17 slides from Brainstorm Cell Therapeutics's technical presentation, Jul 18, 2016
Slide 1: BCT-002 US Phase II Study Topline Results: NurOwn in ALS
ʱʡ ʣ Confidential 1 BCT - 001 - US Phase II Study Topline Results NurOwn ® in ALS July 18, 2016
Slide 2: Safe Harbor Statement
ʱʡ ʣ Confidential 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 state
Slide 3: MSC therapeutic effects
ʱʡ ʣ Confidential MSC therapeutic effects 3 The beneficial effects of MSCs are primarily mediated by paracrine mechanisms Neurogenesis: Induce neuronal growth and differentiation Trophic factors: Secretion of neurotrophic and angiogenic factors Increase reactive astrocytosis: Induce astroglial gro
Slide 4: US clinical trial design
ʱʡ ʣ Confidential 4 US clinical trial A Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study to Evaluate Safety and Efficacy of Transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors (NurOwn ® ) in Patients With Amyotrophic Lateral Sclerosis 1 » 3 In
Slide 5: Subject Disposition & Demographics
ʱʡ ʣ Confidential Subject Disposition & Demographics 5 Disposition MSC - NTF (N=36) Placebo (N=12) All Subjects (N=48) Analysis Populations n (%) n (%) n (%) Safety (ITT) 36 (100.0) 12 (100.0) 48 (100.0) Study Completion Completed 33 (91.7) 10 (83.3) 43 (89.6) Discontinued 3 (8.3) 2 (16.7) 5 (10.4
Slide 6: Medical History
ʱʡ ʣ Confidential Medical History 6 MSC - NTF (N=36) Placebo (N=12) All Subjects (N=48) El Escorial Criteria n (%) n (%) n (%) Possible 3 (8.3) 1 (8.3) 4 (8.3) Laboratory - Supported Probable 5 (13.9) 1 (8.3) 6 (12.5) Probable 16 (44.4) 7 (58.3) 23 (47.9) Definite 12 (33.3) 3 (25.0) 15 (31.3) ALS
Slide 7: Overall Summary of Adverse Events
ʱʡ ʣ Confidential Overall Summary of Adverse Events 7 MSC - NTF N= 36 Placebo N=12 Number of Patients with at least one TEAE 36 (100%) 12 (100%) Number of Patients with at least one Treatment - Related TEAE 35 (97.2%) 9 (75.0%) Number of Treatment - Related Serious TEAEs 0 0 Number of Patients wit
Slide 8: Responder analysis
ʱʡ ʣ Confidential Responder analysis 8 http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3125671/
Slide 9: Efficacy Responder Analysis
ʱʡ ʣ Confidential 9 Efficacy Responder Analysis 26 of 35 5 of 11 19 of 35 4 of 11 12 of 35 4 of 11 10 of 35 1 of 12 7 of 35 0 of 12 4 of 35 0 of 12 T P T P T P T P T P T P 2wks 4wks 8wks 12wks 16wks 24wks T – Treatment; P – Placebo Given the exploratory nature of this study, statistical significan
Slide 10: Efficacy Responder Analysis: ALSFRS-R % Improvement in slope
ʱʡ ʣ Confidential Efficacy Responder Analysis 10 ALSFRS - R % Improvement in post treatment slope compared to pre treatment slope over time T – Treatment; P – Placebo p - values are one - sided from Fisher's Exact test Given the exploratory nature of this study, statistical significance was define
Slide 11: Efficacy Responder Analyses: points change per month
ʱʡ ʣ Confidential Efficacy Responder Analyses 11 p - values using one - sided Fishers Exact Test Given the exploratory nature of this study, statistical significance was defined as a one - sided p value < 0.1 (these p - values are highlighted in red above). In order to see trends in the data p - v
Slide 12: Efficacy Responder Analysis: Change points/month
ʱʡ ʣ Confidential Efficacy Responder Analysis 12 Change points/months 2wks 4wks 8wks 12wks 16wks 24wks T 35 P 11 T 35 P 11 T 35 P 11 T 35 P 12 T 35 P 12 T 35 P 12 ≥0.5 points 64% 36% 47% 36% 42% 36% 36% 17% 25% 8% 22% 17% p=0.1029 ≥1 points 53% 36% 47% 27% 28% 9% 22% 17% 22% 8% 22% 8% ≥1.5 points
Slide 13: Responder Analysis excluding slow progressors
ʱʡ ʣ Confidential Responder Analysis (excluding all patients with pre - treatment slope ≤ - 0.7 (Slow Progressors) 13 0 10 20 30 40 50 60 70 80 90 100 2wks 4wks 8wks 12wks 16wks 24wks % of patients with 100 % improvement ALSFRS - R 100 % improvement excluding slow progressors (pre - treatment slop
Slide 14: ALSFRS-R Slope Change Responder Analyses: Slow Progressors subgroup
ʱʡ ʣ Confidential ALSFRS - R Slope Change Responder Analyses excluding all patients with pre - treatment slope ≤ - 0.7 (Subgroup Analysis: Slow Progressors) 14 % of total (% improvement) 2 wks 4 wks 8 wks 12 wks 16 wks 24 wks T P T P T P T P T P T P 18 5 18 5 18 5 18 5 18 5 18 5 25% improvement 17
Slide 15: Biomarkers
ʱʡ ʣ Confidential Biomarkers 15 » Cerebral - spinal fluid (CSF) samples were collected from patients, as specified per a protocol amendment, after the first 8 patients had already been treated » A total of 35 patients out of 48 had corresponding samples from visits pre - and post - transplantation
Slide 16: Conclusions
ʱʡ ʣ Confidential Conclusions 16 » BCT - 001 achieved its primary objective, demonstrating that NurOwn was safe and well tolerated . » Single administration of NurOwn produced a clinically meaningful beneficial response in terms of both the ALSFRS - R rating scale and CSF biomarkers . » In the ITT
Slide 17: Next Steps
ʱʡ ʣ Confidential Next Steps 17 • Request for FDA Meeting • A Multi - Dose Double Blind Randomized Placebo Controlled Adequately Powered Trial in US and Israel