Fortress Biotech, Inc. · NASDAQ:FBIO
Fortress Biotech — Helocyte — Investor Presentation (March 2025)
38 slides from Fortress Biotech's investor presentation, March 2025
Slide 1: March 2025
March 2025
Slide 2: Investment Highlights
Investment Highlights 1 CMV Vaccine with Positive 7 Phase 2 Data 2 Phase 2 Trial in Liver Potential Peak Sales of $1BN+ Transplant to Start in 2024, Other Trials (HSCT, SOT, HIV) Robust IP, Biologic Exclusivity Ongoing & Planned 6 3 Large Potential Market with Significant Unmet Medical 100K+ Organ T
Slide 3: Company Overview
Company Overview Helocyte is developing novel immunotherapies for the prevention and treatment of cytomegalovirus (“CMV”) Phase 2 Study 2024 2025 Clinically 2027 $1BN+ Met Primary Endpoint Initiation of Initiation of Advanced Topline Data in Potential Peak US/EU in difficult-to-treat Phase 2 in Live
Slide 4: Evolution Since Inception: Groundwork Completed
Evolution Since Inception: Groundwork Completed Helocyte has consistently met critical developmental milestones, paving the way for pivotal studies and approval… Company Inception 2015 - Triplex Phase 1 2016- Triplex Phase 2 2020 - Clear Anticipated 2015 Development Development Development Pathway &
Slide 5: Triplex: Addresses Significant Unmet Medical Need in CMV Control
Triplex: Addresses Significant Unmet Medical Need in CMV Control Prior to approval of Merck’s Letermovir (Prevymis) in HSCT(1) ($370MM in 2021 sales), last CMV antiviral approved 15+ years ago Current Standard of Care (Antivirals) Triplex (Immunotherapy) Four of five approved CMV antivirals carry Bl
Slide 6: Triplex: Market & Commercial Opportunity
Triplex: Market & Commercial Opportunity Triplex targets CMV control in SOT and HSCT; well-defined markets with significant unmet need and no approved immunotherapy Solid Organ Transplant (SOT) Hematopoietic Stem Cell Transplant (HSCT) Procedure US Incidence EU Incidence Peak Sales Procedure US Inci
Slide 7: Triplex: Universal Multi-Antigen MVA-based CMV Vaccine
Triplex: Universal Multi-Antigen MVA-based CMV Vaccine Triplex’s unique clinical profile positions it to potentially be a leading treatment for CMV control in SOT and HSCT recipients Mechanism: Cellular Immunity to Primary CMV Proteins ▪ CD4 & CD8 T Cell Response to: pp65 + IE1 + IE2 — CD8 T Cell Re
Slide 8: Pipeline
Pipeline TRIPLEX Pre-Clinical Phase 1 Phase 2 Phase 3 Liver Transplant (Recipient Vaccination) Kidney Transplant (Recipient Vaccination) CMV-HIV Co-Infection on ART Allogeneic (MRD) Stem Cell Transplant (Donor Vaccination) Allogeneic (Haplo) Stem Cell Transplant (D+R Vaccination) Allogeneic (Haplo,
Slide 9: Completed Studies of Triplex (4)
Completed Studies of Triplex (4) 9
Slide 10: Completed Studies of Triplex
Completed Studies of Triplex Four Completed Trials Demonstrate Triplex Safety, Immunogenicity and Efficacy Phase 1 (Completed) Phase 2 in HSCT (Completed) ▪ Design: multicenter, double-blind, randomized (1:1), placebo-control ▪ Design: open-label, single-arm, single-center, dose-escalation (three le
Slide 11: Completed Studies of Triplex (cont.)
Completed Studies of Triplex (cont.) Four Completed Trials Demonstrate Triplex Safety, Immunogenicity and Efficacy (cont.) Phase 1 in Auto-HSCT (Completed) Phase 1: Donor Vaccination in Allo-HSCT (Completed) ▪ Design: open-label, single-center (University of Minnesota) ▪ Design: open-label, single-c
Slide 12: Ongoing Studies of Triplex (4)
Ongoing Studies of Triplex (4) 12
Slide 13: Ongoing Studies of Triplex
Ongoing Studies of Triplex Pilot Study: Triplex + CD19 CAR T for NHL (ongoing) ▪ Design: open-label, single-center (City of Hope) ▪ Patient Population: adults with intermediate or high Grade B-lineage NHL indicated for autologous HSCT in first relapse after complete remission ▪ Enrollment: 12-15 adu
Slide 14: Ongoing Studies of Triplex (cont.)
