FMTX Forma Therapeutics Holdings Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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FT-4202
Sickle cell disease
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Phase 1
Phase 1
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|
Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
|
FT-7051
Prostate cancer (CRPC)
|
Phase 1
Phase 1
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Phase 1 trial planned for 4Q 2020.
|
|
Olutasidenib FT-2102
Acute myeloid leukemia
|
Phase 2
Phase 2
|
Phase 2 interim data released October 26, 2020. CR+CRh (complete remission plus complete remission with partial hematologic recovery) rate of 33.3% (30% CR and 3% CRh).
|
|
FT-2102
Glioma
|
Phase 1
Phase 1
|
Phase 1 trial ongoing.
|
Latest News
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Strong pipeline progress amid challenging COVID-19 environment
Oral presentation of MAD1 results at upcoming 2020 ASH Virtual Annual Meeting from the randomized, placebo-controlled multi-center Phase 1 trial evaluating FT-4202 in people with sickle cell disease
MAD2 cohort of the Phase 1 trial now enrolling with 600 mg dose
Registrational Phase 2/3 trial of FT-4202 on track to begin enrolling people living with sickle cell disease in the first quarter of 2021
Phase 1 trial evaluating FT-7051 in metastatic castration-resistant prostate cancer on track to begin enrolling patients before end of 2020
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the third quarter ended September 30, 2020. The company also highlighted recent progress and upcoming milestones for its pipeline programs.
"We are very pleased with our strong pipeline progress during the quarter amid such challenging times," said Frank Lee, President and Chief Executive Officer of Forma. "We look forward to presenting new data from our ongoing Phase 1 trial of FT-4202 in sickle cell disease at the ASH meeting in December, as well as beginning enrollment of patients for our Phase 1 trial of FT-7051 in men living with metastatic castration-resistant prostate cancer. The recent positive top-line results from the olutasidenib registrational Phase 2 clinical trial in relapsed/refractory acute myeloid leukemia with an IDH1 mutation further underscores our commitment to developing transformative therapies for patients."
Key Business and Clinical Highlights
PKR Program in Sickle Cell Disease (SCD):
- Both planned dose cohorts enrolling in the multiple ascending dose (MAD) trial. The MAD1 cohort is designed to dose 9-12 SCD patients with 300 mg of FT-4202. Clinical measures being assessed include change in hemoglobin, indirect bilirubin, reticulocytes and lactate dehydrogenase, as well as the monitoring of tolerability and safety during the 14-day dosing and 7-day follow-up period. The MAD2 cohort is assessing a higher 600 mg dose and is now enrolling patients. Patients completing the 600 mg MAD2 cohort may enter the 12-week Open Label Extension (OLE) portion of the trial.
- FT-4202 abstract selected for oral presentation at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition December 5-8, 2020. The FT-4202 abstract describes blinded data from three patients receiving the 300 mg dose, measuring changes in parameters over the 14-day treatment and 7-day follow-up period including hemoglobin and reticulocytes, as well as tolerability and safety. Updated data will be presented at the ASH annual meeting on December 7, 2020.
CPB/p300 Program in Prostate Cancer:
- Phase 1 clinical trial of FT-7051 for the treatment of metastatic castration-resistant prostate cancer (mCRPC) on track to start by year end. This trial will enroll patients who have progressed while on standard anti-androgen therapy. Patients' prostate cancer will be profiled for mutations in the androgen receptor (AR)-signaling pathway that drive resistance to AR-receptor antagonists, such as ARv7 mutations.
