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Roche Provides Updates on New Compound and Moonfish Trial

11 January 2016

Roche, PTC Therapeutics, and the SMA Foundation have announced that they are to begin testing a second splice-modifying drug with potential to treat SMA.

This new drug, called RG7916, will be tested in healthy volunteers in a Phase I clinical trial to commence in January 2016. This study aims to assess the pharmacokinetics, safety and tolerability of the new compound.

RG7916 is a small molecule drug that acts on the backup Survival of Motor Neuron 2 (SMN2) gene, encouraging it to produce more of the SMN protein, which is lacking in SMA patients. By increasing SMN levels, RG7916 may be able to at least partially relieve some of the symptoms caused by SMA.

The advancement of RG7916 comes very soon after the announcement that the Phase II trial of RG7800, the first SMN2 splice-modifying drug trialled by Roche, has been suspended due to safety concerns (click here for more information). The trial, known as Moonfish, was halted in May 2015 as a precautionary measure because unexpected side-effects were observed in the eyes of long-term treated treated mice. However, these mice were exposed to concentrations above those seen in the patient trial.

In their latest announcement, Roche have stated that the trial remains on hold as investigations into the safety issue are continued.

Further Information

Cure SMA Website 

Moonfish clinical trial page

Information on RG7800 Phase I trial