|
 Biogen will engage with regulators and other stakeholders "to
understand the meaningfulness of this data and potential paths
forward," Toby Ferguson, head of the neuromuscular development unit
at Biogen, told Reuters. The company is treating trial patients in a
follow-on study and recently launched a Phase 3 trial of the drug,
tofersen, in patients who are not yet experiencing ALS symptoms.
Tofersen, administered directly into the spinal canal each month, is
designed to suppress the production of SOD1, a protein that can
accumulate to toxic levels in ALS patients with mutations in a
specific gene. Around 2% of ALS cases are believed to be caused by
the genetic mutation.
After 28 weeks of treatment, the 108-patient trial showed a
1.2-point difference on a scale evaluating functional status for
patients with fast-progressing ALS who were given tofersen compared
to placebo patients, which was not statistically significant. In the
group of patients with slower-progressing disease, the difference
was 1.4 points.
An improvement of at least 2 points would be clinically meaningful,
Guggenheim Partners said in a recent research report.
Biogen also detailed results from secondary trial endpoints,
including breathing ability and muscular strength, indicating that
patients treated with tofersen fared better than placebo patients,
and that placebo patients switched to the drug in the extension
phase of the study experienced similar gains.
"Despite the fact that there was no statistically significant
difference in the primary endpoint, there is a clinical signal
here," said Dr. Timothy Miller, the study's lead investigator and
ALS Center Director at Washington University School of Medicine, St.
Louis. He presented the tofersen data at the annual meeting of the
American Neurological Association.
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 The trial also showed that
patients given tofersen had lower levels of SOD1
protein compared to placebo patients, as well as
lower levels of plasma neurofilament light
chain, a potential marker of nerve cell
degeneration. "That suggests
that there is an effect on clinical function of the person," Dr.
Miller said.
Most side effects in trial patients were mild to moderate, including
headache and back pain, but two patients experienced spinal cord
inflammation, and 5.6% of tofersen patients dropped out of the
study.
 Globally, around 168,000 people have ALS, a
fatal neurological disorder also known as Lou Gehrig’s disease.
Around 10% of cases are linked to genetic mutations, including SOD1.
Biogen, which licensed tofersen from Ionis Pharmaceuticals Inc, now
plans to open early access to the drug to all patients with
SOD1-associated ALS. In countries allowing such programs, patients
can access a medicine free of charge before it is licensed
commercially.
Shares of Biogen have fallen by about a third after hitting $414 in
early June on the U.S. Food and Drug Administration's controversial
approval of Alzheimer's drug Aduhelm. The agency's decision to
approve the drug based on evidence that it removes protein plaques
associated with the brain-wasting disease, rather than proof that it
improves cognition, led to a backlash that has curtailed use of the
medication while Medicare, the U.S. health plan for seniors, works
to develop payment terms.
(Reporting By Deena Beasley; Editing by Andrea Ricci)
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