Roche has opened enrollment in the US on one of their two Phase 2 clinical trials testing RO7034067 (also known as RG7916). Late last year, 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.”
The trial currently recruiting in the US is called Firefish and 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.
A 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. Currently, the trial is recruiting only in Europe, but will soon be adding sites in the US, Canada, 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, have the opportunity to transition into a separate open-label study, called Jewelfish, testing RO7034067.