NASDAQ:BCLI
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Business Update
Ready to Move Ahead with Phase 3b Trial for NurOwn in ALS
On July 8, 2024, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) conducted a conference call to provide an update on the company’s NurOwn™ program. The company is currently preparing for a Phase 3b clinical trial of NurOwn in patients with amyotrophic lateral sclerosis (ALS). We anticipate the trial initiating in the fourth quarter of 2024 or the first quarter of 2025. Over the past few months, BrainStorm has made considerable progress in trial preparation, including the following:
– In March 2024, the company presented the proposed design for the Phase 3b trial in a poster at the MDA Clinical and Scientific Conference. The Phase 3b trial will be a two-part, multicenter study that will enroll ALS patients who are earlier in the course of their disease (onset of ALS symptoms, including limb weakness, within the prior 24 months), with all ALSFRS-R items ≥ 2 at screening, and upright slow vital capacity ≥65% of predicted for gender, height, and age. Part A of the trial will have a double blind, placebo controlled period of 24 weeks. Up to approximately 200 patients are expected to be enrolled and randomized 1:1 to receive NurOwn or placebo. There will be a screening period of six to nine weeks, during which time eligible participants will undergo a single bone marrow aspiration to procure the mesenchymal stem cells (MSCs) to produce NurOwn for the duration of the trial. Participants will receive either NurOwn or placebo via three repeated intrathecal injections once every eight weeks. All participants who complete Part A will be eligible to enroll into Part B, an open label extension period of 24 weeks where all participants will receive three repeated injections of NurOwn once every eight weeks. The primary endpoint of the trial will be the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating (ALSFRS-R) total score from baseline to Week 24. Cerebrospinal fluid (CSF) and blood samples will be collected for analysis of biomarkers of neuroinflammation, neurodegeneration, and neuroprotection. An independent Data Monitoring Committee will be established to monitor the safety of trial participants.
– In April 2024, the company announced it had received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) on the design of the Phase 3b trial. The SPA agreement indicates that the FDA concurs with the adequacy and acceptability of specific elements of a protocols overall design to ensure that the trial conducted under the protocol can be considered an adequate and well-controlled study to support marketing approval.
– In June 2024, the company announced it reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspect of the Phase 3b trial. This means that the company has resolved all previous outstanding CMC issues that the FDA. Given the complexities of cell therapy products, reaching alignment with the FDA ahead of the Phase 3b trial is an additional means to lower the regulatory risk for NurOwn.
The company has selected a contract research organization (CRO) to conduct the Phase 3 trial and is currently in the process of building a network of 12 leading clinical centers in a wide range of locations to maximize patient access and representation. During the quarterly conference call, management also indicated that the company is pursuing various nondilutive funding opportunities, including a promising grant application with the potential to secure up to $15 million.
Financial Update
On August 14, 2024, BrainStorm announced financial results for the second quarter of 2024. As anticipated, the company did not report any revenues during the second quarter of 2024. Net R&D expenses for the second quarter of 2024 were $0.9 million, compared to $2.8 million during the second quarter of 2023. The decrease was primarily due to decreased costs related to the Phase 3 clinical trial and payroll expenses. G&A expenses for the second quarter of 2024 were $2.1 million compared to $2.7 million for the second quarter of 2023. The decrease was primarily due to lower payroll, rent, stock-based compensation, and other costs.
The company exited the second quarter of 2024 with approximately $3.5 million in cash, cash equivalents, and short-term deposits. In June 2024, BrainStorm announced a $4.0 million registered direct offering through the sale of approximately 11.1 million shares of common stock at a purchase price of $0.36 per share along with approximately 16.7 million warrants with an exercise price of $0.3912 per share. As of August 9, 2024, BrainStorm had approximately 79.7 million common shares outstanding and, when factoring in stock options and warrants, a fully diluted share count of approximately 103.8 million.
Conclusion
BrainStorm remains on track to initiate the Phase 3b trial of NurOwn in ALS patients as the regulatory aspects of the program have been derisked through reaching alignment with the FDA on the CMC aspects of the trial and having a SPA in place. We look forward to additional updates on the Phase 3b trial, including the potential for nondilutive funding, as the company gets closer to initiating it. With no changes to our model our valuation remains at $2.40.
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