Genentech Updates Clinical Development of RG7800 for SMA

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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Genentech, a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions, recently announced an update on the clinical development of RG7800, its investigational SMN2 splicing modifier that is being developed for the treatment of patients with spinal muscular atrophy (SMA).

Moonfish is the clinical development program of Genentech’s RG7800 that is being conducted in partnership with PTC Therapeutics and the SMA Foundation.

“As we shared earlier this year, an unexpected eye finding was observed in an animal study evaluating the long term safety of RG7800. As a precaution, Genentech (Roche) suspended dosing in the Moonfish trial in April 2015. The U.S. Food and Drug Administration (FDA) agreed with our decision and action plan to further assess this pre-clinical safety finding, and placed the Moonfish trial on clinical hold. Clinical hold status means that we must inform and gain agreement from the U.S. FDA and the national Health Authorities in European countries before resuming the trial”, the company reported in a recent news release. “We have conducted a comprehensive review of safety information, with the assistance of independent scientific and clinical experts. This review identified no safety issues in any of the patients who were dosed with RG7800 in the trial and subsequently followed up for two months after completing treatment”.

In parallel to the Moonfish trial, the company has been investigating the effects of the long term use of RG7800 in animals. These animal studies are a standard requirement in the development of new medicines. While at this point, the company cannot draw conclusions regarding the pre-clinical safety finding or a potential restart of the trial, they will continue to evaluate the drug safety profile in ongoing animal studies.

“We remain fully committed to developing treatments for SMA and supporting the SMA community. We will provide you with an update on the Moonfish trial and our SMN2 splicing modifier program by the end of this year, at the latest.”