About Us | ScientistFMTX Forma Therapeutics Holdings Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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FT-7051
Prostate cancer (CRPC)
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Phase 1
Phase 1
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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FT-4202
Sickle cell disease
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Phase 2/3
Phase 2/3
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Phase 2/3 trial has been initiated.
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Olutasidenib (FT-2102)
Glioma
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Phase 1
Phase 1
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Phase 1 trial ongoing.
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Olutasidenib (FT-2102)
Acute myeloid leukemia
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Phase 2
Phase 2
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Phase 2 data presented at ASCO June 4, 2021. 33.3% composite complete remission rate (CR/CRh) noted in abstract.
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Latest News
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Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced the appointment of John E. Bishop, Ph.D., to the leadership team as senior vice president and chief technology officer. In this role, Dr. Bishop will lead chemistry, manufacturing and control (CMC)-related functions and quality, encompassing Forma's early pipeline through commercial product.
"We are fortunate to welcome John to Forma at this pivotal time in Forma's history," said Frank Lee, chief executive officer of Forma. "John's extensive expertise in small-molecule pharmaceuticals and proven track record as a leader will help to expedite drug development and commercialization at Forma, speeding us on our mission of transforming the lives of patients with rare hematologic diseases and cancers."
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 (QA) functions. Prior to Epizyme, Dr. Bishop held positions of increasing responsibility, including as senior vice president at Momenta Pharmaceuticals, Inc. While at Momenta, Dr. Bishop planned and oversaw expansion of the company's CMC & QA functions from an ad hoc, three-scientist operation to 130 full-time employees. He also developed and executed successful CMC strategies for Momenta's generic versions of two complex drugs, enoxaparin sodium (LOVENOX®) and glatiramer acetate (COPAXONE®), which led to Momenta's establishment of a broad biosimilar portfolio.
"I look forward to this opportunity to help Forma further establish and scale a truly groundbreaking technical organization that will enable the company to innovate in disease areas in urgent need of new solutions," said Dr. Bishop. "I believe the team at Forma is applying world-class science and a sincere commitment to patients, and I'm delighted to join this passionate group."
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
This press release may contain forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could," "would," "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavors," "potential," "continue," "target" or the negative of such words or other similar expressions can be used to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption "Risk Factors" in Forma's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as other risks detailed in our subsequent filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Forma believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Forma nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release represents Forma's views only as of the date on which it was made. Forma undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210614005118/en/
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Clinical results demonstrated durable improvement in hematologic and hemolytic markers, supporting the potential for improvement of red blood cell functional health in those with sickle cell disease
Initial results from an open-label extension cohort showed sustained hemoglobin increase of >1g/dL in 88% (7 of 8) of patients dosed for at least two and up to 12 weeks, as well as favorable tolerability profile
Improvement in markers of red blood cell functional health were observed, including data on measures of cell membrane integrity and systemic biomarkers of inflammation and coagulation
Forma to host webcast today at 8:00 a.m. ET to discuss etavopivat results presented at EHA
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX) a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced new data from its ongoing Phase 1 trial of etavopivat (formerly referred to as FT-4202) being presented at the 26th Annual European Hematology Association (EHA) 2021 Virtual Congress. The e-poster presentation includes initial data from the open-label extension (OLE) cohort showing etavopivat improved and sustained hematologic and hemolytic parameters for patients living with sickle cell disease (SCD) receiving 400 mg etavopivat once-daily for at least two weeks and up to 12 weeks. Also being presented are the unblinded results from the two multiple ascending dose (MAD) cohorts, which demonstrate once-daily dosing of 300 mg or 600 mg etavopivat for 14 days improved measures of sickle red blood cell (RBC) functional health, with effects persisting in some patients even after treatment discontinuation.
"Data presented today, including initial data from the OLE cohort, demonstrate patient responses improved with more than two weeks of dosing with etavopivat, including hemoglobin and markers of hemolysis, RBC functional health, and systemic inflammation and coagulation that together have the potential to reduce the incidence of vaso-occlusive crises with longer-term treatment," said Patrick Kelly, M.D., chief medical officer of Forma Therapeutics. "These results, along with the favorable tolerability profile we have observed, support our recent initiation of the Hibiscus Study – our Phase 2/3 trial in people living with SCD – and bring us one step closer to a potential new treatment option for those affected by SCD."
