6 of 7 (86%) patients on 300 mg of FT-4202 for 14 days achieved a hemoglobin increase > 1 g\/dL from baseline <\/i><\/p>\n
Hemolytic markers support the hypothesis that FT-4202 improves red blood cell survival and reduces turnover<\/i><\/p>\n
A favorable tolerability profile was observed after once-daily dosing of FT-4202 for 14 days and a 7-day follow up period in patients with SCD<\/i><\/p>\n
Initiated 600 mg MAD2 cohort and 12-week open label extension study <\/i><\/p>\n
WATERTOWN, Mass.–(BUSINESS WIRE<\/a>)–Forma Therapeutics Holdings, Inc. <\/a>(Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that clinical proof-of-concept in patients with sickle cell disease (SCD) has been observed in the ongoing randomized, placebo-controlled, multi-center Phase 1 trial of FT-4202. FT-4202 is a novel, investigational, selective red blood cell (RBC) pyruvate kinase-R (PKR) activator in development as a potential disease-modifying therapy for SCD. In a cohort of nine patients, six of seven patients (86%) who received FT-4202 achieved a hemoglobin increase of greater than 1 g\/dL from baseline with once-daily dosing at 300 mg for 14 days. The data, being presented today at the 62nd<\/sup> American Society of Hematology (ASH) Annual Meeting, also demonstrate improvements in hemolytic parameters showing that activation of PKR by FT-4202 decreases reticulocyte counts and sickle RBC hemolysis in patients with SCD.\n<\/p>\n \n\u201cThese results are remarkable for a Phase 1 study, and they\u2019re encouraging for sickle cell patients,\u201d said Marilyn J. Telen, M.D., director of the Duke Comprehensive Sickle Cell Center, Professor of Medicine, Duke University School of Medicine, and one of the study investigators. \u201cThe absence of serious treatment-related adverse events, together with increased hemoglobin and reduced markers of hemolysis among the group receiving FT-4202, indicate a potential impact on overall red blood cell health and support further studies.\u201d\n<\/p>\n \n\u201cWe are pleased to present our clinical proof-of-concept data and continue to see potential for FT-4202 to improve the lives of people living with sickle cell disease,\u201d said Patrick Kelly, M.D., chief medical officer of Forma. \u201cAs we prepare to initiate Forma\u2019s pivotal Phase 2\/3 trial, we plan to evaluate whether the combined ability of FT-4202 to increase hemoglobin levels in red blood cells and bring about improved red blood cell health will meaningfully reduce the frequent painful vaso-occlusive crises these patients endure.\u201d\n<\/p>\n Presentation Overview<\/b><\/p>\n 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<\/i><\/p>\n \nThe data will be presented in an oral presentation by R. Clark Brown, M.D., Ph.D., Pediatric Hematologist\/Oncologist, Medical Director of Sickle Cell at Scottish Rite, Aflac Cancer and Blood Disorders Center of Children\u2019s Healthcare of Atlanta, and Associate Professor of Pediatrics, Emory University School of Medicine.\n<\/p>\n \nThe results announced today are based on nine patients with SCD (FT-4202 n=7; placebo n=2) randomly assigned to receive a single oral dose of 300 mg daily of FT-4202 or placebo for 14 days. The data show that, from baseline, in patients receiving FT-4202:\n<\/p>\n \nThe tolerability data presented today are based on nine patients enrolled at the time of submission of the presentation to ASH. Since then, findings from these nine patients have been unblinded. Among the seven patients receiving FT-4202, the tolerability analysis indicates:\n<\/p>\n \nNon-treatment-related AEs were consistent with sickle cell disease-related events commonly experienced in this patient population.\n<\/p>\n \nThe ongoing Phase 1 study is evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of a single ascending dose and multiple ascending doses of FT-4202, first in healthy volunteers and now in patients with sickle cell disease. Based on the safety results and tolerability profile observed in the initial multiple dose cohort of patients, a second multiple dose cohort is being enrolled in which patients are randomly assigned to receive a single daily 600 mg oral dose of FT-4202 or placebo for 14 days. Patients who complete this second 14-day dose cohort can then enroll into a 12-week, open label cohort receiving a single daily 400 mg oral dose of FT-4202. For more information, please visit clinicaltrials.gov\/NCT03815695<\/a>.