Slide 1: Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics Nasdaq: BCLI
Slide 2: Safe Harbor Statement
Safe Harbor Statement Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause Brainstorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such f
Slide 3: Brainstorm Cell Therapeutics: A Clinical Stage Biotechnology Company
Brainstorm Cell Therapeutics: A Clinical Stage Biotechnology Company Brainstorm pioneers autologous cell therapies for neurological disorders, focusing on ALS Our Mission and MS Flagship NurOwn® - a cutting-edge autologous cell therapy platform Product Strong Pipeline NurOwn® innovative therapy serv
Slide 4: Pioneering Breakthrough: First-In-Class Therapy to Transform ALS Treatment
Pioneering Breakthrough: First-In-Class Therapy to Transform ALS Treatment Promising data from our previous trials continues to support the therapeutic potential of NurOwn®, demonstrating meaningful improvements in key areas. Robust biomarker data strongly supports NurOwn®’s mechanism of action. Ali
Slide 5: Innovating Cell Therapies for Neurodegenerative Diseases
Innovating Cell Therapies for Neurodegenerative Diseases Indication Preclinical Phase 1 Phase 2 Phase 3 NeurOwn® MSC-NTF Cellular Platform Phase 3B ALS* NurOwn® Phase 3 Progressive MS Parkinson's Disease MSC-NTF Exosomes Platform ARDS 5 *dependent on future funding
Slide 6: Experienced Team in Cell Therapy Development
Experienced Team in Cell Therapy Development Proven Expertise Decades of experience in developing innovative cell therapies across multiple therapeutic areas. Successful Track Led the development of NurOwn®, producing over 470 GMP clinical batches for 190+ ALS trial. Record Extensive experience acro
Slide 7: Proven Leadership with Deep Expertise in Biotechnology
Proven Leadership with Deep Expertise in Biotechnology Chaim Lebovits Haro Hartounian, PhD Bob Dagher, MD Uri Yablonka President and CEO COO CMO CBO Mary Kay Turner Netta Blondheim-Sharga, Yossef Levy, PhD Leticia Tarilonte SVP, Advocacy and Government PhD VP. Cell Production VP, Global Clinical Ope
Slide 8: Looking Ahead: Key Upcoming Milestones
Looking Ahead: Key Upcoming Milestones Clinical Trial Agreements (CTA) Finalize agreements with leading clinical sites, setting the stage for a successful Phase 3b trial. Signed 15 Clinical Sites Officially unveil our partnership with 15 premier clinical sites, ensuring broad geographic coverage and
Slide 9: Our Technology
Our Technology Cutting-Edge Innovation: Advancing the Future of Cell Therapy
Slide 10: Amyotrophic Lateral Sclerosis- ALS
Amyotrophic Lateral Sclerosis- ALS Devastating disease with hope on the horizon through Brainstorm’s groundbreaking treatment Normal motor neuron Motor neuron damaged in ALS 10
Slide 11: Developing NurOwn as a Groundbreaking Platform to Treat ALS
Developing NurOwn® as a Groundbreaking Platform to Treat ALS ALS is a progressive neurodegenerative disease that impairs walking, talking, eating, and breathing, and is currently 100% fatal • Survival is 2 to 5 years from onset, with a lifetime risk of 1 in 300 people. • ALS affects 1.6 per 100,000
Slide 12: NurOwn: Harnessing MSC Characteristics and NTF Production
NurOwn®: Harnessing MSC Characteristics and NTF Production MSCs NTFs Intrinsic immunomodulation properties Multiple evidence for neuroprotection Tropism to sites of damage Deficient in several neurodegenerative In ALS murine models diseases, including ALS • Delay motor neuron degeneration Single NTF
Slide 13: Our Technology: Transforming ALS Treatment with Advanced Cell Therapy
Our Technology: Transforming ALS Treatment with Advanced Cell Therapy NurOwn®: Uses the patient’s own cells, reducing immune rejection risks, contributes to the high tolerance and good safety profile, and enhancing natural neurorepair. Introduced to the spinal cord CSF, MSC-NTF cells secrete bioacti
Slide 14: NurOwn: Tipping the Balance to Favor Neuroprotection
NurOwn®: Tipping the Balance to Favor Neuroprotection Biomarkers in CSF from ALS patients CSF biomarker changes following NurOwn demonstrate pathological hallmarks of treatment suggest a favorable disruption active neurodegenerative and of pathological processes: neuroinflammatory processes • Reduct
Slide 15: Robust biomarker data strongly supports NurOwn's mechanism of action
