RegulationOverseas New Drugs Urgently Needed in Clinical Settings (CN), Special Review Project (CN), Fast Track (US), Orphan Drug (US), Priority Review (US)

Structure/Sequence

Molecular FormulaC20H21F6N7O4S

InChIKeyORZHZQZYWXEDDL-UHFFFAOYSA-N

CAS Registry1650550-25-6

View All Structures (2)

Clinical Trials associated with Enasidenib Mesylate

NCT06672146

/ Not yet recruitingPhase 2IIT

A Randomized Phase II Trial of Oral Decitabine and Cedazuridine and Venetoclax Compared With Oral Decitabine and Cedazuridine, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial studies how well ASTX727 and venetoclax plus enasidenib works compared to ASTX727 and venetoclax alone for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML) or younger patients who are considered unfit for standard treatment, and who have an abnormal change (mutation) in the IDH2 gene. This gene mutation can cause AML to grow and spread. This trial is being done to see if adding enasidenib to the usual treatment can help more patients with the IDH2 gene get rid of AML. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Enasidenib works by stopping the growth and spread of tumor cells that have the IDH2 mutation. Giving ASTX727 and venetoclax plus enasidenib may work better in treating AML patients with the IDH2 mutation.

Start Date03 Feb 2025

Sponsor / Collaborator

National Cancer Institute

NCT06577441

/ Not yet recruitingPhase 2IIT

A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.

Start Date17 Jan 2025

Sponsor / Collaborator

National Cancer Institute

NCT06756308

/ RecruitingPhase 2IIT

A Phase 2 Study of Enasidenib in IDH2-Mutant Angioimmunoblastic T-Cell Lymphoma

The researchers are doing this study to find out whether enasidenib is a safe treatment for people with angioimmunoblastic T-cell lymphoma (AITL) that has an IDH2 mutation. The researchers will look at the safety of enasidenib when it is given alone or in combination with the drug rituximab.

Start Date24 Dec 2024

Sponsor / Collaborator

Memorial Sloan Kettering Cancer Center

[+2]

100 Clinical Results associated with Enasidenib Mesylate

100 Translational Medicine associated with Enasidenib Mesylate

100 Patents (Medical) associated with Enasidenib Mesylate

282

Literatures (Medical) associated with Enasidenib Mesylate

02 Jan 2025·Leukemia & Lymphoma

Enasidenib in relapsed aggressive systemic mastocytosis with IDH2 mutation

Article

Author: Al Darobi, Ali ; Heimbigner, Alex ; Heers, Hayley ; Ulrickson, Matthew ; Del Rio Verduzco, Alejandro

01 Jan 2025·American Journal of Clinical Oncology

Maintenance Therapy in Acute Myeloid Leukemia

Review

Author: Sabakhtarishvili, Giorgi ; Ansari, Amir ; Tabbara, Imad A.

Acute myeloid leukemia (AML) poses significant challenges due to its high relapse rates despite initial successful induction chemotherapy. Maintenance therapy aims to prevent disease recurrence, particularly in high-risk patients. This review explores current maintenance treatments, their impacts on patient outcomes, and ongoing studies shaping the treatment landscape for AML. Hypomethylating agents like azacitidine and decitabine have shown promise in improving relapse-free and overall survival, particularly in older patients with AML ineligible for transplantation. Combination regimens involving azacitidine and venetoclax have demonstrated encouraging outcomes post–hematopoietic stem cell transplantation. Targeted therapies, particularly FLT3 inhibitors like midostaurin and quizartinib, have shown significant benefits in improving survival outcomes, especially in FLT3-mutated AML cases. Gilteritinib and sorafenib also exhibit the potential to reduce relapse rates post-transplant. Isocitrate dehydrogenase inhibitors, including ivosidenib and enasidenib, present novel options for postchemotherapy and posttransplantation maintenance. Immunotherapies, such as Wilms tumor 1 peptide-based vaccines and checkpoint inhibitors, are being explored, although results vary. Despite ongoing research, the role of maintenance chemotherapy remains uncertain, with inconsistent outcomes across trials. The approval of oral azacitidine represents a significant advancement, emphasizing the need for further investigation into personalized maintenance approaches. In conclusion, the evolving landscape of maintenance therapy and integrating targeted therapies in AML offers promising avenues for improving patient outcomes.