IDH1 Program in AML and Glioma:
- Announced positive data for olutasidenib in relapsed/refractory acute myeloid leukemia (R/R AML). In October 2020, Forma announced positive results from the planned interim analysis (IA2) of the Phase 2 registration trial in R/R AML patients with isocitrate dehydrogenase 1 gene mutations (IDH1m). Olutasidenib demonstrated a favorable tolerability profile as a monotherapy, and for the primary efficacy endpoint of composite complete remission (CR+CRh, or complete remission plus complete remission with partial hematologic recovery), achieved a rate of 33.3% (30% CR and 3% CRh). While a median duration of CR/CRh has not been reached, a sensitivity analysis (with a hematopoietic stem cell transplant as the end of a response) indicates the median duration of CR/CRh to be 13.8 months. Safety results are consistent with previously reported Phase 1 clinical trial results.
- Olutasidenib is also being evaluated in an exploratory Phase 1 trial for glioma as presented at the American Society of Clinical Oncology meeting in June 2020, as well as in other IDH1m solid tumor indications.
Corporate:
- In September 2020, Forma announced the appointment of industry veteran Thomas G. Wiggans to Forma's board of directors. Mr. Wiggans has led successful biopharmaceutical companies from start-up stage into the clinic and later global commercialization, served on the boards of numerous public and private companies, and was instrumental in the formation of the Biotechnology Industry Organization, now Biotechnology Innovation Organization (BIO).
Upcoming Milestones
- Results from the ongoing randomized placebo-controlled multicenter Phase 1 trial evaluating FT-4202 in patients with SCD to be presented at ASH. Clinical data on the safety results, PK/PD and laboratory measurements in 9-12 patients from the 300 mg MAD1 cohort will be presented during an oral presentation at the virtual, 62nd American Society of Hematology (ASH) Annual Meeting and Exposition December 5-8, 2020. Subsequently, results from the MAD2 600 mg cohort are expected in the first quarter of 2021, and 12-week OLE results are anticipated in the second quarter of 2021.
- Initiation of registrational trial of FT-4202 for people living with SCD: The global pivotal Phase 2/3 trial is expected to initiate in the first quarter of 2021. This adaptive, randomized, placebo-controlled, double-blind, multi-center study will enroll approximately 344 adults and adolescents with SCD. The trial will evaluate FT-4202 doses of 200 mg and 400 mg administered once daily in the Phase 2 portion. Primary endpoints in the Phase 3 portion of the trial are hemoglobin response rate at week 24 (increase of > 1 g/dL from baseline), and annualized vaso-occlusive crisis rate during the 52-week blinded treatment period.
- Initiation of FT-7051 Clinical Development in mCRPC: Patient enrollment in the Phase 1 trial of FT-7051 in mCRPC patients is expected to begin prior to the end of 2020. Safety and tolerability data from the trial are anticipated in 2021 and clinical activity results in 2022.
- Non-core Partnering Strategy: Following the recent positive registrational trial results in R/R AML with an IDH1 mutation, Forma remains focused on partnership opportunities for olutasidenib, as well as for the non-core FASN inhibitor for NASH (FT-8225).
- Possibility of COVID-19 Impact: The COVID-19 pandemic remains a factor in the successful completion of these milestones. Many clinical trials across the biopharma industry 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.
Upcoming Investor Events
- Dec. 7, 2020: Forma will conduct a conference call and webcast on Dec. 7 at 6 p.m. Eastern Standard Time (EST) to discuss updated results from the ongoing Phase 1 trial of FT-4202 in SCD, as well as an overview of the company's development plans for FT-4202. A live webcast will be available in the "News & Investors" section of Forma's website www.formatherapeutics.com.
Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were $384.3 million as of September 30, 2020, as compared to $173.2 million as of December 31, 2019.
- Research and Development (R&D) Expenses: R&D expenses were $24.8 million for the quarter ended September 30, 2020, compared to $27.6 million for the quarter ended September 30, 2019. The decrease was primarily due to planned reductions in spending on FT-2102, FT-4101, FT-8225, research activities, and internal R&D personnel-related costs, which were partially offset by increases in FT-4202 expenses to conduct the Phase 1 trial, clinical product manufacturing, and preparations for the pivotal Phase 2/3 trial.
