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Roche's SMA Drug Meets Primary Endpoint in Pivotal Study

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Roche (RHHBY - Free Report) announced positive data from the part 2 of the SUNFISH study on pipeline candidate, risdiplam, in patients with spinal muscular atrophy (SMA).

Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN-2) splicing modifier for SMA.

SUNFISH is a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated the motor function, using total score of Motor Function Measure 32 (MFM-32) at 12 months.

The study met its primary endpoint of change from baseline in the MFM-32 scale after one year of treatment with risdiplam compared to placebo, as data showed statistically significant improvements in the overall study population with type 2 or 3 SMA.

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

We remind investors that Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics (PTCT - Free Report) .

The drug is being evaluated in a broad clinical trial program in SMA in newborn babies to 60-year olds and includes patients previously treated with SMA-targeting therapies. Additionally, the company has three ongoing studies — FIREFISH, JEWELFISH and RAINBOWFISH.

The successful development of risdiplam will bode well for Roche. However, competition is stiff in the SMA market from the likes of Biogen’s (BIIB - Free Report) Spinraza and Novartis’ (NVS - Free Report) gene therapy Zolgensma.

Roche’s efforts to develop its portfolio beyond oncology is encouraging. The company has more than a dozen investigational medicines in clinical development for diseases like multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism.

Roche’s shares have gained 21.6% in the year so far compared with the industry's 0.5% growth.


Additionally, Roche announced data from the phase II NOBILITY study, investigating the safety and efficacy of lymphoma drug Gazyva (obinutuzumab) in adults with proliferative lupus nephritis. Data showed that 40% of patients treated with Gazyva plus standard of care achieved complete renal response at week 76 compared to 18% of patients treated with placebo plus standard of care.

Roche currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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