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Feb. 17, 2026 R&D Meeting（PDF/2,493KB）

Feb 17, 2026 at 4:18 AM UTC · Issued by Sumitomo Pharma Co.ltd.

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R&D Meeting

February 17, 2026 Sumitomo Pharma Co., Ltd.

Today's Agenda

R&D Progress and Strategy
Managing Executive Officer Research and Development Division
Senior Vice President, Head of Research and Development Division
Chief Development Officer, Sumitomo Pharma America, Inc. Yumi Sato
Two Key Oncology Compounds in Development
- Selective Menin Inhibitor - Enzomenib
- PIM1 Kinase Inhibitor - Nuvisertib
  Global Oncology Strategy Lead
  Lead Senior Vice President, Sumitomo Pharma America, Inc. Masashi Murata
Q&A Session

R&D Progress and Strategy

Managing Executive Officer Research and Development Division

Senior Vice President, Head of Research and Development Division Chief Development Officer, Sumitomo Pharma America, Inc.

Yumi Sato

Reboot 2027

Rebuild our foundation as an R&D-driven pharmaceutical company while continuing the fundamental structural reforms
Reconstruct the Value Creation Cycle driven by our in-house innovation to pave the way toward recovery

Three key products

Establish the Group's revenue base through sales expansion Expand to 250 billion yen (FY2027)

Value Creation Cycle

Regenerative medicine/cell therapy

Start the iPS cell-based drug business with the approval and launch of iPS-PD Expand the business in collaboration with RACTHERA

Oncology

Dedicate resources as a top priority and promote the fastest development (by leveraging partnerships)

enzomenib launch, nuvisertib NDA submission (FY2027)

CNS

Resume development using accumulated expertise and key technologies Expected to become a revenue base after LOE of the three key products

Stabilize the revenue base through sales expansion of the three key products
© Sumitomo Pharma Co., Ltd. All Rights Reserved.
Initiate the construction of the Value Creation Cycle through the commercialization of Regenerative medicine/cell therapy and Oncology businesses 4

Progress Toward the Milestones Set for FY2025

Progress in Oncology (details will be provided in the next section)
- Enzomenib (DSP-5336) : Initiated the confirmatory part of the monotherapy Ph2 study for relapsed/refractory acute leukemia with KMT2A rearrangements, aiming for approval in Japan and the U.S.
  Presented the latest data, including results from combination therapy with venetoclax/azacitidine (Ven/Aza), at ASH 2025
- Nuvisertib (TP-3654) : Advanced Ph1/2 study of monotherapy and combination therapy with momelotinib for relapsed/refractory myelofibrosis, aiming for approval in Japan and the U.S
  Presented the latest data at ASH
- SMP-3124︓Advanced the Ph1/2 study
Progress in Regenerative Medicine/Cell Therapy
- Raguneprocel: Submitted the manufacturing and marketing authorization application in Japan in August 2025 under the Sakigake Designation, and scheduled for review at the Regenerative Medicine and Biologics Committee on Feb. 19, 2026
  Advanced the investigator-initiated clinical study using non-frozen cells and the company-sponsored clinical study using cryopreserved cells in the U.S.
- HLCR011 (retinal pigment epithelial tear, Japan) and DSP-3077 (retinitis pigmentosa, U.S.): Advanced company-sponsored clinical studies
Infectious Diseases
- Universal Influenza Vaccine (fH1/DSP-0546LP): Observed acceptable tolerability of the novel adjuvant and increases in LAH antibody titers in the interim analysis of the Ph1 study. Continued analyses of cross-reactivity and viral activity

Development Pipeline (as of January 30, 2026)

