BrainStorm Hope to Mimic Biogen's Accelerated Approval of ALS Drug
Following the accelerated approval of Biogen and Ionis' amyotrophic lateral sclerosis (ALS) drug, BrainStorm Cell Therapeutics has made the case for its own cell therapy to be available on the US market.
BrainStorm's autologous cell therapy, NurOwn, has survived two prior FDA rejections to secure a third shot at regulatory approval, with an advisory committee scheduled to review the application in the fall.
Ahead of the meeting, BrainStorm has used the 2023 ALS and Related Motor Neuron Diseases Gordon Research Conference to offer a ‘different look' at the phase III ALS drug data.
This analysis was motivated by the recent accelerated approval of Biogen and Ionis' antisense drug Qalsody for ALS patients.
- Talking to Scott Burger, Principal of Advanced Cell and Gene Therapy
- Addressing the Challenges in Cell Line Manufacturing
- Overcoming Regulatory Hurdles Associated with Good Manufacturing Practices
The FDA has conditionally approved Qalsody on the strength of clinical evidence that it reduces plasma neurofilament light (NfL) chains – biomarkers of neuronal damage in different neurological disorders.
In response, BrainStorm has revisited its phase III data to assess the impact of NurOwn on nerve injury and neurodegeneration.
New analysis from BrainStorm has linked treatment with NurOwn to increases in neuroprotection markers, as well as reductions in neuroinflammation and neurodegeneration markers.
Reductions in NfL chains also correlated to improved patient outcomes in clinical trials.
Ensuring ALS Drug Data Accuracy and Integrity
Fierce Biotech quoted BrainStorm co-CEO Stacy Lindborg as saying the time from first symptom to treatment was an important factor to account for in development.
“Baseline physical function is important to understanding the treatment in clinical trials given the greater heterogeneity of the disease, which can influence prognosis,” said Lindborg.
Other drugs approved by the FDA for the treatment of ALS include Relyvrio and Radicava, which both received approval in 2022.
Currently there is no cure for ALS, but there are approaches which can alleviate its symptoms to some extent.
The ALS Association said in a statement on its website that it had ‘consistently requested' access to the full package of data for NurOwn's Phase III trial after BrainStorm disclosed that the trial had not met its primary or secondary endpoints.
It stated that the ‘amazing testimonials' seen around the treatment online did not align with the data shared by BrainStorm or published in peer-reviewed publications.
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