Biogen Advances Once-Yearly SMA Therapy Salanersen to Phase 3 After Positive Phase 1 Results

Biogen Inc. (NASDAQ:BIIB) is one of the most undervalued US stocks according to analysts. On June 25, Biogen announced positive topline results from an interim analysis of its Phase 1 study for salanersen (BIIB115/ION306), which is an investigational antisense oligonucleotide/ASO for treating spinal muscular atrophy/SMA. Based on these encouraging findings, Biogen is engaging with global health authorities to advance salanersen into registrational (Phase 3) studies.

Salanersen was discovered by Ionis Pharmaceuticals Inc. (NASDAQ:IONS) and licensed to Biogen. It uses the same mechanism of action as SPINRAZA (nusinersen) but is designed for greater potency, with the potential to achieve high efficacy and allow for once-yearly dosing. The Phase 1 study was a single ascending dose trial aimed to evaluate the safety, tolerability, and pharmacokinetics of salanersen. It consisted of two parts: Part A was a randomized and placebo-controlled segment involving healthy adult male volunteers, and Part B was an open-label segment with pediatric SMA participants.

The interim results, specifically from Part B (n=24), focused on individuals who had previously received ZOLGENSMA (onasemnogene abeparvovec) and had reported suboptimal clinical status. Both tested dose levels, 40 mg and 80 mg, administered once a year, were generally well-tolerated. The cumulative safety data indicate that salanersen was generally well-tolerated at both dose levels, with most adverse events being mild to moderate in severity.

Biogen Inc. (NASDAQ:BIIB) discovers, develops, manufactures, and delivers therapies for treating neurological and neurodegenerative diseases internationally. Ionis Pharmaceuticals Inc. (NASDAQ:IONS) is a commercial-stage biotechnology company that provides RNA-targeted medicines in the US.

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Disclosure: None. This article is originally published at Insider Monkey.