Biogen and Ionis’ spinal muscular atrophy drug meets primary endpoint
pharmafile | November 7, 2016 | News story | Manufacturing and Production, Research and Development | Biogen, Ionis, spinal muscular atrophy
Biogen and Ionis Pharmaceuticals have released news that their investigational treatment for spinal muscular atrophy (SMA), Spinraza (nusinersen), met the primary endpoint in interim analysis of their Phase III trial.
Spinal muscular atrophy is a degenerative disorder that is identify by the loss of motor neurons in the spinal cord and lower brain stem, this results in progressive muscular atrophy and weakness. This can then lead onto difficulty with necessary bodily functions, such as breathing and swallowing. The positive interim results of the trial are therefore greatly encouraging both for the companies involved and the patients.
This is of particularl importance as the the clinical trial looked at the treatment of children who suffer from SMA. The interim analysis found that children who received the treatment displayed a mean four point improvement on the Hammersmith Functional Motor Scale Expanded; those that were not treated were found to have declined by a mean of 1.9 points on the same scale.
The study was conducted over the course of fifteen months, with 126 patients who suffer from later-onset SMA. No patients discontinued the trial due to adverse events, indicating that the course of treatment did not induce serious side-effects. The trial was stopped on the interim analysis and the patients will be progressed onto the companies’ Shine extension study.
“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, executive vice president, head of Research and Development at Biogen. “We will make regulators around the globe aware of this data and will continue working closely with them to bring Spinraza to families affected by SMA as quickly as possible.”
“These data further validate the potential of Spinraza as a treatment for patients with SMA,” said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. “We are grateful to all the families and clinicians who have participated in all of the Spinraza studies. Without their commitment and support, this program would not have been able to progress so quickly.”
Ben Hargreaves
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