01 Jan 2025·Leukemia Research

Combination therapy involving azacitidine for acute myeloid leukemia patients ineligible for intensive chemotherapy

Review

Author: Li, Jun ; Li, Juan ; Ye, Chunmei ; Fu, Shuying

Acute myeloid leukemia (AML) is a complex hematological malignancy predominantly affecting the elderly, with a median diagnosis age of 68 years. Despite advances in treatment, elderly AML patients face suboptimal survival outcomes, with an estimated 5-year survival rate below 20 %. Epigenetic dysregulation, notably DNA methylation, is a key factor in the progression of myelodysplastic syndromes (MDS) to AML. This review examines various combination regimens involving azacitidine (AZA), including those with lenalidomide, histone deacetylase inhibitors (HDACi), kinase inhibitors, metabolic enzyme inhibitors, monoclonal antibodies, immune checkpoint inhibitors, and anti-apoptotic protein inhibitors. Notable among these are the combinations with venetoclax, which has demonstrated remarkable efficacy in phase III trials, and the emerging IDH inhibitors ivosidenib and enasidenib, which have shown significant clinical benefits in patients with IDH mutations. While combination therapies with AZA hold great promise, challenges persist, including translating in vitro synergies to in vivo efficacy and identifying optimal regimens for specific patient populations. Cumulative toxicities may also offset clinical benefits, necessitating rigorous clinical trial design. Future research must focus on refining combination strategies, optimizing dosages and sequences, and thoroughly evaluating therapeutic efficacy and safety to advance the treatment of AML and improve patient outcomes.

News (Medical) associated with Enasidenib Mesylate

15 Aug 2024

·www.businesswire.com

Bicara Therapeutics Expands Board of Directors with Appointments of Mike Powell, Ph.D., and Christopher Bowden, M.D.

BOSTON--( BUSINESS WIRE )--Bicara Therapeutics, a clinical-stage biotechnology company developing transformative bifunctional therapies for patients with solid tumors, today announced the appointments of Mike Powell, Ph.D., and Christopher Bowden, M.D., to the company’s board of directors. Dr. Powell currently serves as executive partner of Omega Funds and will serve as Bicara’s chairman of the board. Dr. Bowden is the former chief medical officer of Agios Pharmaceuticals and current chief medical officer of Remix Therapeutics and will serve as an independent director. “ Mike and Chris are distinguished biopharma industry leaders that have contributed to the approvals of multiple innovative oncology medicines. We are thrilled to welcome them to our board of directors,” said Claire Mazumdar, Ph.D., MBA, chief executive officer of Bicara Therapeutics. “ The combination of Mike’s four decades of pharmaceutical clinical development experience and Chris’ strategic medical and corporate expertise will be critical as we continue to advance ficerafusp alfa (BCA101) into late-stage development for recurrent/metastatic head and neck squamous cell carcinoma as well as other indications.” About Mike Powell, Ph.D. Dr. Mike Powell joined Omega Funds as executive partner in 2021 from Sofinnova Investments, where he served as managing general partner since 1997. Previously, Dr. Powell was Group Leader at Genentech from 1990 to 1997, where his focus was on drug delivery, CMC, and formulation of both small molecule and protein drug entities. Before this, he served as the director of product development for Cytel Corp, an immunology biotech company. Prior to this, he was senior scientist and project team leader at Syntex Research (Roche). Dr. Powell was the first person in the biotech industry to become a fellow of the American Association of Pharmaceutical Scientists (AAPS). He was previously board president of the AIDS Vaccine Advocacy Coalition, an advisor to the Institute for One World Health, IAVI, and the Bill and Melinda Gates Foundation, and an adjunct associate professor in the Department of Pharmaceutical Chemistry at the University of Kansas. Dr. Powell holds a Ph.D. in physical chemistry from the University of Toronto, and a postdoc in bio-organic chemistry at the University of California. About Christopher Bowden, M.D. Dr. Chris Bowden currently serves as the chief medical officer of Remix Therapeutics. Prior to Remix, Dr. Bowden served as a strategic advisor and chief medical officer of Agios Pharmaceuticals from 2014-2022. As chief medical officer at Agios, he oversaw clinical development that led to U.S. approvals for the IDH inhibitors IDHIFA™ and TIBSOVO™ in acute myeloid leukemia (AML), for TIBSOVO™ in cholangiocarcinoma and for PYRUKYND™ for adults with pyruvate kinase deficiency. Prior to joining Agios, Dr. Bowden was vice president of product development in oncology, franchise lead (signaling group) at Genentech, Inc. where he was responsible for the successful development of several novel targeted oncology medicines, including Zelboraf™ and Tarceva™. Dr. Bowden received his M.D. from Hahnemann University School of Medicine in Philadelphia after which he completed his internal medicine training at Roger Williams Medical Center and the Providence VA Medical Center, Rhode Island. He conducted his medical oncology fellowship at the National Cancer Institute Medicine Branch. Dr. Bowden is board certified in internal medicine and medical oncology. About Ficerafusp Alfa (BCA101) Ficerafusp alfa is a bifunctional antibody designed to inhibit the epidermal growth factor receptor (EGFR) and disable transforming growth factor beta (TGF-β) directly at the tumor site. This approach is designed with the intent to allow ficerafusp alfa to inhibit tumor proliferation, while restoring the cytolytic activity of the local immune cells. Ficerafusp alfa is currently being evaluated in a dose expansion phase of an open-label Phase 1/1b study in combination with pembrolizumab in HPV-negative patients with R/M HNSCC, advanced squamous non-small cell lung cancer (SqNSCLC), or squamous cancer of the anal canal (SCAC) and as a monotherapy for cutaneous squamous cell carcinoma (cSCC). About Bicara Therapeutics Bicara Therapeutics is a clinical-stage biotechnology company developing bifunctional therapies engineered to combine the precision of well validated, tumor-targeting antibodies with the power of tumor microenvironment modulators. The Company’s bifunctional antibodies are designed to deliver an immunomodulatory payload directly to the tumor microenvironment to ramp up immune cell activity, offering the potential for synergistic therapeutic impact at the site of the tumor. Bicara’s lead product candidate, ficerafusp alfa, is a EGFR/TGF-β-trap bifunctional antibody in clinical development for multiple tumor types. For more information, please visit www.bicara.com or follow us on LinkedIn or X.

