RG7916 FIREFISH trial now enrolling patients (though not in UK)
16 August 2017
Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).
RG7916 is a small molecule, oral drug that targets and encourages the SMN2 backup gene to produce more functional SMN protein, which is lacking in people with SMA. The FDA recently granted Orphan Drug Status to RG7916 (click here for more information).
The trial is being conducted in the US, Belgium, Italy, Switzerland, and Turkey and is principally designed to test the safety, tolerability, and properties of the drug. Approximately 50 infants with SMA Type 1 (aged 1-7 months who have two copies of the SMN2 gene) will be enrolled, and the trial is open label, meaning that there is no placebo group.
Divided into two parts, FIREFISH will, over the first four weeks, aim to find a safe and tolerable does of RG7916, which will then be tested in the second part of the trial over 24 months.
In addition to the FIREFISH trial, RG7916 is being tested in a second Phase II trial, known as SUNFISH, which is recruiting in Europe, but will soon be extending to North America and Australia. SUNFISH is being conducted with SMA Type 2 and Type 3 patients and has been recently reported to show promising early results (click here for more information).
The previously initiated Moonfish clinical trial of RG7800 remains on hold due to safety concerns (click here for more information). Patients enrolled on that trial are allowed to move over to a separate open-label study of RG7916 called Jewelfish.