Forma Therapeutics Announces Four Oral and Poster Presentations on FT-4202 in Sickle Cell Disease at Upcoming 2020 ASH Virtual Annual Meeting
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
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
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
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Novel Treatments for Sickle Cell Disease
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)
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Poster III
Session: 113. Hemoglobinopathies, Excluding Thalassemia - New Genetic Approaches to Sickle Cell Disease: Poster I
Session: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I
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
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.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding FT-4202, our expectations of the therapeutic benefits related thereto, the timing and success of ongoing clinical trials, whether positive interim results from a clinical study are predictive of the results of ongoing or future clinical studies, and our growth as a company. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the advancement of our clinical programs and other risks identified in our
Stern Investor Relations