Presentation Details
- Abstract #EP1201: FT-4202 (Etavopivat) improves hematologic and hemolytic parameters in a phase 1 study of patients with sickle cell disease (Robert Clark Brown, M.D., Ph.D)
The e-poster presentation is available as of Friday, June 11, 2021, at 9:00 Central European Summer Time (CEST) and is accessible for on-demand viewing until Sunday, August 15, 2021, on EHA's virtual congress platform. The abstract and poster presentation are also available on Forma's website.
Clinical Data Results
In the combined MAD1 (300 mg QD) and MAD2 (600 mg QD) cohorts, 73% (11 of 15) of patients achieved a hemoglobin increase of greater than 1g/dL over baseline; significant improvement in hematologic and hemolytic markers also included decreased absolute reticulocytes (100%, or 15 of 15), decreased LDH levels (73%, or 11 of 15) and decreased indirect bilirubin levels (93%, or 14 of 15). The osmoscan and oxygenscan results from 14 patients showed a statistically significant improvement.
Initial results as of May 24, 2021 in the OLE cohort for eight patients receiving etavopivat treatment (400 mg QD) for at least two weeks indicated a hemoglobin increase of greater than 1 g/dL in 88% (7 of 8), with a mean hemoglobin increase of 1.5 g/dL. Patient data indicated a durable response for those patients receiving treatment beyond two weeks, for up to 12 weeks, with improved hematologic and hemolytic parameters. The improvement in RBC functional health extended beyond the 12-week treatment period; in one patient, improved sickle RBC deformability remained for up to four weeks after treatment discontinuation. These initial OLE data support the combined MAD cohort results and show that daily etavopivat treatment also significantly improved hematologic and hemolytic parameters.
The safety profile in the OLE cohort was consistent with underlying disease. Of note, two patients reported serious adverse events, including one vaso-occlusive crisis and acute chest syndrome, which was not considered related to treatment by the trial investigator. A deep-vein thrombosis (DVT) report was described as possibly related.
Additional results being presented at the conference are measures of RBC functional health, with RBC elongation and point-of-sickling data analysis showing improvements in cell deformability, including durable changes for up to four weeks following treatment. The data show benefits beyond activation of the glycolytic pathway, including enhanced activity of enzymes involved in preventing and repairing oxidative damage and reduced levels of phosphatidyl serine (PS), a marker of membrane damage observed on the surface of sickle RBCs. Early data from the 12-week OLE cohort show favorable systemic biomarkers including lower levels of erythropoietin (EPO), reduced evidence of activation of coagulation (Prothrombin 1.2 and D-dimers) and decreased activation of innate immunity (TNF-a). These biomarkers suggest the potential for a broader benefit to people living with SCD, including the potential to reduce vaso-occlusive crises.
Forma Webcast Today
Forma is hosting a webcast today at 8:00 a.m. ET to discuss these etavopivat results being presented at EHA. The webcast can be accessed in the "News & Investors" section of Forma's website at www.formatherapeutics.com.
Ongoing Trials
The blinded, randomized, placebo-controlled portion of the ongoing Phase 1 trial is complete. People with SCD are now directly enrolling into the ongoing 12-week OLE cohort receiving 400 mg etavopivat daily.
Forma is currently enrolling adults and adolescents with SCD into the Hibiscus Study, a registrational Phase 2/3 randomized, placebo-controlled, double-blind, multicenter trial to further evaluate the safety and efficacy of etavopivat in this patient population. For more information, please visit https://hibiscusstudy.com/ or clinicaltrials.gov/NCT04624659.
About Sickle Cell Disease (SCD)
SCD is one of the most common single-gene disorders and is estimated to affect approximately 100,000 people in the United States, as well as approximately 30,000 in France, Germany, Italy, Spain and the United Kingdom. The National Institutes of Health (NIH) reports that prevalence is estimated at more than 20 million individuals globally. From 2010 to 2050, the annual number of newborns with SCD is expected to rise globally by approximately one-third.i Despite recent advances in treatment, most patients with SCD still suffer from pain crises, lifelong disability, significant morbidity and reduced quality of life.
