Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA. RG7916 has been safe and well tolerated at all doses and there have been no drug-related safety findings leading to withdrawal. In addition, data on the ability to swallow and requirements for tracheostomy or permanent ventilation, together with overall survival were also presented. Previously published natural history data indicate that in a comparable historic cohort the median age of event-free survival for SMA Type 1 infants to be between 8 and 10.5 months.

The presentation was given by Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, at the International Scientific Congress on spinal muscular atrophy in Kraków, Poland, and will be made available via a link under the investor relations section of the PTC website (

FIREFISH (NCT02913482) is a multi-center, open-label, seamless pivotal study evaluating the safety and efficacy of RG7916 in babies aged 1-7 months at enrollment with Type 1 SMA and two SMN2 gene copies. The exploratory Part 1 (n=8-24) is assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of RG7916 at different dose levels. In Part 1, patients receive RG7916 for at least 4 weeks (or 2 weeks after steady-state is achieved) of daily administration; patients then enter an extension phase with RG7916. The confirmatory Part 2 (n=40) will assess the safety and efficacy of RG7916 at the dose level selected from Part 1 over 24 months. The primary endpoint for Part 2 is the proportion of infants sitting without support for 5 seconds, assessed by the Gross Motor Scale of the BSID-III, after 12 months of treatment.

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In late 2016, Genentech/Roche first announced plans to begin Phase 2 trials of R07034067, also known as RG7916, in response to favorable results from a Phase 1 study in healthy volunteers. R07034067 is an orally available drug that aims to correct splicing of SMN2, the SMA “backup gene”.

FIREFISH (NCT02913482) assesses RG7916 in babies with Type 1 SMA aged 1 to 7 months. FIREFISH has two parts: Part 1 explored two dose levels of RG7916; Part 2 will evaluate efficacy of the most appropriate dose of RG7916. FIREFISH is open-label, i.e. there is no placebo in this study.

This compound is also being tested in two additional pivotal trials: SUNFISH and JEWELFISH.

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