Mustang Bio, Inc. · NASDAQ:MBIO
Mustang Bio — Investor Presentation (April 2016)
20 slides from Mustang Bio's investor presentation, April 2016
Slide 1: Corporate Presentation
Corporate Presentation April 2016 1
Slide 2: Forward Looking Safe Harbor Statement
Forward Looking Safe Harbor Statement This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements are often, but not always, made through the use of words or phrases such as “anticipates”, expects”, plans”, believ
Slide 3: Corporate Overview
Corporate Overview Founded by Fortress Biotech in 2015 Chimeric Antigen Receptor (CAR) T Cell technology from City of Hope (COH) Based on the research of Stephen Forman and Christine Brown, pioneers of CAR-T technology First two CAR-Ts entered the clinic in 2015 and 2016 Research collaboration
Slide 4: Mission Statement
Mission Statement To provide long-term clinical remissions for patients with aggressive forms of cancer by leveraging best-in-class science from COH to create novel CAR-T therapies 4
Slide 5: What is a CAR-T?
What is a CAR-T? CAR Function: Antibody fragment that identifies and Chimeric Antigen Single-chain variable fragment targets tumors (e.g. CD19, CD20) (scFv) Receptor-T cell Extracellular linker / Transmembrane Links co-stimulation/CD3 to the antibody fragment (can effect efficacy) The CAR recognizes
Slide 6: How is a CAR-T made?
How is a CAR-T made? CAR-T T-Cells extracted T-Cells Engineered Administered back to patient Total time From Blood Draw to Infusion: ~2-4 weeks 6
Slide 7: Lead CAR-T Programs
Lead CAR-T Programs • MB-101 • Targets IL13Rα2 • First being applied for the treatment of Malignant Glioma (GBM) tumors • Potential broad application to other solid tumors • Phase 1 on-going • MB-102 • Targets CD123 • For the treatment of AML and BPDCN (Blastic plasmacytoid dendritic cell neoplasm)
Slide 8: MB-101 – IL13Rα2 an ideal Target for CAR-T
MB-101 – IL13Rα2 an ideal Target for CAR-T Brown et al. CCR 2015 Jonnalagadda et al. Mol Therapy; 2015 8 Wang et al. Immunotherapy; 2011
Slide 9: MB-101 CAR T Is More Potent than 1st generation IL13-targeted CAR-T
MB-101 CAR T Is More Potent than 1st generation IL13-targeted CAR-T D7 D13 D16 D20 D27 tumoronly Mock Tcm 0.1 M 1st gen IL13Ra2 0.3 M CAR-T 1.0 M 0.1 M 2nd gen IL13Ra2 0.3 M CAR-T 1.0 M 9
Slide 10: Phase I Clinical Trial with MB-101 is ongoing
Phase I Clinical Trial with MB-101 is ongoing Patient Population Planned Enrollment Study Objectives Rel/Ref GBM- Arm 1: Resectable 12/arm Assess the feasibility, safety and Arm 2: Non-resectable determine MTD 10
Slide 11: GBM, a Significant Unmet Medical Need
GBM, a Significant Unmet Medical Need Glioblastoma (GBM) is the most common primary malignant brain tumor GBM most aggressive form of brain tumor with extremely poor prognosis and overall survival (OS) Median OS from diagnosis is ~15 months 5 year survival of only 5% ~30,000 newly diagnosed GBM
Slide 12: Competitive CAR-T therapies in development for GBM
Competitive CAR-T therapies in development for GBM Drug Name Company Description Targets Clinical Stage CART-EGFRvIII Novartis Anti-EGFRvIII EGFRvIII Phase I Pilot CART EGFRvIII Kite Pharma Anti-EGFRvIII EGFRvIII Preclinical CART UCART-EGFRvIII Cellectis Anti-EGFRVIII EGFRvIII Discovery “universal C
Slide 13: MB-102 – CAR-T Targeting CD123 Expressing Tumors
MB-102 – CAR-T Targeting CD123 Expressing Tumors • CD123 is expressed on cells of myeloid lineage and is overexpressed on AML, ALL and BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm) • Human proof of principle with fusion toxin directed at target in BPDCN • Limited CAR-T competition (Novartis,
Slide 14: MB-102 (CD123) – Antitumor Activity Against Human Acute Myeloid Leukemia
MB-102 (CD123) – Antitumor Activity Against Human Acute Myeloid Leukemia CD123 CARs MARDIROS et al BLOOD, 31 OCTOBER 2013 x VOLUME 122, NUMBER 18 14 MARDIROS et al.. BLOOD 2013
Slide 15: Phase I Clinical Trial with MB-102 is Open and Recruiting Patients
Phase I Clinical Trial with MB-102 is Open and Recruiting Patients Patient Population Planned Enrollment Study Objectives Assess the feasibility, safety and Rel/Ref AML 18 determine MTD of Single Infusion 15
Slide 16: AML, a Significant Unmet Medical Need
AML, a Significant Unmet Medical Need • Acute Myeloid Leukemia is the most common acute leukemia in adults • Approximately 30,000 newly diagnosed cases of AML per year in the US, Japan, and five major EU markets • Overall five-year survival rate in the US is ~25% 16 *Information taken from Datamonit
Slide 17: Competitive Landscape for CD123 Targeted Therapies
Competitive Landscape for CD123 Targeted Therapies Drug Name Company Drug Description Targets Clinical CART-CD33 Theravectys CD33 CD123 (China) CAR IL3RA Phase I/II CSL-360/362 CSL Mab IL3RA Phase I Bi-specific antibody CD3 GD006 MacroGenics to CD123 and CD3 IL3RA Phase I EEF2, SL-401 Stemline IL3 f
Slide 18: Ultra-Orphan Opportunity: BPDCN
Ultra-Orphan Opportunity: BPDCN BPDCN is a rare but aggressive blood cancer Annual US incidence: <60 (similar or larger number in EU) No standard of care Median OS 9 - 12 months Uniformly very high CD123 expression Proof of Principle: IL-3 target fusion protein In a pilot trial, 7/9
Slide 19: Key Milestones for Mustang in 2016
Key Milestones for Mustang in 2016 Preliminary safety data from dosing at least 6 patients for MB-101 Preliminary safety data from dosing at least 6 patients for MB-102 Additional CAR-Ts from the research collaboration entry into development phase 19
Slide 20: Key Take Home Messages
Key Take Home Messages Robust CAR-T platform technology in partnership with pioneers in CAR-T technologies from COH Lead CAR-T with no current competition for the target (IL13Rα2) already in the clinic with data in 2016 Second CAR-T in pipeline where target CD123(IL3Rα) has been validated in Ultr