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Genentech: Risdiplam Trial Meets Primary Goal In People With Type 2 Or 3 SMA

Genentech, a member of the Roche Group (RHHBY), announced Monday that risdiplam met primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy or SMA.

The Part 2 of the study demonstrated statistically significant improvements in the overall study population of people aged 2-25 years with Type 2 or 3 SMA.

Risdiplam is an investigational, survival motor neuron-2 or SMN2 splicing modifier, designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body.

The study met its primary endpoint of change from baseline in the Motor Function Measure 32 or MFM-32 scale after one year of treatment with risdiplam, compared to placebo.

According to the company, no treatment-related safety findings leading to study withdrawal have been seen in any risdiplam trial to date.

The company noted that SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients.

Levi Garraway, chief medical officer and Head of Global Product Development, stated that the positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated.

Data from the SUNFISH study will be presented at an upcoming medical congress.

Risdiplam is currently being evaluated in four multicenter trials in people with SMA.

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