Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, Roche first announced plans to begin these Phase 2 trials in response to favorable results from a Phase 1 study in healthy volunteers. RO7034067 is an orally available drug that aims to correct the splicing of SMN2, the SMA “backup gene.”
One trial (Firefish) will be enrolling approximately 50 infants with SMA type I at sites across the US and Europe. Firefish is an open-label study consisting of two parts: an exploratory dose finding part (Part 1) for 4-weeks and a confirmatory part (Part 2) of RO7034067 for 24-months.
The second trial (Sunfish) will be enrolling individuals aged 2-25 with SMA type II or III. This trial will recruit nearly 200 individuals across 34 sites in the US, Canada, Europe and Australia. Sunfish is a randomized, double-blind, placebo-controlled study consisting of two parts: an exploratory dose finding part (Part 1) of RO7034067 for 12-weeks and a confirmatory part (Part 2) of RO7034067 for 24-months.
Clinical development of RG7800, a related compound, remains on hold. Individuals who were previously enrolled in “Moonfish,” the clinical trial testing RG7800, will have the opportunity to transition into a separate open-label study testing RO7034067.