On November 12, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease, prostate cancer and other rare hematologic diseases and cancers, reported financial results for the third quarter ended Sept. 30, 2021 (Press release, Forma Therapeutics, NOV 12, 2021, View Source [SID1234595392]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

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"During the third quarter, we made significant progress pursuing our commitment not only to serving the needs of people living with sickle cell disease with our lead program, etavopivat, but also targeting prostate cancer with initial FT-7051 results, in addition to new olutasidenib clinical data accepted for presentation at ASH (Free ASH Whitepaper) next month," said Frank Lee, president and chief executive officer of Forma.

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

Enrollment in etavopivat open label extension completed. Full enrollment of 15 patients in the open label extension (OLE) was completed during the quarter. Patients in the OLE are being administered etavopivat 400 mg once daily for up to 12 weeks and assessed for hematologic and hemolytic response, and improvement in markers of red blood cell (RBC) health, including oxygenation and deformability, as well as systemic biomarkers of SCD.
CPB/p300 Program in Prostate Cancer:

Initial FT-7051 Phase 1 clinical trial results presented at NCI/AACR/EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). Preliminary results included data as of Sept. 1, 2021, from eight men enrolled in the trial. FT-7051 was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. The initial pharmacokinetic (PK) analysis of FT-7051 documented rapid absorption, with drug concentrations that approached the predicted efficacious dose based on estimates from preclinical animal models. Skin biopsies of the men participating in the study demonstrated a reduction in H3K27AC, a marker of activity in the CBP/p300 pathway. The majority of the treatment-emergent adverse events (TEAEs) were mild or moderate, at Grade 2 or lower, with no events leading to treatment discontinuation. The first evaluable patient completing more than 90 days of treatment demonstrated an ongoing response of >80% decline in prostate-specific antigen (PSA80) from baseline with stable disease.
Corporate

Upcoming investor conference participation. Forma will participate in the Jefferies London Healthcare Conference taking place Nov. 16-19. The pre-recorded presentation will be available Nov. 18 at 8:00am Greenwich Mean Time (GMT) in the "News & Investors" section of Forma’s website at www.FormaTherapeutics.com.
Investor briefing to discuss results presented at ASH (Free ASH Whitepaper). Forma will hold an investor briefing Dec. 13 at 8:00 am Eastern Time (ET). The live webcast will be available in the "News & Investors" section of Forma’s website www.FormaTherapeutics.com.
Upcoming Milestones

Updated Phase 1 OLE trial results for etavopivat in SCD to be presented at ASH (Free ASH Whitepaper) Dec. 11-14. Results to be presented will include 15 patients being administered etavopivat 400mg once daily for up to 12 weeks and assessed for hematologic and hemolytic response, and improvements in markers of RBC health including oxygenation and deformability, and systemic markers of SCD.
Patient enrollment continues in Phase 2/3 registrational trial, the Hibiscus Study. The Hibiscus study includes two co-primary end points (hemoglobin and vaso-occlusive crises) that support the traditional approval pathway. Based on ongoing feedback from the FDA, although accelerated approval is still an available regulatory pathway, Forma will need to provide additional information to support hemoglobin response as a surrogate endpoint eligible for accelerated approval for etavopivat. The company plans to continue to seek accelerated approval for etavopivat utilizing hemoglobin response rates as a surrogate endpoint by providing additional data to support that hemoglobin response rates predict for a clinical benefit.
Additional etavopivat trials to begin late 2021/1H:22. Forma plans to initiate a Phase 2 trial in transfusion dependent SCD and both transfusion dependent and independent thalassemia prior to the end of the year, and a pediatric SCD trial beginning in the first half of 2022.
Olutasidenib results in R/R AML to be presented at ASH (Free ASH Whitepaper) Dec. 11-14. Results from the Phase 2 trial will show the impact of the combination of olutasidenib and azacitidine on rates of remission and transfusion independence in patients with mutant isocitrate dehydrogenase 1 (mIDH1) AML. Analyses of safety and efficacy will be presented at the ASH (Free ASH Whitepaper) annual meeting.
Additional FT-7051 clinical results in mCRPC to be presented in 2022. Men with mCRPC continue to be enrolled in the dose escalation portion of the Phase 1 trial. Forma plans to present updated results from the trial at a scientific conference in mid-2022.
Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones and ongoing clinical trials. Many clinical trials across the biopharma industry, including Forma’s, have been impacted by the COVID-19 pandemic. Clinical trial sites implementing new policies in response to COVID-19 have impacted enrollment of clinical trials or and the ability to access sites participating in clinical trials.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $531.8 million as of Sept. 30, 2021, as compared to $645.6 million as of Dec. 31, 2020. Current cash runway is projected through the third quarter of 2024.
Research and Development (R&D) Expenses: R&D expenses were $30.7 million for the quarter ended Sept. 30, 2021, compared to $24.8 million for the quarter ended Sept. 30, 2020. The increase was primarily attributable to an increase in research and development staff to support advancement of etavopivat and other programs, an increase in equity-based compensation, and increases in external predevelopment and preclinical programs, conduct of the Phase II/III etavopivat trial in SCD patients and study start-up costs related to a trial in thalassemia/transfusion dependence.
General and Administrative (G&A) Expenses: G&A expenses were $12.7 million for the quarter ended Sept. 30, 2021, compared to $7.5 million for the quarter ended Sept. 30, 2020. The increase was primarily attributable to equity-based compensation, costs due to executive and staff hiring, legal, consulting, and other professional fee expenses, and other related general and administrative costs.
Net Loss: Net loss was $43.3 million for the quarter ended Sept. 30, 2021, compared to net loss of $27.6 million for the quarter ended Sept. 30, 2020.
Forma will conduct a conference call and webcast Nov. 12 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss third quarter 2021 results and business updates. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 6662686.

The live webcast will be available in the "News & Investors" section of Forma’s website www.formatherapeutics.com.