Pfizer-Peter Mac partnership unlocks new drug combination in leukaemia

A novel drug combination could significantly improve outcomes for children and adults with hard-to-treat subtypes of acute myeloid leukaemia (AML).

The approach pairs Revuforj (revumenib), a first-in-class menin inhibitor already approved for use in AML, and CTx-648 (PF-9363), an investigational KAT6A/B inhibitor currently undergoing trials in estrogen receptor-positive (ER+) breast cancer.

Professor Mark Dawson, Haematologist at Peter MacCallum Cancer Centre, said “In the clinic we often find that people treated with menin inhibitors initially respond well but the treatment stops being effective after some time and the cancer returns.”

To address this limitation, the research team collaborated with Pfizer to explore new drug candidates.

“Our lab had previously identified KAT7 as an enzyme essential for leukaemia cells to grow and survive so we worked in collaboration with Pfizer, to characterise new drugs that are effective at blocking KAT6/7,” said Professor Dawson.

“Importantly, we showed that combining this new drug with menin inhibitors, which are already in the clinic, markedly increases the anti-cancer activity against the most common cause of leukaemia in children,” he said.

Peter Mac PhD student, Shellaina Gordon, stated “This discovery could pave the way for more effective, targeted therapies and excitingly, these findings can be rapidly translated to the clinic. With menin inhibitors already available and drugs targeting KAT7 in development, it is feasible to have a clinical trial in AML patients combining these two treatments soon.”

Momentum for Revuforj continues to build, following last month’s U.S. Food and Drug Administration (FDA) approval for treating relapsed or refractory (R/R) mutant NPM1 AML.

“In our preclinical animal models, we were able to show increased survival using combination treatment, and we also showed that this combination can overcome resistance to menin inhibitors,” said Professor Dawson.

The next step, he noted, is to investigate whether combining Revuforj with CTx-648 can deliver a longer-lasting and more durable response in AML patients.

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