Executive ChangeImmunotherapyDrug Approval

06 Aug 2024

·www.fiercepharma.com

Servier snags FDA approval for Voranigo, the first targeted therapy for a common type of brain tumor

This marks the first FDA approval of a systemic therapy for patients with grade 2, IDH-mutant gliomas. Servier has expanded its IDH drug armamentarium with the FDA approval for an IDH1/2 dual inhibitor. The approval represents the first major treatment advance in low-grade brain cancer in more than two decades.The new FDA approval is for Servier’s Voranigo, or vorasidenib, for adult and children ages 12 and older with low-grade glioma with a susceptible IDH1 or IDH2 mutation. As the FDA noted in its press release, this marks the first approval of a systemic therapy for this type of grade 2 IDH-mutant brain cancer, which includes oligodendrogliomas and astrocytomas.Servier obtained Voranigo through its $2 billion acquisition of Agios Pharmaceuticals’ oncology business in 2021. Royalty Pharma, which earlier this year acquired a stake in Agios’ royalty payments for Voranigo, has projected the drug could reach more than $1 billion in annual peak sales in the U.S.Gliomas are the most common primary malignant brain tumors in adults, and IDH1 or IDH2 mutations are present in nearly all grade 2 diffuse gliomas in adults. By Royalty’s estimates, there are around 1,500 new diagnoses for these two IDH-mutant gliomas each year in the U.S.Current therapeutic interventions for these patients are more than 20 years old and are all used off label, Islam Hassan, M.D., senior medical director at Servier, noted in an interview with Fierce Pharma ahead of Tuesday’s FDA decision.Before Voranigo, Servier already has the IDH1 inhibitor Tibsovo and the IDH2 inhibitor Idhifa. Although doctors have been using the two meds off-label because of the lack of treatment options, neither was approved for brain cancer.Voranigo, for its part, was designed to penetrate the blood-brain barrier to be effective against brain tumors, Hassan explained. In a phase 3 trial coded INDIGO, Voranigo significantly reduced the risk of tumor progression or death by 61% compared with placebo among patients with residual or recurrent grade 2 IDH-mutant glioma. These patients had undergone no previous treatment other than surgery.In addition, Voranigo also showed a statistically significant benefit in delaying the need for the next treatment, slashing the risk for further treatment by 74% in the study.The trial is ongoing to gather overall survival data. Still, Hassan argued that the progression-free survival data are just as important, because these tumors will continue to grow and penetrate the brain and may develop into high-grade disease.Besides the Voranigo monotherapy regimen, Servier has been evaluating the IDH inhibitor alongside Merck’s immunotherapy Keytruda in recurrent or progressive IDH1-mutant gliomas. While anti-PD-1 antibody drugs like Keytruda have been largely unsuccessful in brain tumors, Hassan pointed to a previous study in which patients received Voranigo and Keytruda before surgery. In tissues collected from surgery, Servier noticed some changes in the immune microenvironment that Hassan said warrant further development of this combination.Servier is also testing Voranigo alongside the chemotherapy temozolomide in high-grade IDH-mutant glioma. As Hassan noted, these patients with grade 4 gliomas are typically excluded from other clinical trials.The FDA approval triggers a $200 million milestone payment from Servier to Agios, plus a $905 million payment from Royalty to Agios in exchange for a 15% royalty on U.S. net sales of up to $1 billion for the therapy.

