Profiles of Major Products under Development

New drug candidate compounds under development are listed.

As of May 13, 2025

Psychiatry & Neurology

Small molecule

DSP-0038

Origin in-house (Joint research with Exscientia Ltd.)
Formulation oral
Development stage
  • Alzheimer’s disease psychosis: Phase 1 in the U.S.

DSP-0038 is a novel compound discovered at Sumitomo Pharma using Exscientia’s AI technologies. DSP-0038 is a serotonin 5-HT2A receptor antagonist and a serotonin 5-HT1A receptor agonist. DSP-0038 is expected to demonstrate a greater antipsychotic effect, based on the additive effect of 5-HT2A receptor antagonist and 5-HT1A receptor agonist. The compound could also have a broader efficacy in the treatment of behavioral and psychological symptoms of dementia (BPSD) which include agitation, aggression, anxiety, and depression. Furthermore, DSP-0038 has negligible affinity for dopamine D2 receptors, and therefore it can be expected to show improved safety and tolerability compared to existing antipsychotics.

DSP-0187

Origin in-house
Formulation oral
Development stage
  • Narcolepsy: Phase 1 in Japan

DSP-0187 is an orexin 2 receptor agonist. It is expected to improve excessive daytime sleepiness (EDS) and cataplexy of narcolepsy caused by orexin deficiency. DSP-0187 is also expected to demonstrate an efficacy for EDS other than narcolepsy. Sumitomo Pharma granted Jazz Pharmaceuticals plc the exclusive development and commercialization rights in the territories, except for Japan, China, and certain other Asia/Pacific markets in April 2022.

DSP-3456

Origin in-house
Formulation oral
Development stage
  • Treatment resistant depression: Phase 1 in the U.S.

DSP-3456 is a metabotropic glutamate receptor 2/3 negative allosteric modulator (mGluR2/3 NAM). DSP-3456 is expected to exhibit a ketamine-like antidepressant effect through selective activation of the prefrontal cortex by enhancing the glutamate release, while avoiding side effects (psychotic symptoms, cognitive dysfunction).

DSP-0378

Origin in-house
Formulation oral
Development stage
  • Progressive Myoclonic Epilepsy and Developmental Epileptic Encephalopathy: Phase 1 in Japan

DSP-0378 is a gamma-aminobutyric acid (GABA) A receptor positive allosteric modulator. It acts on various subtypes of GABAA receptors expressed in synaptic and extrasynaptic regions in a manner different from common GABAA receptor potentiators such as benzodiazepines and neurosteroids. It is expected to exhibit an antiepileptic effect against broad epilepsies including Progressive Myoclonic Epilepsy and Developmental Epileptic Encephalopathy.

DSP-2342

Origin in-house (Joint research with Exscientia Ltd.)
Formulation oral
Development stage
  • Phase 1 in the U.S.

DSP-2342 is a novel compound discovered at Sumitomo Pharma using Exscientia's AI technologies. DSP-2342 is a serotonin 5-HT2A and 5-HT7 receptor antagonist. DSP-2342 is expected to demonstrate a broader antipsychotic effect which includes psychosis, anxiety, and depression, based on the additive effect of 5-HT2A and 5-HT7 receptor antagonist. Furthermore, DSP-2342 has high selectivity for 5-HT2A and 5-HT7 receptors, which can be expected to show a high level of safety and tolerability.

Regenerative medicine / cell therapy (Collaboration with RACTHERA Co., Ltd.)

In collaboration with RACTHERA Co., Ltd. and our partners in the industry-academia collaboration, we are developing allogeneic iPS cell-derived products using iPS cells from healthy donors for the treatment of Parkinson’s disease, RPE (retinal pigment epithelium) tear, AMD (age-related macular degeneration), retinitis pigmentosa, and spinal cord injury.

CT1-DAP001/DSP-1083 (Allogeneic iPS [induced pluripotent stem] cell-derived dopaminergic neural progenitor cells)

Partnering Kyoto University CiRA, University of California San Diego School of Medicine
Development stage
  • Parkinson’s disease: Under preparation for the NDA in Japan
  • Parkinson’s disease: Phase 1/2 (Investigator-initiated study, Sponsor: University of California San Diego School of Medicine) in the U.S.
  • Parkinson’s disease: Phase 1/2 (Company-sponsored clinical study) in the U.S.
    The Ministry of Health, Labour and Welfare (MHLW) designated “Sakigake Designation System” product for regenerative medicine & cell therapy for the indication of Parkinson's disease in February 2017.

HLCR011 (Allogeneic iPS cell-derived retinal pigment epithelial cells)

Partnering Healios
Development stage
  • Retinal pigment epithelium tear: Phase 1/2 in Japan

DSP-3077 (Allogeneic iPS cell-derived retinal sheet)

Partnering Massachusetts Eye and Ear in Boston, Massachusetts (Teaching hospital of Harvard Medical School), USA
Development stage
  • Retinitis pigmentosa : Phase 1/2 in the U.S.

