Parkinson’s clinical trial of ALX-001 DMT to start by month’s end

Phase 1a study to test safety, efficacy of Allyx disease-modifying therapy

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by Andrea Lobo |

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Allyx Therapeutics said it will begin a clinical trial by the end of the month to test its lead candidate ALX-001, a disease-modifying therapy for neurodegenerative conditions, in people with Parkinson’s disease.

The trial will test the treatment’s safety when administered for 28 days.

The U.S. Food and Drug Administration (FDA) accepted the company’s investigational new drug application for ALX-001 based on top-line data from a Phase 1a trial (NCT04805983) in Alzheimer’s disease patients. That cleared the way for the clinical trial involving Parkinson’s patients to start.

The previous Phase 1a trial evaluated the safety, tolerability, and pharmacological properties of single, increasing doses of ALX-001 ranging from 10 mg to 200 mg in healthy, older adults. The results, presented at the 16th Clinical Trials on Alzheimer’s Disease last year, showed that the treatment was well tolerated at all doses tested — including at levels that can reach full interaction with its brain target.

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Seeking ‘meaningful benefits’ for people living with disease

Stephen Bloch, MD, Allyx Therapeutics’ co-founder and CEO, said the company is trying to advance ALX-001 for use in patients with both progressive conditions.

“We are working with urgency to understand how the unique mechanism of action of ALX-001 at mGluR5, which preserves and protects synapses, can introduce meaningful clinical benefits for people living with Alzheimer’s disease and Parkinson’s disease,” Bloch said in a company press release.

“Our aim is to deliver the first-ever disease-modifying small molecule for these and other neurodegenerative diseases,” Bloch said.

Formerly known as BMS-984923, ALX-001 is an oral, brain-penetrant small molecule that blocks a receptor called mGluR5 in the brain. The investigational molecule is a highly selective, disease-modifying therapy targeting synapses. Synapses are tiny junctions between nerve cells (neurons) in the brain that allow neurons to communicate with each other by transmitting electrical or chemical signals.

ALX-001 can selectively inhibit the disease-associated receptor activation while preserving the normal signaling needed for normal brain activity.

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Parkinson’s clinical trials parallel Alzheimer’s studies

As patient studies in Parkinson’s disease begin, the company will continue to advance the clinical development of ALX-001 for Alzheimer’s disease. In fact, the first part of a Phase 1b multiple ascending dose trial (NCT05804383), which compared the treatment against a placebo in healthy volunteers, recently was completed. Data will be presented at a conference on neurological disorders being held this week in Portugal and virtually.

This trial is testing the therapy in people with early Alzheimer’s.

The ALX-001 program has received more than $20 million in funding from sources including the National Institutes of Health, the Small Business Innovation Research program, and The Michael J. Fox Foundation for Parkinson’s Research.

“We have the talent and the capital needed to rapidly advance our clinical development program and  progress toward transforming the future for people living with serious and debilitating neurodegenerative diseases,” said Timothy Siegert, PhD, co-founder and chief operating officer of Allyx Therapeutics.

The molecule was originally identified by Bristol Myers Squibb. Allyx scientific founder Stephen Strittmatter, PhD, discovered the mechanism of action of ALX-001 for neurodegenerative diseases at Yale University. An exclusive worldwide license for the therapy’s use was obtained by Allyx.