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Biogen Seeking A Path Forward After Phase III Tofersen Study Fails In ALS

Ionis-Partnered Asset Developed For SOD1 Genetic Mutations

 
• By Mandy Jackson

The drug showed favorable but not significant trends across secondary endpoints, and greater efficacy in slower-progressing patients and those treated earlier in the disease, in a small subpopulation.

Illuminated oil lamp on fence along woodland path
Biogen will seek guidance from regulators and others that may illuminate a path forward for tofersen in ALS • Source: Alamy

Tofersen never was going to be a large-market, major revenue-driving drug for Biogen, Inc. But with multiple setbacks over the last several months plus commercial challenges, disappointing Phase III results for the drug in amyotrophic lateral sclerosis (ALS) patients with a superoxide dismutase 1 (SOD1) gene mutation is another badly timed setback for the company and its partner, Ionis Pharmaceuticals, Inc.

Top-line results from the Phase III VALOR clinical trial and an open-label extension study of tofersen – an antisense oligonucleotide that binds to SOD1 mRNA to prevent production of the...

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