Clinical ResultDrug ApprovalPhase 3Acquisition

12 Jul 2024

·www.biopharmadive.com

Bristol Myers’ leukemia blockbuster set to face generic rivals

Pharmaceutical companies face a looming patent cliff over the next several years, as generic rivals could erode sales of some of the industrys best-selling medicines. For some drugmakers, copycat competitors are already on the doorstep.Bristol Myers Squibb, for one, could soon hit headwinds for its blockbuster Sprycel as the first generic of the leukemia drug is expected to hit the market in September.Sprycel, or dasitinib, is a kinase inhibitor that was approved in 2006 to treat chronic myeloid leukemia, a type of blood cancer thats diagnosed in about 10,000 people in the U.S. each year. The tablet has been a reliable blockbuster for Bristol Myers over the years, reaching $1.9 billion in global sales in 2023. Sales topped $2.1 billion in both 2022 and 2021.Bristol Myers has faced several challenges to Sprycels patents, and filed a patent infringement lawsuit again Swedish biopharma company XSpray Pharma in 2022, as well as a handful of other companies that were challenging two patents listed in the Food and Drug Administrations Orange Book that expire in 2025 and 2026.Bristol Myers settled with XSpray last year, clearing the way for the challenger to launch a generic dasatinib as soon as Sept. 1, 2024. XSpray has an FDA decision date of July 31 for its optimized version of dasatinib, Dasynoc.If Dasynoc is approved, it will join dozens of new generics cleared by the FDA this year, including several copies of cancer drugs. Their entry could lead to price declines in oncology, according to IDP Analytics. Sprycel has a list price of more than $18,000 per month. CML mostly affects adults 65 and older, and Sprycel treatment is therefore often covered under Medicare.Biocon, Teva Pharmaceutical and several other companies also have developed dasitinib generics and secured tentative FDA approval.Sprycels patent expiration isnt the only generic threat Bristol Myers faces. Copycat competitors to Revlimid are available in limited fashion already, while protection for top-sellers Eliquis and Opdivo run out in 2026 and 2028 in the U.S., respectively.In oncology, Bristol Myers is focused on expanding the market for Breyanzi, a CAR-T cell therapy for different types of lymphoma and leukemia, as well as for Abecma, for multiple myeloma. Opdualag, a combination immunotherapy for melanoma, also figures heavily in the companys plans.Elsewhere in Bristol Myers pipeline are two candidates for acute myeloid leukemia, or AML, that are in early clinical stages. The company co-markets with Servier Idhifa for AML, which is considered a hard-to-treat disease, '

ImmunotherapyDrug ApprovalPatent InfringementBiosimilar

100 Deals associated with Enasidenib Mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
-	Enasidenib Mesylate	L01XX59	DB13874

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Acute Myeloid Leukemia	US	Bristol Myers Squibb Co.	01 Aug 2017

