Data published on novel molecules for leukaemia treatment

pharmafile | April 27, 2021 | News story | Manufacturing and Production oncology 

STORM Therapeutics have published new data in Nature on targeting RNA modifying enzymes for the development of new anti-cancer therapeutics, with the expectation that their METTL3 inhibitors will be the first RNA epigenetic drug programme globally to enter clinical trials.

The paper entitled Small molecule inhibition of METTL3 as a strategy against myeloid leukaemia, discusses the company’s progress in the development of inhibitors of METTL3 as therapy treatment for acute myeloid leukaemia (AML) and solid tumours.

The published data show STORM has identified novel, potent, first-in-class inhibitors of METTL3, that are orally bioavailable and show pronounced anti-tumour efficacy in physiologically relevant, proof of concept animal models of AML, as well as solid tumours.

The paper highlights the efficacy of METTL3 small molecule inhibition as a new therapeutic strategy against AML, prolonging survival in a variety of AML models, by specifically targeting key stem cell subpopulations of AML.

Additionally, it confirms anti-tumour activity against different AML driver mutations demonstrating that targeting METTL3 is not limited by specific mutations (in contrast to other approaches such as FLT3 or IDH inhibition) and so may have a broad range of patients who might respond to this therapy.

Professor Tony Kouzarides, Founder of STORM Therapeutics and Director of the Milner Therapeutics Institute, University of Cambridge, said: “We are proud to be leading the field in development of drugs targeting RNA epigenetics and are making rapid progress.

“This paper has provided comprehensive proof of concept that targeting RNA modifying enzymes represents a promising new avenue for anti-cancer therapy.”

In October 2020, STORM’s drug candidate targeting METTL3, STC-15, was selected for development for human trials, which are expected to begin in 2022. STC-15 is an orally bioavailable, small molecule METTL3 inhibitor targeting an entirely new mechanism of action (modulation of RNA epigenetics) to treat AML and other solid and haematological cancers.

Keith Blundy, CEO of STORM Therapeutics, said: “We are excited to be leading the field having selected STC-15, STORM’s first-in-class clinical candidate targeting METTL3 for development towards first in human clinical studies in 2022, addressing AML patients refractory to chemotherapy treatment with limited other options in addition to exploring combinations with standard of care.”

The company’s work was carried out in collaboration with the University of Cambridge and Wellcome Sanger Institute, and was supported by grants from Cancer Research UK.

Kat Jenkins

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