StemRIM Inc. · TSE:4599
StemRIM — FY2023 Earnings Presentation
45 slides from StemRIM's earnings presentation, Sep 15, 2023
Slide 1: Presentation Material, Financial Results for the Fiscal Year Ended July 31, 2023
（TSE Growth︓4599） Presentation Material, Financial Results for the Fiscal Year Ended July 31, 2023 September 15, 2023
Slide 2: Overcoming Refractory Diseases by Regeneration-Inducing Medicine
Stem cell Regeneration-Inducing Medicine Overcoming Refractory Diseases by TM “Regeneration-Inducing Medicine ” StemRIM is a biotech company aiming to develop “Regeneration-Inducing TM Medicine ” a next generation of regenerative medicine. TM "Regeneration-Inducing Medicine " is new class of medicin
Slide 3: Mode of Action of Regeneration-Inducing Medicine
TM Mode of Action of “Regeneration-Inducing Medicine ” Bone marrow mesenchymal stem cells mobilized into the peripheral blood stream induce the tissue regeneration. Administration of “Regeneration- : Damaged site TM Inducing Medicine ” Chemokines produced in necrotic tissue (e.g. SDF-1α) Mobilizing
Slide 4: Agenda
Agenda 1 Summary of Financial Results for the Fiscal Year Ended July 31, 2023 2 Summary of Business Activities for the Fiscal Year Ended July 31, 2023 3 Future Growth Strategies 4 Copyright© StemRIM Inc. All rights reserved
Slide 5: Summary of Financial Results for the Fiscal Year Ended July 31, 2023
１. Summary of Financial Results for the Fiscal Year Ended July 31, 2023 5
Slide 6: Statements of Income
Summary of Financial Results Statements of Income （Millions of yen） FY2021 FY2022 FY2023 Function (Ended July (Ended July (Ended July （FY on FY） 31,2021) 31,2022) 31,2023) Operating revenue 1,400 22 2,350 +2,327 ・2.35 billion yen in operating revenue R&D expenses 1,523 1,421 1,567 +145 from the deve
Slide 7: Balance Sheet / Statements of Cash Flows
Summary of Financial Results Balance Sheet / Statements of Cash Flows （Millions of yen） As of July As of July As of July Function 31, 2021 31, 2022 31, 2023 （FY on FY） Current assets 9,940 9,262 10,440 +1,177 Of which cash and deposits 9,719 8,880 10,217 +1,337 Non-current assets 372 334 266 -68 Tot
Slide 8: Summary of Business Activities for the Fiscal Year Ended July 31, 2023
２. Summary of Business Activities for the Fiscal Year Ended July 31, 2023 8
Slide 9: Summary of Business Activities for the Fiscal Year Ended July 31, 2023
Summary of Business Activities for the Fiscal Year Ended July 31, 2023 TM Multiple Clinical Trials Progress to Next Stage for “Regeneration-Inducing Medicine ” Redasemtide Clinical trial-related progress Month/Year History Disclosure of Trial Data from Phase 2 Clinical Trial of Redasemtide for Acute
Slide 10: Overview of Development Pipeline
Summary of Business Activities Overview of Development Pipeline Development Stage Project Investi- Out-license Development candidate Indication Status Pre- Phase 1 Phase 2 Phase 3 code gator Research clinical study study study partner Epidermolysis Shionogi & Additional P2 Study -01 bullosa Co., Ltd
Slide 11: PJ1-01: Redasemtide (DEB)
Summary of Business Activities PJ1-01:Redasemtide(DEB) Additional Phase 2 Protocol Evaluation of efficacy and safety of Redasemtide in patients with dystrophic Study objectives epidermolysis bullosa having intractable ulcers Study design Single arm, multicenter, open label, uncontrolled Intervention
Slide 12: PJ1-01: Redasemtide (DEB) / Orphan Drug Designation
Summary of Business Activities PJ1-01:Redasemtide(DEB)/Orphan Drug Designation In May 2023, Redasemtide was designated by the Ministry of Health, Labour and Welfare as an orphan drug for the treatment of nutritional-type epidermolysis bullosa. Orphan Drug, Orphan Medical Device, and Orphan Regenerat
Slide 13: PJ1-02: Redasemtide (Acute Ischemic Stroke) Phase 2 Protocol
Summary of Business Activities PJ1-02：Redasemtide(Acute Ischemic Stroke) Phase 2 Protocol ・In the domestic Phase 2 trial, the proportion of To evaluate the efficacy of S-005151 compared to placebo in Main purpose patients with acute ischemic stroke with respect to modified Rankin patients who transi
Slide 14: PJ1-02: Redasemtide (Acute Ischemic Stroke) Global Phase 2b Protocol
Summary of Business Activities PJ1-02：Redasemtide（Acute Ischemic Stroke) Global Phase 2b Protocol Study objectives Verification of efficacy of Redasemtide in patients with acute ischemic stroke • Can be administered within 25 hours from the onset of symptoms to the patients Subject at age 18 or olde
Slide 15: PJ1-05: Redasemtide (Chronic Liver Disease) Phase 2 Protocol
Summary of Business Activities PJ1-05：Redasemtide(Chronic Liver Disease) Phase 2 Protocol Evaluate the safety and exploratory efficacy in patients with chronic liver Main purpose disease Clinical trial Single arm study, Open label, Uncontrolled design Patients with chronic liver disease with liver h
Slide 16: PJ1-05: Redasemtide (Chronic Liver Disease)
Summary of Business Activities PJ1-05：Redasemtide(Chronic Liver Disease) Primary endpoint evaluation Safety endpoints: Presence or absence of adverse events and percentage of the presence No serious adverse events or adverse reactions related to Redasemtide 1 patient experienced a serious adverse
Slide 17: Out-licensing activities for new Regeneration-Inducing Medicine
Summary of Business Activities Out-licensing activities for new “Regeneration-Inducing Medicine ” TM Participation in multiple conferences both domestically and internationally as part of the derivation activities for the next TM generation of “Regeneration-Inducing Medicine ” following Redasemtide.
