Business Wire

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

Share

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 56thAmerican Society of Clinical Oncology (ASCO) Annual Meeting and virtual 25thEuropean 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

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

PerkinElmer Launches Industry First No-Wash Immunoassay For Phospho Ubiquitin Ser65 to Help Drive Neuroscience Disease and Therapeutics Research Forward26.10.2020 20:05:00 CETPress release

PerkinElmer, Inc., a global leader committed to innovating for a healthier world, today launched the life science industry’s first no wash Ubiquitin phosphorylation at Serine 65 immunoassay for studying defective mitophagy biological mechanisms that have been broadly associated with diseases such as Parkinson’s and Alzheimer’s. The https://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.cisbio.eu%2Fphospho-ubiquitin-ser65-cellular-kit-44930&esheet=52314112&newsitemid=20201026005820&lan=en-US&anchor=new+PerkinElmer+Cisbio+HTRF%26%23174%3B+Phospho-Ubiquitin+%28Ser65%29+Cellular+Kit&index=2&md5=c03392c571335691d83031ec057300f1 new PerkinElmer Cisbio HTRF® Phospho-Ubiquitin (Ser65) Cellular Kit takes analysis efficiency and accuracy to the next level compared to existing techniques such as Western blot and is aimed at helping scientists discover new therapeutic candidates earlier in the disease cycle. The innovative assay leverages TR-FRET technology to bring high sensitivity

NetApp Brings Optimization and Enterprise Data Services to the Cloud26.10.2020 17:00:00 CETPress release

Global, cloud-led, data-centric software company NetApp® (NASDAQ: NTAP) today unveiled a groundbreaking serverless and storageless solution for containers from Spot by NetApp, a new autonomous hybrid cloud volume platform, and cloud-based virtual desktop solutions. NetApp’s new capabilities help simplify and optimize multicloud management for the highest performance at a low cost, offering true portability for data-rich cloud applications and delivering comprehensive workplace solutions across hybrid cloud environments. “To thrive in the new normal, digital transformation has become a business imperative. To succeed, businesses need to optimize their hybrid multicloud IT architectures,” said NetApp CEO George Kurian. “No matter where customers are on their transformation journey, NetApp can help them build a data fabric to maximize the value of their data, ensure applications are running optimally, and unlock the best of cloud.” The new features and capabilities that NetApp introduced

omni:us Welcomes Insurance Veteran Antonio Derossi to Drive Growth Across US and Europe26.10.2020 16:00:00 CETPress release

omni:us, the insurance claims AI solution provider, welcomes Antonio Derossi as a member of the omni:us Senior Executive Advisory board. Mr. Derossi, with 20+ years of experience in various roles across insurance, consulting, and venture capital, will function as a key advisor on go-to-market and product optimization strategies for the North American and European insurance markets. omni:us seeks to fortify its position as global pioneer of insurance claims transformation with the expert support of Mr. Derossi. Both parties see automation as a defining element of future claims management systems. Mr. Derossi’s understanding of the intricacies of the American and European insurance space will support increased market penetration for the Berlin-based omni:us. He will also act as an effective liaison to insurance companies, solution providers and consulting partners across both continents. Derossi was born in Trieste, Italy. He holds a Laurea degree in physics from Trieste University, and

Dahua Sponsors Omdia Webinars on Next Generation Network Cameras26.10.2020 14:45:00 CETPress release

Dahua Technology, a world-leading video-centric smart IoT solution and service provider, has sponsored one white paper and two webinars hosted by Omdia. The theme of the webinars held on October 22nd is Solving security challenges: Next-generation network cameras. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201026005466/en/ (Photo: Business Wire) Omdia is the new global technology research powerhouse, established in 2019 with the combination of the Informa Tech’s research brands (Ovum, Heavy Reading, and Tractica) and the acquired IHS Markit technology research portfolio. Collaborating with Dahua Technology, the comprehensive webinar has attracted about 1200 registrations. Audiences are experts from various industries such as energy, finance, education, government, retail, etc. With webinar events experts from Omdia, Dahua Technology’s Senior Project Sales Manager Simon Nash and Omdia’s analysts Josh Woodhouse and Tommy Z

26 th Yiwu Fair Concludes with Success, Reaching Deals Worth Millions in Small Commodities26.10.2020 14:36:00 CETPress release

The 26th China Yiwu International Commodities (Standards) Fair concluded in Yiwu on October 25, 2020. As the first large-scale foreign trade exhibition presented both online and offline in China amid regular epidemic prevention and control, Yiwu Fair 2020 provided more than 3,400 standard booths with an exhibition area of ​​100,000 square meters at the Yiwu International Expo Center, and attracted nearly 2,000 companies from 19 domestic provinces, municipalities and autonomous regions, covering categories of hardware, electromechanical facilities, electronic & electrical appliances, daily necessities, crafts & ornaments, stationery & office goods, toys, sporting & outdoor products, knitwear, and gift packaging. The Fair attracted a total of 106,000 offline visitors and 160 million online views. Seven overseas online matchmaking sessions were held targeting the United Kingdom, Malaysia, Chile, etc., and achieved an intended turnover of $4.66 million. While expanding the international ma

Interactive Brokers Launches Innovative Sustainable Investing Tool26.10.2020 14:18:00 CETPress release

Interactive Brokers Group (Nasdaq: IBKR), a global brokerage firm, today unveiled an innovative, interactive Impact Dashboard designed to help clients evaluate and invest in companies that align with their values. The dashboard, which is free for all clients to use, sets a new standard for tools that can be used for sustainable, socially responsible, and values-based investing. The launch comes as interest in Environmental, Social and Governance (ESG) investing is soaring. ESG assets are expected to top $53 trillion by 2022, according to research released in September by Celent. “The future of sustainable investing begins with the Impact Dashboard, which allows investors to align their portfolios with their values,” said Will Peterffy, ESG Director at Interactive Brokers. “In a world lacking transparency, the dashboard quickly analyzes and grades your stock portfolio based on your values and presents your holdings through a personalized ‘impact lens’. You can then decide whether to kee

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom