- PTC Therapeutics (PTCT +17.2%) perks up on more than triple normal volume in response to Roche's (OTCQX:RHHBY) start of enrollment in a Phase 2 clinical trial, Sunfish, assessing RO7034067 (RG7916) for the treatment of pediatric and adult patients with type 2 and type 3 spinal muscular atrophy (SMA).
- Roche's move has stoked investors because a Phase 1 study, MOONFISH, assessing another SMA candidate, RG7800, was placed on clinical hold by the FDA after an unexpected eye finding in an animal study. As a precaution, patient dosing was suspended in April 2015. No safety signals have been observed in humans, however.
- Roche obtained the exclusive rights to RG7916 and RG7800 under a November 2011 agreement with PTC and the SMA Foundation. RG7916 has different characteristics than RG7800 which may influence how it interacts with the body.
- SMA is an inherited disorder caused a deficiency in a protein called SMN which is coded by a gene called SMN1. A similar gene, SMN2, can code for SMN as well, but not as productively. RG7916 and RG7800 modify the splicing of the SMN2 gene to generate more SMN.