Genentech recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA):
“Moonfish is studying RG7800, an investigational SMN2 splicing modifier that Genentech (Roche) is
developing for the treatment of spinal muscular atrophy (SMA). This program is in collaboration with
the SMA Foundation and PTC Therapeutics.
“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.
“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
“As patient safety is of the utmost importance, we continue to thoroughly assess the safety finding in
ongoing animal studies. At this time, we cannot yet draw a final conclusion regarding the pre-clinical
safety finding or a potential restart of the trial.
“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.”