Mustang Bio, Inc. · NASDAQ:MBIO
Mustang Bio — Investor Presentation (June 2017)
27 slides from Mustang Bio's investor presentation, Jun 9, 2017
Slide 1: Corporate Presentation June 2017
Corporate Presentation June 2017
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”, bel
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 colla
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 to create novel CAR - T therapies. 4
Slide 5: Publicly Traded Companies
Publicly Traded CAR - T Companies 5 Company Name (Ticker) Market Cap (6/1/2017) Kite Pharma (KITE) ~$4.2B Juno Therapeutics (JUNO) ~$2.6B Ziopharm (ZIOP) ~$0.9B Cellectis (CLLS) ~$0.8B
Slide 6: What is a CAR-T Cell?
What is a CAR - T? 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 6 Transmembrane domain CAR Single - chain variable fragment ( scFv ) Co - stimulation domain (4 - 1BB or CD28) CD3 ζ signaling d
Slide 7: How is a CAR-T made?
How is a CAR - T made? 7 CAR - T Blood sample obtained via apheresis T - cells extracted T - cells engineered in cell - processing facility T - cells administered back to patient Total time from apheresis to infusion: ~2 - 4 weeks
Slide 8: Lead Programs
Lead CAR - T Programs • MB - 101 • Targets IL13R ⍺ 2 on surface of cancer cells • For the treatment of glioblastoma multiforme (GBM) tumors • Phase 1 ongoing • MB - 102 • Targets CD123 on surface of cancer cells • For the treatment of AML (acute myeloid leukemia) and BPDCN ( blastic plasmacytoid den
Slide 9: MB-101: IL13Rα2 is an Ideal Target for GBM
MB - 101: IL113R ⍺ 2 is an Ideal Target for GBM 9 • Jonnalagadda et al. Mol Therapy; 2015 • Wang et al. Immunotherapy; 2011 • Brown et al. Manuscript in preparation Target Antigen Normal Brain GBM T - Cells TNFR + / - +++ +++ HER 2 - +++ - EGFR ++ ++++ - EGFRvIII - ++++ (<30%) - IL13R ⍺ 2 - ++++ (>9
Slide 10: MB-101 is More Potent than 1st Generation IL13-Targeted CAR
MB - 101 CAR - T is More Potent than 1st Generation IL13 - Targeted CAR - T 10 D7 D1 3 D1 6 D2 0 D 2 7 1.0 M 0.3 M 0.1 M Mock Tcm T umor O nly 1.0 M 0.3 M 0.1 M 1 st gen IL13R ⍺ 2 CAR - T 2 nd gen IL13R ⍺ 2 CAR - T 1 st gen IL13R ⍺ 2 CAR - T 2 nd gen IL13R ⍺ 2 CAR - T
Slide 11: Phase I Clinical Trial with MB-101 is Ongoing
Phase I Clinical Trial with MB - 101 is Ongoing 11 Patient P opu l at i on Planned E n r o ll m e nt Study Objectives Relapsed/Refractory GBM • Arm 1: R esectable • Arm 2: Non - resectable 1 2/arm • Assess feasibility and safety • Determine MTD
Slide 12: Resection Arm – ICT Treatment Summary
Resection Arm – ICT Treatment Summary 12 Patient # Tx Arm / Dose IL13R ⍺ 2 IHC Manuf. CAR - T Cells Treatment Dose Notes UPN097 Resection / Dose 1 110 64% CAR 16 days Cycles 1, 2: 2M, 10M PD; Off - study due to rapid tumor progression UPN109 Resection / Dose 1 80 64% CAR 18 days Cycles 1, 2, 3 (ICT)
Slide 13: Progression of New Tumors Distant from Cell Infusion Site
Progression of New Tumors Distant from CAR - T Cell Infusion Site 13 IL13R ⍺ 2 CAR - T cell infusion First dose of “systemic” therapy Days Post - Enrollment Max Lesion Area (mm 2 ) Cycles 1 - 6 (ICT) Cycles 7 - 11 (ICV) UPN 109 • 50 yr ; male • GBM – Primary Diagnosis • IDH1 wt ; MGM
Slide 14: Regression of Recurrent Multifocal Glioblastoma After Intraventricular Delivery
Regression of Recurrent Multifocal Glioblastoma After Intraventricular Delivery of IL13R ⍺ 2 - Targeted CAR - T Cells 14 Sagittal MRI (top) and PET (bottom) of the brain Axial MRI of the brain All metastatic tumors in the spine were completely eliminated Maximum lesion area for non - resected tumors
Slide 15: GBM is a Significant Unmet Medical Need
