Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX) is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. The company has a diverse pipeline of product candidates, including its lead candidate etavopivat for the treatment of sickle cell disease (SCD) and other hemoglobinopathies, and FT-7051 for the treatment of metastatic castration-resistant prostate cancer (mCRPC).
Business Overview
Forma Therapeutics was founded in 2007 and is headquartered in Watertown, Massachusetts. The company's drug discovery expertise has generated a pipeline of product candidates focused on indications with significant unmet patient need. Etavopivat, Forma's lead product candidate, is a novel, oral, once-daily, potentially disease-modifying therapy being studied for the treatment of SCD. SCD is a chronic hemolytic anemia that affects hemoglobin and can lead to low hemoglobin levels, painful vaso-occlusive crises, progressive multi-organ damage, and early death. Etavopivat is designed to improve red blood cell metabolism, function, and survival, potentially resulting in both increased hemoglobin levels and reduced vaso-occlusive crises.
Forma's other product candidate, FT-7051, is a potent and selective inhibitor of CREB-binding protein/E1A binding protein p300 (CBP/p300) in clinical development for the treatment of mCRPC. Prostate cancer is a leading cause of cancer death for men, and mCRPC is the most advanced form of the disease. FT-7051 targets the androgen receptor, which drives prostate cancer cell growth, and may play an important role in suppressing mCRPC in patients with androgen receptor-resistant variants.
In addition to its internal pipeline, Forma has out-licensed programs to Rigel Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, and Celgene Corporation (now Bristol-Myers Squibb Company), from which it is eligible to receive potential milestone payments and royalties.
Financials
Forma Therapeutics reported an annual net loss of 172,964,000 in 2022, with no revenue generated during the year. The company's annual operating cash flow was -149,787,000, and its annual free cash flow was -153,966,000.
For the three months ended June 30, 2022, Forma reported a net loss of 52,578,000, with no revenue generated during the quarter. The company's operating expenses for the quarter were 52,998,000, consisting of 39,059,000 in research and development expenses and 13,939,000 in general and administrative expenses. Forma's cash, cash equivalents, and marketable securities totaled 395,903,000 as of June 30, 2022.
Pipeline and Clinical Development
Etavopivat (SCD and Hemoglobinopathies)
Forma's lead product candidate, etavopivat, is currently being evaluated in a global pivotal Phase II/III clinical trial, known as the Hibiscus Study, for the treatment of SCD. The company has also initiated a Phase II trial of etavopivat in patients with either transfusion-dependent SCD, transfusion-dependent thalassemia, or non-transfusion-dependent thalassemia.In June 2021, Forma announced initial data from the 12-week open-label extension cohort of its Phase I trial studying the effects of 400 mg of etavopivat once-daily on SCD patients. The company provided updated results from this trial in December 2021 and May 2022, which showed improvements in hemoglobin levels and reductions in markers of hemolysis.
Etavopivat has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of SCD. The European Medicines Agency has also granted Orphan Drug designation to etavopivat for the treatment of SCD.
FT-7051 (mCRPC)
Forma's other product candidate, FT-7051, is in a Phase I clinical trial for the treatment of mCRPC. In October 2021, the company presented initial results from the first eight patients enrolled in this trial, and in May 2022, Forma announced that 25 patients had been enrolled.Olutasidenib (AML and Glioma)
In addition to its internal pipeline, Forma has out-licensed its compound olutasidenib, a selective inhibitor of mutant isocitrate dehydrogenase 1 (mIDH1), to Rigel Pharmaceuticals, Inc. Olutasidenib has completed a registrational Phase II trial for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML) and is also being evaluated in an exploratory Phase I clinical trial for glioma.Liquidity
As of June 30, 2022, Forma Therapeutics had 395,903,000 in cash, cash equivalents, and marketable securities, which the company believes will be sufficient to fund its operations through the third quarter of 2024. The company has financed its operations primarily through license and collaboration agreements, the sale of preferred shares and preferred stock to investors, and the completion of its initial public offering and follow-on public offering.
Risks and Challenges
Forma Therapeutics faces several risks and challenges common to the biopharmaceutical industry, including the need for additional capital, the risks of failure in preclinical studies and clinical trials, the need to obtain marketing approval and reimbursement for its product candidates, and the need to successfully commercialize and gain market acceptance for its products. The company also faces competition from other companies developing therapies for rare hematologic diseases and cancers.
Outlook
Forma Therapeutics is a promising biopharmaceutical company with a diverse pipeline of product candidates targeting rare hematologic diseases and cancers. The company's lead candidate, etavopivat, has shown promising results in early clinical trials for the treatment of SCD and other hemoglobinopathies, and the company is advancing it through late-stage clinical development. Additionally, Forma's other product candidate, FT-7051, is in clinical development for the treatment of mCRPC, a significant unmet medical need.
While Forma Therapeutics faces the typical risks and challenges associated with drug development, the company's strong financial position, with 395,903,000 in cash, cash equivalents, and marketable securities as of June 30, 2022, provides it with the resources to continue advancing its pipeline. The company's out-licensed programs also have the potential to generate additional revenue through milestone payments and royalties.
Conclusion
Overall, Forma Therapeutics appears to be a well-positioned biopharmaceutical company with a promising pipeline and the financial resources to support its development efforts. As the company continues to progress its lead and other product candidates through clinical trials, investors will be closely watching for updates on the potential regulatory approvals and commercialization of these novel therapies.