Fortress Biotech, Inc. · NASDAQ:FBIO
Fortress Biotech — Investor Presentation (October 2016)
34 slides from Fortress Biotech's investor presentation, Oct 24, 2016
Slide 1: Corporate Overview October 2016
CORPORATE OVERVIEW NASDAQ : FBIO October 2016
Slide 2: Forward-Looking Statements
FORWARD - LOOKING STATEMENTS Statements in this presentation that are not descriptions of historical facts are forward - looking statements within the meaning of the “ safe harbor ” provisions of the Private Securities Litigation Reform Act of 1995 . We have attempted to identify forward - looking s
Slide 3: Fortress Biotech Companies
FORTRESS BIOTECH COMPANIES Acute settings (pain) Multiple cancers/Epigene tics/Antibodies Orphan diseases Immunotherapies for Cancer and Infectious Disease Dermatology (marketed products) CAR - T/ Immuno - oncology (brain cancer, leukemia) 3
Slide 4: Overview & Business Strategy
OVERVIEW & BUSINESS STRATEGY Acquire, develop and commercialize a diversified portfolio of products directly and through subsidiary companies (known as “Fortress Companies”) FBIO holds super - majority voting shares of each Fortress Company Multiple sources of revenue/return for FBIO: direct sales,
Slide 5: Fortress Biotech Business Model
Checkpoint Therapeutics Avenue Therapeutics Escala Therapeutics Helocyte Journey Medical Corporation Mustang Bio Legal Business Development Accounting & Finance Clinical Trials Scientific Affairs Executive Chairman / Board Manufacturing FORTRESS BIOTECH BUSINESS MODEL FORTRESS SERVICES 5
Slide 6: Value Proposition: Accomplishments in Just 2 Years
• CAR - T Program Including Solid Tumor (GBM) Complete Response • CMV Vaccine Program: In 2 Phase 2 Clinical Trials: Positive randomized trial recently published in the Lancet Hematology • Internal Immuno - Oncology program makes Fortress one of the only Life Sciences Companies with its own internal
Slide 7: Pipeline Products
PIPELINE PRODUCTS PRODUCT CANDIDATE COMPANY STAGE OF DEVELOPMENT Preclinical Phase 1 Phase 2 Phase 3 Commercial Targadox (Doxycycline Hyclate USP) 50mg tablets Journey Medical Corporation Ceracade (Eczema Emollient) Journey Medical Corporation Luxamend (Wound Cream) Journey Medical Corporation Derma
Slide 8: Pipeline Products
PIPELINE PRODUCTS PRODUCT CANDIDATE COMPANY STAGE OF DEVELOPMENT Preclinical Phase 1 Phase 2 Phase 3 Commercial IV Tramadol Avenue Therapeutics CNDO - 109 Fortress Biotech Argus NeuroOptics Fortress Biotech Anti - PD - L1 Checkpoint Therapeutics Anti - GITR Checkpoint Therapeutics Anti - CAIX Checkp
Slide 9: Pipeline Products
PIPELINE PRODUCTS PRODUCT CANDIDATE COMPANY STAGE OF DEVELOPMENT Preclinical Phase 1 Phase 2 Phase 3 Commercial ManNAc (GNE Myopathy) Escala Therapeutics ManNAc (Nephropathies) Escala Therapeutics CD123 CAR (AML) Mustang Bio IL13Ra2 - specific CAR (Malignant Glioma) Mustang Bio CGRP Fortress Biotech
Slide 10: A Fortress Biotech Company
A FORTRESS BIOTECH COMPANY 10
Slide 11: GBM, a Significant Unmet Medical Need
