Feb. 17, 2026 R&D Meeting transcript（PDF/5,926KB）

R&D Meeting February 17, 2026

(Total: 106 minutes, Presentation: 50 minutes, Q&A: 56 minutes)

Toru Kimura Representative Director, President and CEO

Motoyuki Sakai Representative Director, Executive Vice President Global Corporate Strategy; Global Finance Administration External Affairs; Corporate Governance; IT Management & Data Analytics

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

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

Koichi Kino Vice President, Head of Corporate Governance

Seiji Wakao JPMorgan Securities

Shinichiro Muraoka Morgan Stanley MUFG Securities Hiroshi Wada SMBC Nikko Securities

I am Kino, Head of Corporate Governance, and I will be your moderator today.

Today's session will be conducted in a hybrid format with a face-to-face meeting at the Osaka headquarters and a Zoom webinar. Analysts and investors are invited to participate online, and media are invited to participate either in person or online. We will also provide a brief opportunity of social gathering for the media.

To ensure smooth proceedings, please change the participant information displayed on your Zoom screen to your company name and your name.

First, I will explain our R&D progress, basic policy, and two major oncology products using the presentation material available on our website, followed by a question-and-answer session. The end time is scheduled for 15:40.

In attendance today are Mr. Kimura, Representative Director, President and CEO, Mr. Sakai, Representative Director, Executive Vice President, Ms. Sato, Managing Executive Officer, and Mr. Murata, Global Strategy Oncology Lead.

First, Mr. Kimura will say a few words.

Last Friday the 13th, the agenda for the Regenerative Medicine Products and Biologics Technology Subcommittee of the Pharmaceutical Affairs Council of the Ministry of Health, Labor and Welfare, scheduled to meet on February 19, was announced. Non-autologous iPS cell-derived dopamine neural progenitor cells, for which we have applied for approval, were also on the agenda. Since the product name AMCHEPRY is to approved by the subcommittee, we had not disclosed it before, but on the 13th, we disclosed that this is our product.

The subcommittee will discuss the approval of AMCHEPRY, and then a final decision will be made by the Minister of Health, Labor and Welfare at a later date. We will keep you informed of any developments in this regard.

First, Sato will explain our R&D progress and basic policy. Ms. Sato, please proceed.

See page four.

Last May we published Reboot 2027. With this, we have declared that, in parallel with the continuation of fundamental structural reforms, we will work to rebuild our foundation as an R&D-oriented firm and pave the way for revival by rebuilding a value creation cycle based on our own innovations.

Here is the status of achievement of R&D milestones aimed for FY2025. Murata will explain the progress in the oncology area later.

In the area of regenerative medicine and cell therapy, As Kimura just mentioned, the Regenerative Medicine and Cell Technology Subcommittee is scheduled to deliberate on our product this week.

In the area of infectious diseases, we are currently conducting Phase I trials of a universal influenza vaccine. We reported last fall that an interim analysis confirmed that the new adjuvant was well tolerated and that antibody titers increased, suggesting the efficacy of the vaccine. We are continuing to analyze cross-reactivity and viral activity to confirm universality.

Here is our pipeline list.

We have shown you about regenerative medicine for Parkinson's disease. You are aware that we are conducting a confirmatory and application study on enzomenib. Other than that, there are no late-stage development items, but rather a pipeline of early-stage development products, and how to move this forward is an urgent and important issue for our company.

We describe our basic R&D strategy here.

We would like to turn the value creation cycle around by discovering new value through in-house drug discovery research, creating value through clinical development, and maximizing value.

Our basic strategy is to maximize and accelerate opportunities by selecting and focusing on our core disease areas of oncology and CNS, with our strengths in small- to mid-molecular drugs and iPS-derived cells as our modality axis. We will implement an agile exit strategy with a strong emphasis on acquiring patient signals early, with a firm focus on identifying the value tipping point.

However, I just mentioned that the core disease areas are oncology and CNS. We recognize that both of these areas have a large number of patients, that unmet medical needs continue to be high, and that the use of small molecule drugs and regenerative cells is advancing in these areas.

