BRAINSTORM CELL THERAPEUTICS INC. Q3 FY2022 earnings call

• The FDA issued a refusal to file letter for the NurOwn BLA, citing clinical (trial missed primary endpoint) and CMC issues; Brainstorm believes the Phase 3 trial's totality of evidence supports approval and will pursue a Type A meeting.
• Presentations at ALS ONE and NEALS Meetings shared new biomarker analyses and sensitivity analyses for the Phase 3 ALS trial, showing NurOwn's consistent biological effects.
• Phase 2 trial in progressive MS published in a peer-reviewed journal and presented at ECTRIMS, demonstrating safety and improvements in neurological function, cognition, and CSF biomarkers.
• Financials: Cash, cash equivalents, and short-term bank deposits were ~$7.4M as of Sept 30, 2022 (down from ~$22.1M in Dec 2021); R&D expenses $3.8M, G&A $3.1M, net loss $6.9M.

No distinct product segments with revenue contributions were discussed; the focus was on the NurOwn therapy and related clinical and financial aspects.

• Pursue a Type A meeting with the FDA within 30 days of receiving the refusal to file letter to discuss path forward, which could include additional data or AdCom request.
• Consider a confirmatory trial for ALS as part of the Type A meeting discussions.
• Evaluate opportunities for filings in other countries but primary focus remains with the FDA.

• Regulatory risk: Uncertainty around FDA approval due to the refusal to file letter and the need to address clinical and CMC issues.
• Clinical trial risk: Dependence on successful outcomes in trials and AdComs for gaining approval.
• Financial risk: Uncertainty in funding future operations and R&D without additional capital.

Q: Can you give more information about the CMC issues and their cost?

A: CMC issues are trivial, not costly, mostly review questions with no red flags outside clinical comments.

Q: Explain the floor effect and why it needs to be accounted for in the trial?

A: Floor effect in ALSFRS-R prevents measuring disease progression in some participants; needs to be controlled for to see true treatment effect.

Q: Narrowing the scope of the application to support ALSFRS-R patients with score ≥26?

A: Legal counsel advises not to go into detail as still before Type A meeting.

Q: Biomarkers for patients with low ALSFRS-R scores?

A: Consistent biomarker patterns seen in all trial participants, with pre-specified analysis linking biomarkers to clinical outcomes.

Q: EAP data and patient benefits?

A: EAP helped patients, but regulatory restrictions prevent detailed comment on specific patient data, but Brainstorm pursues all leads professionally.