WATERTOWN, Mass., March 09, 2022 (GLOBE NEWSWIRE) — C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing the science of targeted protein degradation to develop a new generation of small molecule drugs and transform the way disease is treated, has announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CFT8634 for the treatment of soft tissue sarcoma.
The FDA Orphan Product Development Office grants orphan designation status to drugs and biologicals intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people worldwide we Orphan drug designation offers certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the we upon regulatory approval.
“Patients living with synovial sarcoma currently have limited treatment options. After first-line treatment with chemotherapy, the benefit of which is generally limited in duration, patients with metastatic synovial sarcoma tend to have poor results. 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 urgent medical need faced by patients and their families,” said Adam’s CrystalMD, Ph.D., Chief Medical Officer C4 Therapeutic.
CFT8634 is a BiDAC™ degrader targeting BRD9 for the treatment of cancers that are BRD9 dependent, including synovial sarcoma and SMARCB1-deleted cancers. BRD9 was considered a “non-drug” target because BRD9 bromodomain inhibitors are not effective in treating these cancers. However, C4T’s TORPEDO® The platform was leveraged to discover CFT8634, a selective and orally bioavailable degrader of BRD9. Unlike BRD9 inhibition, BRD9 degradation is effective in preclinical models of synovial sarcoma. In December 2021, the FDA has cleared C4T’s Investigational New Drug (IND) application for CFT8634 to proceed with the proposed Phase 1/2 trial in patients with synovial sarcoma and SMARCB1-null solid tumors. Site activation efforts have begun and the trial remains on track to begin dosing patients in the first half of 2022.
On C4 Therapeutic
C4 Therapeutic (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to fulfilling the promise of the science of targeted protein degradation to create a new generation of medicines that transform patients’ lives. C4T relies on its TORPEDO® platform to efficiently design and optimize small molecule drugs that harness the body’s natural protein recycling system to rapidly degrade pathogenic proteins, offering the potential to overcome drug resistance, non-drug drug targets and d improve patient outcomes. C4T is advancing several targeted oncology programs into the clinic and expanding its research platform to deliver the next wave of drugs for hard-to-treat diseases. For more information, please visit www.c4therapeutics.com.
This press release contains “forward-looking statements” of C4 Therapeutics, Inc. within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may include, but are not limited to, express or implied statements regarding our ability to develop potential therapies for patients; the design and potential effectiveness of our therapeutic approaches; the predictive capacity of our TORPEDO® development platform for new selective and bioavailable degraders orally; the potential timing, design and progress of our preclinical studies and clinical trials, including the potential timing of regulatory approval related to clinical trials; our ability and potential to successfully manufacture and deliver our product candidates for clinical trials; our ability to replicate the results obtained in our preclinical studies or clinical trials in any future studies or trials; and regulatory developments in United States and foreign countries. All forward-looking statements contained in this press release are based on management’s current expectations and beliefs regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially. and adversely from those stated or implied by such statements. – look at the statements. These risks and uncertainties include, but are not limited to: uncertainties relating to the initiation, timing, progress and conduct of preclinical and clinical studies and other development requirements for our product candidates; the risk that one or more of our product candidates will cost more to develop or will not be successfully developed and commercialized; and the risk that the results of preclinical studies and/or clinical trials may or may not be predictive of results in future studies or trials. For a discussion of these and other risks and uncertainties, and other important factors, each of which could cause our actual results to differ materially from those contained in the forward-looking statements, see the section entitled “Risk Factors”. in the most recent publication of C4 Therapeutics. Annual Report on Form 10-K and/or Quarterly Report on Form 10-Q, as filed with the Security and Exchange Commission. All information contained in this press release is as of the date of publication, and C4 Therapeutics undertakes no obligation to update this information except as required by law.
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Source: C4 Therapeutics, Inc.
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