PTC Therapeutics SMA Drug Enters Clinical Development

30 January 2014

PTC Therapeutics has announced that its recently selected lead compound with potential to treat SMA has entered early stage clinical trials.

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

This small molecule drug is able to increase the levels of survival motor neuron (SMN) protein and therefore has potential to at least partially improve the symptoms observed in SMA. The drug will be tested in a Phase I clinical trial in healthy adult volunteers, in order to increase understanding of its safety and tolerability in humans.

This potential treatment is to be taken orally (by mouth), meaning that it can be easily administered without specialist equipment or expertise.

This step towards developing a compound to treat SMA marks a milestone for the PTC/Roche/SMA Foundation collaboration, and has resulted in a $7.5 million payment from Roche to PTC as part of their original agreement.

The PTC compound is now the fifth drug currently in clinical development for SMA, highlighting the progress that has been made over the last decade in pursuit of a treatment for the disease.


Further Information

PTC Therapeutics press release