As of October 30, 2026
Psychiatry & Neurology
Small molecule
DSP-0038
| Origin |
in-house (Joint research with Recursion (formerly Exscientia
Ltd.))
|
| Formulation |
oral |
| Planned indication |
- Alzheimer’s disease psychosis
|
DSP-0038 is a novel compound discovered at Sumitomo Pharma with the
assistance of Recursion’s AI technologies. DSP-0038 is a serotonin
5-HT2A receptor antagonist and a serotonin 5-HT1A
receptor agonist. DSP-0038 may demonstrate a large antipsychotic
effect, based on the additive effect of 5-HT2A receptor
antagonist and 5-HT1A receptor agonist. The compound
could also have broad efficacy in the treatment of behavioral and
psychological symptoms of dementia (BPSD) including Alzheimer’s
disease, such as agitation, aggression, anxiety, and depression
symptoms. Furthermore, DSP-0038 has no dopamine D2
receptor antagonistic activity, and therefore may show improved
safety and tolerability compared to existing antipsychotics.
DSP-0187
| Origin |
in-house |
| Formulation |
oral |
| Planned indication |
|
DSP-0187 is a selective orexin 2 receptor agonist. It may improve
excessive daytime sleepiness (EDS) and cataplexy of narcolepsy
caused by orexin deficiency. DSP-0187 also may have potential in
hypersomnia disorders other than narcolepsy.
DSP-3456
| Origin |
in-house |
| Formulation |
oral |
| Planned indication |
- Treatment resistant depression
|
DSP-3456 is a metabotropic glutamate receptor 2/3 negative
allosteric modulator (mGluR2/3 NAM). DSP-3456 may exhibit a
ketamine-like antidepressant effect through selective activation of
the prefrontal cortex via enhanced glutamate release, while avoiding
ketamine-associated side effects (psychotic symptoms, cognitive
dysfunction).
DSP-0378
| Origin |
in-house |
| Formulation |
oral |
| Planned indications |
-
Progressive Myoclonic Epilepsy and Developmental Epileptic
Encephalopathy
|
DSP-0378 is a gamma-aminobutyric acid (GABA)A receptor
positive allosteric modulator. DSP-0378 acts on various subtypes of
GABAA receptors expressed in synaptic and extra synaptic
regions in a manner different from common GABAA receptor
potentiators such as benzodiazepines and neurosteroids. DSP-0378 may
exhibit an antiepileptic effect against a broad range of epilepsies
including Progressive Myoclonic Epilepsy and Developmental Epileptic
Encephalopathy.
DSP-2342
| Origin |
in-house (Joint research with Recursion (formerly Exscientia
Ltd.))
|
| Formulation |
oral |
| Planned indication |
|
DSP-2342 is a novel compound discovered at Sumitomo Pharma with the
assistance of Recursion’s AI technologies. DSP-2342 is a serotonin
5-HT2A and 5-HT7 receptor antagonist. DSP-2342
may demonstrate a broad antipsychotic effect in psychosis, anxiety,
and depression, based on the dual antagonism of 5-HT2A
and 5-HT7 receptors. Furthermore, DSP-2342 has high
selectivity for 5-HT2A and 5-HT7 receptors and
may exhibit a high level of safety and tolerability.
DSP-0551
| Origin |
in-house |
| Formulation |
oral |
| Planned indication |
- Tremor associated with Parkinson’s disease
|
DSP-0551 is a multi-ion channel modulator identified through
phenotype-based drug discovery. It exhibits inhibitory activity
against multiple calcium and sodium ion channels that have been
implicated in tremor. Through these mechanisms, DSP-0551 may
modulate abnormal neuronal firing and excessive synchronization in
neural circuits associated with tremor, thereby improving tremor
associated with Parkinson’s disease.
Regenerative medicine / cell therapy (Collaboration with RACTHERA
Co., Ltd.)
In collaboration with RACTHERA Co., Ltd., and our partners in
industry-academia collaborations, we are advancing the research and
development of regenerative medicine / cell therapy using iPS cells
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 cell-derived dopaminergic neural
progenitor cells)
| Partnering |
Kyoto University, UC San Diego, and others |
| Planned indication |
Parkinson’s disease |
In Japan, CT1-DAP001/DSP-1083 obtained manufacturing and marketing
approval under the conditional and time-limited approval framework
in March 2026 for the indication of the improvement of motor
symptoms in patients with Parkinson’s disease who have an inadequate
response to existing pharmacological therapies, including
levodopa-containing products. The product name is AMCHEPRY® (INN:
raguneprocel).
In the U.S., an investigator-initiated clinical study is being
conducted at UC San Diego and other institutions, while a
company-sponsored study is also being pursued in parallel.
HLCR011 (Allogeneic iPS cell-derived retinal pigment epithelial
cells)
| Partnering |
HEALIOS K.K., Kyushu University, and others |
| Planned indication |
Retinal pigment epithelium tear |
DSP-3077 (Allogeneic iPS cell-derived retinal sheet)
| Partnering |
Massachusetts Eye and Ear in Boston, Massachusetts (Teaching
hospital of Harvard Medical School) and the University of
Minnesota, and others
|
| Planned indication |
Retinitis pigmentosa |
The U.S. Food and Drug Administration (FDA) granted Orphan Drug
Designation for the indication of retinitis pigmentosa in March
2026.
