BrainStorm Cell Therapeutics to Present Data on Longer-Term NurOwn Benefits for ALS Patients

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a prominent developer of stem cell therapies targeting neurodegenerative diseases, is set to present new biomarker data at the 3rd Annual ALS Drug Development Summit. This key event, focusing on identifying critical ALS targets and fostering clinical approvals, will take place from May 21 to 23, 2024, in Boston, MA. Dr. Stacy Lindborg, a member of BrainStorm's Board of Directors, will present findings from the NurOwn Expanded Access Program (EAP) and Phase 3 trial, highlighting potential long-term benefits for ALS patients treated with debamestrocel (NurOwn®).

The data reveal a consistent reduction in neurofilament light (NfL) levels among participants treated with NurOwn in the Phase 3 study. NfL is a significant biomarker for ALS, indicating neurodegeneration and neural cell death. The Phase 3 trial showed an 11% decline from baseline in NfL for patients receiving NurOwn, while those on placebo maintained baseline levels. In the EAP, patients who continued treatment with NurOwn exhibited further reductions: a 27% decrease in Period 1 and a 36% decrease in Period 2 from baseline. This suggests that extended NurOwn treatment may provide continuing benefits.

Participants initially randomized to placebo demonstrated a 37% increase in NfL at the end of the Phase 3 study, but upon receiving NurOwn in the EAP, these patients showed a 17% increase in Period 1 and a 5% decrease in Period 2. These outcomes suggest potential stabilizing effects with extended NurOwn treatment. BrainStorm plans to confirm these findings in a forthcoming Phase 3b trial.

Dr. Lindborg’s presentation, titled "Promising Longer-Term Biomarker Data from NurOwn Program in ALS: Spotlight on NfL in EAP Extension Cohort," will take place on May 22 at 9 am ET at the Hyatt Regency in Boston, MA. The EAP allowed participants from the Phase 3 trial to receive additional doses of NurOwn over two 28-week periods, providing opportunities to measure long-term effects.

BrainStorm’s NurOwn technology platform utilizes autologous MSC-NTF cells, harvested from the patient and processed to secrete neurotrophic factors targeting neurodegenerative pathways. This approach is designed to slow ALS progression, supported by extensive preclinical research and clinical studies. NurOwn has received Orphan Drug designation from both the U.S. FDA and the European Medicines Agency for ALS treatment.

The data to be presented at the ALS Summit underscore BrainStorm's commitment to advancing ALS research and treatment. Antonio Trejo, BrainStorm's VP of Regulatory Affairs, will participate in a workshop on regulatory endpoints meaningful to ALS patients on May 21, while Mary Kay Turner, Senior VP of Global Patient Advocacy & Public Affairs, and Kylan Morris, Senior Manager of Patient Advocacy, will join a panel on trial selection insights from ALS patients on May 22.

BrainStorm's NurOwn program has generated significant insights into ALS pathology and treatment, with findings shared through peer-reviewed publications. The company continues to explore the therapeutic potential of MSC-NTF cells, aiming to bring new hope to ALS patients through ongoing research and rigorous clinical trials.