Press releases

STORM Therapeutics publishes data in Nature showing its first-in-class inhibitor of METTL3 is effective as a new therapeutic strategy against AML

26 April 2021, Cambridge, UK: STORM Therapeutics, the biotechnology company focused on the discovery of small molecule therapies modulating RNA epigenetics, today announces that it has published a scientific paper in the internationally recognised scientific journal Nature.

The paper entitled Small molecule inhibition of METTL3 as a strategy against myeloid leukaemiareveals findings that further establish and validate STORM’s ground-breaking work on targeting RNA modifying enzymes for the development of new anti-cancer therapeutics and describes recent progress made with the METTL3 inhibitor programme.

STORM has identified novel, potent and selective 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 Acute Myeloid Leukaemia (AML), as well as solid tumours. The paper demonstrates that METTL3 small molecule inhibition is effective 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: “At STORM 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 and confirms the findings in our 2017 Nature paper made using genetic approaches.”

In October 2020, STORM announced that STC-15, its first-in-class drug candidate targeting METTL3, had been selected for development towards first in human clinical studies 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. This publication utilises STM2457, an earlier compound than STC-15.

Keith Blundy, CEO of STORM Therapeutics, said: “I am delighted to see the publication of our ground-breaking research on STM2457 in a world leading journal. 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.”

STORM’s work was carried out in collaboration with the University of Cambridge (Gurdon and Milner Institutes) and Wellcome Sanger Institute, and was supported by grants from Cancer Research UK.

For further information

STORM Therapeutics Ltd
Keith Blundy
T: +44 (0)1223 804174

Optimum Strategic Communications
Mary Clark, Shabnam Bashir
Tel: +44 (0)203 950 9144


STORM Therapeutics
STORM Therapeutics, founded in 2015, is a University of Cambridge spin-out translating the ground-breaking work of Professors Tony Kouzarides and Eric Miska in RNA epigenetics into the discovery of first-in-class drugs in oncology and other diseases. Storm is the leading company tackling disease through modulating RNA modifying enzymes and is developing a unique platform and pipeline to address these enzyme classes, including RNA methyltransferases.

STORM is backed by blue chip investors Cambridge Innovation Capital, M Ventures, Pfizer Ventures, Taiho Ventures LLC, Seroba Life Sciences and IP Group, who share the team’s ambitions to build a world-leading company in the field.

The Wellcome Sanger Institute
The Wellcome Sanger Institute is a world leading genomics research centre. We undertake large-scale research that forms the foundations of knowledge in biology and medicine. We are open and collaborative; our data, results, tools and technologies are shared across the globe to advance science. Our ambition is vast – we take on projects that are not possible anywhere else. We use the power of genome sequencing to understand and harness the information in DNA. Funded by Wellcome, we have the freedom and support to push the boundaries of genomics. Our findings are used to improve health and to understand life on Earth. Find out more at or follow us on Twitter, Facebook, LinkedIn and on our Blog.

About the University of Cambridge
The mission of the University of Cambridge is to contribute to society through the pursuit of education, learning and research at the highest international levels of excellence. To date, 110 affiliates of the University have won the Nobel Prize.

Founded in 1209, the University comprises 31 autonomous Colleges and 150 departments, faculties and institutions. Cambridge is a global university. Its 19,000 student body includes 3,700 international students from 120 countries. Cambridge researchers collaborate with colleagues worldwide, and the University has established larger-scale partnerships in Asia, Africa and America.

The University sits at the heart of the ‘Cambridge cluster’, which employs more than 61,000 people and has in excess of £15 billion in turnover generated annually by the 5,000 knowledge-intensive firms in and around the city. The city publishes 316 patents per 100,000 residents.

About Cancer Research UK

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