The Menarini Group, an international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc., a wholly-owned subsidiary of the Menarini Group, along with clinical stage generative artificial intelligence (AI)-driven biotechnology company Insilico Medicine, entered into an exclusive licensing agreement granting Stemline the global rights to develop and commercialize a novel, small molecule KAT6A inhibitor designed using Insilico’s AI platform, as a potential treatment for hormone sensitive cancers and other oncology indications.

Stemline will provide a $12 million upfront payment to Insilico. The combined deal, including all development, regulatory, and commercial milestones, is valued at $500 million.

KAT6A is known to play an important role in several cancers. Overexpression of KAT6A correlates with poor clinical outcomes in patients with ER+/HER2- breast cancer, the most common subtype of breast cancer.  In preclinical studies, the molecule has demonstrated potent inhibition against KAT6A in multiple CDX and PDX models with good efficacy and safety.

The novel molecule was designed by Insilico with the help of its Pharma Generative AI platform to inhibit KAT6A and block endocrine receptor (ER) at the transcriptional level, giving it the potential to overcome resistance to endocrine therapies due to mutation or ligand-independent constitutive activation of ER. Currently, endocrine therapy in combination with CDK4/6 inhibitors is the standard treatment for ER+/HER2- breast cancer patients with advanced or metastatic disease. Novel combinations with CDK4/6 inhibitors and/or new oral SERDs are needed to further extend outcomes.

“We are delighted to enter a collaboration with Insilico that harnesses the power of generative AI as a leader in the field, to explore a promising new treatment approach and potentially unlock transformative new cancer therapies,” said Elcin Barker Ergun, CEO of the Menarini Group. “Having brought the first innovation in endocrine therapy after almost 20 years to the U.S. and Europe with elacestrant for ER+, HER2- breast cancer patients, our aim is to further augment patient outcomes, and targeting KAT6A can potentially serve that in breast cancer and beyond.”

Alex Zhavoronkov, founder, and co-CEO of Insilico Medicine said, “We are excited by the promise of our latest generative AI-designed therapy to provide a new potential treatment option for breast cancer patients. With their innovative vision and deep focus in bringing transformational therapeutics in oncology, Stemline is the ideal partner to lead this molecule into development and through clinical trials.”