BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, has presented new biomarker data from the Phase 3 trial of its late-stage investigational ALS treatment, NurOwn, at the 2023 ALS and Related Motor Neuron Diseases Gordon Research Conference.
The data showed that treatment with NurOwn significantly elevated markers of neuroprotection and lowered markers of neuroinflammation and neurodegeneration, including neurofilament light (NfL) over time compared to a placebo in all trial participants.
The new data analysis was motivated by the regulatory precedent set by a recent FDA drug approval in ALS, suggesting that the blood-based biomarker neurofilament light (NfL) is associated with disease prognosis and progression in patients with ALS and potentially other neurodegenerative diseases. Reductions in plasma NfL are believed to be likely to predict clinical benefit in ALS.
The analysis examined biomarkers that were pre-specified in the NurOwn study: 16 pro-inflammatory/anti-inflammatory, eight neurodegeneration, and nine neuroprotection biomarkers. This analysis is the largest biomarker study conducted in people living with ALS. The data provide further evidence of the importance of NfL as a prognostic and predictive biomarker.
The Phase 3 pivotal trial NurOwn did not reach statistical significance on the primary or secondary endpoints, likely due to a “floor effect,” which confounds measurement of disease progression in patients with more advanced disease.
However, a thorough analysis of NurOwn Phase 3 data shows evidence of clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression.
The NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. BrainStorm previously announced the FDA intention to hold an ADCOM meeting to review NurOwn for the treatment of ALS.
The company filed a BLA for NurOwn on September 9, 2022, and received a Refusal to File (RTF) letter from FDA on November 8, 2022. Following a Type A meeting and subsequent discussions with the FDA BrainStorm requested that CBER utilize the FDA’s “File Over Protest” procedure, which offers the shortest amount of time to complete the regulatory process.
The BLA was filed over protest, allowing completion of the regulatory process in the shortest time possible, and active review resumed on February 7, 2023.