- General and Administrative (G&A) Expenses: G&A expenses were $7.5 million for the quarter ended September 30, 2020, compared to $7.0 million for the quarter ended September 30, 2019. The increase in general and administrative expense was primarily attributable to a $1.3 million increase in equity-based compensation, and a $0.6 million increase in insurance related expense, and a $0.5 million increase in other related general and administrative costs, partially offset by a reduction of $2.0 million related to legal, consulting and other professional fee expenses.
- Net Income/Loss: Net loss was $27.6 million for the quarter ended September 30, 2020, compared to $31.0 million for the quarter ended September 30, 2019.
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 and FT-7051, including expectations regarding timing and success of the current ongoing clinical trials, therapeutic potential and clinical benefits thereof, and upcoming milestones for the company's other product candidates; growth as a company and the anticipated contribution of the members of our board of directors to our operations and progress; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the potential commercial and collaboration opportunities, including potential future collaborators and parties, as well as value and market, for our product candidates; uses of capital, expenses and other 2020 financial results or in the future, and the potential impact of COVID-19 on patient retention, strategy, future operations, clinical trials or IND submissions. 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 impact of the COVID-19 pandemic on the company's business, operations, strategy, goals and anticipated milestones; the therapeutic potential of FT-4202, and the timing associated with the initiation or continuation of any of FT-4202 trials; the initiation of our phase I clinical trial of FT-7051; Forma's ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in the United States and foreign countries; 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 the final prospectus dated June 22, 2020 and filed pursuant to Rule 424(b) under the Securities Act of 1933, as amended, with the United States Securities and Exchange Commission (SEC) and elsewhere in Forma's filings and reports 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: Three Months Ended
September 30,Nine Months Ended
September 30,2020
2019
2020
2019
Revenue $ -
$ 3,377
$ -
$ 93,113
Operating expenses Research and development 24,780
27,558
68,501
84,273
General and administrative 7,460
7,025
22,841
17,631
Restructuring charges -
545
63
5,620
Total operating expenses 32,240
35,128
91,405
107,524
Loss from operations (32,240)
(31,751)
(91,405)
(14,411)
Other income, net 818
766
23,050
3,057
Loss before taxes (31,422)
(30,985)
(68,355)
(11,354)
Income tax benefit (3,806)
-
(26,529)
(1,217)
Net loss $ (27,616)
$ (30,985)
$ (41,826)
$ (10,137)
Preferred return and accretion of preferred return and
cumulative dividends on preferred securities-
(607)
(3,736)
(2,395)
Distribution to holders of preferred securities in excess of
accrued preferred return-
-
-
(11,347)
Tax distribution to holders of Enterprise.1 Incentive Shares -
(60)
-
(60)
Net loss allocable to shares of common stock, basic $ (27,616)
$ (45,562)
Change in fair value attributable to warrants to purchase
common stock(8)
-
Net loss allocable to shares of common stock, diluted $ (27,624)
$ (45,562)
Net loss allocable to shares of Common 1, basic $ (31,652)
$ (23,939)
Change in fair value attributable to warrants to purchase
preferred securities(198)
(515)
Net loss allocable to shares of Common 1, diluted $ (31,850)
$ (24,454)
Net loss per share of common stock: Basic $ (0.67)
$ (2.74)
Diluted $ (0.67)
$ (2.74)
Net loss per share of Common 1: Basic $ (12.42)
$ (9.40)
Diluted $ (12.50)
$ (9.60)
Weighted-average shares of common stock outstanding: Basic 41,088,261
16,616,143
Diluted 41,088,924
16,616,143
Weighted-average shares of Common 1 outstanding, basic
and diluted2,547,924
2,547,924
Selected Balance Sheet Items: September 30, December 31, 2020
2019
Cash, cash equivalents, and marketable securities $
384,346
$
173,180
Total Assets $
447,396
$
183,035
Accounts payable, accrued expenses, and other
current liabilities$
30,215
$
23,629
Redeemable convertible and convertible preferred stock outside
of stockholders' equity-
$
138,131
Total stockholders' equity $
415,602
$
18,246
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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 European Commission granted Orphan Drug designation to Forma's FT-4202 for the treatment of sickle cell disease (SCD), based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA). FT-4202 was previously granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of patients with SCD.
