Innovative, first-in-class compound for the treatment of rare pediatric cancers expands AI Therapeutics product pipeline
GUILFORD,CT, April 25, 2022 — AI Therapeutics, Inc., an AI driven clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, today announced the acquisition of EntreChem, S.L.’s investigational drug candidate EC-8042 (redesignatedAIT-102), a novel targeted cancer therapy in development for rare pediatric and other cancers.
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 notable 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.
“As a physician treating children with cancer on a daily basis, I am acutely aware of the need for more effective and less toxic therapies,” said Patrick Grohar, M.D., Ph.D., Director of Translational Research with the Center of Childhood Cancer Research at Children’s Hospital of Philadelphia, a leader in the study and treatment of pediatric tumors. “For many tumor types, the biological culprit is well understood, but has proven difficult to target with pharmaceuticals. I have dedicated the last 15 years of my professional career to understanding the mechanism of tumor biology in children and finding new, targeted, therapies to treat these tumors. I am particularly impressed with the strong preclinical data from our studies of AIT-102 in rhabdoid tumors and Ewing sarcoma, and I believe that AIT-102 has the potential to transform the treatment of these cancers, as well as many others.”
“AIT-102 is a powerful addition to AI Therapeutics’ rare disease treatment pipeline,” said Brigette Roberts, M.D., Chief Executive Officer of AI Therapeutics. “Cancer is the leading cause of death by disease in children in the US today. With AIT-102, we hope to change these statistics. Beyond pediatrics, AIT-102 has the potential to treat up to 20% of cancers with altered SWI/SNF activity, either through mutation or dysregulation, including epithelioid sarcoma, synovial sarcoma, lung adenocarcinoma, colorectal cancer, and others. We are thrilled to have this opportunity to bring forward a promising new agent to help fight these tumors.”
Financial terms of the transaction were not disclosed.
About SWI/SNF and AIT-102
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% 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).
About Rhabdoid Tumor
Rhabdoid tumor is a rare and rapidly progressive pediatric cancer, with an average age at diagnosis of 15 months. In 90% of cases, rhabdoid tumors are due to a mutation in SMARCB1, a tumor suppressor gene encoding a member of the SWI/SNF remodeling complex. There is currently no widely accepted, effective standard of care therapy for the treatment of Rhabdoid Tumors. Patients have an average four-year survival of 10–40%.
About Ewing Sarcoma
Ewing Sarcoma is a rare aggressive malignant bone tumor most commonly diagnosed in adolescents and young adults between the age of 10-20. In 85% of cases, Ewing sarcoma is caused by a translocation between chromosomes 11 and 22which creates a fused EWS-FLI oncogenic transcription factor. Standard of care treatment for Ewing Sarcoma includes the five-chemotherapy regimen VDC/IE with surgery and/or radiation. These therapies are associated with significant short and long-term side effects. The three-year survival rate for patients diagnosed with metastatic Ewing Sarcoma is approximately 30%. For relapsed Ewing Sarcoma, 6-monthprogression-free survival is just 12.7%.
About AI Therapeutics
AI Therapeutics was founded by Dr. Jonathan Rothberg, serial entrepreneur and Recipient of the National Medal of Technology and Innovation for inventing highspeed “Next-Gen” DNA sequencing, with the goal of utilizing artificial intelligence to accelerate the clinical development of drugs for rare diseases. The company is building out an expansive rare disease pipeline with the help of its Guardian AngelTM Platform, a suite of artificial intelligence tools that use deep learning to understand complex disease biology and the action of potential new therapeutics. To learn more, visit: AI-Therapeutics.com.
AI Therapeutics is part of biotechnology accelerator 4Catalyzer, which has launched Butterfly Network, Quantum-Si, Hyperfine and Liminal Sciences, Detect, Tesseract, and Protein Evolution. All of the 4Catalyzer companies strive to transform 21st century medicine and improve our planet by solving today’s most challenging problems across life science research tools, medical devices, therapeutics and the environment. To learn more, visit: 4Catalyzer.com.