About Etavopivat
Etavopivat is a novel investigational selective red blood cell (RBC) pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD). Employing a multimodal approach, etavopivat is designed to work upstream by activating the RBCs' natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. The downstream activity of etavopivat is designed to increase ATP levels, the fuel that provides energy to cells, to improve RBC functional health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, etavopivat did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development. Etavopivat has been granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency for the treatment of patients with SCD.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
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 our beliefs and expectations regarding: initial results to date for the etavopivat open label extension cohort of our Phase 1 clinical trial; ; the therapeutic potential and clinical benefits and safety related to etavopivat; whether initial results from our clinical trials are predictive of final trial results or future clinical studies; and our planned presentation of data at the 2021 EHA Virtual Congress;. 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.
Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related our ability to execute on our strategy; the therapeutic potential and safety of etavopivat; the timing and completion of our Phase 1 study of etavopivat and final audit and quality controlled verification of initial data and related analyses; the timing and success of our Phase 2/3 Hibiscus Study of etavopivat in SCD patients; positive results from initial data analyses may not be predictive of final results; risks related to our planned regulatory submissions and developments; and other risks identified in our SEC filings, including those risks discussed under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as other risks detailed in our subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.
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i Piel, F. B., Hay, S. I., Gupta, S., Weatherall, D. J., & Williams, T. N. (2013). Global burden of sickle cell anaemia in children under five, 2010-2015: Modelling based on demographics, excess mortality, and interventions. PLOS Medicine, 10(7). Retrieved from link.View source version on businesswire.com: https://www.businesswire.com/news/home/20210611005206/en/
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Olutasidenib demonstrated a 33.3% composite complete remission rate (CR/CRh) in people living with R/R AML with the IDH1 mutation
Among those with CR/CRh, estimated 18-month survival is 87%; median overall survival has not yet been reached
Duration of response of 13.8 months is longest reported in this treatment setting to date
Favorable tolerability profile following continuous oral treatment with olutasidenib 150mg BID
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX) today announced that topline interim data from its Phase 2 trial of olutasidenib in relapsed/refractory acute myeloid leukemia (R/R AML) will be presented at the upcoming 2021 American Society of Clinical Oncology (ASCO) Annual Meeting taking place June 4-8. Olutasidenib, Forma's selective inhibitor for cancers with mutations in isocitrate dehydrogenase 1 (IDH1m), demonstrated positive efficacy and a favorable tolerability profile as a monotherapy in patients with IDH1m R/R AML, achieving the primary endpoint of a composite complete remission (CR) or CR plus CR with partial hematologic recovery (CRh) rate of 33.3% (30% CR and 3% CRh).
The presentation is based on an interim analysis from a pivotal trial arm evaluating continuous treatment with 150 mg twice daily of oral olutasidenib. The data indicate the duration of CR/CRh for people on treatment was 13.8 months. Among patients with a complete remission (CR) who were transfusion-dependent at baseline, 56-day transfusion independence was achieved in 100% of patients as measured by platelets and 83% as measured by red blood cells.
"The data being presented at ASCO showcase olutasidenib's meaningful progress for this patient population, which currently has limited options to extend life expectancy," said Patrick Kelly, M.D., chief medical officer of Forma Therapeutics. "The safety data from the treatment cohort are consistent with the findings from our Phase 1 evaluation in this high-risk AML patient population. The data highlight the duration of response, which is nearly six months longer than current standard of care."
Oral Abstract Session – June 4, 2:30 p.m. ET
- Abstract #7006: Effect of olutasidenib (FT-2102) on complete remissions in patients with relapsed/refractory (R/R) mIDH1 acute myeloid leukemia (AML): Results from a planned interim analysis of a phase 2 clinical trial
About the Phase 1/2 Study
The Phase 1/2 study is a multicenter, open-label, multi-cohort evaluation of the safety, efficacy and pharmacokinetics/pharmacodynamics (PK/PD) of olutasidenib for patients with AML or myelodysplastic syndrome (MDS) with an IDH1 mutation. Phase 1 of the trial, 2102-HEM-101, was an open-label, dose-escalation and expansion study of olutasidenib alone and in combination with azacitidine (AZA). The Phase 2 portion was an open-label, fixed-dose study of olutasidenib as a monotherapy and in combination with AZA in multiple IDH1m AML/MDS populations. The primary efficacy evaluable population is comprised of 123 R/R AML patients enrolled in Cohort 1, who received 150 mg of olutasidenib BID at least six months prior to the interim analysis cutoff date of June 18, 2020. The primary endpoint of the Phase 2 pivotal study is a complete remission (CR) plus a complete remission with partial hematological recovery (CRh) that is defined as <5% blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (platelets >50,000/microliter and ANC >500/microliter).
