leading genetic cause of mortality in infants and young children. To date, RG7916 remains well-tolerated in patients at all doses and there have been no drug-related safety findings leading to withdrawal.


"We are excited to report that in  SMA  patients who are deficient in  SMN  protein, RG7916 treatment led up to a median 2.5-fold increase in  SMN  protein," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. " SMN  protein is a critical factor that is needed both in the central nervous system and in a number of other tissues and organs throughout the body. Increasing  SMN  protein levels throughout the body has the potential to impact every aspect of this disorder. These results have allowed us to determine the dose for the pivotal phase of the SUNFISH trial to evaluate the efficacy and safety of RG7916 versus placebo."


SUNFISH is a double-blind, two-part, placebo-controlled trial. Part 1 of SUNFISH enrolled patients with Type 2/3  SMA  to evaluate safety, tolerability, and PK/PD of several RG7916 dose levels. The pivotal SUNFISH Part 2, in non-ambulant patients with Type 2/3  SMA , will evaluate safety and efficacy of the RG7916 dose level selected from Part 1. 


The U.S. Food and Drug Administration granted orphan drug designation and fast track designation to RG7916 for the treatment of patients with  SMA  earlier this year. RG7916, an orally administered  SMA ll molecule, directly targets the underlying cause of  SMA  by modulating  SMN 2 splicing to increase expression of full-length  SMN 2 mRNA and increases  SMN  protein levels.


The  SMA  program was initially developed by PTC Therapeutics in partnership with the  SMA  Foundation in 2006 to accelerate the development of a treatment for  SMA . In November 2011, Roche gained an exclusive worldwide license to the PTC/ SMA  Foundation  SMN 2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the  SMA  Foundation.