PTC Therapeutics SMA drug enters Phase Ib/IIa trial

26 November 2014

PTC Therapeutics has announced that its SMN2 splice-modifying drug, RG7800, has entered a Phase Ib/IIa clinical trial in adults and children with SMA.

This is the first drug to reach this stage of development coming from the joint SMA initiative between PTC, Roche, and the SMA Foundation, started at the end of 2011 (for more information on this program click here).

RG7800 is a small molecule drug that is able to increase the levels of survival motor neuron (SMN) protein and therefore has the potential to at least partially relieve some of the symptoms caused by SMA.

The compound has already proven safe and well tolerated in healthy volunteers, and was shown to cause an increase in the availability of SMN protein produced by the SMN2 gene.

This new Phase Ib/IIa trial will be placebo-controlled, involve multiple drug doses, and be conducted over 12 weeks. The safety and tolerability of RG7800 will be tested in about 50 SMA patients, who will be randomly allocated to one of the different treatment groups.

RG7800 can be taken orally, meaning that it is easily administered without requiring specialist equipment.

This step marks a milestone for the PTC/Roche/SMA Foundation collaboration, and has resulted in a $10 million payment from Roche to PTC as part of their original agreement.

Further Information

PTC Therapeutics press release