Area	Generic name/Product code	Mechanism of action, etc.	Planned indication(s)	Development stage
Psychiatry & Neurology	DSP-0038	Serotonin 5-HT2A receptor antagonist and serotonin 5-HT1A receptor agonist	Alzheimer's disease psychosis	Phase 1
	DSP-0187＊	Selective orexin 2 receptor agonist	Narcolepsy	Phase 1
	DSP-3456	Metabotropic glutamate receptor 2/3 negative allosteric modulator (mGluR2/3 NAM)	Treatment resistant depression	Phase 1
	DSP-0378	Gamma-aminobutyric acid (GABA) A receptor positive allosteric modulator	Progressive Myoclonic Epilepsy Developmental Epileptic Encephalopathy	Phase 1
	DSP-2342	Serotonin 5-HT2A and 5-HT7 receptor antagonist	To be determined	Phase 1
	CT1-DAP001/DSP-1083 (Japan)	Allogeneic iPS [induced pluripotent stem] cell-derived dopaminergic neural progenitor cells	Parkinson's disease/Investigator-initiated study	MAA submitted in August 2025
	CT1-DAP001/DSP-1083 (U.S.)	Allogeneic iPS cell-derived dopaminergic neural progenitor cells	Parkinson's disease/Investigator-initiated study, Company- sponsored clinical study	Phase 1/2
	HLCR011(Japan)	Allogeneic iPS cell-derived retinal pigment epithelial cells	Retinal pigment epithelium tear	Phase 1/2
	DSP-3077(U.S.)	Allogeneic iPS cell-derived retinal sheet	Retinitis pigmentosa	Phase 1/2
Oncology	Enzomenib/DSP-5336	Selective menin inhibitor	Acute leukemia	Phase 2
	Nuvisertib/TP-3654	PIM1 kinase inhibitor	Myelofibrosis	Phase 1/2
	SMP-3124	CHK1 inhibitor	Solid tumors	Phase 1/2
	DSP-0390	EBP inhibitor	Glioblastoma	Phase 1
Others	KSP-1007	β-lactamase inhibitor	Complicated urinary tract and intraabdominal infections, Hospital-acquired bacterial pneumonia	Phase 1
Others	fH1/DSP-0546LP	Split, Adjuvanted vaccine	Influenza virus prophylaxis	Phase 1

＊Development rights: Japan, China, and certain Asian countries

Basic Strategy and Key Success Factors (KSFs)

Drive the Value Creation Cycle by discovering new value through in-house drug discovery research and creating and maximizing value through clinical development

Basic Strategy

Leverage our strengths in small- to medium-molecule drug discovery and regenerative medicine as core modalities, while maximizing and accelerating opportunities through strategic focus on Oncology and CNS as our priority disease areas
Prioritize early detection of patient signals, identify the Value Inflection Point, and execute agile exit strategies

Our KSFs

Define priority disease areas and build long-term expertise within these areas to strengthen organizational execution capability and increase the probability of success
Define core functions, enhance their capabilities, and gain flexibility to adapt to change in order to strengthen competitiveness
Reduce R&D risk through collaborative research and development, while leveraging partnerships to validate and strengthen our organizational execution capability

SMP's Priority Disease Areas and Breakthrough Drug Discovery Capabilities

Priority Disease Areas: Oncology and CNS

Capitalize on Market Size and Medical Needs
- Capitalize on the large market size and high unmet medical needs in each area by identifying new opportunities for expanding opportunities for use of small molecules and regenerative cell therapies
Fully Leverage Our Drug Discovery Strengths
(see right figure)
- Leverage strong capabilities in the design and development of synthetic small- to medium-molecule compounds to address highly challenging molecular targets
- Target areas where conventional modalities have struggled by leveraging our technological strengths through the enhanced functionality of small-molecule compounds and cutting-edge iPS cell-based modalities
- Enhance the probability of clinical success by leveraging robust translational capabilities, including PDX models*, non-rodent nonclinical evaluation systems, and iPSC-derived disease models
Ensure Continuity of the R&D Pipeline
- Further expand and reinforce the oncology R&D pipeline centered on ORGOVYX®, enzomenib, and nuvisertib
- Advance the regenerative cell product raguneprocel toward commercialization and establish a sustainable CNS pipeline

FDA Priority Review Designation Track Record

(Since 2012)

・Nuvisertib ・Enzomenib ・KSP-1007 ・DSP-7888 ・Ulotaront

Oncology

Fast Track (2025)

Oncology

Fast Track (2024)