<\/span><\/p>\n \nThe data presented today, along with the results from the single 700 mg dose arm of the study presented in June 2020 at the European Hematology Association (EHA) Annual Congress, support initiation of a randomized, placebo-controlled, double-blind, global multicenter Phase 2\/3 registrational study to evaluate the safety and efficacy of FT-4202 in adults and adolescents with SCD in the first quarter of 2021.\n<\/p>\n Investor Event<\/b><\/p>\n \nForma Therapeutics will conduct a webcast today, Dec. 7, at 6 p.m. Eastern Standard Time (EST) to further discuss the results from the ongoing study and to provide additional details of the company\u2019s development plans for FT-4202. A live webcast will be available in the \u201cNews & Investors\u201d section of Forma\u2019s website: https:\/\/ir.formatherapeutics.com\/<\/a>.\n<\/p>\n About Sickle Cell Disease<\/b><\/p>\n \nSickle cell disease (SCD) is one of the most common single-gene disorders and is estimated to affect approximately 100,000 people in the United States, as well as approximately 30,000 in France, Germany, Italy, Spain and the United Kingdom. The National Institutes of Health (NIH) reports that prevalence is estimated at more than 20 million individuals globally. From 2010 to 2050, the annual number of newborns with SCD is expected to rise globally by approximately one-third.1<\/sup> Despite recent advances in treatment, most patients with SCD still suffer from pain crises, lifelong disability, significant morbidity and reduced quality of life.\n<\/p>\n About FT-4202<\/b><\/p>\n \nFT-4202 is a novel investigational 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, FT-4202 is designed to work upstream by activating the RBCs\u2019 natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. The downstream activity of FT-4202 is designed to increase 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, FT-4202 did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development. FT-4202 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.\n<\/p>\n About Forma Therapeutics<\/b><\/p>\n \nForma 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<\/a> or follow us on Twitter @FORMAInc and LinkedIn.\n<\/p>\n Forward-looking Statements<\/b><\/p>\n \nThis 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 our beliefs and expectations regarding: our ability to complete our ongoing Phase 1 clinical trial for FT-4202, including its timing and success, our expectations of the therapeutic benefits related to FT-4202, , whether positive interim results from a clinical study are predictive of the results of ongoing or future clinical studies, our expectations around our future regulatory filings and our growth as a company. The words \u201cmay,\u201d \u201cwill,\u201d \u201ccould,\u201d \u201cwould,\u201d \u201cshould,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201canticipate,\u201d \u201cintend,\u201d \u201cbelieve,\u201d \u201cestimate,\u201d \u201cpredict,\u201d \u201cproject,\u201d \u201cpotential,\u201d \u201ccontinue,\u201d \u201ctarget\u201d and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.\n<\/p>\n \nAny forward-looking statements in this press release are based on management\u2019s 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 \u201cRisk Factors\u201d in our Quarterly Report on Form 10-Q for the quarter ended September 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.\n<\/p>\n \n_______________________ View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20201207005857\/en\/<\/a><\/span><\/p>\n Media Contact: Investor Contacts: Stephanie Ascher, +1 212-362-1200 KEYWORDS:<\/b> Massachusetts United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Biotechnology Health Genetics Pharmaceutical Clinical Trials<\/p>\n\n
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1<\/sup>Piel, F. B., Hay, S. I., Gupta, S., Weatherall, D. J., & Williams, T. N. (2013). Global burden of sickle cell anaemia in children under five, 2010-2015: Modelling based on demographics, excess mortality, and interventions. PLOS Medicine, 10(7). Retrieved from link<\/a>.\n<\/p>\n<\/p>\n
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Megan McGrath, +1 781-591-3488
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MacDougall
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mmcgrath@macbiocom.com
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Mario Corso, +1 781-366-5726
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Forma Therapeutics
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mcorso@formatherapeutics.com
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Stern Investor Relations
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stephanie.ascher@sternir.com<\/a><\/p>\n
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