Risdiplam has been designed to be a survival motor neuron-2 (SMN2) splicing modifier. The drug works by durably increasing and maintaining SMN protein levels throughout the central nervous system and also in the peripheral tissues of the body.
The primary endpoint met in the part 2 of the SUNFISH trial is change from baseline in the Motor Function Measure 32 (MFM-32) scale following a year of treatment with risdiplam, in comparison to placebo.
Roche said that safety for the investigational drug was on par with its known safety profile with no new safety signals detected.
Roche chief medical officer and global product development head Levi Garraway said: “The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated.
“SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients. We thank the SMA community for their partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition.”
Roche is heading the clinical development of the investigational spinal muscular atrophy treatment as part of a collaboration with the PTC Therapeutics and SMA Foundation.
The drug is being investigated in a broad clinical trial programme in SMA, featuring patients ranging from birth to 60 years old. The clinical programme also has patients who were treated previously with SMA-targeting therapies.
In another development, Roche said that the phase 2 NOBILITY trial, which is assessing the safety and efficacy of Gazyva (obinutuzumab) for adults with proliferative lupus nephritis, has met the primary endpoint.
Gazyva is being evaluated in combination with standard of care, consisting of mycophenolate mofetil or mycophenolic acid and corticosteroids. The Gazyva and standard of care combination showed superiority compared to placebo and standard of care.
The investigational combo more than doubled the percentage of lupus nephritis patients showing complete renal response, compared to standard of care alone, in the mid-stage trial. Roche is planning to start enrolment in a phase 3 programme for Gazyva.