Hemogenyx Pharmaceuticals Plc · LSE:HEMO
Hemogenyx Pharmaceuticals — Investor Presentation (October 2024)
24 slides from Hemogenyx Pharmaceuticals's investor presentation, October 2024
Slide 1: Bringing Breakthrough Therapies to Patients
BRINGING BREAKTHROUGH THERAPIES TO PATIENTS October 2024
Slide 2: Disclaimer Notice
Disclaimer Notice The information described in this presentation is information that is confidential, price- This presentation is issued on behalf of Hemogenyx Pharmaceuticals plc (the “Company”) sensitive and which has not been publicly disclosed. By your receipt of solely for the purposes of preli
Slide 3: Company Overview
° “20 HEMOGENYX ete PHARMACEUTICALS ee e a life saving company Company Overview
Slide 4: Company Overview
Company Overview ▪ Founded in 2013 and listed on LSE since 2017 (LSE: HEMO), with its operations located in New York City at its state-of-the-art research and manufacturing facility ▪ Our mission is to develop breakthrough therapies for the treatment of blood diseases, including Acute Myeloid Leukem
Slide 5: Leadership Team
Leadership Team Sir Marc Feldmann Vladislav Sandler, PhD Chairman Chief Executive Officer, Co-Founder ■ Medicine and PhD in Immunology from the Walter ■ Widely published stem cell scientist with decades and Eliza Hall Institute of Medical Research of experience in scientific research at world leadin
Slide 6: Scientific, Clinical, Business Advisors and Team Principals
Scientific, Clinical, Business Advisors and Team Principals H. Michael Shepard, PhD Koen van Besien, MD Alan Walts, PhD ■ PhD in Cellular Molecular Developmental ■ Professor of Medicine and Director of ■ Venture Partner at Advent Life Sciences, Biology the Stem Cell Transplant Program at the Directo
Slide 7: Pipeline Overview
Pipeline Overview ▪ Our Lead asset, HEMO-CAR-T for relapsed/refractory (r/r) AML is expected to enter the clinic in H2 2024 Lead IND Enabling Modality Area Program Target Indication Discovery Phase 1 Phase 2 Optimisation studies HEMO-CAR-T FLT3 r/r AML IND opened in Q1 2024 HG-CT-1 Ph1 initiation in
Slide 8: Established Capability for Program Execution
Established Capability for Program Execution Floorplan of Hemogenyx's New York facility Science/Quality/Analytical/CMC/Pre-clinical/Regulatory/Clinical Capabilities ▪ R&D and manufacturing – In-House ▪ Quality system – In-House ▪ cGMP cell manufacturing – In-House ▪ cGLP analytical release testing –
Slide 9: AML Market and Treatment Paradigm
AML Market and Treatment Paradigm Estimated 61,090 cases of leukemia 20,240 new cases of AML diagnosed in the US in 2021* diagnosed in the US in 2021* AML Front-line Therapy Remission 30% Relapsed Refractory 70% Consolidation Therapy HSCT C/D remission 20% Died 80% Bone Marrow Transplant Global mark
Slide 10: Current Unmet Needs in AML and HSCT
Current Unmet Needs in AML and HSCT r/r AML is Almost Universally Fatal Conditioning regimens have safety concerns ▪ All current conditioning regimens for HSCT are very toxic ▪ Most patients lack sensitivity to currently available therapies and have severe side effects that can be life-threatening ▪
Slide 11: CAR-T program for treatment of relapsed/refractory Acute Myeloid Leukemia
CAR-T program for treatment of relapsed/refractory Acute Myeloid Leukemia
Slide 12: HEMO-CAR-T: a novel CAR-T therapy for AML
HEMO-CAR-T: a novel CAR-T therapy for AML CAR-T mediated cytotoxicity Autologous 3rd generation CAR-T therapies: Perforin and Granzyme ▪ Mechanism of Action: cytotoxic T lymphocyte (CTL)-mediated cytolysis Cytotoxic CAR-T FLT3+ cell ▪ Clinically Proven safety FLT3 HEMO-CAR-T: Activation signaling ▪
Slide 13: HEMO-CAR-T: Pathway to the clinic
HEMO-CAR-T: Pathway to the clinic Current Status ▪ Preclinical development is complete ✓o Preparation cGMP material, release testing and stability ✓o IND-enabling studies (e.g. UPENN, Hemogenyx Pharmaceuticals) ✓o Safety studies ✓o Potential for functional activity (elimination of AML and HSC/HP) ✓
Slide 14: HEMO-CAR-T – Clinical Plan
HEMO-CAR-T – Clinical Plan Clinical Plan ▪ Initial clinical study will be conducted in relapsed or refractory FLT3 + adult AML patients pre-qualified for HSC/HP transplantation to obtain preliminary data on safety (dose escalation), tolerability and partial efficacy – up to 18 patients ▪ The study w
Slide 15: CDX program for treatment of relapsed/refractory Acute Myeloid Leukemia
CDX program for treatment of relapsed/refractory Acute Myeloid Leukemia
Slide 16: CDX: a Novel Humanized FLT3-CD3 Bispecific Antibody
CDX: a Novel Humanized FLT3-CD3 Bispecific Antibody ▪ An “off the shelf” (non patient-specific) product ▪ Eliminates AML-derived cells transplanted into humanized mice and conditions humanized mouse bone marrow in vivo ▪ High affinity binding to FLT3 ▪ No FLT3 Ligand (FLT3L) competition 1 ▪ Unique b
Slide 17: CDX: Potential Path to IND and Clinical Plan
CDX: Potential Path to IND and Clinical Plan Activity Year 1 Year2 Preclinical ADME/toxicology studies Rhesus monkeys (cross-reactive species) to demonstrate: • Safety • Potential for functional activity (elimination of HSC/HP) Establishment of master cell line, process development and formulation P
Slide 18: Chimeric Bait Receptor Platform
Chimeric Bait Receptor Platform
Slide 19: CBR Program
CBR Program A novel paradigm for targeting Cancer, Neurodegenerative disease treatments and creating Antivirals Mechanism of action: Programming or redirection of myeloid immune cells such as macrophage using novel synthetic proteins Expected significant advantages: As CBR-programmed macrophages: a.
Slide 20: Synthetic Macrophage Receptors - Design Concepts
Synthetic Macrophage Receptors - Design Concepts Cell Therapy Biologics Malignancy Malignancy MACROPHAGE MACROPHAGE Infection Infection Viral Viral 20
Slide 21: Intellectual Property
° %e6 HEMOGENYX ete PHARMACEUTICALS ee e a life saving company Intellectual Property
Slide 22: Intellectual Property Position
Intellectual Property Position ▪ Hemogenyx has a strong Intellectual Property position and know-how ▪ Patents cover compositions of matter and methods of use for the CAR-T and CDX programs, in major jurisdictions ▪ In addition, other granted US patents are: o “Method of eliminating hematopoietic ste
Slide 23: Summary
Summary Advancing product candidates towards the clinic ▪ HEMO-CAR-T therapy for the treatment of relapsed/refractory AML and subset of ALL in adults and pediatric patients; IND cleared by US FDA and Phase I trial is projected to be initiated in H2 2024 ▪ CDX bi-specific antibody for the treatment o
Slide 24: Thank You!
Thank You! Contact information: Hemogenyx Pharmaceuticals Vladislav Sandler, Ph.D. Co-founder and CEO vladislav.sandler@hemogenyx.com +1 (347) 735-8380
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