#USA #New York #BrainStorm #ALS #NurOwn

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BrainStorm Cell Therapeutics Inc. has recently announced groundbreaking survival data from its Expanded Access Program (EAP) related to its innovative therapy, NurOwn®, aimed at combating amyotrophic lateral sclerosis (ALS). This compelling study, focusing on ten participants who had previously joined the Phase 3 clinical trial, showcases a remarkable survival rate that defies common expectations for ALS patients.

Key Findings of the EAP

Analyzing the survival data from the EAP, the findings suggest that 90% of the participants (9 out of 10) survived more than five years from the onset of ALS symptoms. This statistic is exceptionally striking considering that conventional estimates indicate only about 10% of ALS patients survive beyond five years. The only reported death among the participants happened following elective euthanasia, a fact further emphasizing the improved survival prospects of those involved in the program.

The median survival rate observed among the EAP participants was 6.8 years (with a range of 6 to 7 years) from the initial onset of symptoms. This significant information suggests that the outcomes witnessed in this cohort are improbable to be mere chances of random occurrence.

Professional Insights

Chaim Lebovits, President and CEO of BrainStorm, remarked, "These survival data provide encouraging real-world insights into the long-term experience of ALS patients treated with NurOwn under expanded access." He highlighted that these findings bolster the rationale for an upcoming Phase 3b clinical trial of NurOwn that will proceed under FDA Special Protocol Assessment (SPA).

Dr. Bob Dagher, Chief Medical Officer of BrainStorm, underlined the clinical relevance of the survival rates. He stated, "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, six out of the ten patients are still alive after crossing the seven-year mark from symptom onset."

The EAP was initiated to provide compassionate access to NurOwn for eligible ALS patients who had completed the Phase 3 trial. The information derived from this program underscored the necessity of advancing toward the upcoming registrational Phase 3b controlled clinical trial.

Understanding NurOwn®

NurOwn® utilizes a unique platform that harnesses autologous MSC-NTF cells, derived from a patient’s own bone marrow. These cells are cultivated and differentiated externally to ensure they produce high levels of neurotrophic factors (NTFs). The aim of the therapy is to deliver multiple NTFs and immunomodulatory cytokines directly to the damaged areas within the body. This strategic approach is designed to induce a biological response that can stabilize or slow down the progression of neurodegenerative diseases like ALS.

Looking Ahead

BrainStorm Cell Therapeutics, listed on NASDAQ under the symbol BCLI, is committed to advancing powerful therapies for debilitating neurodegenerative disorders. The company's groundbreaking work with NurOwn® has prioritization from both the FDA and EMA as an Orphan Drug, granting it special designation intended to expedite the development of treatments for rare diseases.

A significant culmination of their research is evident through the insights gained, including pharmacogenomic responses associated with specific genotypes and biomarker data collected at various time intervals throughout the treatment period. Additionally, BrainStorm is exploring the potential of a proprietary, allogeneic exosome-based platform aimed at delivering therapeutic proteins and nucleic acids, reaffirming their dedication to innovative therapies in regenerative medicine.

As the company gears up for the Phase 3b trial, BrainStorm reiterates its commitment to providing conclusive evidence that can lead to profound benefits for ALS patients worldwide. To learn more about their initiatives, please visit BrainStorm Cell Therapeutics.