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Forma Therapeutics Announces Four Oral and Poster Presentations on FT-4202 in Sickle Cell Disease at Upcoming 2020 ASH Virtual Annual Meeting

·7 min read

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:

Oral Presentation

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
Abstract: 679
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

Poster Presentations

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
Abstract: 2622
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
Abstract: 784
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
Abstract: 786
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.

About FT-4202

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.

Forward-looking Statements

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 SEC filings, including those risks discussed under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as other risks detailed in our subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.

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Forma Therapeutics

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Stern Investor Relations