We recognize our strength in the design, synthesis, and development of small to mid-molecular drugs and our ability to handle highly challenging target molecules. On the right side, we have shown the results of obtaining priority review designation from the FDA since 2012. 5 items are listed here, items for which we have acquired the designation. They are all low to medium molecular weight. We recognize that this number is one of the highest among domestic pharmaceutical companies.

In oncology and CNS, there are many targets that have been difficult to target with traditional modalities. We believe that we will be able to take advantage of our technological capabilities through the functionalization of small molecule drugs and the use of iPS cells, a cutting-edge modality. In addition, as we have shown here, we have several substantial translational technologies that we believe will allow us to proceed with development with a high degree of certainty of clinical success.

We will further expand and augment our oncology R&D pipeline with existing pipelines such as ORGOVYX, enzomenib, and nuvisertib. We would then like to establish a CNS research pipeline with continuity while monitoring the progress of development of raguneprocel.

Here is some information from the Policy Research Institute's newsletter about the competitiveness of synthetic small molecule drug discovery. We chose Japan and the US as the countries of creation. The vertical axis shows the number of approved products globally, and the breakdown by modality is shown by time period.

What this shows is that Japan's synthetic small molecule drug discovery capabilities are declining as a result of shifting its focus to new modalities. Although new modalities such as antibodies have been introduced, the current number of approved drugs does not significantly compensate for this.

Meanwhile, synthetic small molecules, shown in black, continue to be approved in the United States. The number of approved synthetic small-molecule drugs has been maintained while the number of new modalities has increased. We recognize that the information supports the possibility that synthetic low-molecular-weight drugs may continue.

Here you see our expansion strategy in the field of oncology.

Tier one is "leverage our in-house products." We are working to ensure the continuity in the field of prostate cancer franchise as a next-generation drug creation starting from ORGOVYX.

Tier two is "leverage our in-house pipeline." We will continue to expand our hematopoietic malignancy pipeline by utilizing the knowledge and information obtained through the promotion of enzomenib and nuvisertib development and indication expansion activities.

Tier three is "leverage our in-house technology platform (here, liposomal nanomedicine technology)." We intend to build a pipeline utilizing this technology while continuing to validate the technical basis of the SMP-3124 technology and the targets of the encapsulated compounds.

Here is a brief history of the creation of enzomenib.

We have discovered drug targets through co-creation with academia. enzomenib was created by combining the discovery of new target binding sites, the ability to design compounds that bind efficiently, and the ability of organic synthesis to actually create compounds that can be industrialized.

In addition, in anticipation of fierce competition, we have created this compound by promoting drug discovery activities that emphasize high pharmacological efficacy and avoidance of cardiotoxicity.

This is our drug discovery strategy for acute myeloid leukemia (AML).

We are currently developing enzomenib for KMT2A reconstitution and NPM1 mutation.

Next-generation menin inhibitors are intended for patients who do not respond to existing menin inhibitors or who relapse. We are also working on the discovery of another compound that can cover a different mutation than enzomenib. Ultimately, we hope to build a pipeline group that will cover about 70% of the total.

SMP-3124.

In this regard, we discovered a drug target through co-creation with academia, and found that CHK1 inhibitors are the optimal target for ovarian cancer.

Development of existing prior CHK1 inhibitors has stalled. Focusing on the fact that the lack of efficacy is not the reason for this, but rather the narrow safety zone, we have developed the concept of using liposomal nanomedicine technology to ensure a safe zone through sustained release and drug accumulation effects on cancer tissue.

In addition, we found this compound based on the concept of designing and synthesizing an encapsulated compound suitable for the physical properties of liposome formulations. We are currently confirming this concept in a Phase I study.

I would like to move on to the drug discovery strategy in the CNS area.

