Stemrim Inc. · TSE:4599
Stemrim — Investor Presentation (October 2024)
60 slides from Stemrim's investor presentation, Oct 31, 2024
Slide 1: Presentation Material for Business Plan and Growth Potential
（TSE Growth：4599） Presentation Material for Business Plan and Growth Potential StemRIM aims to br ing a br ighter futur e to patients suffer ing fr om intr actabl e diseases. October 31, 2024
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 TM “Regeneration-Inducing Medicine ” a next generation of regenerative medicine. TM “Regeneration-Inducing Medicine " is new class of medicin
Slide 3: Business Model and R&D Structure
１ Business Model and R&D Structure 3
Slide 4: Our Business Model
Our Business Model About a 10-year development period until the launch of pharmaceutical products Discovery/ Candidates Non-clinical Clinical NDA/ Basic identification/ research Check efficacy studies studies Launch Our business domain Joint research Exclusive license Lump-sum Joint patent applicati
Slide 5: Main Contracts with Pharmaceutical Companies
Main Contracts with Pharmaceutical Companies Total Received Contract Name Company Contract Details (Excerpt) Contract Contract (Date of Agreement) Amount Amount Implementation Shionogi A license is granted for the exclusive development, Not 4,046 License & Co. Ltd. manufacturing, use, or sale of pri
Slide 6: Summary of Financial Results
Summary of Financial Results ・For FY 2024, there were no recognition of milestone revenues related to research progress or upfront payments from contracts. As a result, operating revenue was none . Since we are a drug discovery bio- venture, we have an unstable revenue structure considering our busi
Slide 7: Scientific founder: Prof. Katsuto Tamai
Scientific founder : Prof. Katsuto Tamai ＜Academic Society＞ The Japanese Dermatological Association The Japanese Society of Investigative Dermatology The Japanese Society for Matrix Biology and Medicine The Japanese Cancer Association The Japanese Society for Regenerative Medicine The Japanese Socie
Slide 8: R&D Structure and Facilities
R&D Structure and Facilities Osaka University and StemRIM Institute of Regeneration-Inducing Medicine promote joint research and TM development in “Regeneration-Inducing Medicine ”. TM ・Basic Research in “Regeneration-Inducing Medicine ”. ・Proof of mechanism of action Other domestic and internationa
Slide 9: Mode of Action of Regeneration-Inducing Medicine
2 Mode of Action of “Regeneration-Inducing MedicineTM” 9
Slide 10: 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-Inducing : Damaged site TM Medicine ” Chemokines produced in necrotic tissue (e.g. SDF-1α) Mobilizing S
Slide 11: Discovery of in-vivo mechanism inducing tissue regeneration
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 congenita (Fig
Slide 12: Discovery of in-vivo mechanism inducing tissue regeneration
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 blood flow disrup
Slide 13: Discovery of in-vivo mechanism inducing tissue regeneration
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 Necrosis of epidermal cells Peeled epidermis HMGB1 released Damaged skin Cytoplasm Epidermal cells Nucleus HMGB1
Slide 14: HMGB1 peptide drugs with improved safety
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 Identifying th
Slide 15: Advantages of Regeneration-Inducing Medicine
3 Advantages of “Regeneration-Inducing Medicine ” TM 15
Slide 16: Advantages of Regeneration-Inducing Medicine
TM Advantages of “Regeneration-Inducing Medicine ” 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 mass ⇒ ES ce
Slide 17: Advantages of Regeneration-Inducing Medicine
TM Advantages of “Regeneration-Inducing Medicine ” 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 differentiation is
Slide 18: Expanding Indications and Markets (Number of patients)
Expanding Indications and Markets(Number of patients) Targeting all areas where mesenchymal stem cell ：Clinical trial on going therapy can be effective Other countries and regions Traumatic brain Cardiomyopathy injury 121K in US 13.5M in US In Japan Cerebral infarction Traumatic brain (acute phase)
Slide 19: Functions of mesenchymal stem cells
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 growth bloodstream fact
Slide 20: In vitro culture reduces the functions of MSCs
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 ” Mesenchymal stem cel
Slide 21: Advantages in global expansion: transportation and manufacturing
TM Advantages of “Regeneration-Inducing Medicine ” TM “Regeneration-Inducing Medicine ” products have an advantage over conventional regenerative medicine products in terms of global expansion Issues in the Global Development of Regenerative Medicine Products Using Living Cells Transportation Manufa
Slide 22: Summary of advantages of Regeneration-Inducing Medicine
