8-K: BrainStorm Cell Therapeutics Secures FDA Agreement on Phase 3b Trial Design for NurOwn in ALS

BrainStorm Cell Therapeutics has received FDA agreement under a Special Protocol Assessment (SPA) for its Phase 3b trial of NurOwn in amyotrophic lateral sclerosis (ALS).

Summary

• BrainStorm Cell Therapeutics has obtained a Special Protocol Assessment (SPA) agreement from the FDA for its Phase 3b trial of NurOwn in ALS.
• The SPA agreement validates the clinical trial protocol and statistical analysis for the Phase 3b trial, which is intended to support a future Biologics License Application (BLA).
• The Phase 3b trial, named Study BCT-006-US, will be a two-part, multicenter study to assess the efficacy and safety of NurOwn in ALS patients.
• Part A of the trial is a 24-week double-blind, placebo-controlled study, enrolling up to 200 patients randomized 1:1 to NurOwn or placebo.
• Patients will receive three intrathecal injections of NurOwn or placebo every eight weeks.
• All eligible patients completing Part A can enter Part B, a 24-week open-label extension where all participants receive NurOwn.
• The primary efficacy endpoint is the change in the ALSFRS-R total score from baseline to Week 24.
• The primary inference will be based on a combined assessment of function and survival (CAFS).
• The trial will enroll patients with ALS symptoms onset within the prior 24 months, ALSFRS-R items >=2 at screening, and upright slow vital capacity >=65% of predicted.
• The company anticipates commencing the Phase 3b study in 2024.

Sentiment

Score: 8

Explanation: The document conveys a positive sentiment due to the FDA agreement and the progress of the Phase 3b trial. The management's optimism and the detailed trial design contribute to a strong positive outlook.

Positives

• The SPA agreement with the FDA de-risks certain regulatory aspects of the NurOwn clinical program.
• The trial design incorporates feedback from leading neurologists, scientists, and the ALS community.
• The trial will enroll patients earlier in the course of their disease, potentially improving treatment outcomes.
• The company has assembled a highly qualified team to execute the trial.
• The trial includes an open-label extension, allowing all participants to receive NurOwn.

Negatives

• The document does not explicitly mention any negative aspects of the trial or the company's current situation.

Risks

• The company's ability to successfully achieve its goals is subject to risks and uncertainties.
• The company's ability to raise additional capital is a potential risk.
• There are risks associated with the prospects for future regulatory approval of NurOwn.
• The company's future interactions with the FDA may not have productive outcomes.
• The company's ability to continue as a going concern is a risk.

Future Outlook

The company anticipates commencing the Phase 3b study in 2024 and is working towards obtaining regulatory approval for NurOwn as a treatment for ALS.

Management Comments

• Chaim Lebovits, President and Chief Executive Officer, stated that the SPA agreement provides a potential path forward towards obtaining regulatory approval.
• Chaim Lebovits believes that having the SPA in place will help de-risk certain regulatory aspects of the NurOwn clinical program.
• Stacy Lindborg, Co-CEO, mentioned that the trial builds on valuable insights from earlier studies and will enroll people living with ALS who are earlier in the course of their disease.
• Stacy Lindborg stated that they are optimistic the trial will be positive and they are anxious to get started.

Industry Context

This announcement is significant for the ALS treatment landscape, as it represents a step forward in the development of a potential new therapy. The SPA agreement with the FDA is a positive signal for the regulatory pathway of NurOwn.

Comparison to Industry Standards

• The use of a Special Protocol Assessment (SPA) is a common practice for companies seeking regulatory approval for new drugs, particularly in areas with high unmet medical needs like ALS.
• The Phase 3b trial design, including the use of a placebo-controlled arm and a combined assessment of function and survival (CAFS), aligns with industry standards for clinical trials in ALS.
• Other companies developing ALS treatments, such as Amylyx Pharmaceuticals with Relyvrio, have also gone through rigorous clinical trial processes and regulatory reviews.
• The enrollment criteria for the trial, focusing on patients earlier in the course of their disease, is consistent with the trend in ALS research to intervene earlier for better outcomes.
• The use of intrathecal injections for drug delivery is a method being explored by other companies in the neurodegenerative space.

Stakeholder Impact

• Shareholders are likely to view the SPA agreement positively, as it reduces regulatory risk.
• Patients with ALS and their families may have increased hope for a new treatment option.
• The medical community will be interested in the results of the Phase 3b trial.
• Employees of BrainStorm will be involved in the execution of the trial.

Next Steps

• The company will review the protocol with investigators.
• The company will secure study site Institutional Review Board approvals.
• The company will engage with appropriate members of the ALS community.
• The company anticipates commencing the Phase 3b study in 2024.

Key Dates

Keywords

NurOwn, ALS, amyotrophic lateral sclerosis, Special Protocol Assessment, SPA, Phase 3b trial, FDA, Biologics License Application, BLA, stem cell therapy, clinical trial