Ongoing Studies of Triplex (cont.) Expanding Dataset in Other Indications Phase 2: Adults Co-Infected with CMV & HIV (Ongoing) Phase 1: Triplex + Bispecific CMV-HIV CAR (ongoing) ) ▪ Design: randomized, multicenter, placebo-controlled trial ▪ Design: open-label, Phase 1 dose-finding trial at single-
Slide 15: Planned Studies of Triplex (4)
Planned Studies of Triplex (4) 15
Slide 16: Planned Studies of Triplex
Planned Studies of Triplex Targeting Largest Segments of Solid Organ Transplant: Liver and Kidney Phase 2: Liver Transplant (2024) Phase 2: Kidney Transplant (2025) ▪ Design: randomized, double-blind, placebo-control ▪ Design: randomized, double-blind, placebo-control ▪ Patient Population: seronegat
Slide 17: Planned Studies of Triplex (cont.)
Planned Studies of Triplex (cont.) Expanding Dataset in HSCT via Vaccination of HSC Donor Phase 2: Donor Vaccination in Phase 1b: Donor + Recipient Vaccination in High-Risk Matched Related Donor (MRD) Allo-HSCT (2023) Haploidentical Allo-HSCT (2024) ▪ Design: randomized, double-blind, placebo-contro
Slide 18: Robust IP Protection & Orphan Market Exclusivity
Robust IP Protection & Orphan Market Exclusivity Regulatory Triplex IP ➢ Orphan Exclusivity: 7 ➢ HCMV Antigens Expressed in MVA, Methods (7,163,685), expires Years (US), 10 Years 2024 (EU) ➢ rMVA Vaccines and Methods of Prep Thereof (8,580,276), expires ➢ Biologic Exclusivity: 12 2031 Years (US), 11
Slide 19: Experienced Management Team with Proven Track Record
Experienced Management Team with Proven Track Record Board of Directors Lindsay A. Rosenwald, M.D. Key Management Tenor Overview Prior Experience Executive Chairman Lindsay A. Rosenwald, M.D. 20+ years Life Sciences Entrepreneur Co-Founder and Executive Chairman experience and Investor Michael S. We
Slide 20: World-Class Key Opinion Leaders
World-Class Key Opinion Leaders Helocyte has partnered with a distinguished group of key opinion leaders Key Opinion Leaders Tenor Overview ▪ Professor in the Department of Hematology & Hematopoietic Cell Transplantation at City Don J. Diamond, PhD 35+ years of of Hope Scientific Founder & Chair of
Slide 21: CMV Market Overview and Unmet Need
CMV Market Overview and Unmet Need 21
Slide 22: CMV Overview
CMV Overview CMV infection is ubiquitous and usually benign, but is a major cause of morbidity and mortality in immunosuppressed patients CMV Overview Effects of CMV ▪ CMV is a member of the herpes virus family and the largest among known human viruses …with the ability to …and progress to CMV CMV i
Slide 23: Market Overview & Opportunity: Solid Organ Transplant (SOT)
Market Overview & Opportunity: Solid Organ Transplant (SOT) The number of solid organ transplants, specifically kidney and liver, has risen year-over-year Observations 2019 Transplants by Organ & Region Organ U.S. Europe Globally 40K+ Solid Organ Transplants Kidney 25,490 23,593 92,532 in the US per
Slide 24: Market Overview & Opportunity: Hematopoietic Stem Cell Transplant (HSCT)
Market Overview & Opportunity: Hematopoietic Stem Cell Transplant (HSCT) The number of stem cell transplants worldwide has grown steadily year-over-year, and correlates with an increased CMV burden Observations Stability in Number of Allo-HSCT Transplants in the U.S. 10,800 11,000 ~11,000 10K+ Allog
Slide 25: Limitations of Current Standard of Care…
Limitations of Current Standard of Care… Preemptive and Prophylactic Antivirals (most with Black Box Warnings) Prophylactic vs. Preemptive FDA Approved Antivirals for CMV Prophylaxis: Drug Label Admin / Dosing Black Box Warning ▪ Commence treatment at the time of or Hematologic Toxicity, Impairment
Slide 26: Triplex: Addresses Significant Unmet Medical Need in CMV Control