Key Study Findings
Efficacy (n=123)
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Olutasidenib induced a durable CR/CRh rate of 33.3% (95% CI 25.1, 42.2), which is the primary endpoint of the study:
- The CR rate was 30.0% (37 of 123 patients) and the CRh rate was 3.0% (4 of 123 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 duration of overall response was 11.7 months.
- The median overall survival (OS) was 10.5 months. The median OS for non-CR/CRh responders was 15.0 months. A median OS has not yet been reached for the CR/CRh population, with an 18-month survival estimate of 87.0%.
- Transfusion independence was achieved in all response groups at 56 days, particularly in those achieving CR, with 100% independence for platelet transfusions and 83.0% independence for red blood cells.
Safety (n=153)
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Olutasidenib was well-tolerated, with adverse events (AEs) consistent with the late stage disease and the heavily pre-treated population. A safety analysis for all 153 patients enrolled in the Phase 2 Cohort 1 found the most common grade 3/4 (≥ 20% or ≥ 10%) treatment-emergent adverse events (TEAEs) were febrile neutropenia (20%), anemia (19%), thrombocytopenia (16%), neutropenia (13%), leukocytosis (9%) and fatigue (<1%). AEs of interest were the following:
- 14% of patients reported AEs due to Differentiation syndrome, including 7% Grade 3 and 1% Grade 4 AEs. Most resolved with corticosteroids and treatment interruption. However, 1 fatal event was reported.
- 8% of patients reported AEs due to QTc prolongation, with <1% Grade 3 or 4. No events led to discontinuation.
- Grade 3 or 4 elevation in liver parameters (ALT/AST/total bilirubin) occurred in 10% and 2% of patients, respectively. Most resolved with treatment interruption and dose reduction. Seven patients (<5%) discontinued study treatment due to LFT abnormalities. No Hy's law cases reported.
About Olutasidenib
Olutasidenib is an oral, potent, small-molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes. This targeted treatment has the potential to provide therapeutic benefit by reducing 2-HG levels and restoring normal cellular differentiation. With the conclusion of the Phase 2 R/R AML trial, Forma has begun preparing a new drug application (NDA) for submission to the U.S. Food and Drug Administration (FDA).
IDH1 is a natural enzyme that is part of the normal metabolism of all cells; when mutated, its activity can promote blood malignancies and solid tumors. IDH1 mutations are present in 6-8% of patients with AML and as many as 70 to 80% of patients with grade II/III gliomas and secondary glioblastoma. In gliomas, IDH1 mutations occur early in the tumor pathogenesis and persist throughout progression from a neural stem or progenitor cell. Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors, and high-grade gliomas are associated with poor long-term prognosis. Treatment options for relapsed glioma are limited.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
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 our beliefs and expectations regarding: interim data analysis for olutasidenib in the Phase 2 trial in R/R AML; the therapeutic potential and clinical benefits and safety related to olutasidenib; whether interim results from our clinical trials are predictive of final trial results or future clinical studies; our planned presentation of data at ASCO; and planned regulatory submissions, including the preparation and submission of an NDA for olutasidenib with the FDA. 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.
Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related our ability to execute on our strategy; the finalization of our Phase 2 study in R/R AML and final audit and quality controlled verification of interim data and related analyses; positive results from interim data analyses may not be predictive of final results; risks related to our planned regulatory submissions and developments; and other risks identified in our SEC filings, including those risks discussed under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as other risks detailed in our subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210520005330/en/
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Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that company management will participate in two upcoming investor conferences:
- The Oppenheimer Rare & Orphan Disease Summit taking place Friday, May 21, 2021. Forma's pre-recorded presentation will be available on May 21 at 8:00 a.m. EDT.
- The Jefferies Virtual Healthcare Conference taking place June 1-4, 2021. Forma will present on June 2 at 11 a.m. EDT.
