Roche's Genentech Rounds Out Week of SMA Program Updates

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Genentech, part of the Roche Group, shared new data from its SUNFISH study evaluating the use of Evrysdi (risdiplam) in people ages 2 to 25 years diagnosed with Type 2 or Type 3 spinal muscular atrophy (SMA). 

SMA is a severe and progressive neuromuscular disorder that can be fatal. A leading genetic cause of infant mortality, it affects one in 10,000 babies. The disease is characterized by nerve dysfunction leading to muscle weakness and decline of motor function. 

Researchers observed that Evrysdi was able to increase the Motor Function Measure 32 (MFM-32) score from baseline in the first year and maintained this through the third year. Total scores for the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Lim Module (RULM) were also maintained from the first to the third year. Evrysdi was well-tolerated throughout the observation period, and the overall rate of adverse events declined. 

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat mutations in chromosome 5q, which leads to SMN protein deficiency and then later to SMA. Evrysdi comes in liquid form and is given daily at home orally or via a feeding tube. Treatment is done throughout the patient's lifetime. 

"The positive long-term efficacy and safety results for Evrysdi in this broad SMA population are important for physicians as they consider Evrysdi as a treatment option for their patients. In treating people with SMA, our aim is to enable or preserve their independence and patients in the SUNFISH study reported continuous improvement or stabilization in the level of help needed for daily living," commented Laurent Servais, M.D., Ph.D., professor of pediatric neuromuscular diseases at the MDUK Oxford Neuromuscular Center. 

In the same announcement, Genentech shared positive interim data on the drug's effect on newborns. Partial results from the RAINBOWFISH trial demonstrated safety and efficacy in pre-symptomatic babies with SMA who are under two months old. For this reason, the U.S. Food and Drug Administration granted the drug priority review status in January 2022 for that said indication. 

SUNFISH and RAINBOWFISH are just two of five multi-center trials that Genentech is conducting on SMA. FIREFISH, an open-label, two-part clinical study, is evaluating Evrysdia in infants with Type 1 SMA while JEWELFISH, an open-label exploratory trial, assesses the tolerability, safety and pharmacokinetics of the drug in ages 6 months to 60 years. Another study, MANATEE, a global Phase II/III trial, is looking into the safety and efficacy of using an anti-myostatin molecule called GYM329 plus Evrysdi in treating patients ages 2 to 10 years who have SMA. Recruitment for MANATEE is ongoing. 

Genentech rounds out a week of data revelations in the SMA space. 

Biogen is also conducting a Phase II open-label trial (NURTURE) on the use of Spinraza (nusinersen) for pre-symptomatic patients with the disease. The participants were diagnosed via a genetic test that placed them as highly likely to develop Type 1 or Type 2 SMA. Researchers are observing the patients from six weeks of age to eight years. Spinraza is dosed every four months for the patient's lifetime. 

Meanwhile, Novartis is testing Zolgensma (onasemnogene abeparvovec) as a one-time treatment. Data from its Phase III SPR1NT trial found the drug to be promising in children who were treated presymptomatically as they achieved age-appropriate development milestones like walking and standing, with few or no signs of neuromuscular disease. However, many industry observers have criticized Zolgensma because the price for a single treatment is around $2.1 million. 

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