Slide 1: NurOwn Phase 3 Clinical Program in ALS
NurOwn® Phase 3 Clinical Program in ALS • Poster ID: 2 • Category: Mesenchymal Stem/Stromal Cells Bob Dagher, MD Executive Vice President & Chief Medical Officer May 08, 2025
Slide 2: Background on UNC13A & TDP-43 Pathology in ALS
Background on UNC13A & TDP-43 Pathology in ALS • ALS and UNC13A – UNC13A protein critical for synaptic vesicle priming and neurotransmitter release – UNC13A gene variant (rs12608932 C allele) linked to shorter survival and more severe ALS o Dysfunctional UNC13A protein: impaired synaptic transmissio
Slide 3: NurOwn Development Program in ALS
NurOwn® Development Program in ALS Phase 3b Phase 1/2 Phase 2a Phase 2 Phase 3 EAP planned N=12 N=14 N=48 N=189 N=10 N=200 Screening NurOwn (N = 95) R Placebo (N = 94) Wk -20 Wk 0 Wk 8 Wk 16 Wk 28 Run-in Period 20 weeks 28 Weeks R: Randomization Intrathecal Injection every 8 weeks 3
Slide 4: NurOwn Phase 3 Trial: UNC13A Genetic Substudy
NurOwn Phase 3 Trial: UNC13A Genetic Substudy • Baseline Disease Characteristics by UNC13A Genotype – N= 124 participants (63 NurOwn; 61 placebo) – C risk allele (CC & CA): 77 participants (62%) o Homozygous CC (15%): NurOwn n= 9; Placebo n= 10; no comparative analysis were conducted o Heterozygous
Slide 5: UNC13A Genetic Substudy: Primary & Key Secondary Endpoints
UNC13A Genetic Substudy: Primary & Key Secondary Endpoints NurOwn Placebo n=63 n=61 Endpoints AA AC CC AA AC CC n=31 n=23 n=9 n=16 n=35 n=10 Responders*, % 23% 65% 22% 25% 29% 20% ALSFRS-R, points -6.37 -1.90 -6.91 -4.52 -3.75 -5.18 Change from Baseline to Week 28 Neurofilament Light (NfL), % -15% -
Slide 6: Primary Endpoint: Broad Population & UNC13A AC Genotype
Primary Endpoint: Broad Population & UNC13A AC Genotype • % Responders, Overall Population – NurOwn 33% vs Placebo 28%; p= 0.453 • % Responders, AC Genotype NurOwn Placebo – NurOwn 65% vs Placebo 29% p=0.011 35% 29% 65% 71% Responders Non-Responders 6 * Responder definition: ≥ 1.25 points/month impr
Slide 7: UNC13A Genetic Substudy: Interpretation & Conclusions
UNC13A Genetic Substudy: Interpretation & Conclusions • UNC13A genotype influences response to NurOwn therapy • AC genotype carriers showed significant clinical benefit and biomarker improvement • CC genotype associated with severe disease; small sample size limits conclusions • Informative pharmaco
Slide 8: Thank You
is, brainstorm DO?’ cell therapeutics Thank You