Biogen Drops Ionis-Partnered Assets in ALS, Angelman Syndrome After Early-Stage Readouts

Biogen_iStock, JHVEPhoto

Pictured: Biogen’s signage at its headquarters in Massachusetts/iStock, JHVEPhoto

Biogen and Ionis Pharmaceuticals on Thursday revealed early results from the Phase I/II ALSpire study, demonstrating that their investigational antisense oligonucleotide BIIB105 failed to significantly lower plasma levels of a neurodegeneration biomarker, or improve clinical outcomes, in amyotrophic lateral sclerosis patients.

Given the disappointing outcome, the partners have elected to discontinue the development of BIIB105.

ALSpire was a multiple-ascending-dose trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal BIIB105 in adult patients with amyotrophic lateral sclerosis (ALS). The study consisted of a six-month placebo-controlled study, followed by a three-year, long-term, open-label extension phase.

In Thursday’s announcement, Biogen and Ionis revealed that the antisense oligonucleotide was unable to significantly reduce the levels of plasma neurofilament light chain (NfL), a known indicator of nerve damage and neurodegeneration. BIIB105 also showed…
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