Slide 18: Enhanced Investor Relations and Public Relations activities
Summary of Business Activities Enhanced Investor Relations and Public Relations activities Global outreach and expanded coverage for information dissemination to individual and institutional investors both domestically and internationally. Enhanced video contents Timely Disclosure Sharing informatio
Slide 19: Future Growth Strategies
3. Future Growth Strategies 19
Slide 20: Future Growth Strategies
Future Growth Strategies We will continue to pursue our growth strategy and aim to maximize the potential value of “Regeneration-Inducing Medicine ” TM Development Expansion of Out-licensing development of activities Redasemtide pipeline ・Facilitate out-licensing ・Continued to provide lateral ・Conti
Slide 21: IP Strategy
Future Growth Strategies IP Strategy Patents related to “Regeneration-Inducing Medicine ” granted worldwide. TM Aiming for Global Expansion Canada EU Russia Korea 3 cases Total: 6 pending 39 cases 4 cases 5 cases 113 cases（+5） 12 pending 3 pending 6 pending 102 pending PCT: China 6 cases US 4 pendin
Slide 22: SDGs: Achieving a Sustainable Future
Future Growth Strategies SDGs: Achieving a Sustainable Future Health and Well-Being for All StemRIM is dedicated to achieving a sustainable future by providing therapeutic solutions to people worldwide suffering from refractory diseases through the realization of “Regeneration-Inducing Medicine ”. T
Slide 23: SDGs: Identification of Materiality
Future Growth Strategies SDGs: Identification of Materiality Our company has identified key material issues (Materiality) with the aim of achieving sustained growth while contributing to the resolution of global societal challenges, including the Sustainable Development Goals (SDGs). Out of the 17 S
Slide 24: SDGs: Activities for the Fiscal Year Ended July 31, 2023
Future Growth Strategies SDGs: Activities for the Fiscal Year Ended July 31, 2023 ● Donation to “Towards a Society Where Women with Pelvic Organ Prolapse Can Seek Treatment with Ease”. We contributed to the Osaka University Graduate School of Medicine's crowdfunding project, "Towards a Society Where
Slide 25: Appendix
€2 stemrim Appendix.
Slide 26: Discovery of in-vivo mechanism inducing tissue regeneration
Appendix. Discovery of in-vivo mechanism inducing tissue regeneration Differences between normal skin and epidermolysis bullosa skin In normal skin (Figure A), type 7 collagen functions like an adhesive, bonding the epidermis and dermis, the superficial layers of skin. In epidermolysis bullosa conge
Slide 27: Discovery of in-vivo mechanism inducing tissue regeneration
Appendix. Discovery of in-vivo mechanism inducing tissue regeneration TM The beginning of the research and development on “Regeneration-Inducing Medicine ” : Hypothesis of stem cell recruitment mechanism from bone marrow to damaged skin. Necrotic epidermis with Despite loss of epidermal stem cells,
Slide 28: Discovery of in-vivo mechanism inducing tissue regeneration
Appendix. Discovery of in-vivo mechanism inducing tissue regeneration Discovery of crosstalk mechanism between damaged skin and bone marrow mesenchymal stem cells via necrotic tissue-derived factor Peeled epidermis Necrosis of epidermal cells HMGB1 released Damaged skin Cytoplasm Epidermal cells Nuc
Slide 29: HMGB1 peptide drugs with improved safety
Appendix. HMGB1 peptide drugs with improved safety Designing highly safe, chemically synthesized peptide drug from A-Box domain of HMGB1 protein C23 C45 C106 N C HMGB1 protein A box B box Tail An Important in vivo molecule that activate tissue regeneration Prof. Katsuto Tamai Osaka University Identi
Slide 30: Advantages of Regeneration-Inducing Medicine
Appendix. Advantages of “Regeneration-Inducing Medicine ” TM Epidermis formation during human development Carnegie developmental stage (developmental stage of vertebrate embryos) CS1 10 20 30 40 50 60 fertilized eggs CS9*：Embryo section (ectoderm) epidermis 2-cell blastodermic vesicle (inner cell ma
Slide 31: Advantages of Regeneration-Inducing Medicine
Appendix. Advantages of “Regeneration-Inducing Medicine ” TM Ectodermal mesenchymal stem cells have high pluripotency and differentiation ability to various tissues. Tissues into which differentiation has been confirmed in our collaborative research with Osaka University Tissues into which different
Slide 32: Expanding Indications and Markets
Appendix. Expanding Indications and Markets ：Clinical trial on going Targeting all areas where mesenchymal stem cell therapy can be effective ：Clinical trial in preparation Other countries and regions Traumatic brain Cardiomyopathy injury 121K in US 13.5M in US In Japan Cerebral infarction Traumatic