"I am pleased to see the regulatory community recognize the urgent need to bring therapies to people living with sickle cell disease," said Frank Lee, president and chief executive officer of Forma. "Without effective treatment, sickle cell can affect all organs over time and lead to substantial suffering for those born with this genetic disease. We embrace the potential this designation may provide as we prepare to initiate a global registrational Phase 2/3 trial with FT-4202 in the first quarter of 2021."
Forma is currently enrolling patients with SCD in a randomized, placebo-controlled, multi-center Phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202. For more information on eligibility and study sites for the open Phase 1 study, please visit clinicaltrials.gov/NCT03815695.
About Orphan Drug Designation in the European Union (EU)
The European Commission grants Orphan Drug designation (ODD) to investigational drugs intended to diagnose, prevent or treat a rare disease. To qualify for ODD, the potential therapeutic must target a life-threatening or chronically debilitating disease that affects fewer than five (5) in 10,000 persons in the EU. In addition, the investigational drug must either provide a significant benefit over existing therapies or provide a treatment for patients for whom existing therapies do not work or exist. The designation provides financial and regulatory incentives to the sponsor company such as reduced fees, tax waivers, dedicated funds to reimbursement and 10 years of market exclusivity.
About Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common single-gene disorders and is estimated to affect more than 70,000 in the EU-27, as well as approximately 100,000 people in the United States. 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.1 Despite recent advances in treatment, most patients with SCD still suffer from pain crises, lifelong disability, significant morbidity and reduced quality of life.
About FT-4202
FT-4202 is a novel 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, FT-4202 works 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 FT-4202 increases ATP levels, the fuel that provides energy to cells, to improve RBC health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, FT-4202 did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development.
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 potential regulatory, exclusivity and marketing advantages of Orphan Drug designation by the EU Commission and previously granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) of FT-4202 for the treatment of patients with SCD,the advancement of our sickle cell disease program, our expectations of the therapeutic benefits related thereto, the timing and success of ongoing clinical trials, and our growth as a company and the anticipated contribution of our executives and employees to our operations and progress. 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 to the advancement of our clinical programs 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 June 30, 2020, 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.
____________________
1Piel, 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.
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Updated data from an ongoing randomized, multi-center, placebo-controlled Phase 1 clinical trial of FT-4202 in sickle cell disease selected for an oral presentation
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that four abstracts – including one oral presentation and three poster presentations – have been accepted for presentation at the 62nd American Society of Hematology (ASH) Virtual Annual Meeting taking place December 5-8, 2020.
The oral presentation will feature clinical data from the multiple ascending dose cohort of a randomized, multi-center, placebo-controlled Phase 1 trial of FT-4202 in people living with sickle cell disease (SCD). While the trial is currently enrolling patients in the second dose escalation cohort of 600 mg FT-4202 daily, data will be presented on 9-12 patients who have completed the 300 mg FT-4202 daily cohort. A "Trials in Progress" poster presentation will highlight key aspects of the planned registrational Phase 2/3 clinical trial. In addition, two collaborative posters will report the findings of research of FT-4202 in a mouse model of sickle cell anemia and in an ex vivo analysis of blood samples from patients with SCD, respectively.
"We're pleased the FT-4202 data have been selected for multiple presentations at ASH 2020," said Frank Lee, president and chief executive officer of Forma. "Forma has a deep commitment to advancing science in sickle cell disease. We look forward to sharing data that will further characterize FT-4202 as an investigational treatment for people living with sickle cell disease."
