AI Therapeutics Announces Acquisition of New Cancer

GUILFORD, Conn., April 25, 2022 (GLOBE NEWSWIRE) — AI Therapeutics, AI-focused clinical-stage biopharmaceutical company developing novel therapies for rare diseases, today announced the acquisition of EntreChem, SL, investigational drug candidate EC-8042 (renamed AIT-102), a new targeted cancer therapy in development for rare pediatric cancers and other cancers.

Despite recent advances in understanding tumor biology, treatment for many childhood cancers remains limited to surgery, radiotherapy 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’s sarcoma, and has the potential to treat a broader family of tumors that share a common mechanism of mutated or deregulated SWI/SNF activity.

“As a physician who treats children with cancer daily, I am acutely aware of the need for more effective and less toxic therapies,” said Patrick Grohar, MD, Ph.D., director of translational research at 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’s sarcoma, and believe that AIT-102 has the potential to transform the treatment of these cancers, as well than many others.

“AIT-102 is a powerful addition to AI Therapeutics’ rare disease treatment pipeline,” said Brigette Roberts, MD, Chief Executive Officer of AI Therapeutics. “Cancer is the leading cause of death by disease in children in the United States today. With AIT-102, we hope to change those statistics. Beyond pediatrics, AIT-102 has the potential to treat up to 20% of cancers with impaired SWI/SNF activity, either by mutation or dysregulation, including epithelioid sarcoma, synovial sarcoma, lung adenocarcinoma, colorectal cancer and others. opportunity to propose a promising new agent to help combat these tumours.

Financial terms of the transaction were not disclosed.

About SWI/SNF and AIT-102

SWI/SNF is a protein complex involved in wrapping and unwinding 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 SWI/SNF activity in certain tumors where SWI/SNF is mutated (such as rhabdoid tumors) and in other tumors where SWI/SNF associates with transcription factors oncogenes (such as EWS-FLI in Ewing’s 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 of SMARCB1, a tumor suppressor gene encoding a member of the SWI/SNF remodeling complex. There is currently no widely accepted standard of effective treatment for the treatment of rhabdoid tumors. Patients have an average four-year survival of 10 to 40%.

About Ewing’s Sarcoma

Ewing’s sarcoma is a rare, aggressive malignant bone tumor most commonly diagnosed in adolescents and young adults between the ages of 10 and 20. In 85% of cases, Ewing’s sarcoma is caused by a translocation between chromosomes 11 and 22 that creates a fused oncogenic transcription factor EWS-FLI. The standard treatment for Ewing’s sarcoma includes the five VDC/IE chemotherapy regimen with surgery and/or radiotherapy. These therapies are associated with significant short- and long-term side effects. The three-year survival rate for patients diagnosed with metastatic Ewing’s sarcoma is approximately 30%. For recurrent Ewing’s sarcoma, progression-free survival at 6 months is only 12.7%.

About AI Therapeutics

AI Therapeutics was founded by Dr. Jonathan Rothberg, serial entrepreneur and National Medal of Technology and Innovation recipient for inventing high-speed “next-generation” DNA sequencing, with the goal of using artificial intelligence to accelerate the clinical development of drugs for rare diseases. diseases. The company is developing a broad portfolio of rare diseases using its Guardian Angel™ platform, a suite of artificial intelligence tools that use deep learning to understand the complex biology of diseases and the action of new therapies potential. To learn more, visit:

About 4Catalyser

AI Therapeutics is part of the 4Catalyzer biotechnology accelerator, which launched Butterfly Network, Quantum-Si, Hyperfine and Liminal Sciences, Detect, Tesseract and Protein Evolution. All 4Catalyzer companies are striving to transform 21st century medicine and improve our planet by solving today’s toughest problems across life science research tools, medical devices, therapeutics and the environment. To learn more, visit:


Irene B. Bowles