BRAINSTORM CELL THERAPEUTICS INC. Q1 FY2025 earnings call

• The company announced U.S. FDA cleared initiation of the Phase 3b trial for NurOwn to confirm its therapeutic benefit for early-stage ALS, with the trial design agreed under a Special Protocol Assessment (SPA) which derisks the regulatory pathway. • Chief Operating Officer Dr. Haro Hartounian discussed manufacturing plans, including initial manufacturing at Tel Aviv Sourasky Medical Center and technology transfer to Pluri, and securing a leading U.S. clinical site. • Chief Medical Officer Dr. Bob Dagher summarized the Phase 3b trial design changes, including eliminating the run-in period and shortening the screening period. • Senior Vice President of Research & Development Dr. Netta Blondheim-Shraga discussed biomarkers analysis, preclinical experiments showing NurOwn's immunomodulatory, neuroprotective, and neuroregenerative properties, and progress in the exosome program with promising preclinical data. • Emphasized the team's dedication despite financial constraints and focus on securing funding for the trial.

• Actively pursuing multiple funding avenues including strategic investments and non-dilutive grants to initiate the trial as swiftly as responsibly possible. • Advancing the exosome program towards clinical development through strategic partnerships and expanding intellectual property portfolio.

• Financial constraints, a reality for small biotech companies, which could impact trial initiation. • Regulatory uncertainties related to clinical trial execution and approval. • Need for proper funding to confidently initiate and complete the clinical study.

Q: Can you start the trial without proper funding?

A: While progress has been made with limited resources, initiating and executing the trial demands robust and sustainable cash flow. Securing proper funding is essential, and the company is actively pursuing multiple funding avenues including a $15 million non-dilutive grant and strategic partnerships.

Q: What's the meaning of the trial name ENDURANCE?

A: It pays tribute to the strength, tenacity, and spirit of ALS patients and their families, and underscores the company's commitment to persevere in scientific endeavors for regulatory approval of NurOwn.

Q: Will the company also be producing in the U.S.?

A: Haro Hartounian stated they will announce a letter of intent with a U.S.-based facility with FDA inspection experience, a key step in commercialization and supply chain security.

Q: Update on exosome program?

A: The exosome program shows promising preclinical data in respiratory and inflammatory diseases, with a manuscript on COPD efficacy in preparation, and active pursuit of strategic partnerships to advance to clinical development.

Q: Communications with FDA regarding UNC13A stratification in Phase 3b?

A: Under SPA, protocol details are agreed, but UNC13A stratification is exploratory. The company will post hoc analyze and explore further in the Phase 3b trial.

Q: Mechanism of action from hypoxic stress co-culture and support for BLA?

A: Media from NurOwn cell cultures restored viability of motor neuron cells under hypoxic stress, which is supportive of the IND and may contribute to the BLA data package.

Q: Manufacturing capacity of Tel Aviv facility?

A: It will serve initially, with Pluri and U.S. site to follow, with rolling enrollment in the trial.

Q: Number of clinical trial sites and enrollment estimation?

A: Actively negotiating with ~15 leading clinical centers, but exact enrollment numbers depend on funding and manufacturing progress, with the plan to enroll 200 patients over three years.