8-K: BrainStorm Reports Remarkable 90% Five-Year Survival Rate for ALS Patients in NurOwn Expanded Access Program

BrainStorm Cell Therapeutics announced new survival data from its NurOwn Expanded Access Program for ALS, showing 90% of participants survived over five years from symptom onset, significantly exceeding typical ALS survival rates.

Summary

• BrainStorm Cell Therapeutics Inc. announced new survival data from 10 participants in its Expanded Access Program (EAP) for NurOwn (autologous MSC-NTF cells) in amyotrophic lateral sclerosis (ALS).
• 90% of participants (9 out of 10) survived more than five years from the onset of ALS symptoms.
• This survival rate significantly contrasts with published estimates, which indicate that approximately 10% of individuals with ALS survive beyond five years.
• The median survival observed in the EAP cohort was 6.8 years, with a range of 6 to 7 years from symptom onset.
• The single death in the cohort occurred following elective euthanasia.
• 6 out of 10 patients are still alive, even post seven years from symptom onset.
• EAP participants had a Mean ALSFRS-R at Baseline of 35.8 (Range: 32-40) at the start of the Phase 3 trial, and 31.4 (Range: 27-38; with one outlier score of 13) at the start of the EAP.
• These findings are expected to support the upcoming Phase 3b clinical trial of NurOwn, which will be conducted under an FDA Special Protocol Assessment (SPA).

Sentiment

Score: 9

Explanation: The survival data for NurOwn in ALS patients from the Expanded Access Program is exceptionally positive, showing a 90% five-year survival rate compared to a typical 10% rate for ALS patients. This represents a significant potential breakthrough for a devastating disease.

Positives

• 90% of participants (9 out of 10) in the Expanded Access Program survived more than five years from the onset of ALS symptoms.
• This survival rate is remarkably higher than the published estimate of approximately 10% for ALS patients surviving beyond five years.
• The median survival observed in the EAP cohort was 6.8 years (range: 6 to 7 years) from symptom onset.
• 6 out of 10 patients are still alive more than seven years from symptom onset.
• The data provides strong real-world insights into the long-term experience of ALS patients treated with NurOwn.
• The positive survival data is expected to support the upcoming Phase 3b clinical trial of NurOwn under an FDA Special Protocol Assessment (SPA).

Risks

• Management's ability to successfully achieve its goals.
• BrainStorm's ability to raise additional capital.
• BrainStorm's ability to continue as a going concern.
• Prospects for future regulatory approval of NurOwn.
• Whether BrainStorm's future interactions with the FDA will have productive outcomes.
• Inherent uncertainties, risks, and assumptions associated with forward-looking statements.

Future Outlook

The company plans to advance an upcoming Phase 3b clinical trial of NurOwn in ALS, to be conducted under an FDA Special Protocol Assessment (SPA), with the goal of generating high-quality and conclusive data to demonstrate the benefit of NurOwn. They also continue to advance a proprietary, allogeneic exosome-based platform.

Management Comments

• "These survival data provide encouraging real-world insights into the long-term experience of ALS patients treated with NurOwn under expanded access. We believe these findings add further evidence to support the upcoming Phase 3b clinical trial of NurOwn, to be conducted under an FDA Special Protocol Assessment (SPA). Our goal remains to generate high quality and conclusive data to demonstrate the benefit of NurOwn in people living with ALS." Chaim Lebovits, President and CEO of BrainStorm Cell Therapeutics.
• "The fact that 9 out of 10 EAP participants surpassed five years of survival from first symptom in contrast with the very modest expected survival pattern typically seen in ALS represents a clinically meaningful observation. Furthermore, 6 out of 10 patients are still alive, even post seven years from symptom onset. These data further strengthen the scientific rationale to pursue our pivotal Phase 3b trial of NurOwn in ALS, under full alignment with the FDA guidance." Bob Dagher, MD, Chief Medical Officer of BrainStorm Cell Therapeutics.

Industry Context

This announcement is significant within the neurodegenerative disease and biotechnology sectors, particularly for ALS, a devastating disease with limited treatment options and a typically poor prognosis. The reported extended survival rates, if replicated in controlled trials, could represent a major breakthrough, positioning NurOwn as a potentially transformative therapy. The focus on a Phase 3b trial under FDA SPA indicates a clear regulatory path forward, which is crucial for biotech companies.

Comparison to Industry Standards

• The observed 90% survival rate beyond five years for NurOwn-treated ALS patients in the EAP cohort significantly surpasses the published industry standard of approximately 10% survival beyond five years for individuals with ALS, as referenced from the National Institute of Neurological Disorders and Stroke (NINDS), NIH.
• This data suggests a potentially superior outcome compared to current standard of care or other investigational therapies for ALS, which typically show only modest improvements in disease progression or survival.
• While specific comparable companies or projects are not named in the document, the stark contrast to the general ALS prognosis highlights NurOwn's potential differentiation in a field where extended survival is rare.

Stakeholder Impact

• Shareholders: Potentially very positive due to highly encouraging clinical data, which could lead to increased stock value if the Phase 3b trial is successful and regulatory approval is achieved.
• ALS Patients: Highly positive, as NurOwn could offer a significant improvement in survival and quality of life for a devastating neurodegenerative disease with limited treatment options.
• Employees: Positive, as the company's prospects improve, potentially leading to job security and growth opportunities.
• Regulatory Authorities (FDA): The data strengthens the rationale for the upcoming Phase 3b trial and continued engagement with the FDA.

Next Steps

• Launch of a pivotal Phase 3b clinical trial of NurOwn in ALS under a Special Protocol Assessment (SPA) agreement with the FDA.
• Generate high quality and conclusive data to demonstrate the benefit of NurOwn in people living with ALS.
• Continue advancing the proprietary, allogeneic exosome-based platform.

Key Dates

Recommendation

Keywords

ALS, Amyotrophic Lateral Sclerosis, NurOwn, MSC-NTF cells, Stem Cell Therapy, Neurodegenerative Diseases, Clinical Trial, Expanded Access Program, Survival Data, BrainStorm Cell Therapeutics, FDA Special Protocol Assessment, Biotechnology, Orphan Drug, Multiple Sclerosis, Exosome Technology