GBM, A SIGNIFICANT UNMET MEDICAL NEED ~30,000 newly diagnosed GBMs annually in the US, Japan and five major EU markets GBM most aggressive form of brain tumor with extremely poor prognosis and overall survival (OS) Glioblastoma (GBM) is the most common primary malignant brain tumor • Median OS from
Slide 12: CAR-T Program (Mustang Biotech)
CAR - T PROGRAM (MUSTANG BIOTECH) 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 Research collaboration between Mustang and COH to identify additional CAR - T clinical candidates
Slide 13: IL13Rα2 an Ideal Target for CAR-T
GBM – CART MB - 101 – IL13R α 2 AN IDEAL TARGET FOR CAR - T TARGET ANTIGEN NORMAL BRAIN GBM T CELLS TNF R +/ - +++ +++ Her 2 - +++ - EGFR ++ ++++ - EGFRvIII - ++++ (<30%) - IL - 13R α 2 - ++++ (>90%) - Source: Brown et al. CCR 2015 Jonnalagadda et al. Mol Therapy; 2015 Wang et al. Immunotherapy; 201
Slide 14: Patient Treatment Data
PATIENT # TX ARM / DOSE IL13RA2 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): 2M, 10M, 10M Cycles 4, 5, 6 (ICT): 10M, 10M,
Slide 15: Progression of New Tumors Distant from CAR-T Infusion Site
PROGRESSION OF NEW TUMORS DISTANT FROM CAR - T CELL INFUSION SITE 15 FIRST DOSE OF “SYSTEMIC” THERAPY -50 0 50 100 150 200 250 300 350 400mm^2 0 60 80 100 120 140 160 180 200 Days Post Enrollment Max Legion Area Lesion 4 Lesion 5 Lesion 6 Lesion 7 Lesion Spine UPN 109 50 YEAR OLD MALE GBM - PRIMARY
Slide 16: ICV Delivery Mediates Regression of Multifocal GBM
ICV DELIVERY OF IL13BB ζ T CELLS MEDIATES REGRESSION OF MULTIFOCAL GBM REGRESSION OF DISTANT CRANIAL METASTASES REGRESSION OF SPINE METASTASES PRE ICV POST ICV PRE ICV POST ICV 16
Slide 17: 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 18: A Fortress Biotech Company
A FORTRESS BIOTECH COMPANY 18
Slide 19: PepVax for Post Allo-HSCT (Helocyte)
19 PEPVAX FOR POST ALLO-HSCT ( HELOCYTE ) • Antigen target applicable to ~35% of Patients • Healthy volunteer Phase 1 completed - Safe, well tolerated at all dose levels - Immunogenic • Phase 1b published in Lancet, Dec. 2015 • Multicenter Phase 2 in 96 Patients - Data by 1H2018 - NCI Funding: >$5M
Slide 20: Phase 1b in Stem Cell Transplant Recipients
Phase 1b in 36 Stem Cell Transplant Recipients: published with editorial (12/2015) Vaccine (n=18) Observation (n=18) P Patients with serious adverse events 4 (22%) 9 (50%) 0.16 Patients with serious adverse events related to vaccine 1 NA .. Grade 3 - 4 adverse events 54 91 0.2 Patients with acute GV
Slide 21: Triplex for Post Allo-HSCT (Helocyte)
TRIPLEX FOR POST ALLO-HSCT ( HELOCYTE ) • 3 Antigen targets applicable to ~100% of Patients • CMV MVA Vaccine (pp65, IE1, IE2) HEALTHY VOLUNTEER PHASE 1 PRESENTED AT ASH 2015 Phase 2 (Enrolling As Of 11/2015) • Safe, well tolerated at all dose levels • Immunogenic • Multicenter Phase 2 in 115 Patien
Slide 22: A Fortress Biotech Company
A FORTRESS BIOTECH COMPANY 22
Slide 23: Immuno-Oncology Agents
IMMUNO - ONCOLOGY AGENTS • A fully human agonistic antibody binding GITR resulting in downregulation of T - regs • Pre - clinical data demonstrates synergy with anti - PD - L1 • IND expected before YE17 • A fully human antagonistic antibody that binds PD - L1 • 1 St Gen Maintains ADCC • Developing 2
