$1 million grant supports trial of experimental treatment for ALS

The ALS Association has awarded Mitochon Pharmaceuticals a $1 million grant to support a Phase 2 clinical trial testing the company’s experimental therapy MP-101 in people with amyotrophic lateral sclerosis (ALS).

The study is still in the planning stages and is expected to kick off next year.

“We are honored to receive such a significant award,” Robert Alonso, CEO of Mitochon, said in a company press release. “It is a great endorsement of the idea of using a mitochondrial uncoupler as a treatment for ALS and will help fund the Phase II study planned for early 2027.”

Mitochon developing therapy as potential ALS, MS treatment

ALS is a neurological disease marked by the dysfunction and death of motor neurons, the nerve cells that control movement. The causes of ALS are not fully understood, but a large body of data suggests that the disease may be driven in part by problems with mitochondria.

Mitochondria, known as the powerhouses of the cell, are small structures that produce much of the energy cells need to function. Motor neurons require a huge amount of energy for the busy work of sending electrical signals, so problems with mitochondria can cause substantial dysfunction of these nerve cells.

MP-101 is designed to improve mitochondrial function and support energy production, thus protecting motor neurons from damage.

The investigational therapy contains 2,4-dinitrophenol (DNP), a compound that was used as a weight loss drug in the 1930s. However, the high doses used for this purpose were toxic, and the medication was pulled from the market.

By funding programs at this critical stage, we are striving to accelerate the development of therapeutic candidates that can help make ALS a livable disease.

MP-101 uses a very low dose of DNP that may have neuroprotective effects to help prevent the stress overload that can cause mitochondrial dysfunction in ALS.

Mitochon is developing the therapy as a potential treatment for ALS and other neurological conditions where problems with mitochondria may play a role in disease biology, including Huntington’s disease and multiple sclerosis.

“ALS is a truly insidious disease that typically strikes people in the prime of their life,” said John Geisler, PhD, chief scientific officer and co-founder of Mitochon. “Appearing rooted in mitochondrial dysfunction, ALS has been top of our priorities for many years.”

In animal models of ALS, daily oral treatment with MP-101 has been shown to improve motor function and preserve the connections between motor neurons and muscle cells.

According to Mitochon, recent clinical studies in Europe showed that MP-101 led to a rapid decrease in levels of the nerve damage biomarker neurofilament light in people with sporadic ALS.

The ALS Association is funding further development of the investigational therapy via a Hoffman ALS Clinical Trial Award, a program that supports early clinical research into potential ALS treatments.

“We are pleased to support the continued evaluation of MP-101 through our Hoffman ALS Clinical Trial Awards,” said Kuldip Dave, PhD, chief scientist at the ALS Association. “By funding programs at this critical stage, we are striving to accelerate the development of therapeutic candidates that can help make ALS a livable disease.”