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	CA	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	BR	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	CN	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	CZ	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	AT	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	AU	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	FR	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	BE	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	DE	Celgene Corp.	30 Dec 2015

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04281498 (MPN-RC 119, CTgov)	Phase 2	Primary Myelofibrosis	6	Enasidenib+Ruxolitinib	eimoigkjmq(zkqeowdfgo) = bthjlpkftr mysqpteinl (ldsmbwjztg, dbsgxnouku - kxrpuzdimc) View more	-	18 Dec 2024
NCT05010772 (4277, ASH2024)	Phase 1	Acute Myeloid Leukemia complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... View more	31	ASTX727 alone	(wsrctmoeah) = apfadxcrgm mcgbgpwxbo (nmywpxbtai ) View more	Positive	09 Dec 2024
				ASTX727 plus venetoclax	(wsrctmoeah) = vtbyhlejpy mcgbgpwxbo (nmywpxbtai ) View more
NCT01915498 \| NCT02677922 \| NCT02577406 \| NCT02632708 (Pubmed \| Blood Adv) Manual	Not Applicable	Acute Myeloid Leukemia	643	enasidenib ± azacitidine or chemotherapy	zxfaeunjbh(rsswvidssi) = jacaqczyvt pipszageei (ypqtorvhqh ) View more	Positive	20 Mar 2024
				Enasidenib plazacitidineine	zxfaeunjbh(rsswvidssi) = uhcauoxarj pipszageei (ypqtorvhqh )
NCT03728335 (Tandem Meetings ASTCT and CIBMTR2024) Manual	Phase 1	IDH2 mutation Acute Myeloblastic Leukemia Maintenance	15	Enasidenib 100 mg/day	(vmawypufpx) = ooqhocoyqi vlgeipjdgp (iuetstkztv ) View more	Positive	01 Feb 2024
NCT03013998 (Pubmed) Manual	Phase 1/2	Acute Myeloid Leukemia First line	60	Enasidenib monotherapy	(lhnnsieefk) = kzwasdfmsb rtscjauilr (kxpbtjmhwm ) View more	Positive	23 Jan 2024
				Enasidenib + azacitidine combination therapy	(lhnnsieefk) = rjgicepgpk rtscjauilr (kxpbtjmhwm ) View more
NCT05010772 (ASH2023)	Phase 1	Acute Myeloid Leukemia Maintenance	23	ASTX727 35/100 mg	(oqkbqylepc) = vqyivvxhjg yqfgiypoqg (rakempmzfy ) View more	-	10 Dec 2023
				ASTX727 35/100 mg + Venetoclax 400 mg	(oqkbqylepc) = uazefldpis yqfgiypoqg (rakempmzfy ) View more
NCT04092179 (Phase Ib/II Study, ASH2023)	Phase 1/2	Myeloid Tumor	27	Enasidenib + Venetoclax	(yfqluianwe) = scnesqkvwp brttshqarm (jojkxmnphi, 8.2 - NR) View more	-	09 Dec 2023
INNV-07 (SNO2023)	Not Applicable	Adjuvant	1	Enasidenib	aqjtmaxoar(dzzzoeznrn) = mzlazppfje lsxmrbhius (pqqbguyfdb )	Positive	10 Nov 2023
NCT02677922 (AG-221-AML-005 \| AG221-AML-005, CTgov)	Phase 1/2	Acute Myeloid Leukemia	130	AZA+AG-221 (Phase 1b Dose-finding Stage: AG-221 (100mg) + AZA)	ohrhazjjmd(aqxwvurius) = zlwssvnyoq cdmkmzgtfx (lbnjhtoift, gahrkmjvot - nwpidystnw) View more	-	22 Nov 2022
				AZA+AG-221 (Phase 1b Dose-finding Stage: AG-221 (200mg) + AZA)	ohrhazjjmd(aqxwvurius) = glfomgpmqw cdmkmzgtfx (lbnjhtoift, byvwvfhftb - aptkcyidch) View more
NCT04281498 (ASH 12022 \| ASH 22022) Manual	Phase 2	Myeloproliferative Disorders IDH2 Mutation	6	Ruxolitinib Phosphate+Enasidenib Mesylate	(uyotpdxuyv) = xhamianfdz qyybrgbifq (xoldsxojot ) View more	Positive	15 Nov 2022

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis