On January 11, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancer reported a review of corporate highlights from 2020 and outlined anticipated key clinical program milestones for 2021 (Press release, Forma Therapeutics, JAN 11, 2021, View Source [SID1234573778]).
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"While 2020 was marked by tremendous challenges due to the COVID-19 pandemic, Forma made important strides due to the courage of patients and the dedication of our employees. We reported promising results our from our clinical programs including FT-4202 in sickle cell disease, olutasidenib in both AML and glioma, and FT-7051 in prostate cancer, and also raised approximately $695 million in public equity offerings," said Frank Lee, president and chief executive officer of Forma. "In this coming year, we anticipate continued progress, and look forward to advancing drug candidates that we believe have the potential to significantly impact patients living with rare hematologic diseases and cancers."
Clinical Program Highlights and Milestones
PKR Program in Sickle Cell Disease (SCD):
FT-4202 is a novel investigational selective red blood cell pyruvate kinase R (PKR) activator Forma is actively evaluating in a multi-center, placebo-controlled Phase 1 trial in individuals with sickle cell disease ages 12 years and older.
In June 2020, encouraging single dose cohort data were reported at the 25th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress. Initial findings from Forma’s Phase 1 trial of FT-4202 in patients with SCD demonstrated a favorable tolerability profile and biologic effects, with evidence of pharmacodynamic activity.
In December 2020, clinical proof-of-concept data were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. Forma presented data from a multi-dose cohort of its Phase 1 trial of FT-4202 in patients with SCD, showing that 86% of patients dosed with 300 mg of FT-4202 for 14 days achieved a hemoglobin increase of greater than 1 g/dL from baseline. The observed reduction in hemolysis in conjunction with the biomarker analysis showing improved deformability and hydration of RBCs supports the hypothesis that pyruvate kinase activation may have an impact on vaso-occlusive crises (VOCs).
Additional clinical data anticipated from the ongoing Phase 1 randomized placebo-controlled trial in SCD patients. Data from the MAD2 600 mg cohort of the Phase 1 trial is expected to be reported in the first quarter of 2021, followed by results from the 12-week open-label extension in the second quarter of 2021.
Expanding FT-4202 development program. Forma has initiated a global pivotal Phase 2/3 trial of FT-4202 in SCD patients, with plans to initiate a Phase 2 trial in thalassemia in the second half of 2021, and a pediatric SCD trial in the first half of 2022.
IDH1 Program in AML and Glioma:
Olutasidenib (FT-2102) is a selective inhibitor for cancers with IDH1 mutations Forma is evaluating for the treatment of acute myeloid leukemia (AML) and glioma. Olutasidenib is currently being studied in a registrational Phase 2 trial for relapsed/refractory (R/R) AML and an exploratory Phase 1 trial for glioma.
In May 2020, positive data for olutasidenib in glioma was announced at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). Forma announced positive preliminary Phase 1 data for olutasidenib in refractory, enhancing glioma at ASCO (Free ASCO Whitepaper) 2020, suggesting the potential for response and prolonged disease control in relapsed/refractory IDH1-mutated glioma patients.
In October 2020, positive data for olutasidenib in a registrational trial for R/R AML was announced. Forma announced positive results from the planned interim analysis of the Phase 2 registration trial of olutasidenib in R/R AML patients with IDH1 gene mutations. Olutasidenib as a monotherapy demonstrated a favorable tolerability profile and achieved a composite complete remission (CR/CRh) rate of 33.3% (30% CR and 3% CRh), the primary efficacy endpoint. While a median duration of CR/CRh has not yet been reached, a sensitivity analysis indicated the median duration of CR/CRh to be 13.8 months. Further data analysis indicates an estimated 87% survival rate at 18 months in patients who respond to treatment with olutasidenib. Olutasidenib’s potential transformative treatment profile is based upon three key indicators: durability of response and increased survival for responders, favorable tolerability suggesting ability to combine with other therapies, and properties to support indication expansion to other IDH1 mutated cancers.
Forma has begun preparing for a new drug application for olutasidenib in R/R AML.
CPB/p300 Program in Prostate Cancer:
FT-7051 is a potent and selective CBP/p300 inhibitor Forma is evaluating for the treatment of metastatic prostate cancer resistant to androgen receptor (AR) signaling inhibitor therapy.
In April 2020, preclinical data on FT-6876 (a research compound related to FT-7051) in breast cancer was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper). Forma presented preclinical data that demonstrated antitumor activity of FT-6876 in AR-dependent breast cancer cell lines, suggesting that FT-6876 could serve as a treatment for patients with other AR-dependent tumors, such as prostate cancer.
Phase 1 underway in 2021. In December 2020, Forma initiated recruitment in a Phase 1 trial of FT-7051 in men with metastatic castration-resistant prostate cancer including those with AR-v7 splice variants. Dosing in this trial is anticipated to begin in early in the first quarter of 2021. Forma plans to disclose initial safety, tolerability and preliminary response data in the second half of 2021.