Webcasts/audio recordings of the conference presentations will be available in the "News & Investors" section of Forma's website at www.formatherapeutics.com.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210517005098/en/
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Development pipeline progressing to key upcoming clinical milestones
Multiple-ascending dose (MAD) cohorts of FT-4202 Phase 1 trial in sickle cell disease completed; hemoglobin increased ≥ 1 g/dL in approximately 71% of patients and improved hematologic and hemolytic markers consistent with improved red blood cell (RBC) health
Updated MAD results and initial open-label extension trial (OLE) results to date to be presented at European Hematology Association (EHA) Virtual Congress in June
Phase 1 trial of FT-7051 in metastatic castration-resistant prostate cancer (mCRPC) ongoing, initial results expected fourth quarter of 2021
Olutasidenib relapsed/refractory acute myeloid leukemia (R/R AML) results presentation in June at American Society of Clinical Oncology (ASCO), new drug application (NDA) preparation ongoing
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the first quarter ended March 31, 2021. The company also highlighted recent progress and upcoming milestones for its pipeline programs.
"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 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 Annual Meeting taking place from June 4-8, 2021, and the EHA 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.
The live webcast will be available in the "News & Investors" section of Forma's website www.formatherapeutics.com.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Our R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. For more information, please visit www.FormaTherapeutics.com or follow us on Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
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 FT-4202, FT-7051 and olutasidenib, including expectations regarding timing and success of the current ongoing clinical trials, therapeutic potential, clinical benefits and safety thereof, planned regulatory submissions, including an NDA for olutasidenib, and upcoming milestones for the company's other product candidates; growth as a company; presentation of additional data at upcoming scientific conferences, and other preclinical data and potential data publications in 2021; the potential commercial and collaboration opportunities, including potential future collaborators and parties, as well as value and market, for our product candidates; uses and need of capital, expenses and other 2021 financial results currently or in the future, and the potential impact of COVID-19 on patient retention and enrollment, future operations, clinical trials or investigational new drug (IND) applications. 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.
Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties associated with the following: the impact of the COVID-19 pandemic on the company's business, operations, patient enrollment and retention , strategy, goals and anticipated milestones; the therapeutic potential of FT-4202, FT-7051, and olutasidenib, and the timing associated with the initiation or continuation of any trials and success of ongoing clinical trials of FT-4202 and FT-7051; Forma's ability to execute on its strategy; the submission and acceptance of a new drug application (NDA) for submission to the U.S. Food and Drug Administration (FDA) for olutasidenib; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; any one or more of Forma's product candidates may not be successfully developed and commercialized; regulatory developments in the United States and foreign countries; Forma's ability to protect and maintain our intellectual property position; the impact of COVID-19 affecting countries or regions in which we have operations or do business, including potential negative impacts on our employees, customers, supply chain and production as well as global economies and financial markets; Forma's ability to fund operations; Forma's ability to identify satisfactory collaboration opportunities, as well as those risks and uncertainties set forth more fully under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, filed with the United States Securities and Exchange Commission (SEC) and subsequent filings with the SEC.. Forma disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent Forma's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Forma explicitly disclaims any obligation to update any forward-looking statements.
Selected Financial Information
(in thousands except share and per share data)
(unaudited)
Statement of Operations Items: For the Three Months
Ended March 31,
2021
2020
Collaboration revenue $
—
$
—
Operating expenses: Research and development 26,343
23,210
General and administrative 9,867
8,933
Restructuring charges —
83
Total operating expenses 36,210
32,226
Loss from operations (36,210
)
(32,226
)
Other income, net 258
23,971
Loss before taxes (35,952
)
(8,255
)
Income tax expense (benefit) 8
(19,485
)
Net (loss) income $
(35,960
)
$
11,230
Accretion of cumulative dividends on Series D redeemable convertible preferred stock —
(1,936
)
Undistributed earnings allocable to participating securities —
(3,456
)
Net (loss) income allocable to shares of common stock, basic $
(35,960
)
$
5,838
Change in fair value attributable to warrants to purchase Series B-3 convertible preferred shares —
(20
)
Accretion of cumulative dividends on Series D redeemable convertible preferred stock —
1,936
Net (loss) income allocable to shares of common stock, diluted $
(35,960
)
$
7,754
Net (loss) income per share of common stock: Basic $
(0.76
)
$
2.29
Diluted $
(0.76
)
$
0.36
Weighted-average shares of common stock outstanding: Basic 47,295,013
2,548,079
Diluted 47,295,013
21,392,760
Selected Balance Sheet Items: March 31, 2021 December 31, 2020 Cash, cash equivalents, and marketable securities $
603,724
$
645,588
Total assets $
650,236
$
680,971
Accounts payable, accrued expenses, and other current liabilities $
25,142
$
31,399
Total stockholders' equity $
(93,370
)
$
(57,410
)
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