Slide 33: Functions of mesenchymal stem cells
Appendix. Functions of mesenchymal stem cells In-vivo mesenchymal stem cells have 5 distinctive capabilities 1. Cell migration ability 3. Trophic factor secretion ability Mesenchymal stem cells migrate to damaged tissue via the Promotes cell proliferation and tissue repair by secreting bloodstream g
Slide 34: In vitro culture reduces the functions of MSCs
Appendix. In vitro culture reduces the functions of MSCs TM “Regeneration-Inducing Medicine ” can avoid functional degradation of mesenchymal stem cells due to in vitro culture TM Manufacturing process of conventional cellular medicine Induction of MSC in “Regeneration-Inducing Medicine ” Mesenchyma
Slide 35: Summary of advantages of Regeneration-Inducing Medicine
Appendix. Summary of advantages of “Regeneration-Inducing Medicine ” TM TM “Regeneration-Inducing Medicine ” includes advantages in both cell therapy and chemicals “Regeneration-Inducing Medicine ” Cell therapy Chemicals TM Applicable for large tissue Tissue Applicable for large-scale damage with la
Slide 36: Summary of advantages of Regeneration-Inducing Medicine
Appendix. Summary of advantages of “Regeneration-Inducing Medicine ” TM TM “Regeneration-Inducing Medicine ” can solve the four major problems of conventional cell therapy Cancerization risk Risk of cancer depending on the site of gene insertion iPS cell Somatic ES cell “Regeneration-Inducing stem c
Slide 37: PJ3 Novel Regeneration-Inducing Peptide for Local Administration
Appendix. PJ3 Novel Regeneration-Inducing Peptide for Local Administration Developing protein drugs that accumulate mesenchymal stem cells at the site of injury Small injury Efficient accumulation of mesenchymal stem cells Large injury or chronic phase by topical administration of PJ3 drug PJ3 drug
Slide 38: PJ4 Autologous cell collection device for treatment
Appendix. PJ4 Autologous cell collection device for treatment Developing devices to collect mesenchymal stem cells mobilized in vivo Devices Bone marrow mesenchymal stem cell mobilization factor Normal skin （1）Skin incision （2）Subcutaneous （3）Stationary injection (a few days) （4）Cell accumulation （5
Slide 39: PJ4 Autologous cell collection device for treatment
Appendix. PJ4 Autologous cell collection device for treatment Confirmation of mesenchymal stem cell harvesting in a rat model Devices for Rat Tests Single-cell transcriptome analysis by next-generation sequencers for cells accumulated on the device bottom cover Mesenchymal stem cells accumulated on
Slide 40: PJ5 Stem cell gene therapy
Appendix. PJ5 Stem cell gene therapy Aim to cure intractable genetic disease by stem cell gene therapy Concept Ex vivo gene therapy involving the introduction of correct type VII collagen gene into autologous mesenchymal stem cells (MSCs) and administration of the cells in the blisters of the patien
Slide 41: PJ5 Stem cell gene therapy
Appendix. PJ5 Stem cell gene therapy Ex vivo gene therapy with minimal invasiveness, high efficacy, and persistent effect This therapy employs a novel method of isolating Bf-MSCs from a patient, efficient delivery of functional type VII collagen gene to the cells, and novel administration method to
Slide 42: PJ5 Stem cell gene therapy
Appendix. PJ5 Stem cell gene therapy Verification of therapeutic efficacy and duration of drug effect of this treatment using RDEB model mice Restoration of the type-VII collagen protein （C7） at the basement membrane on RDEB model mouse Bf-MSCs from C7 expression Skin tissue of RDEB model mouse af
Slide 43: PJ5 Stem cell gene therapy
Appendix. PJ5 Stem cell gene therapy Therapeutic effects on RDEB skin model We confirmed the effect of gene therapy using patient-derived Bf-MSCs by RDEB skin model and artificially forming blisters by suction method. RDEB skin equivalents RDEB skin equivalents with gene transduced Bf-MSC Negative
Slide 44: Corporate information
Appendix. Corporate information Month/ Company History StemRIM Inc. Year name Established a company aiming to develop new drugs based on the discovery Oct. 2006 of bone marrow multi-potent stem cell mobilization factors identified by Chief Kensuke Tomita (Representative Director) Professor Katsuto T
Slide 45: StemRIM Management
Appendix. StemRIM Management Director Noriko Sawai, External director Kensuke Tomita, Chairman and CEO Head of healthcare team, Social Innovation and Investment Foundation (Aug. 2022 – present) Chairman and CEO, StemRIM Inc. (March 2019 – Present) Impact Officer, Social Innovation and Investment Fou