Infectious Diseases Fast Track (2022)

Oncology

Breakthrough Therapy (2020)

CNS

Breakthrough Therapy (2019)

Pursue the potential of small-molecule drug discovery amid declining capabilities across Japanese pharma, and maintain a top-tier position in Japan ,building on a consistent track record of FDA Priority Review designations (synthetic products: 4 small molecules, 1 synthetic peptide)

* PDX models: Patient-Derived Xenograft models, A preclinical model in which tumor tissue derived from a patient is transplanted into an immunodeficient mouse to evaluate drug efficacy

Competitiveness Comparison of Synthetic Small-Molecule Drug Discovery (U.S. vs Japan)

Annual Trend of Modalities in the U.S.

OPIR Views and Actions No.70 Nov. 2023; Nationality of Patent-Generating Institutions for NME Approvals in Japan, the U.S., and Europe - Comparison of Approved New Molecular Entity Pharmaceuticals Containing New Active Substances in Japan, the U.S., and Europe -

Source: Prepared by the Office of Pharmaceutical Industry Research (OPIR) based on publicly available information from PMDA, FDA, and EMA, and on Clarivate Analytics Cortellis Competitive Intelligence

Annual Trend of Modalities in Japan

*1 ADC: Antibody-drug conjugate

*2 CAR-T: Chimeric Antigen Receptor T-cell

Note1: The numbers represent the count of products. When multiple institutions are listed as applicants, the count is evenly allocated by nationality. Note2: Products that were approved in two or more regions (Japan, the U.S., and Europe) and received their first approval from any regulatory authority in or after 2013.

U. S.: Continually pursuing the potential of small-molecule drug discovery

Despite the increasing variety of new modalities, the number of approvals for synthetic small-molecule drugs has been maintained
Japan: A significant decline in synthetic small-molecule drug discovery capability following a shift toward new modalities
Although new modalities such as antibodies have been adopted, They have not filled the gap and approvals have declined overall
© Sumitomo Pharma Co., Ltd. All Rights Reserved. CONFIDENTIAL
© Sumitomo Pharma Co., Ltd. All Rights Reserved. 9

Expansion Strategy in Oncology
- Leverage our in-house products, pipeline assets, and technology platforms to drive both pipeline enhancement and the creation of next-generation therapies
- In parallel, explore new targets and technological foundations to build R&D structure capable of sustainable growth
  Leverage Our In-House Products
  Create next-generation therapies originating from ORGOVYX®
  Ensure continuity in the prostate cancer franchise
  Tier
  01
  Leverage Our In-House Pipeline
  Tier
  Identify new indication opportunities for
  enzomenib and nuvisertib
  Expand the hematologic
  02
  malignancy pipeline
  03
  Leverage Our In-House Technology Platform (Liposomal NM*)
  Tier
  Advance development of SMP-3124
  - Verify technological platforms
  - Validate targets of encapsulated compounds

Drive expansion from both the "technology" and "target" perspectives

* Liposomal NM: Liposomal Nanomedicine

Enzomenib（DSP-5336）

Leveraged co-creation with academia as a starting point, combining our precise

compound-design and synthesis capabilities with forward-looking competitive profile design to generate well-differentiated assets

A small-molecule compound with inhibitory protein-protein binding activity for a highly challenging target
Compound design with a complex structure that breaks from conventional norms for orally available small molecules
A robust patent portfolio amid intense competition

Discovery of drug targets driven by co-creation

with academia

Engaged Dr. Akihiko Yokoyama, an early pioneer in menin science, as Principal Investigator through the DSK Project*1
Compound design capabilities and MD simulation＊2
- Discovered new target binding sites and designed compounds with efficient binding properties
- Organic synthesis capability enabled the practical production of industrially scalable compounds
  Profile design anticipated intense competitive environments
- Prioritized strong pharmacological activity while minimizing cardiotoxicity in light of competitive pressures
- Developed biomarkers to enable patient stratification and clinical efficacy prediction
  *1 DSK Project: A cancer drug discovery project under an industry-academia collaboration between Kyoto University and Sumitomo Pharma, from fiscal year 2011 (start of the first term) to fiscal year
  © Sumitomo Pharma Co., Ltd. All Rights Reserved.
  2020 (end of the second term) 11
  *2 MD simulation: Molecular Dynamics simulation A computational method that reproduces the movements of compounds and proteins at the atomic level and evaluates binding modes and stability
  
