– 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
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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
