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

"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...