BRAINSTORM CELL THERAPEUTICS INC. Q4 FY2024 earnings call

• 2024 was a year of foundational work positioning for the Phase 3b trial of NurOwn for ALS.
• Maintained strong collaborative relationship with FDA, secured special protocol assessment in 2024.
• Actively negotiating clinical trial agreements with ~15 leading US clinical centers.
• Partnered with IQVIA for clinical research and Pluri Inc. for clinical manufacturing of NurOwn; planning to bring additional US-based manufacturing center online.
• Pursuing multiple funding avenues including warrant inducement, licensing non-core assets, and non-dilutive grants.

Research and development expenditures net for the year ended December 31, 2024 were $4.7 million, compared to $10.7 million in 2023. General and administrative expenses for the 12 months ended December 31, 2024 were approximately $7 million, down from $10.7 million in 2023. Net loss for the 12 months ended December 31, 2024 was approximately $11.6 million or $2.31 per share, compared to a net loss of approximately $17.2 million or $6 per share in 2023. Cash, cash equivalents and restricted cash were approximately $0.4 million as of December 2024, down from $1.5 million in 2023.

• Anticipates needing $20 million to $30 million annually for the Phase 3b trial.
• Believes market valuation will increase with key milestones like IND model submission, FDA clearance, CTA signings, and first patient enrollment.
• Anticipates announcing submission of IND models shortly.

• Intricate and time-consuming regulatory processes for updating and submitting IND models.
• Financial constraints in raising upfront capital for the trial given market conditions for non-revenue biotech.
• Skepticism regarding the efficacy of NurOwn.

Q: Some investors are concerned about delays in initiating the Phase 3b trial. Can you provide more clarity on the timeline and reasons for perceived delays?

A: Perceived delays are due to intricate regulatory processes like updating and submitting IND models (technical transfer, QA/QC), and time required for negotiating clinical trial agreements with multiple sites.

Q: Can you elaborate on the financial situation and how you plan to secure necessary funding for the trial?

A: Actively pursuing multiple funding avenues including a $1.64 million warrant inducement, licensing non-core assets, and non-dilutive grants like the CIRM grant; confident market valuation will improve with key milestones to secure additional funding.

Q: Some analysts and investors are skeptical about the efficacy of NurOwn. How do you plan to address these concerns?

A: The Phase 3b trial is designed to provide definitive evidence of efficacy; it's de-risked with Special Protocol Assessment, and key opinion leaders in ALS support the program.

Q: What is the status of manufacturing facilities and how will you ensure consistent supply of NurOwn?

A: Contracted with Pluri Inc. for clinical manufacturing, complementing leased facility in Tel Aviv; planning to bring additional US-based manufacturing center online to support production needs.

Q: Given financial constraints, how is the company ensuring operational continuity and starting the Phase 3b trial?

A: Implemented cost-saving measures like salary reductions, senior leadership working without compensation; dedicated team focused on trial progress.

Q: Why not refile the original BLA?

A: Refiling BLA not strategic; focused on generating robust data through Phase 3b trial as per SPA with FDA to support new BLA and approval.

Q: Thoughts on changing environment in cell therapy space and impact on BrainStorm?

A: FDA's special protocol assessment is favorable; working closely with partners, undervalued currently, and confident in ability to pull off trial.

Q: Status of US manufacturing facility and impact on investors?

A: Planning to announce US manufacturing sites soon; partners see upside of product, and manufacturing in US mitigates some investor concerns.

Q: Percentage of financing in place before starting trial and timeline for first patient enrollment?

A: Working closely with partners, other fundings in pipeline including non-dilutive grants in fall; once preparations finalized, first patient enrollment will follow soon