The abstracts, currently available on the ASH conference website, are:
Oral Presentation
Title: FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease
Date/Time: Monday, December 7, 2020 at 2:00 p.m. PT
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Novel Treatments for Sickle Cell Disease
Abstract: 679
Presenter: R. Clark Brown, MD, PhD, Pediatric Hematologist/Oncologist, Medical Director of Sickle Cell at Scottish Rite, Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, and Associate Professor of Pediatrics, Emory University School of MedicinePoster Presentations
Title: An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)
Date: Monday, December 7, 2020
Session: 114. Hemoglobinopathies, Excluding Thalassemia - Clinical: Poster III
Abstract: 2622
Presenter: Kenneth W. Wood, Executive Director, Project Leadership at Forma Therapeutics, Inc.Title: Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels
Date: Saturday, December 5, 2020
Session: 113. Hemoglobinopathies, Excluding Thalassemia - New Genetic Approaches to Sickle Cell Disease: Poster I
Abstract: 784
Presenter: Archana Shrestha, PhD, Research Associate at Cincinnati Children's Hospital Medical CenterTitle: Ex-Vivo FT-4202 Treatment Improves Hemoglobin Oxygen Affinity and Membrane Health in Red Blood Cells of Patients with Hemoglobin SS and Hemoglobin SC Disease Irrespective of Prior Hydroxyurea Use
Date: Saturday, December 5, 2020
Session: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I
Abstract: 786
Presenter: Diamantis Konstantinidis, Research Associate at Cincinnati Children's Hospital Medical CenterAbout Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common disorders caused by a single gene mutation. Prevalence of SCD is approximately 100,000 people in the U.S. and approximately 30,000 people in France, Germany, Italy, Spain and the UK. While reporting limitations complicate stating an exact number, the National Institutes of Health reports that prevalence is estimated at over 20 million individuals worldwide. In people living with SCD, red blood cells, or RBCs, spontaneously deform in low oxygen conditions, taking on a sickle-like shape. Sickle cells are stiff and have damaged membranes, causing the RBCs to clump and burst in small blood vessels, resulting in inflammation and vaso-occlusive crises. Repeated deformation also depletes the RBC energy supply, called ATP. One important consequence of this energy depletion is increased levels of a metabolite, 2,3-DPG, that further reduces the RBCs' affinity for oxygen and exacerbates the cycle of repeated deformation and anemia.
About FT-4202
FT-4202 is a novel, oral, once-daily pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD). Early studies and trials have shown that FT-4202 works upstream by employing a multi-modal approach and activating the red blood cells' (RBC) natural PKR activity to decrease 2,3-DPG levels, which we believe leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. FT-4202 has also shown downstream activity by increasing ATP levels, the fuel that provides energy to cells, which we believe may improve RBC health and survival. Together, these effects have the potential to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, FT-4202 did not inhibit aromatase activity, important biological processes responsible for sexual development.
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 FT-4202, our expectations of the therapeutic benefits related thereto, the timing and success of ongoing clinical trials, whether positive interim results from a clinical study are predictive of the results of ongoing or future clinical studies, and our growth as a company. 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 to the advancement of our clinical programs 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 June 30, 2020, 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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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 three upcoming investor conferences.
- The Credit Suisse 29th Annual Healthcare Conference taking place November 9-12. Forma will present via webcast on Wednesday, November 11 at 2:45 p.m. Eastern Standard Time (EST).
- The Jefferies 2020 Virtual London Healthcare Conference taking place November 17-19. Forma will present via webcast on Wednesday, November 18 at 7:55 p.m. Greenwich Mean Time (GMT).
- The Virtual SVB Leerink Oncology 1X1 Day taking place November 19. Forma will participate virtually in one-on-one meetings during this event on Thursday, November 19.
A live webcast of the presentations at the Credit Suisse and Jefferies conferences 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.
