Phase 1 Trial Opens into Potential Parkinson’s Therapy, Anle138b

A Phase 1 clinical trial of MODAG Neuroscience Solutions’ lead candidate anle138b is now underway and recruiting healthy participants at its one site in Nottingham, England, to study the compound’s safety.

The small molecule is being developed to treat multiple system atrophy (MSA), but holds the potential to be used in other diseases caused by clumping of the protein alpha-synuclein, such as Parkinson’s disease.

“With the start of this trial, we are on track to run the tests necessary to bring anle138b one step closer to patients,” Torsten Matthias, PhD, chief executive officer of MODAG, said in a press release.

Many neurodegenerative disorders involve the aggregation of misfolded (harmful) proteins in the brain. Anle138b works by binding to harmful forms of alpha-synuclein — a key protein involved in Parkinson’s disease — to clear the brain of existing clumps and to prevent new clumps, also known as Lewy bodies, from forming.

The compound has been shown to reduce the buildup of these toxic clumps and delay disease progression in models of MSA, Parkinson’s, and Alzheimer’s disease. Moreover, anle138b was found to reverse Parkinson’s-related motor symptoms in mice models of the neurodegenerative disorder.

This Phase 1 study’s primary goal is to evaluate the safety and tolerability of oral anle138b in healthy volunteers. Secondary objectives include dose-finding evaluations and assessments of its pharmacokinetics  — essentially how a compound is absorbed, distributed, metabolized, and excreted by the body.

Recruitment is ongoing and being overseen by Quotient Sciences in Nottingham.

“Anle138b has the potential to become a tangible treatment option to stop MSA, a highly underserved indication, in its tracks. MSA patients are severely impacted by progressing movement, balance and autonomic function impairments and as with many neurodegenerative diseases, there are no disease-halting treatment options available,” said Johannes Levin, MD, chief medical officer of MODAG.

“If successful, the Phase 1 trial also opens the opportunity for MODAG to investigate anle138b in other Parkinsonian disorders and Parkinson’s itself,” Levin added.

The biotech company has already secured a worldwide patent for anle138b.