– Designation Based on Phase 2 Data and Reinforces Significant Treatment Need for HR-MDS –
– Pevonedistat Could be First Novel Treatment Option for HR-MDS in More
Than a Decade –
CAMBRIDGE, Mass. & OSAKA, Japan-Thursday 30 July 2020 [ AETOS Wire ]
(BUSINESS WIRE) -- 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.
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.
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.
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.
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200730005233/en/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.
Contacts
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
No comments:
Post a Comment