On May 29, 2020 Forma Therapeutics reported that kit has developed a pipeline of small molecules aimed at rare blood disorders and cancers, but it aims to advance two—investigational treatments for sickle cell disease and for advanced prostate cancer—and find partners for the rest of them (Press release, Forma Therapeutics, MAY 29, 2020, View Source [SID1234558743]).

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Now the Watertown, MA-based company is considering tapping the public markets to add enough funds to move one of its pair of core drug candidates further along in the clinic and to start human tests of the other. Forma set a preliminary $150 million goal for its IPO and says it plans to apply for a Nasdaq listing under the stock symbol "FMTX."

The company priorities are its oral, once-daily drug, FT-4202, which it is evaluating in a Phase 1 trial as a treatment for sickle cell disease, and FT-7051, a preclinical-stage treatment for metastatic castration-resistant prostate cancer (mCRPC).

Forma’s sickle cell drug is designed to increase hemoglobin levels and reduce the episodes of acute pain known as vaso-occlusive crises, caused when sickle-shape cells aggregate and block the supply of oxygen to tissues. The drug is intended to activate an enzyme, pyruvate kinase-R, that plays a role in the metabolism, function, and survival of red blood cells.

The biotech is studying the drug in patients age 12 and older who have the blood disorder. About 100,000 people in the US are affected by the inherited condition. A slew of companies are also pursuing new treatments for the disease, including Cambridge, MA-based Imara (NASDAQ: IMRA), which went public in March to raise funds to advance its investigational small molecule; Bluebird Bio (NASDAQ: BLUE), which is developing a gene therapy; and a handful of competitors with experimental gene-editing fixes. Late last year Global Blood Therapeutics (NASDAQ: GBT) won FDA approval for its once-daily sickle cell disease drug.

In its preliminary prospectus, Forma said it also aims to expand clinical development of FT-4202 into beta thalassemia, another rare blood disorder.

Forma’s prostate cancer treatment is designed to inhibit a protein complex, CBP/p300, that plays a role in the activity of the androgen receptor, which drives prostate cancer cell growth. The FDA in April cleared Forma to begin human tests of the drug.

Prostate cancer is the second leading cause of cancer death for men in the US; the type that Forma’s drug is intended to treat is the most advanced form of the disease. The UK’s CellCentric is also aiming to treat mCRPC with a CBP/p300 inhibitor of its own; Cambridge, MA-based Constellation Pharmaceuticals (NASDAQ: CNST) is advancing a clinical-stage drug for the indication, too.

Money raised in an IPO would be deployed to get the sickle cell drug through Phase 1 and, if results are as anticipated, proceed to a pivotal Phase 2/3 clinical trial. The funds would also be used to advance the cancer therapy into a planned Phase I clinical trial.

Some cash would also go toward Forma’s non-core programs—those already partnered and those seeking partners to take them forward. Programs still in search of partners include its most advanced, olutasidenib, which is currently in a pivotal study testing it as a treatment for acute myeloid leukemia. The investigational cancer drug is also being studied in an exploratory Phase I trial for glioma. In addition, Forma is advancing two potential treatments for nonalcoholic steatohepatitis, the liver disease better known as NASH.

Forma previously licensed a molecule it discovered, a potential treatment for non-Hodgkin lymphoma, to Celgene (now a subsidiary Bristol Myers Squibb (NYSE: BMY)) and a KRAS inhibitor, meant to treat solid tumors, to Boehringer Ingelheim.

The company, which was founded in 2007, is headed by president and CEO Frank Lee, who previously ran global development and commercial strategy for Genentech’s immunology, ophthalmology and infectious diseases divisions. Its chief medical officer, Patrick Kelly, who has been with the company since 2016, served previously in the oncology unit at Takeda Pharmaceutical (NYSE: TAK). Forma last month added to its C-suite David Cook, who was most recently at Seres Therapeutics (NASDAQ: MCRB), as its chief scientific officer.

Forma’s founding backers were the investment arms of Eli Lilly (NYSE: LLY) and Novartis (NYSE: NVS. Today, according to the prospectus, RA Capital owns 21.16 percent of the company; Novartis holds 12.63 percent; Baker Bros. Advisors, 10.58 percent; and Lilly, 10.15 percent. Other shareholders include Cormorant Asset Management and Singapore’s Biomedical Sciences Investment Fund.

As of the end of March the company reported about $142 million in cash, cash equivalents, and short-term investments.