On May 14, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported financial results for the first quarter ended March 31, 2021 (Press release, Forma Therapeutics, MAY 14, 2021, View Source [SID1234580013]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

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"During the first quarter, we successfully completed the multiple ascending dose portion of our Phase 1 trial in sickle cell disease, and despite challenges from the COVID-19 pandemic also began enrolling patients in the Phase 2/3 trial of FT-4202, called The Hibiscus Study, as well as in the Phase 1 trial of FT-7051 for metastatic castration resistant prostate cancer," said Frank Lee, President and Chief Executive Officer of Forma. "We look forward to sharing additional pipeline results over the course of 2021 in our mission to transform the lives of people living with rare hematologic diseases and cancers."

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

MAD cohorts completed with approximately 71% of participants achieving hemoglobin increase ≥ 1 g/dL from baseline, and improvement across markers of RBC health. Doubling the dose of FT-4202 to 600 mg daily for 14 days compared to the previous 300 mg cohort was well-tolerated with no dose-limiting toxicities or treatment-related adverse events observed. Improvements in hematologic (hemoglobin and reticulocytes) and hemolytic (bilirubin and lactate dehydrogenase) parameters were comparable to that observed with the 300 mg dose, with best response typically observed at the end of the 14-day treatment period. In the combined cohorts, 10 of 14 (71%) patients on FT-4202 achieved a hemoglobin increase ≥ 1 g/dL from baseline to Day 14. Improvement in RBC health was evidenced by increased sickle RBC survival and reduced intravascular hemolysis in patients with SCD based on a reduction in reticulocytes, bilirubin and LDH levels.
Patient enrollment began in Phase 2/3 registrational trial, the Hibiscus Study. The Phase 2/3 Hibiscus Study is currently enrolling people living with SCD. This adaptive, randomized, placebo-controlled, double-blind, multi-center trial is expected to enroll approximately 344 adults and adolescents with SCD. FT-4202 doses of 200mg and 400mg administered once-daily are being evaluated in the Phase 2 portion of the trial. Primary endpoints in the Phase 3 portion of the trial are hemoglobin response rate at week 24 (increase of > 1 g/dL from baseline), intended to support accelerated approval, and annualized vaso-occlusive crisis rate during the 52-week blinded treatment period, which if positive is expected to support full approval.
CPB/p300 Program in Prostate Cancer:

FT-7051 Phase 1 clinical trial initiated for the treatment of mCRPC. In January 2021, Forma announced that the first patient was dosed in the ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment of mCRPC. The trial is a multicenter, open-label evaluation of the safety and tolerability, preliminary anti-tumor activity (prostate specific antigen (PSA) and radiographic responses), and pharmacokinetics/pharmacodynamics (PK/PD) of FT-7051 in men with mCRPC who have progressed despite prior therapy with at least one anti-androgen therapy. The trial will include genetic mutation analysis to identify the basis of resistance to standard-of-care and will also evaluate expression of the AR-v7 splice variant, for which there are no approved therapies. The trial utilizes an adaptive trial design, intended to accelerate the escalation to potentially therapeutic doses and yield important safety information, as well as to identify biomarkers of clinical benefit such as PSA response.
IDH1 Program in AML and Glioma:

Olutasidenib NDA preparation for R/R AML. With the conclusion of the Phase 2 R/R AML trial, Forma has begun preparing an NDA for submission to the U.S. Food and Drug Administration (FDA).
Upcoming Milestones

Presentation of updated Phase 1 FT-4202 results in SCD. A poster presentation on FT-4202 in SCD is scheduled for the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021. The presentation will include combined unblinded data from the two-week MAD cohorts as well as initial OLE results to date. In addition, full results from the MAD dose cohorts and the OLE are expected to be presented at a scientific congress in late 2021.
Initial Phase 1 clinical results from FT-7051 in mCRPC anticipated later this year. This adaptive trial is assessing multiple doses of FT-7051 with dose escalation dependent upon safety and tolerability. Initial results anticipated in the fourth quarter of 2021 may include safety/tolerability, PK/PD results and preliminary biomarker data.
Olutasidenib results presentation in R/R AML. Phase 2 registrational results of olutasidenib in R/R AML will be presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place from June 4-8, 2021, and the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021.
Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones. Many clinical trials across the biopharma industry, including ours, have been impacted by the COVID-19 pandemic, with clinical trial sites implementing new policies in response to COVID-19, resulting in potential delays to enrollment of clinical trials or changes in the ability to access sites participating in clinical trials.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $603.7 million as of March 31, 2021, as compared to $645.6 million as of December 31, 2020. Current cash runway is projected through the third quarter of 2024.
Research and Development (R&D) Expenses: R&D expenses were $26.3 million for the quarter ended March 31, 2021, compared to $23.2 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in FT-4202 development expenses, partially offset by reduced spending on olutasidenib development.
General and Administrative (G&A) Expenses: G&A expenses were $9.9 million for the quarter ended March 31, 2021, compared to $8.9 million for the quarter ended March 31, 2020. The increase in general and administrative expense was primarily attributable to stock compensation expense and insurance, partially offset by a reduction in professional fees.
Net Income/Loss: Net loss was $36.0 million for the quarter ended March 31, 2021, compared to net income of $11.2 million for the quarter ended March 31, 2020.
Forma will conduct a conference call and webcast May 14th at 8 a.m. Eastern Daylight Time (EDT) to discuss first quarter 2021 results and business update. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 8597396.