RARE Daily

AI Therapeutics Acquires Novel Cancer Drug Candidate for the Treatment of Rare Pediatric Cancers

May 2, 2022

AI Therapeutics, an AI driven clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, said it acquired EntreChem’s investigational drug candidate EC-8042 (redesignated AIT-102), a novel targeted cancer therapy in development for rare pediatric and other cancers.

Photo: Brigette Roberts, CEO of AI Therapeutics

Financial terms of the transaction were not disclosed.

Rhabdoid tumor is a rare and rapidly progressive pediatric cancer, with an average age at diagnosis of 15 months. In 90 percent of cases, rhabdoid tumors are due to a mutation in SMARCB1, a tumor suppressor gene encoding a member of the SWI/SNF remodeling complex. Patients have an average four-year survival of 10 to 40 percent.

Ewing Sarcoma is a rare aggressive malignant bone tumor most commonly diagnosed in adolescents and young adults between the age of 10 and 20. In 85 percent of cases, Ewing sarcoma is caused by a translocation between chromosomes 11 and 22 which creates a fused EWS-FLI oncogenic transcription factor. The three-year survival rate for patients diagnosed with metastatic Ewing Sarcoma is approximately 30 percent. For relapsed Ewing Sarcoma, 6-month progression-free survival is just 12.7 percent.

Despite recent advances in the understanding of tumor biology, treatment for many pediatric cancers remains limited to surgery, radiation and chemotherapy which often have poor outcomes and are associated with significant side effects. AIT-102 specifically targets the mutations responsible for the formation and progression of two pediatric tumors, rhabdoid tumors and Ewing sarcoma, and holds the potential to treat a broader family of tumors that share a common mechanism of mutated or dysregulated SWI/SNF activity.

SWI/SNF is a protein complex involved in the winding and unwinding of DNA around proteins called chromatin, thereby regulating the expression of thousands of genes. SWI/SNF is mutated in approximately 20 percent of all cancers. Mutations either in SWI/SNF itself or in transcription factors that associate with SWI/SNF alter the expression of a wide range of genes leading to cancer.  AIT-102 is a targeted cancer therapy that inhibits the activity of SWI/SNF in certain tumors where SWI/SNF is mutated (such as Rhabdoid tumors) and in other tumors where SWI/SNF associates with oncogenic transcription factors (such as EWS-FLI in Ewing Sarcoma).       

“AIT-102 is a powerful addition to AI Therapeutics’ rare disease treatment pipeline,” said Brigette Roberts, CEO of AI Therapeutics. “Cancer is the leading cause of death by disease in children in the U.S. today. With AIT-102, we hope to change these statistics. Beyond pediatrics, AIT-102 has the potential to treat up to 20 percent of cancers with altered SWI/SNF activity, either through mutation or dysregulation, including epithelioid sarcoma, synovial sarcoma, lung adenocarcinoma, colorectal cancer, and others.”

Author: Rare Daily Staff

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