Triplex: Addresses Significant Unmet Medical Need in CMV Control Prior to approval of Merck’s Letermovir (Prevymis) in HSCT(1) ($370MM in 2021 sales), last CMV antiviral approved 15+ years ago Current Standard of Care (Antivirals) Triplex (Immunotherapy) Four of five approved CMV antivirals carry Bl
Slide 27: Triplex Clinical Trials (Detailed)
Triplex Clinical Trials (Detailed) 27
Slide 28: Triplex: CMV Immunotherapy with Best-in-Class Potential
Triplex: CMV Immunotherapy with Best-in-Class Potential Differentiated Product Profile Addresses Significant Limitations of Standard of Care Antivirals Indication ▪ Indicated for prophylactic control of CMV in post allogeneic hematopoietic stem cell transplant (HSCT) in oncology Description / ▪ Univ
Slide 29: Triplex: Phase 1 Safety and Tolerability Study
Triplex: Phase 1 Safety and Tolerability Study Phase 1 data confirms safety, tolerability and immunogenicity of Triplex Phase 1 Trial (Completed) Design Method ▪ Open-label, single arm, dose-escalating trial to assess safety and ▪ Eligible Subjects: 18-60 years old, CMV(+) and CMV(-) healthy volunte
Slide 30: Triplex: Phase 2 Trial in Allogeneic HSCT
Triplex: Phase 2 Trial in Allogeneic HSCT Phase 2 data demonstrates Triplex safety, immunogenicity and efficacy Phase 2 Trial (Completed) Design Method ▪ Multi-Center ▪ Eligible Subjects: CMV-seropositive undergoing allogeneic HSCT from — City of Hope (PI, Nakamura) matched related and unrelated don
Slide 31: Triplex for CMV Control in Liver Transplant
Triplex for CMV Control in Liver Transplant Phase 2 Study Anticipated to Commence in 2024 Phase 2 Trial (Planned) Overview Objectives ▪ Induce the expansion of CMV-pp65, IE1, and IE-2-specific functional T-cells in CMV Primary seronegative and CMV seropositive patients with advanced liver disease wh
Slide 32: Triplex for CMV Control in Kidney Transplant
Triplex for CMV Control in Kidney Transplant Phase 2 Study Anticipated to Commence in 2025 Phase 2 Trial (Planned) Overview Objectives ▪ Induce the expansion of CMV-pp65, IE1, and IE-2-specific functional T-cells in CMV Primary seronegative and CMV seropositive patients with advanced kidney disease
Slide 33: Pathway to Approval in HSCT
Pathway to Approval in HSCT Generating Additional Data in HSCT to Support Broad Phase 3 Inclusion Phase 3 Trial Overview Phase 3 Trial Highlights ▪ Phase 3 study for Triplex will be an expanded version of the Phase 2 study with the same Design: Randomized 2:1 between vaccine endpoint and enrollment
Slide 34: Triplex vs. HOOKIPA’s HB101
Triplex vs. HOOKIPA’s HB101 HOOKIPA’s HB101 is a key comparable to Triplex for CMV control in solid organ transplant Triplex HB-101 Met Primary Endpoint in Multi-Center Phase 2 in difficult- Failed Phase 2 in less difficult-to-treat kidney transplant recipients Clinical Stage to-treat allogeneic ste
Slide 35: Helocyte Summary
Helocyte Summary 35
Slide 36: Commercial Opportunity
Commercial Opportunity Well-defined, Orphan disease markets with modest sales and marketing requirement SOT/HSCT Market Opportunity Current Transplant Center Landscape Commercial Infrastructure Required Number of SOT by Organ & Region Organ U.S. Europe Globall y SOT ▪ ~200 major transplant centers i
Slide 37: Investment Highlights Overview
Investment Highlights Overview Key Investment Highlights 1 ▪ Triplex: vaccine engineered to prophylactically reduce the occurrence of early and late CMV after Allogeneic Hematopoietic Stem Cell Transplant (HSCT) and Solid Organ Transplant (SOT) Two Novel Biologics ▪ Recent positive Ph. 2 data for HS
Slide 38: Investment Highlights Overview (cont’d)
Investment Highlights Overview (cont’d) Key Investment Highlights 4 ▪ Safe and well-tolerated in transplant recipients with no adverse impact on GvHD, relapse, survival ▪ Efficacy: equivalent or better than standard of care antivirals in Phase 2 Triplex: Differentiated ▪ Convenient dosing of 2-3 IM