Roche Initiates New Phase 2 Clinical Trials

Roche has announced that the company is initiating two new Phase 2 studies in patients with Spinal Muscular Atrophy (SMA) to evaluate RG7916—SUNFISH and FIREFISH. RG7916 is an orally available drug that directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA and SMN protein from the SMN2 gene.

SUNFISH is a two-part study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RG7916 in Type II and III adult and pediatric SMA patients. Part 1 of the study is a double-blinded, placebo-controlled, randomized exploratory dose-finding study in approximately 36 patients for 12 weeks to evaluate the safety of RG7916 and to select the dose for Part 2 of the study. Part 2 is a double-blinded, placebo-controlled, randomized confirmatory study in approximately 150 non-ambulant Type II and Type III SMA patients for 24 months. After 12-month interim analysis, placebo participants will be switched to active treatment (RG7916). The primary objective of Part 2 is to evaluate the efficacy of RG7916 compared to placebo. The study will be followed by an open-label extension. SUNFISH will begin recruitment in October.

The second study, FIREFISH, is aimed to assess safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in infants with Type I SMA. The study is planned to initiate in the coming months. It is also a two-part study with the first part being an open-label dose escalation study in at least 8 infants for 4 weeks to evaluate the safety profile of RG7916 and to determine the dose for Part 2. Part 2 is an open-label, single-arm confirmatory study in approximately 40 infants with Type I SMA for 24 months, followed by an open-label extension.

For more information about these trials, please visit www.clinicaltrials.gov.

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