- **FDA Milestone Achieved**: Phase 3b trial cleared with SPA, positioning NurOwn for potential approval if endpoints met.
- **Manufacturing Expansion**: U.S. manufacturing partnerships secured, though initial production limited to Tel Aviv.
- **Biomarker Promise**: NfL reductions and UNC13A genotype correlations suggest NurOwn's potential efficacy in specific patient subsets.
- **Funding Critical**: $15M grant and strategic deals needed to initiate enrollment, with no trial start without adequate capital.
- **Exosome Potential**: Preclinical success in lung disease could diversify pipeline if partnerships materialize.

**【CEO Summary Section】**
- FDA cleared the Phase 3b ENDURANCE trial for NurOwn in early-stage ALS patients, with regulatory alignment via Special Protocol Assessment (SPA) and CMC Type C meeting.
- 15 U.S. clinical sites are in advanced negotiation, with one leading site nearing finalization.
- Manufacturing expansion includes technology transfer to Pluri and U.S. cleanroom facilities, with Tel Aviv Sourasky Medical Center for initial production.
- Securing $15M non-dilutive grant and strategic partnerships is critical to initiate enrollment.

**【COO Operational Update】**
- Signed LOI with Pluri for U.S. manufacturing, pending definitive agreement.
- Secured FDA-inspected U.S. clinical site, with details to be announced.
- Scaling manufacturing capacity to support 200-patient trial enrollment over 3 years.

**【CMO Trial Design Details】**
- Phase 3b features 24-week double-blind (Part A) and 24-week open-label (Part B) phases.
- Primary endpoint: ALSFRS-R score change at Week 24, with SPA-agreed statistical plan.
- Simplified protocol removes 3-month run-in and shortens screening to 9 weeks.

**【R&D Biomarker Insights】**
- NurOwn shows anti-inflammatory, neuroprotective, and neuroregenerative effects in preclinical models.
- NfL biomarker analysis suggests NurOwn may stabilize neurodegeneration in early-treated patients.
- UNC13A genotype may predict treatment response, with heterozygous carriers showing better outcomes.

**【Q&A Key Takeaways】**
- FDA not yet ready to approve UNC13A stratification; post-hoc analyses planned.
- Exosome program shows promise in preclinical lung disease models, with IP expansion underway.
- Manufacturing capacity constrained initially, with U.S. sites to ramp up in 2026.
- Enrollment pace dependent on funding and site activation, targeting 200 patients over 3 years.

Brainstorm Cell Therapeutics Inc. (BCLI) Q1 2025 Earnings Call Transcript

- **Trial Readiness**: Phase 3b trial nearing initiation with FDA-aligned design; IND submissions and CTA signings imminent.
- **Funding Strategy**: Secured $1.64M; pursuing grants, asset sales, and partnerships to meet $20-30M annual funding needs.
- **Manufacturing Strength**: GMP-compliant production via Pluri Inc. and planned U.S. expansion for commercial scalability.
- **Team Commitment**: Leadership and staff working reduced pay to advance trial; 35% YoY cost reduction achieved.

**【CEO Opening Remarks】**
- **Phase 3b Trial Progress**: NurOwn's Phase 3b trial for early-stage ALS patients is nearing initiation, with 15 U.S. clinical centers engaged. FDA's Special Protocol Assessment (SPA) reduces regulatory risk, and IND model submissions are expected shortly.
- **Regulatory & Manufacturing Strategy**: Partnering with IQVIA for clinical execution and Pluri Inc. for GMP-compliant manufacturing. A U.S.-based manufacturing center is planned for broader production.
- **Funding Outlook**: Anticipates $20-30M annual funding needs, pursuing non-dilutive grants (e.g., CIRM), asset licensing, and strategic partnerships. Recent $1.64M warrant inducement closes April 1, 2025.
- **Team Dedication**: Senior leadership and staff have reduced or deferred compensation to prioritize trial execution.

**【CFO Financial Highlights】**
- **2024 Financials**: R&D expenses $4.7M (down 56% YoY), G&A $7M (down 35% YoY), net loss $11.6M, cash balance $0.4M.
- **Liquidity Update**: Secured $1.64M via warrant inducement; exploring non-core asset licensing for near-term capital.
- **Cost Management**: Achieved 35% YoY reduction in operating expenses through strategic cost-saving measures.

**【Q&A Key Takeaways】**
- **Trial Timeline Concerns**: Delays due to complex regulatory submissions (e.g., technical transfer, QA/QC) and CTA negotiations. IND model submission and first patient enrollment announcements imminent.
- **Efficacy Addressing Skepticism**: Phase 3b trial designed to replicate positive early-stage data from prior trials. Leading ALS clinicians and FDA alignment support trial design.
- **Manufacturing Assurance**: Pluri Inc. and Sourasky Medical Center facilities ensure GMP compliance. U.S. manufacturing expansion planned for commercial readiness.
- **Financial Resilience**: Team working without full pay to conserve cash. Milestone-driven funding strategy aims to increase market valuation as trial progresses.

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