BrainStorm Cell Therapeutics Advances NurOwn® with FDA Submission
BrainStorm Cell Therapeutics Drives Forward with IND Amendment Submission
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a prominent name in biotechnology focusing on innovative cell therapies for neurodegenerative diseases, recently made a significant move by submitting an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for their groundbreaking treatment, NurOwn®. This pivotal step not only reinforces their commitment to advancing ALS therapies but also positions them for the highly anticipated Phase 3b clinical trial.
Exciting Developments for Phase 3b Trial of NurOwn®
NurOwn® is an autologous mesenchymal stem cell therapy aimed at addressing amyotrophic lateral sclerosis (ALS). The FDA has collaborated with BrainStorm to ensure the trial’s design meets the necessary standards through a Special Protocol Assessment (SPA). This agreement underlines that the trial's design and statistical plan are aligned with FDA expectations, which is crucial for the ultimate goal of submitting a Biologics License Application (BLA) in the future.
CEO Insights on Trial Progress
"The submission of this IND amendment and the accompanying FDA agreement represent a landmark milestone toward making NurOwn available to those affected by ALS," stated Chaim Lebovits, the CEO of BrainStorm Cell Therapeutics. He expressed optimism regarding a swift regulatory review and emphasized the eagerness to begin the Phase 3b study, which aims to support regulatory approval effectively.
Trial Details and Objectives
The Phase 3b trial is set to enroll around 200 participants battling ALS, employing a dual-part structure. Initially, in Part A, participants will be administered either three doses of NurOwn or a placebo during a 24-week double-blind period. Subsequently, everyone—including those who received placebo—will have the opportunity to enter Part B, where they will receive additional doses of NurOwn over another 24 weeks. A key objective of the trial will be to assess changes in ALSFRS-R scores, tracking disease progression and comparing results between the treatment and placebo groups.
Commitment to ALS Community
BrainStorm acknowledges the pressing need for effective treatments for ALS—a disease marked by limited therapeutic options. They are dedicated to collaborating with regulatory agencies, clinical investigators, and the ALS community to ensure a rigorous and expedited evaluation process for NurOwn®. The company plans to provide ongoing updates as the FDA review advances, keeping all stakeholders informed about this important journey.
About NurOwn® Technology Platform
NurOwn® technology represents an innovative therapeutic strategy that targets critical disease pathways in neurodegenerative disorders. This platform utilizes autologous bone marrow-derived mesenchymal stem cells (MSCs) that are expanded and differentiated under specific conditions, inducing them to produce high levels of neurotrophic factors (NTFs). The MSCs are transformed into MSC-NTF cells, which are engineered to deliver multiple NTFs and immunomodulatory cytokines directly to areas of damage, with the aim of slowing or stabilizing disease progression.
Corporate Overview of BrainStorm Cell Therapeutics
BrainStorm Cell Therapeutics Inc. is at the forefront of developing innovative therapeutics using adult stem cells to combat debilitating neurodegenerative diseases. With exclusive rights to the NurOwn® technology platform, BrainStorm is focused on the clinical development and commercialization of autologous MSC-NTF cells. This platform has received Orphan Drug designation from the FDA and the European Medicines Agency (EMA) specifically for the treatment of ALS. With a successful Phase 3 pivotal trial already completed, BrainStorm continues to explore additional applications of their technology.
Frequently Asked Questions
What is the purpose of the IND amendment submitted by BrainStorm?
The IND amendment aims to advance the clinical trial for NurOwn®, facilitating its progression through regulatory processes and towards potential approval.
How many participants will be involved in the Phase 3b trial of NurOwn®?
The trial is expected to enroll approximately 200 ALS patients, ensuring a robust dataset for evaluating the treatment's efficacy.
What is the structure of the Phase 3b trial?
The trial will be divided into two parts: Part A includes three doses of either NurOwn or placebo, followed by an open-label extension allowing all participants to receive treatment in Part B.
What is NurOwn® and how does it work?
NurOwn® is an autologous mesenchymal stem cell therapy designed to deliver neurotrophic factors that aim to promote recovery and alleviate the symptoms of ALS.
What is BrainStorm Cell Therapeutics' mission?
BrainStorm's mission is to develop cutting-edge cell therapies for neurodegenerative diseases, with a focus on addressing the unmet medical needs of patients suffering from these conditions.
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