Robust biomarker data strongly supports NurOwn®’s mechanism of action Changes in biomarkers across main-pathways following NurOwn treatment Inflammatory pathway Anti-Inflammatory pathway MCP-1 Fetuin-A OPG IL-37 S100B LAP (TGF-β1) SDF-1a MSR1 CHI3L1/YKL-40 hsa-miR-146a-5p Chitotriosidase-1 hsa-miR-1
Slide 16: Biomarkers Change Following NurOwn Treatment
Biomarkers Change Following NurOwn Treatment Illustrative approach to demonstrate the cumulative biomarker changes over time following NurOwn treatment, compared to placebo: Change from placebo, neuroprotective markers Change from placebo, neurodegenerative and neuroinflammatory markers 16 Lindborg
Slide 17: The NurOwn Process: Step by Step
The NurOwn® Process: Step by Step The patient’s bone marrow is harvested and Mesenchymal Stromal Cells (MSCs) are isolated from the total bone marrow population. The MSCs are expanded ex-vivo and cryopreserved. Cryopreserved MSCs are thawed, expanded and induced to differentiate into MSC-NTF cells (
Slide 18: Manufacturing Expertise: Delivering Quality and Scale for NurOwn
Manufacturing Expertise: Delivering Quality and Scale for NurOwn ® 470+ GMP Batches Produced: Proven ability to deliver clinical-grade materials at scale, dosing over 190 patients in multiple trials. Successfully executed technology transfer across multiple clinical sites for the production of NurOw
Slide 19: NurOwn: A Breakthrough in ALS Treatment with Promising Clinical Data
NurOwn ® A Breakthrough in ALS Treatment with Promising Clinical Data
Slide 20: Our Milestones: NurOwn Development Program in ALS
Our Milestones: NurOwn® Development Program in ALS Phase 1/2 Phase 2a Phase 2 Phase 3 EAP Phase 3b N=12 N=14 N=48 N=189 N=10 N=200 Orphan Drug Fast Track FDA SPA Designation Designation Agreement 20
Slide 21: Primary Endpoint ALS Functional Rating Scale- Revised (ALSFRS-R)
Primary Endpoint ALS Functional Rating Scale- Revised (ALSFRS-R) Change in ALSFRS-R slope (rate of disease progression) >20-25% is clinically meaningful2 Bulbar Function Validated approvable questionnaire-based tool Respiratory Function • Dyspnea Used as basis for approval of Radicava in 2017 with m
Slide 22: NurOwn: Phase 3 Clinical Trial in ALS (BCT-002-US)
NurOwn®: Phase 3 Clinical Trial in ALS (BCT-002-US) Subgroup analysis: positive results in early disease population Pre-specified subgroup with baseline ALSFRS-R ≥35 Sensitivity analyses across different cutoffs: (N=58) Baseline ALSFRS-R Threshold from ≥26 to ≥35 • Change from baseline in ALSFRS-R t
Slide 23: Clinical Data Summary
Clinical Data Summary NurOwn® demonstrated a significant treatment effect in subgroup of patient with less advanced disease. More advanced ALS patients did not show a treatment benefit, likely due to a floor effect. Data reinforces the safety of repeat intrathecal administration. The next trial will
Slide 24: NurOwn: Optimized Phase 3b Trial in Early ALS Disease
NurOwn®: Optimized Phase 3b Trial in Early ALS Disease
Slide 25: NurOwn: Phase 3 Clinical Trial in ALS (BCT-006-US)
NurOwn®: Phase 3 Clinical Trial in ALS (BCT-006-US) Part A: Part B: Double Blind Open Label NurOwn® NurOwn® Screening R Placebo NurOwn® Randomization 9 weeks 24 Weeks 24 Weeks Intrathecal Injection every 8 weeks Upon successful completion of our double-blind Phase 3b study, we will be positioned to
Slide 26: NurOwn Phase 3b (BCT-006): Key Design Criteria
NurOwn® Phase 3b (BCT-006): Key Design Criteria Significant changes made to upcoming Phase 3b clinical trial Phase 3 (BCT-002-US) Phase 3b (BCT-006) Patient ≥25, followed by 20-week “run-in” period, allowing the Early-disease participants: ALSFRS-R criteria targeting less population possibility to e
Slide 27: NurOwn: Positioned for Success
NurOwn®: Positioned for Success Proven Expertise Our team has extensive experience in conducting clinical trials, including Phase 1, 2, and 3 studies. Regulatory Strong alignment from the FDA with Special Protocol Assessment (SPA) approval, ensuring trial design Alignment meets regulatory standards
Slide 28: Looking Ahead: Key Upcoming Milestones
Looking Ahead: Key Upcoming Milestones Clinical Trial Agreements (CTA) Finalize agreements with leading clinical sites, setting the stage for a successful Phase 3b trial. Signed 15 Clinical Sites Officially unveil our partnership with 15 premier clinical sites, ensuring broad geographic coverage and
Slide 29: Thank you
Thank you Chaim Lebovits President & CEO Brainstorm Cell Therapeutics 1325 Avenue of Americas NY, NY 10019 www..Brainstorrm-cell.com