Slide 24: CK-101, 3rd Generation EGFR Rationale
CK - 101, 3RD GENERATION EGFR RATIONALE ‒ Success of 1 st generation EGFR’s have led to acquired resistance through further mutations to EGFR (T790M) ‒ One 3 rd generation EGFR inhibitor ( Tagrisso ™) is approved for patients with lung cancer with T790M mutation ‒ AZN’s drug, Tagrisso ™, has signifi
Slide 25: CK-101, 3rd Generation EGFR Rationale
CK - 101, 3RD GENERATION EGFR RATIONALE 0 500 1,000 1,500 2,000 Tumor Volume (mm^3) 12 15 18 21 24 27 Days Post Implantation RX518, 150mg/kg, ig, QD RX518, 60mg/kg, ig, QD Vehicle Control CELL LINE A431 H1975 HCC827 Mutation EGFR WT L858R/ T790M Del 19 Erlotinib 392 2,500 9.3 AZD9291 64 2.2 1.5 CO -
Slide 26: CK-101, 3rd Generation EGFR Status
CK - 101, 3RD GENERATION EGFR STATUS In Phase 1/2 clinical trials Will explore accelerated approval strategy, similar to AZN Targeting first - in - class combination as AZN terminated combo program and CLVS is shutting program Develop as a monotherapy and in combination with synergistic I/O agents 2
Slide 27: A Fortress Biotech Company
A FORTRESS BIOTECH COMPANY 27
Slide 28: IV Tramadol for Post-Op Pain
• Fits important niche, more potent pain relief than acetaminophen and NSAIDs. Less side effects than other opioids • Phase 3 ready • Oral tramadol is one of the most prescribed pain medications in the U.S. • IV tramadol approved and widely used outside the U.S. • IV acetaminophen sells ~$250MM - ~2
Slide 29: Immuno/Oncology Platform Synergy Among the Pipeline
Anti - EGFr PARP inhibitor Anti - CAIX BET Inhibitor Anti - CMV Vaccine for GBM IL13 for GBM CD123 for AML more to come IMMUNO/ONCOLOGY PLATFORM SYNERGY AMONG THE PIPELINE CAR - Ts Targeted therapy 29
Slide 30: Immuno/Oncology Platform Synergy Among the Pipeline
Anti - EGFr PARP inhibitor Anti - CAIX BET Inhibitor Anti - CMV Vaccine for GBM IL13 for GBM CD123 for AML more to come IMMUNO/ONCOLOGY PLATFORM SYNERGY AMONG THE PIPELINE CAR - Ts Targeted therapy 30
Slide 31: Immuno/Oncology Platform Synergy Among the Pipeline
Anti - EGFr PARP inhibitor Anti - CAIX BET Inhibitor Anti - CMV Vaccine for GBM IL13 for GBM CD123 for AML more to come IMMUNO/ONCOLOGY PLATFORM SYNERGY AMONG THE PIPELINE CAR - Ts Targeted therapy 31
Slide 32: Immuno/Oncology Platform Synergy Among the Pipeline
Anti - EGFr PARP inhibitor Anti - CAIX BET Inhibitor Anti - CMV Vaccine for GBM IL13 for GBM CD123 for AML more to come IMMUNO/ONCOLOGY PLATFORM SYNERGY AMONG THE PIPELINE CAR - Ts Targeted therapy 32
Slide 33: Other Programs
OTHER PROGRAMS • Rare Disease Program… ManNac Phase 2 • NK Program • Anti - CAIX Program • PARP Inhibitor Program • CK - 103 BET Inhibitor • Our business development engine identifies multiple opportunities weekly 33
Slide 34: Key Take Home Messages
KEY TAKE HOME MESSAGES Diverse pipeline with multiple shots on goal and multiple revenue streams (sales, equity, royalties, fees) CAR - T program with solid tumor response reported Emerging I/O pipeline with differentiating features and multiple combinations possible Vaccine program for CMV with hum
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