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RG7916 (RO7034067) is a code name for an oral drug being developed as a treatment for SMA by Roche, PTC Pharmaceuticals, and SMA Foundation. The RG7916 molecule modifies alternative splicing of the SMN2 gene in such a way that the gene starts producing only the SMN protein, which is deficient in all those with SMA.

RG7916 follows an earlier RG79800 molecule whose development was stopped in 2015 due to animal toxicity.

Clinical trials

RG7916 was tested for safety in healthy adult volunteers in early 2016 and in October 2016 phase 2-3 clinical trials in SMA patients started. The drug’s properties are being tested in three trials:

  1. Firefish – a clinical trial for babies 1–7 months old diagnosed with SMA type 1. This is an open-label trial where all participants receive the active substance for 24 months. The trial consists of two parts: The ongoing Part I tries to establish the optimum dose and monitors for safety. Part II, which is yet to start, will primarily test for the compound’s efficacy. Firefish Part I is open in Belgium, France, Germany, Italy, Switzerland, Turkey and United States. UK babies may be enrolled in the Firefish trial in those centres and Roche will bear most of the cost.
  2. Sunfish – a clinical trial for children and young adults aged 2–25 with SMA types 2 and 3. Like Firefish, this trial consists of two parts: the ongoing Part I which is testing for safety and optimal dose, and Part II, which is only starting, which will test for efficacy. Likewise, Sunfish is conducted abroad but in contrast to Firefish, international recruitment is not foreseen.
  3. Jewelfish – a clinical trial for participants age 12–60 who receive nusinersen.

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