Biogen shares positive results for higher dose nusinersen in spinal muscular atrophy

Biogen has announced promising top-line results from a phase 2/3 study focusing on a higher dose regimen of its antisense oligonucleotide nusinersen. Known globally under the brand name Spinraza, this drug is already approved in more than 71 countries for the treatment of spinal muscular atrophy (SMA) in infants, children, and adults.

The latest investigation involves a new dosing regimen that includes a more rapid loading phase with two 50mg doses administered two weeks apart, followed by a higher maintenance dose of 28mg every four months. SMA is a debilitating and progressive genetic disease that affects approximately one in every 10,000 newborns worldwide. The condition is marked by inadequate levels of the survival motor neuron (SMN) protein, leading to muscle weakness and issues with breathing and swallowing.

Nusinersen is administered directly into the central nervous system and targets the root cause of motor neuron loss by increasing the production of full-length SMN protein in the body. The drug’s efficacy was examined in Part B of a three-part DEVOTE trial, which involved treatment-naïve children with infantile-onset SMA. Participants were randomly assigned either the investigational higher-dose regimen or the approved 12mg regimen.

The study achieved its primary endpoint at the six-month mark, showing a statistically significant improvement in motor function among patients receiving the higher dose compared to a matched, untreated control group from the phase 3 ENDEAR study. The higher dose regimen also showed benefits over the currently approved 12mg dose in key biomarker and efficacy measures and was generally well tolerated.

Biogen plans to submit regulatory applications for this investigational regimen, believing it has the potential to advance SMA treatment. Stephanie Fradette, head of the neuromuscular development unit at Biogen, emphasized the ongoing need for improved SMA treatments. She highlighted the well-established profile of nusinersen over the past decade and expressed the company’s commitment to exploring ways to maximize efficacy while ensuring safety. Fradette added that Biogen is eager to share the detailed results with the SMA community and health authorities.

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