eClinical Technology and Industy News

Forma Therapeutics Highlights Etavopivat Development Expansion and Introduces New Oncology Program from Research Pipeline at Inaugural Research and Development (R&D) Day

 Expanding etavopivat development in 2022 with Phase II trial exploring transfusion burden across sickle cell disease (SCD), thalassemia and myelodysplastic syndromes (MDS)

Phase 1 trial of FT-7051 in mCRPC proceeding with predicted efficacious dose range under evaluation and exploring alternative dosing schedule ​

FT-3171 (USP1 inhibitor) introduced targeting BRCA mutant tumors with investigational new drug (IND) filing expected in the first half of 2023 ​

Early stage research pipeline focusing on red blood cell (RBC) health and novel mechanisms in rare hematology and targeted oncology indications

Forma well-positioned with $441 million in cash to progress programs in rare hematologic diseases and cancers

Excerpt from the Press Release:

WATERTOWN, Mass.–(BUSINESS WIRE)–May 26, 2022– Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease (SCD), prostate cancer and other rare hematologic diseases and cancers, will hold a virtual R&D Day today to provide a comprehensive update on its pipeline and strategic vision.

The R&D event is taking place today at 8:00 a.m. ET. A live webcast will be available in the “News & Investors” section of Forma’s website.

“We are pleased to share insights into Forma’s unique commitment to patients and expansion of etavopivat development into transfusion-dependent populations that have tremendous unmet need across sickle cell disease, thalassemia and MDS,” said Frank Lee, president and chief executive officer of Forma.

“In our early stage research pipeline, we are excited to introduce a USP1 program that targets a novel DNA damage repair pathway relevant to a broad range of tumor types,” said David Cook, chief scientific officer of Forma. “We are also capitalizing on our knowledge of the emerging science of red blood cell health by exploring areas beyond hematologic disease.”

Etavopivat (oral PKR activator) Program in SCD, thalassemia and MDS:

In addition to ongoing enrollment in the Phase II/III Hibiscus Study in SCD, Forma will outline ongoing and anticipated future development plans:

  • A Phase II trial is enrolling patients in three cohorts: SCD with chronic transfusion, and both transfusion-dependent and non-transfusion-dependent thalassemia. Etavopivat has the potential to address RBC health, hemolytic anemia and/or ineffective erythropoiesis in these populations, leading to reduction of transfusion burden and associated iron overload and improvement of anemia.
  • A Phase II trial in lower-risk MDS is planned to commence in the second half of 2022. Dr. Michael Savona of Vanderbilt University will review the significant unmet need in lower-risk MDS and the potential for etavopivat to provide a well-tolerated oral treatment that may be able to improve the ability of bone marrow to produce healthier RBCs.
  • Analyses from the completed Phase I trial in SCD show benefit in both pain events reported in the trial and vaso-occlusive crises (VOC’s) occurring during treatment.
  • Enrollment in the Phase II/III Hibiscus Study in SCD is on track for the first interim analysis (IA1) in late 2022.

FT-7051 (oral CBP/p300 inhibitor) in prostate cancer:

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