Research & Development

Nippon Shinyaku’s Basic Policy for R&D

Offering quality pharmaceuticals at the earliest possible time

True to Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” in the pharmaceuticals division we aspire to offer innovative quality pharmaceutical drugs to people troubled by ill health, thus alleviating their suffering and enhancing their wellbeing. We strive to do this by focusing on the areas where Nippon Shinyaku’s strengths shine in particular.

FY2022 Overview

Expanding the pipeline in our focal areas

In March 2022, we obtained manufacturing and marketing approval for MonoVer (code no.: NS-32, generic name: ferric derisomaltose), an iron deficiency anemia treatment in-licensed from Pharmacosmos A/S in 2016, and it launched in March 2023.
Uptravi (code no.: NS-304, generic name: selexipag), a pulmonary arterial hypertension (PAH) treatment launched in 2016, is in a Phase II trial for pediatric indications. In addition, a late-stage Phase II clinical trial is underway to add arteriosclerosis obliterans to the indications for Uptravi.
A global Phase III clinical trial is underway for Viltepso (code no.: NS-065/NCNP-01, generic name: viltolarsen), a Duchenne muscular dystrophy (DMD) treatment launched in Japan and the U.S. in 2020.
In December 2022, Chugai Pharmaceutical Co., Ltd. obtained approval for an additional indication of chronic lymphocytic leukemia for humanized anti-CD20 monoclonal antibody Gazyva (code no.: GA101, generic name: obinutuzumab), and in June 2022, we initiated a Phase III clinical trial for lupus nephritis in collaboration with them. In addition, in March 2023 we initiated a Phase III clinical trial for pediatric nephrotic syndrome in collaboration with Chugai.
In 2019, we entered into an exclusive distribution agreement in Japan with UCB S.A. (formerly Zogenix, Inc.) for Fintepla Oral Solution 2.2 mg/mL (code no.: ZX008, generic name: fenfluramine hydrochloride), a treatment for epileptic seizures associated with Dravet syndrome. In September 2022, UCB S.A. obtained approval for manufacturing and marketing, and Nippon Shinyaku began distribution of the product in November 2022.
NS-161 and NS-025 are now in Phase I clinical trials in Japan for inflammatory and urological diseases, respectively.

Promoting licensing activities

In February 2023, we entered into an exclusive partnership for the Japan distribution of CAP-1002 for the expected indication of DMD with U.S. company Capricor Therapeutics Inc. This agreement follows the exclusive partnership for U.S. distribution we entered with them in January 2022. CAP-1002 is comprised of human allogeneic cardiosphere-derived cells and is expected to be effective in a wide range of DMD patients, regardless of the type of genetic mutation. Capricor Therapeutics Inc. is currently conducting a Phase III clinical trial in the U.S.

FY2023 Initiatives

In the area of intractable and rare diseases, in addition to the DMD treatments to follow Viltepso (NS-089/NCNP-02, NS-050/NCNP-03, and NS-051/NCNP-04), we will develop NS-229 and NS-161 for inflammatory diseases.
As for Nippon Shinyaku’s focal areas and fields, in hematologic malignancies, we will promote the development of a treatment for high-risk acute myeloid leukemia (NS-87) and for myelofibrosis (NS-018). In gynecology, we will promote the development of a treatment for endometriosis (NS-580), and in urology, we are developing NS-025.
As for licensing activities, we will promote the introduction of unique products and developments that complement our R&D activities and expand our R&D pipeline.

Fields of Focus

We strive to do this by concentrating our managerial resources on promoting R&D and maximizing the product value in four fields of focus: urology, hematology, intractable and rare diseases (e.g., pulmonary arterial hypertension [PAH], muscular dystrophy), and gynecology.

Fields of Focus Fields of Focus

Fundamental Technologies

For the future, on our drug discovery foundation that has given birth to the small molecule drug Uptravi, we are adding new modalities and technologies, including nucleic acid molecules, gene therapy, and nucleic DDS technology, further building distinctive drug discovery capabilities not found among other pharmaceutical companies.

Product development

CMC (Chemistry, Manufacturing and Controls) activities are indispensable to develop candidate compounds identified in explorative research into drugs and bring them to patients who need them. CMC activities include process research to establish technologies to mass-produce active pharmaceutical ingredients, research into analytical methods for quality assurance, and formulation development to facilitate product use by patients.