In the CNS area, drug development leading to treatment is stagnant, and medical needs remains over the long term. We recognize the high degree of difficulty in drug discovery and the high barriers to entry for other companies. We would like to steadily develop drug discovery by utilizing our long and abundant experience, as well as the assets and strengths we possess.

tier one is a group of products that are expected to have a high probability of clinical success. These are compounds that have unique actions that are clearly supported by their therapeutic effects and that differentiate them from existing drugs. We would like to proceed to confirm the effectiveness concisely by utilizing objective indicators. We will soon begin clinical development of DSP-0378, a drug for rare epilepsy, and a drug candidate for improving motor symptoms of Parkinson's disease.

tier two is for disease-modifying drugs for neurodegenerative diseases. This is an area where pathophysiology is being elucidated, and it is becoming possible to identify drug targets to be addressed. We are in the process of creating several disease-modifying drug candidates, mainly for Parkinson's disease.

tier three is a group of drugs for the treatment of psychiatric symptoms associated with neurological disorders. The experience and assets accumulated in LATUDA and ulotaront will be used for neurodegenerative diseases. We would like to detect efficacy signals early by utilizing biomarkers in diseases with homogeneous backgrounds.

These are all at very young development stages, and our urgent task and goal is to focus on early clinical development and generate late-stage development items.

Here is the history of the discovery of DSP-0378.

Our experience with the older antiepileptic drug EXCEGRAN, as well as other development experience, has provided us with a library of compounds suitable for this classification, from which we found the starting compound for this agent.

We have also identified optimal compounds and points of action for refractory epilepsy through clinically relevant phenotypic screening and multiple pharmacological evaluation systems.

It acts on targeted GABAA receptors with clinically proven efficacy. We confirmed that this has a unique action that is different from existing drugs and also identified translational biomarkers. We are currently in the process of initiating a study to look at efficacy signals while confirming safety for patients.

Based on the above, the world's first practical application of iPS cell-derived products will be approved at the end of this fiscal year, with a time limit on the condition that it be in Japan. We hope to steadily proceed with Phase IV testing based on this.

In addition, we will steadily advance the development of two oncology products, enzomenib and nuvisertib, on which we are focusing our efforts, in order to bring them to the market as valuable drugs.

In the first half of the 2030s, we will continue development so that we can launch the iPS cell-PD program in the US, as well as DSP-0378, which I explained earlier, and a drug that improves the symptoms of Parkinson's disease.

We will continue to make steady progress in the next generation of oncology and CNS products, as well as in the expansion of our regenerative and cellular medicine business, and make a strong contribution to the rebuilding of the value creation cycle of our group.

That's all from me. Thank you very much.

Next, Murata will explain about the two major oncology developments. Mr. Murata, please proceed.

Sato has just introduced the expansion strategy for the cancer strategy. Today, I would like to introduce our efforts to obtain and expand the indications for enzomenib and nuvisertib in tier two, "Leverage our in-house pipeline."

As for data for enzomenib and nuvisertib, we are mainly using data presented at the American Society of Hematology meeting last December. So perhaps some of you have already heard about its contents. I would like to take this opportunity to explain again how we perceive the data and to present the possibilities of these two drugs.

The first is enzomenib.

The mechanism of action is selective menin inhibition. The development phase is currently in Phase II. The planned indication is acute leukemia. Among them, we believe that patients with acute leukemia, such as those with specific genetic mutations called KMT2A rearrangements and NPM1 mutations, will be targeted.

First, let me briefly introduce the diseases those cover.

We have summarized it here under the title Disease Background of Acute Myeloid Leukemia. The cause of this disease is that hematopoietic stem cells in the bone marrow contain progenitor cells that differentiate into myeloid cells, which become cancerous when accompanied by some genetic mutation, leading to leukemia.

As the name "acute" implies, this is an intractable disease that progresses very quickly and has a very poor prognosis if it relapses.

Although the number of new patients is small (21,000 in the US and 8,000 in Japan), once the disease develops, about 50% of patients will have recurrence, and the 5-year survival rate is about 30%, making it a disease with a very poor prognosis. So the medical need for this is very high.