Over $500 Million Deal: Insilico Medicine's AI-Designed KAT6 Inhibitor Marks Second Out-Licensing in Three Months

InSilico Medicine Announces AI Drug Out-Licensing Deal with Italian Multinational Pharmaceutical Company Menarini Group and Its Wholly Owned Subsidiary Stemline Therapeutics; Menarini to Acquire Global Exclusive Rights for the Development and Commercialization of InSilico Medicine’s Proprietary KAT6 Small-Molecule Inhibitor. The Total Transaction Value Exceeds $500 Million, Including a $12 Million Upfront Payment and Subsequent Development, Regulatory, and Commercial Milestones. Additionally, InSilico Medicine Is Eligible for Potential Double-Digit Royalty Rates.

This collaboration marks another out-licensing of InSilico Medicine’s internally developed pipeline, following the September 2023 agreement in which the company licensed its USP1 small-molecule inhibitor to Nasdaq-listed Exelixis for an upfront payment of $80 million. Compared with the USP1 program, which has entered Phase I clinical trials in both China and the United States, the KAT6 project covered by this new partnership is at an earlier stage. In December 2022, InSilico Medicine nominated ISM5043 as the preclinical candidate compound for this project. The program remains in the preclinical translational medicine research phase, including indication exploration and combination therapy development supported by PandaOmics.

ISM5043 is an oral, highly selective small-molecule inhibitor targeting KAT6, with potential for the treatment of ER+/HER2- breast cancer and other malignancies. This subtype represents a substantial patient population, accounting for approximately 70% of all breast cancer cases. In preclinical studies, ISM5043 demonstrated potent inhibition of KAT6A in multiple cell line-derived xenograft (CDX) and patient-derived xenograft (PDX) models, exhibiting remarkable efficacy and a favorable safety profile. InSilico Medicine presented the latest data from this innovative program at the 2023 San Antonio Breast Cancer Symposium (SABCS).

Dr. Alex Zhavoronkov, Founder and CEO of InSilico Medicine“The successful out-licensing of our in-house AI-discovered drug once again validates InSilico Medicine’s unique business model. This KAT6 small-molecule inhibitor, currently under development, holds promise for providing new clinical treatment options for breast cancer patients. Stemline, a Menarini company, with its forward-looking vision in oncology and deep understanding of transformative therapies, is the ideal partner to bring ISM5043 into clinical trials and complete its clinical development.”

Stemline Therapeutics, a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on the development and commercialization of innovative oncology therapies, and has achieved commercial availability of ORSERDU in the United States and the European Union.^®(elacestrant) commercialization. It is an oral endocrine therapy approved for postmenopausal women or adult men with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer who have experienced disease progression following at least one prior line of endocrine therapy. This collaboration may help pave the way for the clinical combination of KAT6 inhibitors with elacestrant.

Elcin Barker Ergun, CEO of Menarini Groupstated, “InSilico Medicine is a global leader in the field of AI-driven drug discovery, and we are delighted to have entered into this collaboration. We aim to explore a promising avenue for innovation and launch transformative anti-cancer therapies. Our goal is to further improve clinical outcomes for patients, and KAT6-targeted candidate drugs hold the potential to achieve this in breast cancer and other oncology indications.”