Updated data from an ongoing randomized, multi-center, placebo-controlled Phase 1 clinical trial of FT-4202 in sickle cell disease selected for an oral presentation
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that four abstracts – including one oral presentation and three poster presentations – have been accepted for presentation at the 62nd American Society of Hematology (ASH) Virtual Annual Meeting taking place December 5-8, 2020.
The oral presentation will feature clinical data from the multiple ascending dose cohort of a randomized, multi-center, placebo-controlled Phase 1 trial of FT-4202 in people living with sickle cell disease (SCD). While the trial is currently enrolling patients in the second dose escalation cohort of 600 mg FT-4202 daily, data will be presented on 9-12 patients who have completed the 300 mg FT-4202 daily cohort. A "Trials in Progress" poster presentation will highlight key aspects of the planned registrational Phase 2/3 clinical trial. In addition, two collaborative posters will report the findings of research of FT-4202 in a mouse model of sickle cell anemia and in an ex vivo analysis of blood samples from patients with SCD, respectively.
"We’re pleased the FT-4202 data have been selected for multiple presentations at ASH 2020," said Frank Lee, president and chief executive officer of Forma. "Forma has a deep commitment to advancing science in sickle cell disease. We look forward to sharing data that will further characterize FT-4202 as an investigational treatment for people living with sickle cell disease."
The abstracts, currently available on the ASH conference website, are:
Title: FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease
Date/Time: Monday, December 7, 2020 at 2:00 p.m. PT
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Novel Treatments for Sickle Cell Disease
Presenter: R. Clark Brown, MD, PhD, Pediatric Hematologist/Oncologist, Medical Director of Sickle Cell at Scottish Rite, Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, and Associate Professor of Pediatrics, Emory University School of Medicine
Title: An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)
Date: Monday, December 7, 2020
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Poster III
Presenter: Kenneth W. Wood, Executive Director, Project Leadership at Forma Therapeutics, Inc.
Title: Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels
Date: Saturday, December 5, 2020
Session: 113. Hemoglobinopathies, Excluding Thalassemia - New Genetic Approaches to Sickle Cell Disease: Poster I
Presenter: Archana Shrestha, PhD, Research Associate at Cincinnati Children’s Hospital Medical Center
Title: Ex-Vivo FT-4202 Treatment Improves Hemoglobin Oxygen Affinity and Membrane Health in Red Blood Cells of Patients with Hemoglobin SS and Hemoglobin SC Disease Irrespective of Prior Hydroxyurea Use
Date: Saturday, December 5, 2020
Session: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I
Presenter: Diamantis Konstantinidis, Research Associate at Cincinnati Children’s Hospital Medical Center
About Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common disorders caused by a single gene mutation. Prevalence of SCD is approximately 100,000 people in the U.S. and approximately 30,000 people in France, Germany, Italy, Spain and the UK. While reporting limitations complicate stating an exact number, the National Institutes of Health reports that prevalence is estimated at over 20 million individuals worldwide. In people living with SCD, red blood cells, or RBCs, spontaneously deform in low oxygen conditions, taking on a sickle-like shape. Sickle cells are stiff and have damaged membranes, causing the RBCs to clump and burst in small blood vessels, resulting in inflammation and vaso-occlusive crises. Repeated deformation also depletes the RBC energy supply, called ATP. One important consequence of this energy depletion is increased levels of a metabolite, 2,3-DPG, that further reduces the RBCs’ affinity for oxygen and exacerbates the cycle of repeated deformation and anemia.
FT-4202 is a novel, oral, once-daily pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD). Early studies and trials have shown that FT-4202 works upstream by employing a multi-modal approach and activating the red blood cells’ (RBC) natural PKR activity to decrease 2,3-DPG levels, which we believe leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. FT-4202 has also shown downstream activity by increasing ATP levels, the fuel that provides energy to cells, which we believe may improve RBC health and survival. Together, these effects have the potential to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, FT-4202 did not inhibit aromatase activity, important biological processes responsible for sexual development.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
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