WATERTOWN, MA, USA I May 16, 2022 I C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, today announced that the first patient has been dosed in its Phase 1/2 clinical trial of CFT8634, an orally bioavailable BiDAC™ degrader targeting BRD9 for the treatment of SMARCB1-perturbed cancers, including synovial sarcoma and SMARCB1-null tumors.
“The initiation of our first clinical trial of CFT8634 is a significant milestone for C4 Therapeutics as we apply our TORPEDO® platform to an oncology target currently considered ‘undruggable’ and work to provide a new treatment option for patients living with synovial sarcoma and SMARCB1-null tumors,” said Adam Crystal, M.D., Ph.D., chief medical officer of C4 Therapeutics. “Based on our pre-clinical research showing that the BRD9 degrader CFT8634 is potent, selective and efficacious in models of synovial sarcoma and malignant rhabdoid tumors, we believe CFT8634 may offer an effective targeted treatment for patients who currently have limited therapeutic options.”
The Phase 1/2 trial will primarily investigate safety, tolerability and anti-tumor activity with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT8634. The Phase 1 portion of the study will evaluate CFT8634 as an oral, single agent therapy for patients with synovial sarcoma and SMARCB1-null tumors to identify a recommended Phase 2 dose. Following identification of recommended dosage, the Phase 2 portion of the trial is expected to expand to the following investigational arms: one in synovial sarcoma, and one in SMARCB1-null tumors. Across the Phase 1/2 trial, C4T plans to enroll approximately 90 patients.
CFT8634 is C4T’s second oncology program to enter clinical studies from its internally developed research pipeline. CFT8634 is C4T’s first BiDAC degrader candidate to be evaluated in the clinic.
To learn more about the CFT8634 clinical trial, visit clinicaltrials.gov (identifier: NCT 05355753).
About CFT8634
CFT8634 is a BiDAC™ degrader targeting BRD9 for the treatment of cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1-null cancers. BRD9 has been considered an “undruggable” target due to the inability of bromodomain inhibitors to effectively treat these cancers. Unlike BRD9 inhibition, BRD9 degradation has been shown to be efficacious in pre-clinical models of synovial sarcoma. By leveraging C4T’s TORPEDO® platform, C4T developed CFT8634, an orally bioavailable, selective degrader of BRD9. In March 2022, C4T announced the U.S. Food and Drug Administration had granted orphan drug designation (ODD) to CFT8634.
About Synovial Sarcoma and SMARCB1-null Tumors
Synovial sarcoma is a rare and aggressive subtype of soft tissue sarcoma. It accounts for approximately 10 percent of all sarcoma diagnoses. An estimated 900 people are diagnosed with synovial sarcoma in the U.S. each year. Approximately one-third of patients are diagnosed under the age of 30.
SMARCB1-null tumors include malignant rhabdoid tumor, poorly differentiated chordoma, epithelioid sarcoma and other rare cancers; some subtypes are most commonly diagnosed in children and young adults.
Both synovial sarcoma and SMARCB1-null tumors are believed to be dependent on BRD9 and, as a result, CFT8634 may be an effective treatment.
About C4 Therapeutics
C4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transforms patients’ lives. C4T is leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines that harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. C4T is advancing multiple targeted oncology programs to the clinic and expanding its research platform to deliver the next wave of medicines for difficult-to-treat diseases. For more information, please visit www.c4therapeutics.com.
SOURCE: C4 Therapeutics
Post Views: 317
WATERTOWN, MA, USA I May 16, 2022 I C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, today announced that the first patient has been dosed in its Phase 1/2 clinical trial of CFT8634, an orally bioavailable BiDAC™ degrader targeting BRD9 for the treatment of SMARCB1-perturbed cancers, including synovial sarcoma and SMARCB1-null tumors.
“The initiation of our first clinical trial of CFT8634 is a significant milestone for C4 Therapeutics as we apply our TORPEDO® platform to an oncology target currently considered ‘undruggable’ and work to provide a new treatment option for patients living with synovial sarcoma and SMARCB1-null tumors,” said Adam Crystal, M.D., Ph.D., chief medical officer of C4 Therapeutics. “Based on our pre-clinical research showing that the BRD9 degrader CFT8634 is potent, selective and efficacious in models of synovial sarcoma and malignant rhabdoid tumors, we believe CFT8634 may offer an effective targeted treatment for patients who currently have limited therapeutic options.”
The Phase 1/2 trial will primarily investigate safety, tolerability and anti-tumor activity with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT8634. The Phase 1 portion of the study will evaluate CFT8634 as an oral, single agent therapy for patients with synovial sarcoma and SMARCB1-null tumors to identify a recommended Phase 2 dose. Following identification of recommended dosage, the Phase 2 portion of the trial is expected to expand to the following investigational arms: one in synovial sarcoma, and one in SMARCB1-null tumors. Across the Phase 1/2 trial, C4T plans to enroll approximately 90 patients.
CFT8634 is C4T’s second oncology program to enter clinical studies from its internally developed research pipeline. CFT8634 is C4T’s first BiDAC degrader candidate to be evaluated in the clinic.
To learn more about the CFT8634 clinical trial, visit clinicaltrials.gov (identifier: NCT 05355753).
About CFT8634
CFT8634 is a BiDAC™ degrader targeting BRD9 for the treatment of cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1-null cancers. BRD9 has been considered an “undruggable” target due to the inability of bromodomain inhibitors to effectively treat these cancers. Unlike BRD9 inhibition, BRD9 degradation has been shown to be efficacious in pre-clinical models of synovial sarcoma. By leveraging C4T’s TORPEDO® platform, C4T developed CFT8634, an orally bioavailable, selective degrader of BRD9. In March 2022, C4T announced the U.S. Food and Drug Administration had granted orphan drug designation (ODD) to CFT8634.
About Synovial Sarcoma and SMARCB1-null Tumors
Synovial sarcoma is a rare and aggressive subtype of soft tissue sarcoma. It accounts for approximately 10 percent of all sarcoma diagnoses. An estimated 900 people are diagnosed with synovial sarcoma in the U.S. each year. Approximately one-third of patients are diagnosed under the age of 30.
SMARCB1-null tumors include malignant rhabdoid tumor, poorly differentiated chordoma, epithelioid sarcoma and other rare cancers; some subtypes are most commonly diagnosed in children and young adults.
Both synovial sarcoma and SMARCB1-null tumors are believed to be dependent on BRD9 and, as a result, CFT8634 may be an effective treatment.
About C4 Therapeutics
C4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transforms patients’ lives. C4T is leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines that harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. C4T is advancing multiple targeted oncology programs to the clinic and expanding its research platform to deliver the next wave of medicines for difficult-to-treat diseases. For more information, please visit www.c4therapeutics.com.
SOURCE: C4 Therapeutics
Post Views: 317
