BrainStorm Cell Therapeutics Inc. is a leading biotechnology company committed to the development and commercialization of best-in-class autologous cellular therapies for the treatment of neurodegenerative diseases. The company's proprietary NurOwn® technology platform leverages the power of the patient's own mesenchymal stem cells (MSCs) to produce high levels of neurotrophic factors, modulate neuroinflammation, and promote neuronal survival and function.
Business Overview
BrainStorm's primary focus is the development of NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. ALS is a fatal neurodegenerative disorder that affects motor nerve cells in the brain and spinal cord. The company has completed a Phase 3 clinical trial of NurOwn® in ALS and is now preparing to initiate a Phase 3b trial.In addition to ALS, BrainStorm is also exploring the potential of NurOwn® in other neurodegenerative diseases, such as progressive multiple sclerosis (PMS) and Alzheimer's disease (AD). The company recently completed a Phase 2 study of NurOwn® in PMS, which demonstrated promising results in terms of improving motor and cognitive function.
Financials
BrainStorm has not generated any revenue to date, as the company is still in the clinical development stage of its lead product candidate, NurOwn®. For the fiscal year ended December 31, 2023, the company reported a net loss of $17,192,000, with no revenue generated. The company's annual operating cash flow was -$20,458,000, and its annual free cash flow was -$20,476,000.In the first quarter of 2024, BrainStorm reported a net loss of $3,401,000, with no revenue generated. The company's quarterly research and development expenses were $961,000, down from $2,924,000 in the same period of the previous year. General and administrative expenses were $1,513,000 in the first quarter of 2024, compared to $2,227,000 in the first quarter of 2023.
As of March 31, 2024, BrainStorm had approximately $1,000,000 in cash, cash equivalents, and short-term deposits. In June 2024, the company announced a $4.0 million registered direct offering, which will provide additional funding to support the upcoming Phase 3b trial of NurOwn® in ALS.
Regulatory Progress and Clinical Development
BrainStorm's lead product candidate, NurOwn®, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug status in the U.S. and Europe for the treatment of ALS. The company has completed a Phase 3 clinical trial of NurOwn® in ALS, which, while not meeting the primary endpoint, did demonstrate a clinically meaningful benefit in a pre-specified subgroup of patients with less advanced disease.Following the Phase 3 trial, BrainStorm engaged in extensive discussions with the FDA to determine the best path forward for NurOwn®. In December 2023, the company announced a productive meeting with the FDA, during which the agency agreed to a Special Protocol Assessment (SPA) for the design of a Phase 3b trial of NurOwn® in ALS. The SPA agreement validates the clinical trial protocol and statistical analysis, demonstrating the company's adequacy in addressing the FDA's objectives to support a future Biologics License Application (BLA) submission.
The upcoming Phase 3b trial of NurOwn® in ALS will enroll patients with earlier-stage disease, with the goal of demonstrating a more robust treatment effect. The trial will have a double-blind, placebo-controlled period followed by an open-label extension, and will assess changes in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) as the primary endpoint. BrainStorm expects to initiate the Phase 3b trial before the end of 2024.
In addition to the progress in ALS, BrainStorm has also reported positive results from a Phase 2 study of NurOwn® in PMS. The open-label trial demonstrated improvements in various measures of motor and cognitive function, as well as favorable changes in cerebrospinal fluid biomarkers.
Intellectual Property and Manufacturing
BrainStorm holds a comprehensive intellectual property portfolio related to the NurOwn® technology, with patents granted in the United States, Canada, Europe, Israel, Japan, Hong Kong, Brazil, and Australia. The company has also made significant investments in its manufacturing capabilities, including the establishment of a GMP-certified cleanroom facility in Israel to support the production and distribution of NurOwn® for the European and Israeli markets.Risks and Challenges
As with any biotechnology company, BrainStorm faces several risks and challenges, including the inherent uncertainty of clinical development, regulatory approval, and commercialization. The company's success is heavily dependent on the continued development and potential approval of NurOwn® for the treatment of ALS and other neurodegenerative diseases. Additionally, the company will need to secure additional funding to complete the upcoming Phase 3b trial and prepare for potential commercialization.Outlook
BrainStorm has made significant progress in advancing its NurOwn® technology and preparing for the next stage of clinical development. The company's upcoming Phase 3b trial in ALS, with the SPA agreement from the FDA, represents an important milestone that could further derisk the program and bring the company closer to potential regulatory approval and commercialization.The positive results from the Phase 2 study in PMS also highlight the broader potential of the NurOwn® platform in other neurodegenerative diseases. As BrainStorm continues to execute on its clinical and regulatory strategy, the company's ability to secure additional funding and navigate the complex landscape of cell therapy development will be critical to its long-term success.