Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference

-- RG7916 demonstrated ~400% increase in SMN2 full length/Δ7 mRNA ratio --

-- RG7916 demonstrated a favorable preliminary safety profile and was generally well tolerated in Type 2/3 SMA patients --

SOUTH PLAINFIELD, NJ, USA I July 1, 2017 I PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the presentation of preliminary clinical data  from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) at the Cure SMA Meeting in Orlando, FL. Preliminary results from an early analysis of Part 1 of the Phase 2 SUNFISH trial evaluating oral RG7916, a small molecule modifier of Survival Mo tor Neuron 2 (SMN2) splicing, were highlighted in an oral presentation titled "Clinical Studies of RG7916 in Patients with Spinal Muscular Atrophy: Study Update." SMA is a rare genetic disorder that results in neuromuscular disability beginning in infancy and is the leading genetic cause of mortality in infants and young children.

"The increase in SMN2 full length transcript in SMA patients is promising and confirms that RG7916 targets the underlying cause of the disease," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "The low levels of SMN protein in SMA patients affect multiple tissues throughout the body, including muscles, bones and nerves. We believe that the ease of administration of an oral therapy and its broad tissue distribution give RG7916 the potential to address the complete spectrum of functional deficits observed in SMA patients. We look forward to advancing RG7916 into pivotal clinical trials in the second half of the year."

Preliminary results from an early analysis from the ongoing Part 1 of the RG7916 SUNFISH study in Type 2/3 SMA patients demonstrated a dose-dependent increase in SMN2 full length/Δ7 mRNA ratio of ~ 400% versus baseline, as measured in whole blood. These results provided proof of mechanism for oral small molecule SMN2 splicing modifiers. No drug-related adverse events leading to withdrawal have been observed to date for RG7916. 

SUNFISH is a two-part phase 2 clinical study. Part 1 is a double-blind, placebo-controlled, randomized, exploratory dose-finding study in Type 2/3 pediatric and adult SMA patients. The primary objective of the first part of the study is to evaluate the safety, pharmacokinetics, and pharmacodynamics of RG7916 in patients, and to select the dose for the second part of the study. The pivotal second part is a double-blind, placebo-controlled, randomized, confirmatory study in Type 2/3 SMA patients followed by an open-label extension. The primary objective of the pivotal second part of the study is to evaluate the safety and efficacy of RG7916 compared to placebo. SUNFISH is one of three ongoing clinical trials of RG7916, along with FIREFISH and JEWELFISH.

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 SMN2 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.

About PTC Therapeutics

PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel medicines using our expertise in RNA biology. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. Since its founding nearly 20 years ago, PTC' mission has focused on developing treatments to fundamentally change the lives of patients living with rare genetic disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey. For more information on the company, please visit our website www.ptcbio.com.

SOURCE: PTC Therapeutics

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