Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS - Seite 2

“Data from the tofersen Phase 3 study and its open-label extension showed signs of slowing disease progression in people with SOD1-ALS, a rare, devastating disease that leads to loss of everyday functions and ultimately death,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “Following discussions with investigators, bioethicists, and having listened to the voice of patient advocacy groups, we will broaden early access to tofersen to all eligible SOD1-ALS patients through our already established expanded access program. We are grateful for the courageous efforts of patients, families, advocates, and the scientific community who have contributed to this important research.”

ALS is a progressive neurodegenerative disease that is uniformly fatal with an average survival of three to five years. The most common cause of death is respiratory failure. SOD1-ALS is a rare, genetic form of ALS that accounts for approximately two percent of the estimated 168,000 people who have the disease globally. Currently, there are no genetically targeted treatment options for ALS.

Click here for a fact sheet to learn more about genetic amyotrophic lateral sclerosis (ALS):
http://ml.globenewswire.com/Resource/Download/ae9c9142-ae2a-4d95-8f6f- ...

In light of the critical unmet need, Biogen will expand eligibility for its ongoing early access program (EAP) to all people with SOD1-ALS, in countries where such programs are permitted by local regulations and future access may be secured. EAP programs enable patients to gain access to a medicine free of charge before the treatment is licensed commercially. If a clear path forward for tofersen is not established, or if another controlled trial is required by regulators, Biogen may revise or discontinue the EAP.

The VALOR and Open-Label Extension Studies

VALOR was a 28-week Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability, pharmacodynamic, and biomarker effects of tofersen 100 mg in adults with ALS associated with a SOD1 mutation. In total, 108 participants were randomized in VALOR (n=72 to tofersen 100 mg and n=36 to placebo). Sixty of these participants met the study’s protocol-defined enrichment criteria for rapid disease progression, comprising the primary analysis population (“faster progressing”). Forty-eight participants did not meet these prognostic enrichment criteria (“slower progressing”).