Kyowa Kirin Co., Ltd. (TYO:4151)
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May 1, 2026, 3:30 PM JST
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J.P. Morgan Healthcare Conference

Jan 9, 2022

Masashi Miyamoto
President and CEO, Kyowa Kirin

I am Masashi Miyamoto, President and CEO of Kyowa Kirin. It's a great honor to participate in the JPMorgan Healthcare Conference. As this is the first time Kyowa Kirin is presenting at this conference, I'd like to take this opportunity to talk about who we are, our contribution to science and the patients, and our vision towards the future. All my comments must be qualified by this statement. This is today's agenda, and let me start from who we are. We are Japan-based global specialty pharmaceutical company with the vision shown here. We will realize the successful creation and delivery of life-changing value that ultimately makes people smile as a Japan-based global specialty pharmaceutical company built on a diverse team of experts with a shared passion for innovation.

In order to make this vision a reality by 2030, we focus on provide pharmaceuticals for unmet medical needs, address patient-centric healthcare needs, and retain the trust of a society. Here is the overview of Kyowa Kirin in 2020, such as annual sales and the number of employees. In 2020, the Kyowa Kirin group is comprised of 38 affiliates in four key regions of Japan, EMEA, North America and Asia, with 48% of consolidated sales revenue coming from outside Japan. Next, let's take a brief look at our history and the contribution we have made to society by delivering important medicines to meet patients' needs. Kyowa Kirin was created from the merger of Kyowa Hakko and Kirin Pharma in 2008.

At that time, both companies had a strong legacy of fermentation technology applied to pharmaceutical manufacturing and also enjoyed long-lasting relationship with research institutions and biotech companies. For example, Kirin Pharma had a 30-year joint venture with Amgen. Both companies established antibody technologies such as POTELLIGENT and fully human antibody producing technologies, which have spurred innovations in our portfolio. After the merger, the company began to seek to expand its operation globally. Over the past 13 years, we have followed that plan, expanding through organic growth and targeted acquisitions. Since 2018, we have received FDA approval on three medicines discovered and developed in-house, Crysvita, Poteligeo, and Nourianz. Here is a list of our major products. In Japan and some Asian countries, we market some products licensed from Kirin-Amgen, a joint venture with Amgen.

These products, known in the U.S. as Aranesp, Neulasta, Nplate, are the great achievement of the joint venture. Here is an overview of our proprietary technologies that support in-house drug discovery. We have POTELLIGENT and fully human antibody producing technology, which are utilized for research and manufacturing. POTELLIGENT is one of our proprietary technologies that create 100% fucose-free monoclonal antibodies, demonstrating the marked increase in antibody dependent cellular cytotoxicity, ADCC. As you may know, ADCC is a critical function of the immune system that enhance the ability of antibodies to kill target cell. This POTELLIGENT technology has been used for our products and drug candidates. In addition, it has been licensed out and utilized by numerous major pharmaceutical and biotech companies in their pipelines.

The marketed product in which we have applied this technology include Poteligeo, our drug for cutaneous T-cell lymphoma, Fasenra, an asthma drug licensed out to AstraZeneca, and GSK's Blenrep, a drug for multiple myeloma. We also have our proprietary human antibody producing technology applied for several drugs and drug candidates. The product launched using this technology include Crysvita, a treatment for X-linked hypophosphatemia. In addition, KHK4083 also known as AMG 451 is being developed for the treatment of atopic dermatitis by applying both technologies. As for our next generation technologies to pursue our life-changing value creation, we are exploring some promising new technologies. The first is our bispecific antibody technology that would create a rich drug pipeline.

We are working hard to have this technology to enter clinical trials. Also, we hope that open innovation will lead to the discovery of innovative new drugs and the creation of fundamental technologies to support them. We value our partnership with outside collaborators as they are essential to reinforce the strengths of our technologies into life-changing value. Now let's take a look at the status of our growing global products. During the previous medium term plan period, we succeeded in launching three in-house drugs, Crysvita, Poteligeo, and Nourianz in the global market. These products are growing steadily, and I am confident that these will drive the company's growth toward 2025. I'm going to introduce each product briefly. The first is Crysvita, an anti-FGF23 antibody showing the most significant growth among the three global products.

