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Takeda Announces U.S. FDA Breakthrough Therapy Designation Granted for Pevonedistat for the Treatment of Patients with Higher-Risk Myelodysplastic Syndromes (HR-MDS)

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Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for its investigational drug pevonedistat for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS). Pevonedistat, a first in class NEDD8-activating enzyme (NAE) inhibitor, could be the first novel treatment for HR-MDS patients in more than a decade, expanding treatment options that have so far been limited to hypomethylating agent (HMA) monotherapy alone. Even with current treatment options, outcomes for people living with HR-MDS remain poor.

The Breakthrough Therapy Designation is based on the final analysis of the Pevonedistat-2001 Phase 2 study, which evaluated pevonedistat plus azacitidine versus azacitidine alone in patients with rare leukemias, including HR-MDS. The FDA considered a number of endpoints, including overall survival (OS), event-free survival (EFS), complete remission (CR) and transfusion independence, as well as the adverse event profile. This designation signals a potential advancement in addressing the needs of people living with HR-MDS, for whom few therapies exist and the benefits are limited.

“Higher-risk MDS is associated with poor prognosis, diminished quality of life and a higher chance of transformation to acute myeloid leukemia, another aggressive cancer. The combination of pevonedistat and azacitidine is a promising therapeutic approach with the potential to be the first novel treatment advancement for higher-risk MDS in more than 10 years,” said Christopher Arendt, Head, Oncology Therapeutic Area Unit, Takeda. “We thank the FDA for recognizing pevonedistat, and the urgency to develop innovative therapies that address critical treatment needs for higher-risk MDS, a patient population with few options.”

Breakthrough Therapy Designation from the U.S. FDA is granted to accelerate the development and regulatory review of investigational drugs that are intended to treat serious or life-threatening ailments. Agents with this designation have shown preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Takeda presented results of the Pevonedistat-2001 trial during oral sessions at the virtual 56th American Society of Clinical Oncology (ASCO) Annual Meeting and virtual 25th European Hematology Association (EHA) Annual Congress.

About Pevonedistat

Pevonedistat is a first in class NEDD8-activating enzyme (NAE) inhibitor. In pre-clinical studies, the inhibition of NAE by pevonedistat blocked the modification of select proteins, which resulted in disruption of cell cycle progression and cell survival, leading to cancer cell death. Pevonedistat in combination with azacitidine demonstrated promising clinical activity in a Phase 2 study of patients with higher-risk myelodysplastic syndromes (HR-MDS), higher-risk chronic myelomonocytic leukemia (HR-CMML) and acute myeloid leukemia (AML) and a Phase 1 study of patients with AML. Pevonedistat is currently being evaluated in Phase 3 studies as a first-line treatment for patients with HR-MDS, HR-CMML and AML, who are ineligible (unfit) for transplant or intensive induction chemotherapy, and in a Phase 2 study in unfit AML in a triple combination with azacitidine and venetoclax. Pevonedistat is an investigational drug for which safety and efficacy have not been established.

About MDS
MDS is a rare form of bone marrow-related cancer caused by irregular blood cell production within the bone marrow. This cancer most commonly affects older patients, with the median age of diagnosis ranging from 60 to 74 years. As a result of this irregular production, a person with MDS does not have enough normal red blood cells, white blood cells and/or platelets in circulation. Symptoms for MDS are often vague and related to low blood counts, and may include fatigue, shortness of breath, easy bruising or bleeding, loss of appetite, weakness, pale skin, fever and frequent or severe infections.

There are several classifications of MDS – very low-risk to very high-risk – determined by blood counts, blast counts, mutations and cytogenetics. Higher-risk disease is defined as intermediate, high or very high risk on the International Prognostic Scoring System – Revised (IPSS-R), and these patients have a poorer prognosis. Approximately 40% of patients with HR-MDS transform to AML, another aggressive cancer with poor outcomes.

Takeda’s Commitment to Oncology
Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com

Important Notice
For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements
This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/reports/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.

Contact information

Japanese Media
Kazumi Kobayashi
kazumi.kobayashi@takeda.com
+81 (0) 3-3278-2095

Media outside Japan
Sara Noonan
sara.noonan@takeda.com
+1 617-551-3683

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