- Micro cap PTC Therapeutics (NASDAQ:PTCT) is up 4% premarket on light volume in response to its announcement that Roche (OTCQX:RHHBY) has initiated a Phase 2 clinical trial, SUNFISH, assessing RG7916 in pediatric and adult patients with Type 2/3 spinal muscular atrophy (SMA).
- The first part of the study will evaluate the safety and tolerability of escalating doses of RG7916 in ~36 subjects. After the optimal dose is selected, the study will move into a pivotal second stage to assess efficacy for up to 24 months followed by an open-label extension. The transition will trigger a $20M milestone to PTC from Roche.
- A trial in Type 1 SMA patients, FIREFISH, should commence in the coming months.
- 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 (an earlier SMA candidate) modify the splicing of the SMN2 gene to generate more SMN.
- Previously: PTC Therapeutics up 17% on licensee Roche's move into mid-stage study in spinal muscular dystrophy (Sept. 30)