Roche Initiate Phase II Trial of RG7916
27 September 2016
Roche have announced that they are to begin recruiting SMA patients in two parallel Phase II clinical trials of their second splice-modifying drug, RG7916 (also known as RO7034067).
RG7916 is a small molecule that encourages the backup Survival of Motor Neuron 2 (SMN2) gene to produce more SMN protein, which is lacking in SMA patients. By increasing SMN levels, RG7916 may be able to improve some of the symptoms caused by SMA. A Phase I trial of RG7916 to assess its safety was initiated in healthy volunteers at the beginning of 2016 (click here for more information). RG7916 is orally bioavailable, which means that it can be taken by mouth.
The two new trials, named Firefish and Sunfish, are designed to test the safety, tolerability, and properties of the drug. On a small scale, the effectiveness of RG7916 will also be assessed. Both trials have been divided into two parts. Over an initial period of a few months, Part 1 aims to find a safe and tolerable dose of RG7916, which will then be tested in Part 2 over 24 months.
The Firefish trial will be conducted with infants with SMA Type 1 (aged 1-7 months with two copies of SMN2) and is open label, meaning that there is no placebo group. Currently, Firefish is scheduled to start recruiting at centers across the USA and France in November.
Contrastingly, the Sunfish trial will be conducted with children, young people and adults aged 2-25 years with SMA Type 2-3. Beginning in October, Sunfish will be a randomised, placebo-controlled, and double blind trial which means that patients will be randomly allocated to either a placebo or treatment group, and that this will be concealed from the patients and people administering the treatments. Sunfish is planned to be conducted at sites worldwide. You can find out where here.
The previously initiated Moonfish clinical trial of RG7800 remains on hold due to safety concerns. Patients enrolled on that trial may soon be eligible to move over to a separate open-label study called Jewelfish.