New £1.2 million funding to advance development of investigational MND drug

Researchers from the University of Sheffield and early-stage biopharmaceutical company Aclipse Therapeutics have been awarded £1.2 million to support the development of an investigational motor neurone disease (MND) drug.

The funding has been awarded from the Congressionally Directed Medical Research Programs (CDMRP) at the United States Department of Defense’s (DOD) U.S. Army Medical Research and Development Command (USAMRDC), as part of its FY21 Amyotrophic Lateral Sclerosis Research Program (ALSRP) Therapeutic Development Award.

The award will fund research conducted at the Sheffield Institute for Translational Neuroscience (SITraN) to further develop M102 – a potentially disease-modifying drug candidate that has shown promise in impeding the progression of MND – also known as Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease.

MND is highly relevant to the health of military service members and veterans. Since September 2001, more U.S. veterans have died from ALS (an estimated 9,500 to 10,000 deaths) than from U.S. combat deaths in Iraq and Afghanistan combined. Currently, there is no cure for the disease, and there are limited treatments to slow its progression.

Aclipse is taking a dual approach for the treatment of MND. M102 activates the NRF2 (nuclear factor erythroid 2-related factor 2) and HSF1 (Heat shock factor 1) signalling pathways, which are understood to impact MND pathophysiology. Based on preclinical results to date, M102 is expected to be mechanistically superior to currently available drugs and may lead to significant slowing of disease progression in patients with both familial and sporadic MND.

Professor Dame Pamela Shaw, Director of SITraN and primary contributor to M102’s development programme, said: “This development funding is wonderful news for MND/ALS patients who are in urgent need of effective therapies to address this life-threatening neurodegenerative disease.

“Along with my SITraN colleagues and co-principal investigators, Professor Laura Ferraiuolo and Dr Richard Mead, we spearheaded the MND/ALS biology research that led to the identification and development of M102, including the discovery of a potential precision medicine approach for its use.

“This funding will allow our team to expand development of precision medicine research and drive M102 into a Phase 1 trial. We are very excited and appreciative of the funding support awarded.”

Raymond K. Houck, Chief Executive Officer of Aclipse Therapeutics, said: “I believe that the award reflects the DOD’s appreciation of the significance of ALS to the military and their belief in our unique M102 drug candidate and approach to treating ALS patients. We are very grateful for their support.”

Dr Ning Shan, Chief Scientific Officer of Aclipse Therapeutics and the award’s principal investigator, said: “This latest award, combined with ongoing financial backing from Australia’s FightMND and the United Kingdom’s Medical Research Council, is allowing our team to accelerate the development of M102 toward first-in-human clinical studies, which is expected to initiate in 2023, and further validates M102’s novel biology and our precision medicine approach.”

This work was supported by the Office of the Assistant Secretary of Defense for Health Affairs endorsed by the Department of Defense, through the Amyotrophic Lateral Sclerosis Research Program, in the amount of $1,475,101, under Award No. W81XWH2210175. Opinions, interpretations, conclusions and recommendations are those of the author and are not necessarily endorsed by the Department of Defense.

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