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
The R&D event is taking place today at
“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
“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
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 Savonaof Vanderbilt Universitywill 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:
Forma’s Phase I trial continues to enroll men with metastatic castration-resistant prostate cancer (mCRPC).
May 12, 2022, 25 patients have enrolled in the Phase I dose escalation trial, assessing the predicted efficacious exposure range supported by target engagement.
- The trial population is heavily pre-treated, with a high AR-v7 positivity rate and mutation burden.
- Future trial enrollment to include less heavily pre-treated patients and alternative dosing schedules to address adverse events, with updated results expected in the first half of 2023.
Forma’s ongoing research efforts are focused on novel mechanisms of action in oncology and expansion in red blood cell health and hematologic diseases:
- Forma’s research pipeline is led by the USP1 program (FT-3171), targeting a novel DNA damage repair pathway with the potential to address multiple tumor types in both poly ADP ribose polymerase inhibitor (PARPi)-sensitive and resistant settings.
- FT-3171 IND filing is expected in the first half of 2023.
- Ongoing pre-clinical research is targeting areas of expansion for RBC health, novel mechanisms that may be complementary to etavopivat in rare hematology, and targeted oncology.
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 the company’s beliefs and expectations regarding its: business plans and objectives; future plans for etavopivat, FT-7051 and FT-3171, including expectations regarding potential development expansion plans as well as regulatory filings, enrollment, timing, success and data announcements of planned and ongoing clinical and pre-clinical trials; therapeutic potential, clinical benefits, mechanisms of action and safety of our product candidates; plans for the USP1 program (FT-3171) and other research pipeline expansion efforts; upcoming milestones and planned additional trials for the company’s product candidates; growth as a company; upcoming presentations of our R&D programs, including the introduction of a new molecule and related studies; uses and need of capital, expenses and other financial results currently or in the future. 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.
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