Roche, PTC Therapeutics, and the SMA Foundation are currently researching two investigational therapies, RG7916 and RG7800, for the treatment of SMA.
How RG7916 and RG7800 work
RG7916 and RG7800 are splicing modifiers. RG7916 is designed to shift the pre-mRNA splicing toward the production of full length SMN mRNA. RG7800 is a more advanced substance, and its mechanism of action may interact differently with the human body.
Clinical trials for RG7916 and RG7800
A study to investigate the safety, tolerability, pharmacokinetics (how it moves in the body), and pharmacodynamics (effect in the body) of RO7034067 (RG7916) of increasing doses, orally taken, in healthy volunteers was completed (NCT02633709) in August 2016. The study’s goal was to assess RG7916’s safety and tolerability in these volunteers, with data collected helping to determine the optimal dose for people with SMA.
A Phase 2 study was planned to test RG7800 in children and adults with SMA, but it was placed on hold as a precautionary measure after an unexpected finding was identified in an animal study exploring long-term treatment. The first cohort of this study has been completed.
Next steps for RG7916 and RG7800
Roche is manufacturing RG7800 and RG7916, with a joint steering committee overseeing development with members from PTC, Roche, and the SMA Foundation.
As of July 2016, Roche was planning a Phase 2 trial for SMA treatment in Europe.
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