Pharma Co. Shares Get a Bang from FDA Orphan Drug Approval

Source: Streetwise Reports   03/09/2022

Shares of C4 Therapeutics Inc. traded 16% higher after the biopharma company reported that the U.S. FDA granted Orphan Drug approval status for its CFT8634 for use in treating soft tissue sarcoma.

Clinical-stage biopharmaceutical company C4 Therapeutics Inc. (CCCC:NASDAQ) (C4T), which is focused on developing next generation small-molecule medicines that exhibit the potential to eliminate disease-causing proteins for use in treatments for cancer and other diseases, today announced that “the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT8634 for the treatment of soft tissue sarcoma.”

The company explained that the FDA assigns ODD status to medicines and biologics used to address rare conditions or diseases that typically affect less than 200,000 people in the U.S. The firm noted that “ODD provides certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.”

C4 Therapeutics’ Chief Medical Officer Adam Crystal, M.D., Ph.D. remarked, “Patients living with synovial sarcoma currently have limited treatment options. After first-line treatment with chemotherapy, the benefit of which is typically of limited durability, patients with metastatic synovial sarcoma tend to do poorly.”

“The FDA’s decision to grant orphan drug designation to CFT8634 is an important recognition of the potential of our targeted protein degrader to address this dire unmet medical need faced by patients and their families,” Dr. Crystal added.

The company described CFT8634 as “a BiDAC™ degrader targeting BRD9 for the treatment of cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1 deleted cancers.”

The firm stated that until recently, BRD9 was believed to be an “undruggable” target as bromodomain inhibitors of BRD9 were not proven to be effective in treating these cancers. C4 Therapeutics advised that “its TORPEDO® platform was leveraged to discover CFT8634, an orally bioavailable, selective degrader of BRD9, and that unlike BRD9 inhibition, BRD9 degradation is efficacious in preclinical models of synovial sarcoma.”

The company mentioned that the FDA recently approved an investigational new drug (IND) application for CFT8634 in December 2021 allowing C4 Therapeutics to go forward with its Phase 1/2 clinical trial in patients with synovial sarcoma and SMARCB1-null solid tumors. The firm indicated that the study is proceeding and stated that it expects to begin dosing patients in the trial in H1/22.

C4 Therapeutics is based in Watertown, Mass. and is a clinical-stage biopharmaceutical company that is focused on the implementation of targeted protein degradation science used to create a new small-molecule medicines to treat cancer and other diseases with the goal of transforming patients’ lives.

C4 Therapeutics Inc. began the day with a market cap of around $1.0 billion with approximately 48.74 million shares outstanding and a short interest of about 6.9%. CCCC shares opened 6% higher today at $22.34 (+$1.34, +6.38%) over yesterday’s $21.00 closing price. The stock has traded today between $21.73 and $24.80 per share and is currently trading at $24.28 (+$3.28, +15.62%).

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