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.