On August 13, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported financial results for the second quarter ended June 30, 2021 (Press release, Forma Therapeutics, AUG 13, 2021, View Source [SID1234586545]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.
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"During the second quarter, we presented positive results from our ongoing Phase 1 trial demonstrating etavopivat’s highly differentiated profile and multimodal mechanism of action to improve markers of sickle cell disease and red blood cell health that are associated with vaso-occlusion," said Frank Lee, president and chief executive officer of Forma. "These results, in addition to the progress on our other clinical programs this quarter, position us well to deliver on our mission of transforming the lives of patients with rare hematologic diseases and cancers."
Key Business and Clinical Highlights
PKR Program in Sickle Cell Disease (SCD):
Clinical data presented at EHA (Free EHA Whitepaper) Virtual Congress support potential of investigational agent etavopivat to significantly impact RBC health and lifespan. Updated results were presented from the two week multiple ascending dose (MAD) cohorts and initial open-label extension (OLE) results administering etavopivat for up to 12 weeks, including:
Sustained increases in hemoglobin levels. In the MAD cohorts 73% of patients (11/15) achieved a hemoglobin increase of ≥ 1 g/dL at the end of two-weeks of treatment. In the OLE, hemoglobin levels increased >1g/dL in 88% of patients (7/8) receiving once daily treatment for at least two weeks, and this increase was sustained in those patients receiving continued treatment for up to 12 weeks.
Improvements in RBC oxygenation and deformability. RBC’s from 14 patients in the MAD cohorts showed increased hemoglobin-oxygen affinity, a significant shift in the point of sickling (POS), and improved deformability.
Significant reduction in hemolysis with markers approaching normal levels. Reticulocyte counts were reduced in 100% of patients (15/15), with normalization in some patients at the end of 2 weeks of treatment. The majority of patients demonstrated a marked decrease in lactate dehydrogenase levels (LDH) and indirect bilirubin levels as compared to baseline levels.
Reduction in systemic biomarkers related to inflammation and hypercoagulability. Initial results from the OLE showed improvement in systemic biomarkers such as lower levels of TNF-alpha, a marker of inflammation and decreases in prothrombin 1.2 and D-dimer, markers of coagulation activation.
Etavopivat was well tolerated with a safety profile consistent with underlying sickle cell disease. Etavopivat was well tolerated at doses up to 600mg daily (150% of the maximum dose in the ongoing Phase 2/3 Hibiscus Study).
CPB/p300 Program in Prostate Cancer:
FT-7051 Phase 1 clinical trial enrollment is ongoing. In January 2021, Forma announced the first patient 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, pharmacokinetics/pharmacodynamics (PK/PD), and preliminary anti-tumor activity, of FT-7051 in men with mCRPC who have progressed despite prior therapy with at least one anti-androgen therapy. The adaptive trial design is intended to accelerate the dose escalation to potentially therapeutic doses and yield important safety information, as well as to identify biomarkers of clinical benefit such as PSA response. Genetic mutation analysis will be conducted to correlate genetic changes with resistance to standard-of-care and will also evaluate expression of the AR-v7 splice variant, for which there are no approved therapies.
IDH1 Program in AML and Glioma:
Phase 2 registrational results for olutasidenib in R/R AML were presented at scientific conferences. Olutasidenib data in R/R AML were presented at both the annual ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) meetings in June 2021. The primary efficacy evaluable population, comprised of 123 patients, received 150 mg of olutasidenib twice daily for at least six months prior to the planned interim analysis. The primary endpoint, a composite complete remission (CR) or CR plus CR with partial hematologic recovery (CRh), was achieved in 33.3% (30% CR and 3% CRh) of patients. While the median duration of response was not yet reached, in a sensitivity analysis with hematopoietic stem cell transplant considered as the end of a response, the median duration was 13.8 months. The median overall survival (OS) was 10.5 months. Although a median OS has not yet been reached for the CR/CRh population, 18-month survival is estimated at 87% for that response category, and median survival is 15.0 months for non-CR/CRh responders. In addition, among patients with a CR who were transfusion-dependent at baseline, 56-day transfusion independence was achieved in 100% of patients as measured by platelets and 80% as measured by RBC’s. Olutasidenib was well-tolerated, and adverse events were consistent with the late stage disease in this heavily pre-treated patient population. Based upon these results, Forma is preparing an NDA for the R/R AML indication.
Corporate
In June 2021, Forma announced the appointment of John E. Bishop, Ph.D., as chief technology officer. Dr. Bishop leads chemistry, manufacturing and control (CMC)-related functions and quality, encompassing Forma’s early pipeline through commercial product. Dr. Bishop’s background includes extensive expertise with CMC development in oncology and hematology. Prior to joining Forma, Dr. Bishop served as senior vice president of pharmaceutical sciences at Epizyme, Inc., where he was a member of the executive team and held overall responsibility for the CMC and quality assurance functions.
Upcoming Milestones
Scientific conference presentation of updated Phase 1 etavopivat results in SCD. Updated results of safety, clinical activity, and biomarkers from the 12-week OLE are expected to be presented at a scientific congress in late 2021. Up to 20 patients are being administered etavopivat 400mg once daily and assessed for hematologic and hemolytic response, improvements in RBC oxygenation and deformability, and systemic markers of SCD.
Initiation of etavopivat trials in thalassemia and pediatric sickle cell patients. Enrollment in a Phase 2 trial of etavopivat in thalassemia patients is expected to begin prior to the end of the year, with results anticipated in 2022. The trial may enroll up to 60 patients with either thalassemia or SCD who are receiving chronic red blood cell transfusions, or thalassemia without chronic red blood cell transfusions. A trial in pediatric sickle cell disease patients is planned to begin in the first half of 2022.
Scientific conference presentation of initial Phase 1 FT-7051 clinical results in mCRPC. An abstract from this ongoing trial has been accepted for presentation at the 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) taking place Oct. 7-10, 2021. The presentation will include preclinical data and initial clinical results on safety, tolerability and PK/PD from patients undergoing dose escalation.
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 may result 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 $570.8 million as of June 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 $31.6 million for the quarter ended June 30, 2021, compared to $20.5 million for the quarter ended June 30, 2020. The increase was primarily attributable to etavopivat development, as well as increases in staff and stock-based compensation.
General and Administrative (G&A) Expenses: G&A expenses were $12.5 million for the quarter ended June 30, 2021, compared to $6.4 million for the quarter ended June 30, 2020. The increase in was primarily attributable to increased stock-based compensation, executive and staff hiring, professional fees, and insurance.
Net Loss: Net loss was $43.6 million for the quarter ended June 2021, compared to net loss of $25.4 million for the quarter ended June 30, 2020.
Forma will conduct a conference call and webcast Aug.13 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss second 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 9155938.
The live webcast will be available in the "News & Investors" section of Forma’s website www.formatherapeutics.com.