Corporate Presentation. April 2016
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1 Corporate Presentation April
2 Forward Looking Safe Harbor Statement This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of These statements are often, but not always, made through the use of words or phrases such as anticipates, expects, plans, believes, intends, and similar words or phrases. Such statements involve risks and uncertainties that could cause Mustang Bio s actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any such statements due to various factors, including the risks and uncertainties inherent in clinical trials, drug development, and commercialization. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Checkpoint Therapeutics undertakes no obligation to update these statements, except as required by law. 2
3 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 between Mustang and COH to identify additional CAR-T clinical candidates 3
4 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
5 What is a CAR-T? CAR Function: Chimeric Antigen Receptor-T cell The CAR recognizes targets on the surface of the malignant cell to direct and activate T-cells to destroy the tumor Single-chain variable fragment (scfv) Extracellular linker / Transmembrane Co-stimulation domain (4-1BB or CD28) Antibody fragment that identifies and targets tumors (e.g. CD19, CD20) Links co-stimulation/cd3 to the antibody fragment (can effect efficacy) Strengthens patient derived T cell signaling and persistence, enhances potency CD3ζ Activates the cytotoxic patient derived T Cell to which CAR is attached 5
6 How is a CAR-T made? CAR-T T-Cells extracted Administered back to patient T-Cells Engineered 6 Total time From Blood Draw to Infusion: ~2-4 weeks
7 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) Phase 1 on-going 7
8 MB-101 IL13Rα2 an ideal Target for CAR-T 8 Brown et al. CCR 2015 Jonnalagadda et al. Mol Therapy; 2015 Wang et al. Immunotherapy; 2011
9 2 nd gen IL13Ra2 CAR-T 1 st gen IL13Ra2 CAR-T MB-101 CAR T Is More Potent than 1st generation IL13-targeted CAR-T tumoronly D7 D13 D16 D20 D27 Mock Tcm 0.1 M 0.3 M 1.0 M 0.1 M 0.3 M 1.0 M 9
10 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
11 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 GBMs annually in the US, Japan and five major EU markets 11
12 Competitive CAR-T therapies in development for GBM Drug Name Company Description Targets Clinical Stage CART-EGFRvIII Novartis Anti-EGFRvIII CART EGFRvIII Kite Pharma Anti-EGFRvIII CART UCART-EGFRvIII Cellectis Anti-EGFRVIII universal CAR through expression on allogeneic T- cells EGFRvIII EGFRvIII EGFRvIII Phase I Pilot Preclinical Discovery 12
13 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, Juno and Kite not in or near clinic) 13 Wang et al Blood Mardiros et al Blood Jonnalagadda et al Mol Ther
14 MB-102 (CD123) Antitumor Activity Against Human Acute Myeloid Leukemia CD123 CARs 14 MARDIROS et al BLOOD, 31 OCTOBER 2013 x VOLUME 122, NUMBER 18 MARDIROS et al.. BLOOD 2013
15 Phase I Clinical Trial with MB-102 is Open and Recruiting Patients Patient Population Planned Enrollment Study Objectives Rel/Ref AML 18 Assess the feasibility, safety and determine MTD of Single Infusion 15
16 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 Datamonitor Healthcare*
17 Competitive Landscape for CD123 Targeted Therapies Drug Name Company Drug Description Targets Clinical CART-CD33 CD123 Theravectys (China) CAR CD33 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 fusion toxin IL3RA Phase I/II Phase I UCART123 Cellectis allogeneic T-cell CAR IL3RA planned 17 *Data taken from Bioseeker Group*
18 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 BPDCN responded: 5 CR, 2 PR Median duration of response: 5 months (1-24) 18
19 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
20 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 Ultra Orphan indication CAR-T collaboration with COH to generate additional CAR-Ts to build worldclass robust CAR-T company 20
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