On November 4, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease, prostate cancer and other rare hematologic diseases and cancers, reported that four abstracts have been accepted for presentation – including three oral presentations and one poster presentation – at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place Dec. 11-14, 2021 (Press release, Forma Therapeutics, NOV 4, 2021, View Source [SID1234594532]).
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Two oral presentations will feature clinical data on a Phase 1 trial of etavopivat, the company’s oral, once-daily, selective pyruvate kinase-R (PKR) activator for the treatment of sickle cell disease (SCD). One abstract evaluates the ability of etavopivat to improve anemia and decrease intravascular hemolysis. A second abstract shows the effects of etavopivat on improving red blood cell health and lifespan, as well as reduction in systemic markers of inflammation and hypercoagulability. A third abstract highlights clinical data from the Phase 2 trial of olutasidenib, the company’s selective inhibitor for cancers with mutations in isocitrate dehydrogenase 1 (IDH1m) being evaluated in patients with relapsed/refractory acute myeloid leukemia (R/R AML).
"We’re pleased that these abstracts were selected for oral and poster presentation at the ASH (Free ASH Whitepaper) annual meeting," said Patrick Kelly, M.D., chief medical officer of Forma Therapeutics. "We look forward to sharing updated data at the meeting on the profile of etavopivat in sickle cell patients to not only increase hemoglobin but also to improve markers of hemolysis, as well as red blood cell health and red blood cell lifespan. In addition, the new olutasidenib results indicate potential for use as a single agent and in combination therapy with azacytidine to provide benefit in patients with relapsed/refractory AML."
The abstracts are currently available on the ASH (Free ASH Whitepaper) website: View Source
Oral Presentations:
Title: FT-4202, Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks
Date/Time: Saturday, Dec. 11, at 9:45 AM ET
Session: 114
Abstract: 147091
Presenter: R. Clark Brown, MD, PhD
Title: FT-4202, Etavopivat, an Allosteric Activator of Pyruvate Kinase-R, Improves Sickle RBC Functional Health and Survival and Reduces Systemic Markers of Inflammation and Hypercoagulability in Patients with Sickle Cell Disease: An Analysis of Exploratory Studies in a Phase 1 Study
Date/Time: Saturday, Dec. 11, at 10:00 AM ET
Session: 114
Abstract: 147078
Presenter: Theodosia A. Kalfa, MD, PhD
Title: FT-2102, Olutasidenib (FT-2102) in Combination with Azacitidine Induces Durable Complete Remissions in Patients with mIDH1 Acute Myeloid Leukemia
Date/Time: Monday, Dec. 13, at 3:00 PM ET
Session: 616
Abstract: 144905
Presenter: Jorge E. Cortes, MD
Poster Presentation:
Title: FT-2102, Molecular Characteristics of Response to Olutasidenib (FT-2102) in Patients with Relapsed/Refractory mIDH1 Acute Myeloid Leukemia
Date/Time: Sunday, December 12th at 6:00-8:00 PM ET
Session: 616
Abstract: 144912
Presenter: Stéphane de Botton, MD, PhD
About Etavopivat
Etavopivat is an investigational, once-daily, selective red blood cell (RBC) pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD). Employing a multimodal approach, etavopivat is designed to work by activating the RBCs’ natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce polymerization and RBC sickling. Etavopivat-mediated PKR activation also increases ATP levels, the fuel that provides energy to cells, to improve RBC health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, etavopivat did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development. Etavopivat has been granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency for the treatment of patients with SCD.
About Olutasidenib
Olutasidenib is an oral, potent, small-molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes. This targeted treatment has the potential to provide therapeutic benefit by reducing 2-HG levels and restoring normal cellular differentiation. With the conclusion of the Phase 2 R/R AML trial, Forma has begun preparing a new drug application (NDA) for submission to the U.S. Food and Drug Administration (FDA).
IDH1 is a natural enzyme that is part of the normal metabolism of all cells; when mutated, its activity can promote blood malignancies and solid tumors. IDH1 mutations are present in 6-8% of patients with AML and as many as 70 to 80% of patients with grade II/III gliomas and secondary glioblastoma. In gliomas, IDH1 mutations occur early in the tumor pathogenesis and persist throughout progression from a neural stem or progenitor cell. Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors, and high-grade gliomas are associated with poor long-term prognosis. Treatment options for relapsed glioma are limited.