Novel breakthrough in hematological cancer treatment: first-in-class Wiskott-Aldrich syndrome protein activator, EG-011

The Institute of oncology research (IOR), affiliated to USI, is pleased to announce the publication of a study describing a new compound designed at IOR, targeting the Wiskott-Aldrich syndrome protein (WASp) with anti-cancer activity in various hematological cancers. The research, led by Dr Eugenio Gaudio (former IOR senior investigator) and Prof. Francesco Bertoni (IOR group leader and deputy director, USI adjunct professor), involved prominent institutions, including the Institute for research in biomedicine (IRB) and various European and North-American institutions.

Researchers at the Institute of oncology research (IOR) have announced the development of EG-011, a pioneering small molecule activator of the Wiskott-Aldrich syndrome protein (WASp), which exhibits significant anti-tumor activity in various hematological cancers. Hematological cancers, including lymphomas, leukemias, and multiple myelomas, continue to present substantial therapeutic challenges despite advancements in treatment. The novel compound EG-011 represents a possible breakthrough in targeting these cancers. Unlike traditional therapies, mechanistic studies revealed that EG-011 activates the auto-inhibited form of WASp, leading to enhanced actin polymerization and subsequent tumor cell death.

Preclinical studies have demonstrated that EG-011 effectively reduces tumor growth in lymphoma, leukemia, and multiple myeloma models, including some resistant to standard treatments like BTK, PI3K, and proteasome inhibitors. "We are excited about the potential of EG-011 as a novel therapeutic agent for patients with hematological malignancies, especially those with resistant forms of the disease", said Prof. Francesco Bertoni. "This first-in-class WASp activator, designed in our lab by Dr. Eugenio Gaudio, could pave the way for new treatment paradigms in oncology". Importantly, the IOR patent covering EG-011 has been licensed to BIMINI Biotech, a start-up company that is now collaborating with IOR to move forward EG-011 (now also known as BM-011). BIMINI Biotech is currently incubated at the USI Startup Centre.

For more information, please refer to the full article, titled "A first-in-class Wiskott-Aldrich syndrome protein activator with anti-tumor activity in hematologic cancers", publised in the Haematologica journal.

For further information, please contact:

Prof. Francesco Bertoni

Institute of oncology research

Phone: +41 58 666 7206

E-mail: [email protected]