We aim to realize growth by steadily delivering Crysvita to more patients while establishing its position as the standard treatment for XLH and unresectable TIO. Through three global observational studies on XLH, we are obtaining better understanding of patients that can be applied for market expansion. Next, let me talk about Poteligeo, an anti-CCR4 humanized antibody to treat patients with indications shown in the slide. Poteligeo is the world's first marketed drug that uses Potelligent that I mentioned earlier, and it can eliminate CCR4-positive tumor cells from patients' bodies. Poteligeo is steadily gaining market penetration and sales territories are expanding. Now let's move on to Nourianz. It is known that dopaminergic drug used in the treatment of Parkinson's disease gradually became ineffective over a long treatment period, resulting in the complication of Parkinson's disease.

Nourianz is a first new mechanism of action drug for Parkinson's disease treatment indication and is an innovative adenosine A2A receptor antagonist that we discovered. We are also developing the next generation strategic products that are expected to follow the existing 3 global products, and I am confident that these products will continue to accelerate our growth in 2025 and beyond. We will proceed with careful and rapid development to maximize the potential of these 5 candidate drugs. KHK4083, AMG 451 is a human anti-OX40 monoclonal antibody created with our POTELLIGENT technology and human antibody producing technology. As is commonly known, activation of TH2 cells is central in atopic dermatitis. KHK4083 blocks the OX40 signaling pathway, which plays a major role in upstream T cell activation by OX40 inhibition or OX40 positive cell depletion.

As expected, clinical results based on this mechanism of action have been obtained to date. The phase II clinical trial of KHK4083 was conducted in a placebo-controlled manner. We are pleased to see that the primary endpoint was met in all those groups, and there were no serious safety concerns shown. Continued treatment with KHK4083 was found to increase clinical efficacy beyond week 16. In particular, the improvement in clinical symptom persists even after treatment. This is an innovative feature that may differentiate KHK4083 from other biologics. To maximize the product's value of KHK4083 AMG 451 for atopic dermatitis and its potential in other autoimmune disease treatment, Kyowa Kirin and Amgen have entered into an agreement to develop and commercialize KHK4083 AMG 451 jointly. Our agreement to co-development and co-commercialize KHK4083 made news in 2021.

With Amgen, we are planning to initiate a phase III study of KHK4083 in the first half of 2022. We will also explore the potential use of KHK4083 in indications beyond atopic dermatitis. Next, let me talk about Zandelisib. Zandelisib is a good fit with our hemato oncology portfolio, which includes Poteligeo. Zandelisib is a selective PI3K delta inhibitor being studied initially as a third-line treatment of relapsed or refractory follicular lymphoma in the U.S. where there are more than 8,000 patients. Zandelisib has innovative molecular and biologic properties that show the potential to avoid safety issues that are common among other PI3K treatments. On November 4, 2021, FDA granted orphan drug designation to Zandelisib for follicular lymphoma. For the pivotal phase II TIDAL study, top-line data was obtained in fourth quarter last year.

We see this as a very positive result, and I am looking forward to this drug. Together with MEI Pharma, we look forward to sharing more details of the study with you at one of the medical meetings this year. We are actively planning and considering additional studies to explore the full potential of Zandelisib as a backbone therapy for various B-cell malignancy treatment. Next, I would like to talk about Bardoxolone methyl, known as RTA 402. The number of patients on dialysis is increasing in Japan and diabetic nephropathy is the leading cause of the new dialysis cases.

Given this bardoxolone methyl has opportunities as it activates the system that plays an important role in defense responses against oxidative stress as its convincing clinical data and priority review designation support its potential market opportunity in Japan and as it could be the first drug addressing the need for renal function protection and improvement. The phase II TSUBAKI study showed improvement in glomerular filtration rate, GFR, a major indicator of kidney filtration function by direct measurement, and there were no severe safety issues. We are pleased to be able to create a drug with such a new effect as no drug has ever shown data to improve GFR until now. The phase III AYAME study is scheduled to be complete this year, and then the top-line data is available. There are various catalysts for 2022, so please stay tuned.

In particular, I hope you will look forward to starts of several phase III clinical trials, including KHK4083 and KW-6356. We will announce in a timely manner when phase III studies actually start. Here is today's last section. We will make a firm commitment to sustainable growth through 2030. On top of the three main products that we continue to expand, we hope to introduce next generation products and more innovative technologies to support the future of Kyowa Kirin. In order to do so, we will work on open innovation by developing our own technologies as well as successfully incorporating useful technologies from outside. We will surely produce life-changing value that will make patients smile. We will continue to strive for this goal.

For the next four years, we will manage our business according to the quantitative KPIs shown here. We are continuously carrying out activities to deliver life-changing value to even more people by challenging ourselves in the ongoing creation of innovation required by patients. Thank you very much for your time and attention for Kyowa Kirin.

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