Summary of advantages of “Regeneration-Inducing MedicineTM” TM “Regeneration-Inducing Medicine ” includes advantages in both cell therapy and chemicals “Regeneration-Inducing TM Medicine ” Cell therapy Chemicals Applicable for large Tissue Applicable for large- tissue damage with large No regenerati
Slide 23: Solving the four major problems of conventional cell therapy
Summary of advantages of “Regeneration-Inducing MedicineTM” 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- stem cells Inducing Medici
Slide 24: Summary of advantages of Regeneration-Inducing Medicine
TM Summary of advantages of “Regeneration-Inducing Medicine ” The “Regeneration-Inducing MedicineTM” technology platform aims to be a game-changer in the field of cell therapy. Efficient mobilization of ectodermal mesenchymal stem cells into Effective Aiming to change the common the bloodstream. acc
Slide 25: Business Areas and Pipeline Progress Status
４ Business Areas and Pipeline Progress Status 25
Slide 26: Overview of Development Pipeline
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. Ongoing * Acute Ischemic Shio
Slide 27: PJ1-01: Redasemtide (Dystrophic Epidermolysis Bullosa)
PJ1-01:Redasemtide(Dystrophic Epidermolysis Bullosa) Additional Phase 2 Protocol Evaluation of efficacy and safety of Redasemtide in patients with dystrophic epidermolysis Study objectives bullosa having intractable ulcers Study design Single arm, multicenter, open label, uncontrolled Intervention R
Slide 28: PJ1-02: Redasemtide (Acute Ischemic Stroke)
PJ1-02：Redasemtide（Acute Ischemic Stroke) Developing a stroke treatment that alleviates time constraints compared to conventional therapies. Acute Ischemic Stroke Market Size in Japan 187 thousand people Mechanism of action Suppression of inflammation MSCs Promotion of angiogenesis and secretion
Slide 29: PJ1-02: Global Phase 2b Protocol
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 at age 18 or Subject older population • Baseline NIHSS s
Slide 30: PJ1-03: Phase 2 Clinical Trial in Ischemic Cardiomyopathy
PJ1-03:Phase 2 Clinical Trial in Ischemic Cardiomyopathy Ischemic Cardiomyopathy Phase 2 Protocol Study Evaluation of the efficacy and safety of Redasemtide in objectives patients with ischemic cardiomyopathy Study design Multicenter, Randomized, Double-blind, Placebo-controlled Infarct site Ectoder
Slide 31: PJ1-04: Redasemtide (Knee Osteoarthritis)
PJ1-04：Redasemtide(Knee Osteoarthritis) Developing a treatment for osteoarthritis of the knee that does not rely on surgical interventions. Knee Osteoarthritis（OA） Market size in Japan thigh thigh ・Number of patients with symptoms: arthrocyte bone bone bone cyst approximately 8 million Outside ・Esti
Slide 32: PJ1-04: Redasemtide (Knee Osteoarthritis) Phase 2 Results
PJ1-04：Redasemtide(Knee Osteoarthritis) Phase 2 Protocol Study Evaluation of efficacy and safety of Redasemtide in Phase 2 Clinical Trial Results objectives patients with Osteoarthritis (OA) of the knee Safety Evaluation Subject Patients with knee OA who have undergone high tibial No severe adverse
Slide 33: PJ1-05: Redasemtide (Chronic Liver Disease)
PJ1-05：Redasemtide(Chronic Liver Disease) Developing a treatment for chronic liver disease that works by inhibiting fibrosis. Chronic Liver Disease Market size in Japan Healthy Chronic Liver Disease Approximately 400,000 to 500,000 people Fibrosis Mechanism of action progresses Supply of due to MSC
Slide 34: PJ1-05: Phase 2 Clinical Trial Results
PJ1-05：Redasemtide(Chronic Liver Disease) Phase 2 Protocol Phase 2 Clinical Trial Results Evaluate the safety and exploratory efficacy in Main purpose Safety Evaluation: Presence and Incidence of Adverse Events patients with chronic liver disease 2 mild adverse events (voice disorder and fever) with
Slide 35: PJ3 Novel Regeneration-Inducing Peptide for Local Administration
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 act like S
Slide 36: PJ4 Autologous cell collection device for treatment
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）Take out
Slide 37: PJ4 Confirmation of MSC harvesting in a rat model
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 device Dev
Slide 38: PJ5 Stem cell gene therapy
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 patient. Collect
Slide 39: PJ5 Stem cell gene therapy SR-GT1
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 the patien
Slide 40: PJ5 Efficacy and duration in RDEB model mice
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 after admini
Slide 41: PJ5 Therapeutic effects on RDEB skin model
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 epidermis
Slide 42: Future Growth Strategy
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Slide 43: Future Growth Strategy
Future Growth Strategy Development of next-generation “Regeneration-Inducing MedicineTM” ・Promote licensing activities for new compounds with significant activity in non-clinical trials. ・Expand business development activities with a global development perspective, not limited to domestic companies.
