Therapeutics Discovery division advances novel small-molecule to clinic

In Clinical Studies News by Barbara Jacoby

By: Clayton R. Boldt, Ph.D.


The Therapeutics Discovery division at MD Anderson Cancer Center has advanced a new small-molecule inhibitor of cancer metabolism to Phase I clinical trials. This new drug candidate marks the fourth novel therapeutic brought from concept to clinical trial by Therapeutics Discovery, a drug-discovery engine built within the walls of MD Anderson to eliminate the bottlenecks that hamper traditional drug development.

The drug candidate, IPN60090, targets glutaminase (GLS1), an important enzyme for metabolic energy production in the cell. The small molecule, now being developed through a collaboration with the global biopharmaceutical company Ipsen Biopharmaceuticals, was first discovered and developed by researchers in MD Anderson’s Institute for Applied Cancer Science (IACS) and Translational Research to Advance Therapeutics and Innovation in Oncology (TRACTION) platforms, both engines within Therapeutics Discovery.

The first-in-human Phase I clinical trial in patients with advanced solid tumors, led by Tim Yap, M.B.B.S., Ph.D., associate professor of Investigational Cancer Therapeutics and medical director of IACS, began in March by enrolling and treating its first patient.

“We are proud to see IPN60090 advance into clinical testing in partnership with Ipsen. This drug is the result of a significant collaborative effort to address unmet needs raised by our clinical colleagues, and it represents an innovative therapeutic opportunity for patients at MD Anderson,” says Phil Jones, Ph.D., vice president of Therapeutics Discovery and head of drug discovery for IACS.

Cancer treatment innovation

The IPN60090 program involves more than 25 Therapeutics Discovery team members across multiple platforms of oncology research. The IACS team, responsible for development of the small-molecule drug is led by medicinal chemist Mick Soth, Ph.D., institute group leader. The TRACTION team, which performed extensive translational biology work to clarify how the drug works and which patients are most likely to benefit, is led by biologist Jeffrey Kovacs, principal institute research scientist.

“Our unique approach to drug development, working with the bench at the bedside, affords us the opportunity to collaborate with MD Anderson physician-scientists, who helped guide our research and enabled us to rapidly advance this drug to the clinic,” saysTim Heffernan, Ph.D., executive director of TRACTION. “Now, through our partnership with Ipsen, we were able to further accelerate our efforts to investigate this drug in clinical trials and, hopefully, provide an effective new medicine for patients.”

Much of the development of IPN60090 was made possible through collaborative relationships with researchers in the Lung Cancer Moon Shot™ and Ovarian Cancer Moon Shot™, both part of MD Anderson’s Moon Shots Program™. The Moon Shots Program, which also supports the Therapeutics Discovery platforms, is a collaborative effort designed to accelerate the development of scientific discoveries into clinical advances that save patients’ lives.

MD Anderson and Ipsen announced their drug development partnership in 2018. Through the agreement, MD Anderson will progress IPN60090 through Phase I clinical development and Ipsen will be responsible for further global development and commercialization. MD Anderson and Ipsen also have collaborated to expand upon knowledge of the drug’s mechanism of action and possible expansion to additional indications.  (MD Anderson has implemented an Institutional Conflict of Interest Management and Monitoring Plan to manage its financial conflict of interest regarding this research. You can read more here.)

In addition to IPN60090, Therapeutics Discovery has several other therapies now in clinical trials. The first small molecule was IACS-10759, which is now in Phase I trials for patients with acute myeloid leukemia and a variety of solid tumors. Two antibodies developed by the Oncology Research for Biologics and Immunotherapy Translation (ORBIT) platform also are now in clinical development.

“The robust pipeline and rapid development within Therapeutics Discovery speaks to the value of the group’s unique model of drug discovery”, says Jones. “We continue to strive each day to bring forward life-saving small-molecules, biologics and cellular therapies, inspired by the needs of our patients. This is our singular mission here – to improve the lives of those we treat at MD Anderson.”