Oncology
Enzomenib
| Origin |
in-house (Joint research with Kyoto University) |
| Formulation |
oral |
| Planned indication |
Acute leukemia |
-
Enzomenib (DSP-5336) is an oral small molecule inhibitor of the
menin and lysine methyltransferase 2A (KMT2A) protein interaction.
Acute myeloid leukemia with KMT2A rearrangement or nucleophosmin 1
(NPM1) mutation relies on the menin-KMT2A interaction for
upregulation of genes instrumental to leukemogenesis. Enzomenib
has been shown to have anti-cancer activity through downregulation
of these genes by inhibition of menin-KMT2A interaction in
preclinical studies. The FDA granted Orphan Drug Designation for
enzomenib for the indication of acute myeloid leukemia in June
2022 and for acute lymphoblastic leukemia in July 2026, and
granted Fast Track Designation for the treatment of relapsed or
refractory acute myeloid leukemia with KMT2A rearrangement or NPM1
mutation in June 2024. In October 2026, the FDA also granted
Breakthrough Therapy Designation for the treatment of relapsed or
refractory acute myeloid leukemia with KMT2A rearrangement.
Furthermore, the MHLW granted Orphan Drug Designation for
enzomenib in September 2024 for the treatment of relapsed or
refractory acute myeloid leukemia with KMT2A rearrangement or NPM1
mutation. In August 2026, the MHLW granted Orphan Drug Designation
for acute myeloid leukemia in an expanded patient population,
including newly diagnosed patients with KMT2A rearrangement or
NPM1 mutation, and for the treatment of relapsed or refractory
acute lymphoblastic leukemia with KMT2A rearrangement.
Nuvisertib
| Origin |
in-house (former Tolero Pharmaceuticals, Inc.) |
| Formulation |
oral |
| Planned indication |
Myelofibrosis |
-
Nuvisertib (TP-3654) inhibits the inflammatory signaling pathways
through inhibition of PIM1 (proviral integration site for Moloney
murine leukemia virus 1) kinase. PIM1 kinase is frequently
overexpressed in various hematologic malignancies and solid
tumors, which could cause evasion of cancer cells' apoptosis and
promotion of tumor growth. The FDA, the MHLW, and the European
Medicines Agency (EMA) granted Orphan Drug Designation for
nuvisertib for the indication of myelofibrosis in May 2022,
November 2024, and July 2025, respectively. Additionally, the FDA
granted nuvisertib Fast Track Designation in June 2025, also for
the indication of myelofibrosis.
SMP-3124
| Origin |
in-house |
| Formulation |
injection (Liposomal Nanomedicine) |
| Planned indication |
Solid tumors |
-
SMP-3124 is an injectable formulation of a CHK1 inhibitor
encapsulated in PEGylated liposomes. CHK1 is a serine/threonine
kinase activated by the DNA damage response, leading to cell-cycle
arrest and DNA repair. CHK1 inhibition leads cancer cells with
high replication stress to apoptosis by inducing further DNA
damage. SMP-3124 may enhance the anti-tumor activity and reduce
side effects of the CHK1 inhibitor through optimized
pharmacokinetics of the compound by liposomal nanoparticle
formulation.
DSP-0390
| Origin |
in-house |
| Formulation |
oral |
| Planned indication |
Glioblastoma |
-
DSP-0390 is an inhibitor of Emopamil Binding Protein (EBP), an
endoplasmic reticulum membrane protein involved in cholesterol
biosynthesis. EBP mediates de novo cholesterol synthesis for cell
membrane structure and signaling, enabling aberrant growth of
tumors. Inhibition of EBP causes depletion of cellular
cholesterol, which may lead to anti-cancer activity. The FDA
granted Orphan Drug Designation for DSP-0390 for the indication of
brain tumor in May 2022.
Others
KSP-1007
| Origin |
in-house (Joint research with The Kitasato Institute) |
| Formulation |
injection |
| Planned indications |
Complicated urinary tract infections and complicated
intra-abdominal infections, hospital-acquired bacterial
pneumonia including ventilator-associated bacterial pneumonia
|
-
KSP-1007 broadly and strongly inhibits β-lactamases, enzymes
produced by bacteria that can degrade carbapenem antibiotics.
KSP-1007 may become an effective treatment option against
carbapenem-resistant bacterial infections as a component of a
combination drug with meropenem hydrate, a carbapenem antibiotic
in general use worldwide (name of Sumitomo Pharma’s product for
the Japanese 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 |
| Planned indication |
Influenza virus prophylaxis |
fH1/DSP-0546LP is a next-generation candidate vaccine formulation composed of the post-fusion hemagglutinin antigen (fH1) that may be effective against a broad range of influenza viruses, and TLR7 adjuvant “DSP-0546LP” that enhances the quantity, quality, and durability of immune responses. 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 preclinical study of fH1/DSP-0546LP demonstrated broad cross-protection against influenza viruses antigenically different from those used in vaccine formulations, and the significance of the TLR7 adjuvant, DSP-0546LP. fH1/DSP-0546LP may improve the breadth and durability of protection against seasonal influenza viruses and be effective against novel and potentially pandemic strains.