Brainstorm Cell Therapeutics (BCLI) Status Update summary

Regulatory and clinical trial progress

• Achieved alignment with the FDA on CMC aspects for the upcoming phase III-B ALS trial after a productive Type C meeting in June 2024.

• The phase III-B trial design, endpoints, and statistical analysis are set under an SPA agreement with the FDA, ensuring data acceptance for a future BLA if expectations are met.

• A leading CRO with ALS and cell therapy expertise has been contracted, with clinical sites lined up and preparations underway to dose the first patient before year-end.

• Entry criteria for the trial focus on early-stage ALS patients, with a shortened screening period and higher baseline ALSFRS scores to target the subgroup most likely to benefit.

• Fast enrollment is anticipated due to increased trial sites and strong patient interest.

Manufacturing and operational updates

• Secured a commercial manufacturing partner to ensure BLA readiness and reduce time to market if the trial is successful.

• Technology transfer and agreements with the manufacturer are in progress, following FDA alignment on release criteria and product specifications.

• Dr. Haro Hartounian, with extensive cell and gene therapy experience, has joined as COO to oversee operations, CMC, and commercialization.

Financial and strategic developments

• Raised $4 million in gross proceeds from a single institutional investor to fund near-term milestones, including trial initiation.

• Additional financing options, including non-dilutive grants, are being pursued to fully fund the phase III-B trial.

• Strategic focus on achieving milestones to enhance company value and avoid a reverse stock split.