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FT2102-HEM-101 clinical trial data demonstrate a 30% CR and 3% CRh rate with olutasidenib monotherapy in 123 relapsed or refractory IDH1m AML patients
While median duration of CR/CRh has not been reached, sensitivity analysis indicates the median duration of CR/CRh to be 13.8 months
Favorable tolerability profile consistent with Phase 1 study results
Data will be submitted for discussion at an upcoming medical meeting
Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced positive top-line data from a planned interim analysis of a registrational Phase 2 clinical trial of olutasidenib, Forma's selective inhibitor for hematological malignancy cancers with mutations in isocitrate dehydrogenase 1 (IDH1m). Olutasidenib demonstrated a favorable tolerability profile as a monotherapy in patients with IDH1m relapsed/refractory acute myeloid leukemia (R/R AML), and achieved a composite complete remission (CR+CRh, or complete remission plus complete remission with partial hematologic recovery) rate of 33.3% (30% CR and 3% CRh), the primary efficacy endpoint. While a median duration of CR/CRh has not been reached, a sensitivity analysis (with a hematopoietic stem cell transplant or HCST as the end of a response) indicates the median duration of CR/CRh to be 13.8 months.
Safety results are consistent with previously reported Phase 1 clinical trial results1,2. The most common adverse events (AEs) observed were nausea, constipation, increased white blood cell count, decreased red blood cell count, fever, febrile neutropenia and fatigue.
"We are pleased to announce these compelling top-line data," said Patrick Kelly, MD, chief medical officer of Forma Therapeutics. "The safety profile and the duration of the response we're seeing supports the potential for olutasidenib to become a leading therapy for R/R IDH1m AML patients. While the multi-cohort Phase 2 trial is ongoing, this specific cohort was designed to serve as a pivotal study; these efficacy data support an early stop in enrollment in favor of moving the program forward."
Additional analyses and other outcome measures will be presented at an upcoming medical meeting.
Study Design
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, FT2102-HEM-101, was an open-label, dose-escalation and expansion study of olutasidenib alone and in combination with azacitidine (AZA). The pivotal Phase 2 study is an open-label, fixed-dose study of olutasidenib as a monotherapy in IDH1m AML patients. The Phase 2 study includes other cohorts of olutasidenib in combination with AZA in IDH1m AML/MDS populations. The primary efficacy-evaluable population of the pivotal phase 2 study is comprised of 123 R/R AML patients, who received olutasidenib 150 mg BID at least six months prior to the interim analysis cutoff date of June 18, 2020. The primary endpoint is a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh), defined as less than 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).
About Olutasidenib
Olutasidenib is an oral, potent and 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. Forma is currently evaluating olutasidenib in a registrational Phase 2 trial for relapsed/refractory AML and in an exploratory Phase 1 trial for glioma and other solid tumors.
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 guidance regarding our business plans and objectives for olutasidenib, including the therapeutic potential and clinical benefits thereof as well as the planned study design, the interim results of the Phase 2 clinical trial of olutasidenib, including its initial primary efficacy, safety and tolerability results, the planned additional analyses of the 2102-HEM-101 study, the timing and success of ongoing clinical trials, our growth as a company, and the potential impact of COVID-19 on patient retention, strategy, future operations and clinical trials. 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 to the advancement of our clinical programs, our ability to execute on our strategy, that positive interim results from a clinical study may not be necessarily predictive of the results of future or ongoing clinical studies, the regulatory developments in the United States, the risks related to the competitive landscape, 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 June 30, 2020, 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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1 J. Watts et al., ASH 2019
Olutasidenib, an IDH1 Inhibitor as a single agent or in combination with azacitadine, induces deep clinical remissions with mutation clearance in patients with acute myeloid leukemia treated in a Phase I dose escalation and expansion study.2 J. Cortes et al., ASH 2019
Olutasidenib induces rapid remissions in patients with IDH1-mutant myelodysplastic syndrome: Results of Phase 1/2 single-agent treatment and combination with azacitidineView source version on businesswire.com: https://www.businesswire.com/news/home/20201026005229/en/