  Drug Discovery Coverage for Acute Myeloid Leukemia (AML)
  Expect our pipeline to address approximately 70% of AML genetic mutation subtypes, building a comprehensive portfolio for AML treatment
  Others
  30%
  Mutation X
  10%
  KMT2Ar
  5%
  NPM1
  Enzomenib
  Compound A

KMT2A rearrangements (KMT2Ar) and NPM1 mutations (NPM1m)
Monotherapy and combination therapy with VEN/AZA*
Ph2 study ongoing
Next-generation menin inhibitor:
AML Genetic Mutation Subtypes
15%
- Non-response/relapse (acquired resistance) to menin inhibitors
  menin inhibitor

Next-generation

Enzomenib

Monotherapy and combination therapy
Mutation Z
10%
Mutation Y
15%
NPM1+Mutation W
15%
Nonclinical studies in progress
Compound A / B / C:
Treatment-resistant (Mutations X / Y / Z)
Monotherapy and combination therapy
Nonclinical studies in progress
Compound B／Ｃ

*Ven: Venetoclax / Aza: Azacitidine

SMP-3124

Leveraged established precedents in product design to improve the probability of clinical success
Validated Liposomal Nanomedicine technologies through the development of SMP-3124
Discovery of drug targets driven by co-creation
- Encapsulated a small-molecule
  CHK1 inhibitor, whose target relevance in tumors has been clinically validated, into a liposomal formulation
- Utilized liposome/nanoparticle platforms with substantial accumulated clinical evidence
SMP-3124
SMP-3124(Non-encapsulated) Immature Endothelial Cell Immature Endothelial Cell Cancer Cell
Normal Cell
with academia
- Through collaborative research with the Department of Obstetrics and Gynecology at Kyoto University (Dr. Mandai and
  Dr. Hamanishi), evaluated our kinase HTS library using clinical ovarian cancer specimens
- Identified CHK1 inhibition as an optimal therapeutic target for ovarian cancer
  Profile design based on prior precedents
- Recognized that the primary development challenge of
  first-generation CHK1 inhibitors was treatment-related side-effects rather than insufficient efficacy
  Liposomal Nanomedicine technology
- Achieved sustained in-vivo release and preferential tumor accumulation to maintain local drug concentrations, reduce side effects, and enhance efficacy
- Capability for the design and synthesis of encapsulated compounds (selective CHK1 inhibitors) suited to the physicochemical properties of liposomal formulations
- Expanded applicability across a broad range of therapeutic targets through these capabilities

Drug Discovery Strategy in CNS

CNS area continues to show persistent unmet medical needs, as therapeutic development has long since stagnated
Although CNS drug discovery is highly challenging and presents high entry barriers for competitors,
steadily advancing our programs by leveraging our extensive experience, robust assets, and unique strengths
Compounds with strong predicted clinical success
- DSP-0378（GABAA receptor PAM*）
- Candidate compound for improving motor symptoms in Parkinson's disease
  - Ensure clear evidence supporting therapeutic effects
  - Leverage unique mechanisms that differentiate from existing therapies
  - Verify effectiveness concisely by using objective measures
    Tier
    01
    Disease-modifying therapies for neurodegenerative diseases
    - Identify actionable drug targets informed by advancing
  Tier
  - Multiple disease-modifying

candidates centered on Parkinson's disease

disease biology

Leverage the advantages of small molecules in removing intracellular brain aggregates
Detect early efficacy signals in patients using advanced biomarker technologies
Therapies for psychiatric symptoms associated with neurological diseases
- DSP-2342（5-HT

・5-HT

Apply the experience and assets accumulated from the drug
Tier
2A 7
receptor antagonist）
- Multiple candidate compounds for improving multiple psychiatric symptoms