Oncology

enzomenib/DSP-5336

Origin in-house (Joint research with Kyoto University)
Formulation oral
Development stage
  • Acute leukemia: Phase 2 in the U.S. and Japan
  • Enzomenib (DSP-5336) is a small molecule inhibitor against the binding of menin and mixed-lineage leukemia (MLL) protein. Acute myeloid leukemia with MLL rearrangements or nucleophosmin 1 (NPM1) mutations rely on the menin-MLL interaction for upregulation of genes instrumental to leukemogenesis. Enzomenib has been shown to have anti-cancer activity through downregulation of the genes by inhibition of menin-MLL interaction in pre-clinical studies. The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022 and granted Fast Track Designation for the treatment of relapsed or refractory acute myeloid leukemia with MLL rearrangement or NPM1 mutation in June 2024. Furthermore, the Ministry of Health, Labour and Welfare in Japan granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLL rearrangement or NPM1 mutation in September 2024.

nuvisertib/TP-3654

Origin in-house (former Tolero Pharmaceuticals, Inc.)
Formulation oral
Development stage
  • Myelofibrosis: Phase 1/2 in the U.S. and Japan
  • Nuvisertib (TP-3654) inhibits the inflammatory signaling pathways through inhibition of PIM1 (proviral integration site for Moloney murine leukemia virus 1) kinases. PIM1 kinases are frequently overexpressed in various hematologic malignancies and solid tumors, allowing cancer cells to evade apoptosis and promoting tumor growth. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in May 2022. In addition, the Ministry of Health, Labour and Welfare in Japan granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in November 2024.

DSP-0390

Origin in-house
Formulation oral
Development stage
  • Glioblastoma: Phase 1 in the U.S. and Japan
  • DSP-0390 is an inhibitor of Emopamil Binding Protein (EBP), which is one of cholesterol biosynthetic enzymes. EBP is an endoplastic reticulum membrane protein involved in cholesterol biosynthesis. When functional, EBP mediates de novo cholesterol synthesis for cell membrane structure and signaling, enabling aberrant growth of tumors. Inhibition of EBP causes an efficient cellular cholesterol depletion and it is expected to show anti-cancer activities. The FDA granted Orphan Drug Designation for DSP-0390 for the indication of brain cancer in May 2022.

SMP-3124

Origin in-house
Formulation injection (Liposomal Nanomedicine)
Development stage
  • Solid tumors: Phase 1/2 in the U.S. and Japan
  • SMP-3124 is an injection, a liposomally encapsulated CHK1 (checkpoint kinase 1) inhibitor. CHK1 is activated by DNA damage response, then arrests the cell cycle, and induces DNA repair via serine-threonine kinase. CHK1 inhibition leads cancer cell with high replication stress to apoptosis by inducing further DNA damages. SMP-3124 is expected to strengthen the anti-tumor activity and weaken side effects by changing pharmacokinetics of the compound with liposomal nanomedicinal encapsulation.

Others

KSP-1007

Origin in-house (Joint research with The Kitasato Institute)
Formulation injection
Development stage
  • Complicated urinary tract and intra-abdominal infections, Hospital-acquired bacterial pneumonia including ventilator-associated bacterial pneumonia: Phase 1 in the U.S. and Japan
  • KSP-1007 can broadly and strongly inhibit β-lactamases, enzymes produced by bacteria that can degrade carbapenem antibiotics. KSP-1007 is expected to become an effective treatment option against carbapenem-resistant bacterial infections in a combination drug with meropenem hydrate, a carbapenem antibiotic in general use worldwide (name of Sumitomo Pharma’s product for the domestic market: MEROPEN®). The FDA granted Qualified Infectious Disease Product (QIDP) status and Fast Track Designation for KSP-1007 for the indications of complicated urinary tract infections, complicated intra-abdominal infections, and hospital-acquired bacterial pneumonia including ventilator-associated bacterial pneumonia in August 2022.

fH1/DSP-0546LP

Origin in-house (Joint research with the National Institutes of Biomedical Innovation, Health and Nutrition)
Formulation injection
Development stage
  • Influenza: Phase 1 in Europe
  • fH1/DSP-0546LP is the next-generation candidate vaccine formulation composed of the post-fusion hemagglutinin antigen (fH1) that is expected to be effective against a broad range of influenza viruses, and TLR7 adjuvant “DSP-0546LP” that enhances the quantity, quality, and durability of immune response. Conventional influenza vaccines lose effectiveness due to viral mutations, making it necessary to select, produce, and inoculate a vaccine to immunize against strains predicted to circulate each year. They may also not respond well to emerging strains of influenza. The pre-clinical study of fH1/DSP-0546LP demonstrated the broad cross protection against influenza viruses antigenically different from those used in vaccine formulations, and indicated the significance of the TLR7 adjuvant, DSP-0546LP. It is expected that fH1/DSP-0546LP improves the breadth and durability of protection against seasonal influenza viruses and is effective against novel and potentially pandemic strains.