Disarm Therapeutics Receives Key Funding for ALS Therapy Development

Research into the development of new therapies to treat several traumatic, inflammatory, and neurodegenerative diseases including amyotrophic lateral sclerosis (ALS) received an important financial boost with the announcement by Disarm Therapeutics that it will receive $30 million in financing to develop treatments for axonal degeneration.

Disarm was founded in 2016 as a joint effort by venture capital firm Atlas Venture, and Jeffrey Milbrandt, MD, PhD, and Aaron DiAntonio, MD, PhD, of Washington University in St. Louis.

The $30 million comes from a Series A financing round, which is part of an early development stage for start-up companies that are raising capital. The round was led by Atlas Venture, with co-investors including Lightstone Ventures and AbbVie Ventures.

Axonal degeneration is involved in several traumatic, inflammatory, and neurodegenerative diseases. The process occurs early in a disease’s course, which means there is great potential for therapeutic strategies. Axonal degeneration is seen in chronic and acute diseases of the central, ocular, and peripheral nervous systems, and ultimately leads to disability and disease progression.

Disarm’s approach focuses on inhibiting the SARM1 protein, which recently was found by the company’s scientific founders to promote axonal degeneration.

In a study published in the journal Neuron, scientists “demonstrated that SARM1 itself is the central driver of axonal degeneration,” and its enzymatic activity makes it possible to target with new therapeutics.

The study was titled, “The SARM1 Toll/Interleukin-1 Receptor Domain Possesses Intrinsic NAD⁺ Cleavage Activity that Promotes Pathological Axonal Degeneration.”

In a series of experiments using injury models in neurons, the researchers observed that a specific portion of SARM1 is able to deplete axonal nicotinamide adenine dinucleotide (NAD⁺, an enzyme found in all living cells), a hallmark of degeneration.

“Solving the puzzle of axonal degeneration has challenged scientists and physicians for more than a century,” Jason Rhodes, a partner at Atlas Venture and co-founder, chairman, and acting CEO of DisarmTherapeutics, said in a press release. “Now, for the first time, we have a therapeutic approach to directly prevent the loss of axons in patients with diseases including multiple sclerosis, ALS, glaucoma, and peripheral neuropathies.”

Disarm has licensed exclusive rights to SARM1 findings from Washington University in St. Louis. This strategy could be the basis of breakthrough treatments for patients with neurological diseases, the company believes.

“Using our proprietary product engine, the Disarm team has discovered novel, potent SARM1 inhibitors,” said Rajesh Devraj, PhD, co-founder and CSO of Disarm Therapeutics. “We plan to translate these potential therapeutics to human proof of concept in a range of neurological diseases, supported by non-invasive biomarkers.”