$2.2M Grant Aims to Move M102, Potential ALS Therapy, Into Trials

Work into moving M102, an investigative treatment for amyotrophic lateral sclerosis (ALS), into testing in people has been given a boost by a £1.6 million research grant awarded to Aclipse Therapeutics and the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield in the U.K.

The award, worth roughly $2.2 million, was given by the U.K.’s Medical Research Council (MRC), and adds to a recent $700,000 grant from FightMND, a nonprofit organization in Australia that supports research into motor neuron diseases (MND), such as ALS.

“This development funding from MRC is wonderful news for ALS/MND patients who are in dire need of an effective therapy to address this life-threatening neurodegenerative disease,” Pamela Shaw, MD, director of SITraN and a primary contributor to M102’s development, said in a press release.

“We spearheaded the ALS/MND biology research that led to the development of M102, including the discovery of a potential precision medicine approach for M102 in ALS/MND, so we are very appreciative of MRC’s funding support,” Shaw added.

M102, discovered by SITraN scientists, is a potentially disease-modifying therapy that has been shown to slow ALS disease progression in a range of preclinical models. 

The medicine activates two signaling pathways — NRF2 and HSF1 — known to impact the development and progression of ALS, a neurodegenerative disease that affects nerve cells in the brain and spinal cord that control muscle movement.

The NRF2 pathway mediates oxidative stress, which refers to an imbalance between the production of toxic free radicals by metabolic processes and their clearance, while the HSF1 pathway helps in protein folding, which counteracts the formation of protein clumps. Both oxidative stress and protein clumping contribute to disease progression in ALS.

M102 is also reported to lower inflammation, improve the working of mitochondria (the powerhouses of cells), and to help clear already formed protein clumps. Due to its action on a broad range of disease mechanisms, M102 is expected to be superior to available therapies, and significantly slow disease progression in both familial and sporadic ALS, the company said. 

Focusing on a personalized medicine approach, the MRC grant will support additional research into patient biomarkers that will be applied in clinical trials, with the goal of identifying those ALS patients most likely to benefit from treatment.

“We greatly appreciate the support from MRC for our novel and broad multi-disease patho-mechanism approach to treating ALS patients,” said Raymond K. Houck, CEO of Aclipse. “The MRC award, coupled with our recent FightMND grant award, accelerates M102’s development into its first-in-human clinical studies and validates M102’s biology and potential for a precision medicine approach for the treatment of ALS.”

The therapy, given its dual mechanism of action, might treat other neurodegenerative diseases as well.

“The research funding from these programs will be key as they will support … the regulatory filings for first-in-human studies,” Houck said. “Importantly, M102 may have applications in a wide array of conditions associated with impaired neuronal function such as Friedreich’s ataxia, Huntington’s disease, and Parkinson’s disease.”