Slide 44: Next-generation Regeneration-Inducing Medicine TRIM3, TRIM4
TM Next-generation “Regeneration-Inducing Medicine ” TRIM3, TRIM4 We have identified several peptides that mobilize mesenchymal stem cells from the bone marrow into the bloodstream, accumulate in damaged tissues, and induce functional regeneration. Among them, two peptides with particularly prominen
Slide 45: Out-licensing activities for new Regeneration-Inducing Medicine
TM Out-licensing activities for new “Regeneration-Inducing Medicine ” FY 2024.7 Conference Participation BioJapan 2023 2023.10.11～13 ＠Yokohama 42nd Annual J.P. Morgan Healthcare Conference 2024.1.8～11 ＠San Francisco, CA BIO International Convention 2024 2024.6.3～6 @San Diego, CA FY 2023.7 Conference
Slide 46: IP Strategy
IP Strategy TM Patents related to “Regeneration-Inducing Medicine ” have been granted in various countries. We are steadily promoting the intellectual property protection of our research outcomes, paving the way for global expansion. Patent Grant Status Number of IP （Case） Total Patents 125（+12） +12
Slide 47: Activities of StemRIM Institute of Regeneration-Inducing Medicine, Osaka University
Activities of “StemRIM Institute of Regeneration-Inducing Medicine, Osaka University” In June 2020, StemRIM Institute of Regeneration-Inducing Medicine, Osaka University (covering an area of 1,540 square meters) was established on the 6th and 7th floors of the Techno Alliance Building at Osaka Unive
Slide 48: Pipeline Development Plan
Pipeline Development Plan Given the long development timeline for pharmaceuticals, a pipeline development plan is formulated based on a long-term management vision. Pipeline Indication Status Development Scheｄule Add. Phase 2 End Epidermolysis Additional P2 Study Application bullosa Ongoing Global P
Slide 49: Forecast for the fiscal year ending July 2025
Forecast for the fiscal year ending July 2025 Research and Development Progress for the Fiscal Year Ended July 2024 Previous Disclosure (October 31, 2023) Actual Results Research Progress Outlook for the Fiscal Year Ending July 2024 Research Progress Results for the Fiscal Year Ended July 2024 Resea
Slide 50: Allocation Status of Raised Funds
Allocation Status of Raised Funds (Amount: million yen) Planned Amount Actual Disbursement Undisbursed Funding Method Use of Funds Disbursement Raised Amount(Timing) Amount(Timing) Amount(Timing) Funds for establishing the 7,195 ― ― “Regeneration-Inducing MedicineTM” Stock issuance research institut
Slide 51: Approach to Sustainability
Approach to Sustainability TM Under the corporate philosophy of ‘Overcoming Refractory Diseases by “Regeneration-Inducing Medicine ” ,’ we are actively engaged in the development of next-generation pharmaceuticals, “Regeneration-Inducing TM Medicine ” , which overcome the challenges of conventional
Slide 52: Sustainability Initiatives
Sustainability Initiatives 1 Fostering a workplace culture where diverse talent can thrive We recognize that securing and continuously developing diverse talent with advanced expertise, skills, and experience, as well as fostering a workplace culture where employees can thrive, are critical to achie
Slide 53: Sustainability Initiatives
Sustainability Initiatives ２ Creating a Safe and Comfortable Working Environment We believe that supporting our employees' growth to help them thrive leads to sustainable and stable organizational development. With the aim of creating a more comfortable working environment, we regularly review and u
Slide 54: Sustainability Initiatives
Sustainability Initiatives 3 Initiatives related to intellectual property Our company operates a business model in which we license out the development and sales rights of the pharmaceuticals we develop to pharmaceutical companies, generating revenue through upfront payments, milestone payments, and
Slide 55: Risk Information
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Slide 56: Risk Information
Risk Information Possibility and Risk Timing of Countermeasures Materialization A) Uncertainty in the ・Risk of development delays or discontinuation due to the Medium / As ・We aim to maintain multiple development of inability to discover beneficial effects in clinical trials. needed pipelines in cli
Slide 57: Appendix
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Slide 58: StemRIM Management
StemRIM Management Director Noriko Sawai, External director Masatsune Okajima, Head of healthcare team, Social Innovation and Investment President and CEO Foundation (Aug. 2022 – present) Impact Officer, Social Innovation and Investment President and CEO, StemRIM Inc. (Oct. 2023 – Present) Foundatio
Slide 59: Corporate information
Corporate information Month/ Company History StemRIM Inc. Year name Established a company aiming to develop new drugs based on the Oct. 2006 discovery of bone marrow multi-potent stem cell mobilization factors Chief Masatsune Okajima (Representative identified by Professor Katsuto Tamai of the Gradu
Slide 60: Disclaimer
Disclaimer This document is based on economic, regulatory, market, and other conditions as of its publication date, and neither the company nor its representatives guarantee the accuracy or completeness of the information contained herein. The information may change without prior notice, and such ch