GBM is a Significant Unmet Medical Need • Glioblastoma multiforme (GBM) is the most common primary malignant brain tumor • GBM is also the most aggressive form of brain tumor, and is associated with extremely poor prognosis and survival • Median overall survival from diagnosis is approximately 15 mo
Slide 16: Competitive Therapies in Development for GBM
Competitive Therapies in Development for GBM 16 Drug Name Company Description Target Clinical Stage AU105 Aurora BioPharma AU105: Bispecific anti - HER2/anti - CMV pp65 antigen HER2 and CMV pp65 antigen Phase I CART - EGFRvIII NIH Anti - EGFRvIII CART EGFR Phase I CART - EGFRvIII University of Penns
Slide 17: MB-102 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 • Human proof - of - principle with fusion toxin directed at target on BPDCN cells • Limited CAR - T competition for this target (Novartis, Juno and Kite ar
Slide 18: MB-102 (CD123) – Antitumor Activity Against Human AML
MB - 102 (CD123) – Antitumor Activity Against Human Acute Myeloid Leukemia 18 Mardiros et.al. Blood . 2013;122:3138 - 3148. CD123 CARs
Slide 19: Phase I Clinical Trial with MB-102 is Open and Recruiting Patients
Phase I Clinical Trial with MB - 102 is Open and Recruiting Patients 19 Patient Population Planned Enrollment Study Objectives Refractory / Relapsed AML 18 Assess the feasibility / safety and determine MTD of single infusion
Slide 20: AML is a Significant Unmet Medical Need
AML is 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 European markets • Overall five - year survival rate in the US is ~25% 20
Slide 21: Competitive Landscape for CD123 Targeted Therapies
Competitive Landscape for CD123 Targeted Therapies 21 Drug Name Company Drug Description Targets Clinical CART - CD33 CD123 Theravectys (China) CAR CD33, CD123 Phase I/II SL - 401 Stemline IL3 fusion toxin CD123 Phase I/II CSL - 360/362 CSL MAb CD123 Phase I GD006 MacroGenics Bi - specific antibody
Slide 22: Ultra–Orphan Opportunity: BPDCN
Ultra – Orphan Opportunity: BPDCN • Blastic plasmacytoid dendritic cell neoplasm is a rare but aggressive blood cancer • Annual US incidence: <60 (similar or larger number in Europe) • No standard of care • Median overall survival: 9 – 12 months • Uniformly very high CD123 expression • Proof - of -
Slide 23: New Programs
New CAR - T Programs ▪ MB - 103 • Targets HER2 on surface of cancer cells • Will be initially applied to the treatment of GBM tumors • Preclinical • Other HER2 CAR - Ts: Aurora BioPharma’s HER2 CAR - T is in phase 1 development ▪ MB - 104 • Targets CS1 on surface of cancer cells • For the treatment
Slide 24: Key Milestones and Deliverables for Mustang in 2017: GBM
Key Milestones and Deliverables for Mustang in 2017: GBM CAR - T • Amend GBM protocol to allow continued intracerebro - ventricular dosing with MB - 101 • Collect safety and efficacy data at COH • Conduct in - depth root - cause analysis for all suboptimal responses and assess how therapy might be i
Slide 25: Additional Key Milestones and Deliverables for Mustang in 2017
Additional Key Milestones and Deliverables for Mustang in 2017 • Continue to dose AML patients with MB - 102 and assess for safety and efficacy; plan for Mustang IND in 2018 • In - license additional CAR - Ts from academic institutions • Secure facility to build internal cell processing capabilities
Slide 26: 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 currently known competition for the target (IL13R ⍺ 2) already in the clinic at COH with 1 complete response • Second CAR - T also accruing patients at COH
Slide 27: Corporate Presentation June 2017 27
Corporate Presentation June 2017 27