*PAM: Positive Allosteric Modulator

discovery of LATUDA® and ulotaront to neurodegenerative diseases

Detect early efficacy signals by leveraging biomarkers in biologically homogeneous disorders

DSP-0378

Applied a proprietary symptom-based screening strategy to a compound library designed for antiepileptic drugs
Identified a unique mechanism of action against a clinically validated therapeutic target
- Unique pharmacological effects
- Favorable brain penetration
- Desirable safety profile
  GABAA
  Experience from previous antiepileptic drug programs
- Leveraged compound libraries optimized for antiepileptic drugs discovery, built through development of EXCEGRAN® and DSP-0565
- Utilized these libraries to efficiently identify optimal candidate

DSP-0378

GABA

receptors in extrasynaptic regions

Cl-

Excessive neuronal firing

*Image

Glutamatergic excitatory neurons

compounds

Identification of clinically validated targets through a proprietary pharmacological evaluation strategy

Identified optimal candidate compounds for refractory epilepsy through phenotype screening*1 that reflects clinical manifestations and subsequently characterized their mechanism and site of action through multiple
GABAergic nerve terminals
GABAA
receptors in
Glutamic acid
pharmacological evaluation systems
Observed unique activity on the clinically validated target, the
synaptic regions
*1 Phenotype screening: A method for narrowing down compounds by evaluating their effects on disease-relevant features observed in cells or animal models
*2 NHP: Non-human primate
GABAA receptor, that is distinct from existing therapeutics
Design of translational biomarkers
- Identified a translational biomarker (EEG) using NHP*2 models that reliably translates pharmacological effects into clinical outcomes
  © Sumitomo Pharma Co., Ltd. All Rights Reserved. 15
  
  R&D Goals for the Medium- to Long-Term
  FY2025 FY2026
  FY2027
  FY2028-FY2030
  FY2031-FY2033
  Launch of next-generation pipelines
  - Hematological malignancies, Rare
    Sumitomo Pharma's R&D capabilities
    - Capabilities of medicinal chemistry for challenging targets
    - World-leading technology and experience in iPS cells
    - R&D cycle based on data and expertise obtained from enzomenib and nuvisertib
    - Drug discovery and translational research platform in CNS
    - AI and digital technology to drive innovation
      Development and CMC organization that can execute through to commercialization
      neurological & degenerative diseases
      - Launch of SMP-3124
        Expansion of the regenerative medicine/cell therapy business
      - HLCR011, DSP-3077, etc.
    Launch of CNS pipelines
iPS-PD program in the U.S.
DSP-0378
Therapeutic agent for improving symptoms in Parkinson's disease
Commercialization by partners Ulotaront, DSP-0187, infectious disease products, etc.

NDA submission and launch of two oncology compounds

Launch of enzomenib
NDA submission of nuvisertib

World's first practical use of iPS cell-derived products

Obtain domestic conditional and time-limited approval for iPS-PD program

Two Key Oncology Compounds in Development

Global Oncology Strategy Lead Lead Senior Vice President, Sumitomo Pharma America, Inc.

Masashi Murata

Expansion Strategy in Oncology

Leverage our in-house products, pipeline assets, and technology platforms to drive both pipeline enhancement and the creation of next-generation therapies
In parallel, explore new targets and technological foundations to build R&D structure capable of sustainable growth
Leverage Our In-House Products
Create next-generation therapies originating from ORGOVYX®
Strengthen continuity in the prostate cancer franchise
Tier
01
Leverage Our In-House Pipeline
Tier
Advance indication acquisition and
expansion for enzomenib and nuvisertib
Expand the hematologic
02
malignancy pipeline
03
Leverage Our In-House Technology Platform (Liposomal NM*)
Tier
Advance development of SMP-3124
- Verify technological platforms
- Validate targets of encapsulated compounds

Drive expansion from both the "technology" and "target" perspectives

* Liposomal NM: Liposomal Nanomedicine

Enzomenib

Mechanism of action	Selective menin inhibitor
Development stage	Phase 2
Planned indication	Acute leukemia（KMT2A rearrangements, NPM1 mutations）

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