Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — Investor Presentation (August 2017)

19 slides from Brainstorm Cell Therapeutics's investor presentation, August 2017

  1. Slide 1: BrainStorm Cell Therapeutics Company Update

    BrainStorm Cell Therapeutics NASDAQ: BCLI COMPANY UPDATE August 2017 Chaim Lebovits, CEO Ralph Z. Kern MD, MHSc, COO/CMO

  2. 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

  3. Slide 3: NurOwn technology

    NurOwn® technology • NurOwn® cells are autologous (vs. allogeneic) • NurOwn® cells consistently secrete high levels of multiple NTFs and are immunomodulatory • NurOwn® cells are Photomicrograph Courtesy Prof D. Offen, Tel Aviv University delivered by intrathecal injection Bone marrow is MSCs are MSC

  4. Slide 4: NurOwn shows enhanced in-vitro NTF secretion

    NurOwn® shows enhanced in-vitro NTF secretion (ALS patients and donor cell paired samples) Enhanced in-vitro neurotrophic-factor secretion differentiates NurOwn® from MSC cells of origin in ALS patients GDNF BDNF MSC NurOwn® Individual participant Individual participant VEGF HGF Individual participa

  5. Slide 5: NurOwn successfully evaluated in preclinical models of neurodegenerative disease

    NurOwn® Parkinson’s successfully Disease evaluated in preclinical models of neurodegenerative disease Huntington’s Multiple Disease Sclerosis ALS Autism 5

  6. Slide 6: NurOwn Phase 2a ALS Study: Improvement in ALSFRS-R slope and FVC

    NurOwn® Phase 2a ALS Study: Improvement in ALSFRS-R slope and FVC The mean (±SEM) of the ALSFRS-R score and FVC (% predicted) of patients with ALS treated by combined intrathecal and intramuscular administration in the phase 2a study. β is the slope determined by linear regression analysis of the pr

  7. Slide 7: BCT-001-US Phase 2 Clinical Trial Study Design

    NurOwn® BCT-001-US Phase 2 Clinical Trial Study Design Pre-treatment ALSFRS-R slope Post-treatment slope (mean, responder analysis, excluding slow progressors) Screening BMA Transplantation End of Study (V1) (V4) (V5) (V10) -3-5 weeks 0 24-36 weeks -12-16 weeks CSF Collection (V5 and V6) ~ 9 - 10 mo

  8. Slide 8: Phase 2 ALS Study participant disposition and demographics

    Phase 2 ALS Study participant disposition and demographics- Baseline characteristics balanced MSC-NTF Placebo All Participants Disposition (N=36) (N=12) (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.

  9. Slide 9: BCT-001-US ALS Clinical Trial: Endpoints

    NurOwn® BCT-001-US ALS Clinical Trial: Endpoints • Primary: – Safety and tolerability • Secondary: – ALSFRS-R – Slow vital capacity (SVC) • Exploratory: – Muscle strength (HHD) – CSF Biomarkers (added after the 1st 8 participants) 9

  10. Slide 10: Treatment was Safe and Well-Tolerated

    NurOwn® BCT-001-US ALS Clinical Trial: Treatment was Safe and Well-Tolerated  No deaths  No treatment-related SAEs observed  No dropouts related to AEs  Most common adverse events (>15%; most mild/mod severity): Adverse Event NurOwn® (%) Placebo (%) Headache and Procedural Headache 80.6 66.7 Bac

  11. Slide 11: Phase 2 ALSFRS-R efficacy outcomes

    Phase 2 ALSFRS-R efficacy outcomes: ALSFRS-R- 48 points in 12 Mean change (post transplantation- pre transplantation) subscales 1.5 point/month responder analysis- reversal 100%+ improvement in ALSFRS-R slope- halt 11

  12. Slide 12: Phase 2 ALSFRS-R Slope Change

    NurOwn® Phase 2 ALSFRS-R Slope Change: Cell therapy produced improvement in mean ALSFRS-R slope All participants (n=46) Excluding Slow progressors (n=21) Pre-treatment Change from post-treatment to pre treatment slope Pre-treatment Change from Post-treatment to pre treatment slope 4 4 * 3 3 2 2 * LS

  13. Slide 13: Responder Analysis: ≥1.5 points/month improvement

    NurOwn® Phase 2 Responder Analysis: More participants in the NurOwn® cell therapy group showed ≥1.5 points/month improvement All participants (n=46) Excluding slow progressors (n=21) ALSFRS-R ≥1.5 points improvement/month in post- ALSFRS-R ≥1.5 points improvement excluding % of patients with ≥1.5 po

  14. Slide 14: ALS Study Responder Analysis: >=100% slope improvement

    NurOwn® Phase 2 ALS Study Responder Analysis: >=100% slope improvement All participants (n=46) Excluding slow progressors (n=21) ALSFRS-R ≥100% improvement in post-treatment ALSFRS-R ≥100%% improvement excluding slope compared to pre-treatment slope slow progressors 100% % of patients with 100% impr

  15. Slide 15: CSF NTFs Significantly Increased 2 Weeks Post-Treatment

    NurOwn® Phase 2 ALS Study: CSF NTFs Significantly Increased 2 Weeks Post-Treatment VEGF VEGF VEGF HGF HGF HGF LIF LIF LIF 1000 1000 1000 600 600 600 18 18 18 * * * ** ** ** *** *** *** 900 900 900 16 16 16 500 500 500 800 800 800 14 14 14 700 700 700 400 400 400 12 12 12 600 600 600 pg/ml pg/ml pg/m

  16. Slide 16: CSF Inflammatory Markers Decreased Post-Treatment

    NurOwn® Phase 2 ALS Study: CSF Inflammatory Markers Decreased Post-Treatment Pre transplantation CSF MCP-1, SDF- markers of neuroinflammation Post transplantation CRP CRPCRP CRP MCP-1 MCP-1 MCP-1 MCP-1 SDF-1 SDF-1 SDF-1 SDF-1 MIP-1b MIP-1b MIP-1b MIP-1b 1600 1600 1600 1600 50 50 50 50 250 250 250 25

  17. Slide 17: NurOwn Phase 3 ALS Study (BCT-002-US)

    NurOwn® Phase 3 ALS Study (BCT-002-US) • Randomized, double-blind, placebo-controlled, multi-dose trial • WCT selected as CRO, Patient Webinar July 17th • 200 participants; 1:1 randomization; 6 leading clinical sites • Study design – 3 month baseline to determine ALSFRS-R slope – 3 doses over first

  18. Slide 18: 2017 Calendar

    2017 Calendar May 2017 June 2017 July 2017 December 2017 ISCT, Gordon MSC-NTF London Conference NTFs, miRNA cell Newport, RI characterization World Advanced publication and Bio 2017 Gordon Motor Neuron CSF Regenerative Phase 2 study San Diego, CA Conference ALS, Disease biomarker Medicine results St

  19. Slide 19: Senior Management

    Senior Management Chaim Lebovits Yael Gothelf PhD. President and CEO VP, Scientific and Regulatory Affairs Ralph Kern, MD, MHSc. Yossef Levy PhD. COO and CMO VP, Cell Production Uri Yablonka Revital Aricha PhD. Exec. VP, Chief Business VP, R&D Officer Mary Kay Turner VP, Patient Advocacy and Governm

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