In-house Drug Discovery

Priority areas of drug discovery research

Our drug discovery policy is "developing original and innovative new drugs." We focuses on the areas of oncology, immunology, neurology and specialties; all of which include diseases with high medical needs.

A Drug Discovery System in Each of the Four Priority Areas

Strengthening competitiveness in drug discovery by accumulating and using knowledge and expertise of diseases in each domain.


Research Center of Oncology

As a pioneer in cancer immunotherapy, the Center works toward discovering innovative drugs for cancer patients with the experience, expertise, and know-how we nurtured through R&D of the immune checkpoint inhibitor OPDIVO. The Center is pursuing original and unique drug seeds and new drug modalities not only through open innovation with academia and bio-pharmaceutical companies around the world with cutting edge technologies, but also through promoting translational research.


Research Center of Immunology

Based on many years of its experiences in immunology research, which contributed to creating OPDIVO, the Center is working toward drug discovery in both fields of cancer immunotherapy and autoimmune & allergy therapy by having a research structure with main focus on biopharmaceutical development in immunology. The Center is operated in accordance with the policy of advancing unique research with strong awareness of serendipity and the insight not to miss it.


Research Center of Neurology

The Center focuses on not only neurons as major components of the nervous system, but also glial cells, which maintain and support the environment necessary for the survival and function of neurons.
Through its intensive analysis of patient-derived tissues and iPS cells, the Center is dedicated to discovering innovative drugs to provide disease-modifying therapies, as well as symptomatic treatments, to patients with neurodegenerative diseases, which are becoming serious problems in the aging society, and those with mental disorders or chronic pain, which are quite detrimental to society.


Research Center of Specialty

The Center is working toward discovery of clinically valuable pharmaceutical products for diseases for which treatment is high in unmet needs, regardless of the disease indication. The Center has taken up the challenge of accurately identifying those needs in patients, medical professionals, and society, and then leveraging this knowledge to discover and develop highly original new drugs.

Case Examples

Research Center of Oncology

ONO-4578/EP4 antagonist

Prostaglandin E2(PGE2)produced by cancer cells acts on PGE2receptor EP4 expressed on various immune cells to suppress the action of cancer immunity. ONO-4578 is expected to restore cancer immunity and attack cancer cells by blocking EP4.

COX-2 enzymes expressed on cancer cells produce PGE2, which suppresses the action of cancer immunity through EP4 expressed on various immune cells.
ONO-4578 is a selective antagonist of EP4. The nonclinical experiments have shown that ONO-4578 exerts its antitumor effect by suppressing the action of PGE2 and restoring cancer immunity.
It is also expected to enhance the antitumor effect of our anti-PD-1 antibody nivolumab (Opdivo®).

Research Center of Immunology

ONO-4685/PD-1×CD3 bispecific antibody

PD-1 is a receptor expressed on activated T cells that inhibits activation signals of immune system. CD3 is a component of T-cell activating receptor.
ONO-4685 is a bispecific antibody that targets both PD-1 and CD3, uniquely designed by Research Center of Immunology based on a hypothesis. Clinical trials of ONO-4685 are underway with an expectation of a new treatment of autoimmune diseases.

Using our developed genetically modified mice, Research Center of Immunology has verified the hypothesis that ONO-4685 suppresses activated T cells and shows efficacy in an autoimmune disease model.

Research Center of Neurology

ONO-2910/Schwann cell differentiation promoter

Schwann cells, a kind of glial cell, wrap around axons of peripheral nerve cells to form a myelin sheath and support the function of nerve cells. ONO-2910, which acts on Schwann cells to promote myelination, is expected to improve various symptoms associated with peripheral neuropathy by protecting and promoting regeneration of peripheral nerve cells.

Schwann cell (blue) wraps around the axon of peripheral nerve cell (orange) to form a myelin sheath. ONO-2910 promotes Schwann cell differentiation and myelination.

Research Center of Specialty

Velexbru/BTK inhibitor

Velexbru is an oral Bruton's tyrosine kinase (BTK) inhibitor with high selectivity created by ONO and was approved as the world’s first BTK inhibitor for the treatment of primary central nervous system lymphoma. Velexbru is now under development for diseases other than cancer.

In autoimmune diseases, the signal transduction of B cell receptors (BCRs), which play important roles in immunity, is known to be constitutively activated.
By inhibiting BTK, which plays a central role in BCR signaling, Velexbru is expected to offer advantages also in autoimmune diseases including Pemphigus.