BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, has announced the outcome of its meeting with the U.S. Food and Drug Administration (FDA) regarding its investigational treatment for amyotrophic lateral sclerosis (ALS), called NurOwn®. The meeting focused on discussing key considerations for a Special Protocol Assessment (SPA) for a planned Phase 3b registrational trial for NurOwn.
The purpose of the SPA is to reach an agreement with the FDA on the design and size of certain clinical trials that adequately address scientific and regulatory requirements. The goal is to secure the FDA’s agreement on critical elements of the trial’s protocol design, such as entry criteria, endpoints, and planned analyses, to support a future marketing application.
BrainStorm intends to submit the necessary documentation as outlined by the FDA to support the SPA. The company views the SPA process as an opportunity to de-risk certain regulatory aspects of the NurOwn clinical program. BrainStorm is dedicated to providing ALS patients with safe and effective treatments and is grateful for the FDA’s ongoing support and guidance.
Chaim Lebovits, the President and CEO of BrainStorm, expressed optimism about the meeting, stating that it provided valuable input on the Phase 3b trial design and outlined clear steps forward. Lebovits emphasized the company’s commitment to reaching a regulatory path forward for NurOwn and ensuring ALS patients have access to the treatment.
A Special Protocol Assessment (SPA) is an essential process for drug developers to gain FDA agreement on the design and size of clinical trials. This agreement indicates the FDA’s concurrence with critical elements of the trial’s overall protocol design. The feedback received during this process aids companies in planning their late-phase development strategy, while it does not imply FDA concurrence on every detail of the protocol.
NurOwn® is BrainStorm’s innovative technology platform that utilizes autologous MSC-NTF cells to target disease pathways associated with neurodegenerative disorders like ALS. These cells are harvested from each ALS patient and manufactured using a proprietary process to secrete neurotrophic factors that target specific neurodegenerative diseases.
The NurOwn clinical program has provided valuable insights into ALS pathology, disease progression, and treatment. BrainStorm has shared the extensive dataset from the program, including an in-depth exploration of the Floor Effect, evaluation of pre-specified biomarkers, and analysis of genetic data. These findings contribute to a better understanding of ALS and hold promise for future treatment development.
BrainStorm Cell Therapeutics Inc. is dedicated to developing innovative autologous adult stem cell therapeutics for neurodegenerative diseases. The company holds the rights to the NurOwn technology platform and has received Orphan Drug designation status from the FDA and the European Medicines Agency for the treatment of ALS.
While awaiting further updates on the SPA, BrainStorm remains committed to advancing the development of NurOwn as a therapy for ALS. The company has completed a Phase 3 trial in ALS and a Phase 2 trial in progressive multiple sclerosis (MS). BrainStorm’s focus on cutting-edge research and collaboration with regulatory agencies demonstrates its dedication to improving the lives of patients with neurodegenerative diseases.
