Phase 2 Trial of NLY01 in Slowing Disease Progression Fully Enrolled

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by Steve Bryson, PhD |

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Enrollment is complete in the Phase 2 study evaluating Neuraly‘s NLY01, an investigational disease-modifying agent designed to slow or stop Parkinson’s disease progression by protecting nerve cells and limiting neuroinflammation.

Top-line results from the trial, which enrolled 255 people with early Parkinson’s at 60 sites in the U.S. and Canada, are expected in the first half of 2023.

“We are pleased to announce the completion of enrollment in this important study,” Seulki Lee, PhD, president and CEO of Neuraly, said in a press release. “Based on the novel mechanism of action of NLY01 in neurodegenerative diseases, we believe the compound has the potential to represent a breakthrough in the treatment of Parkinson’s disease by offering the ability to slow, or even halt, the progression of the disease.”

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In Parkinson’s, inflammatory changes in the brain trigger the death of nerve cells (neurons) that release dopamine, a molecule that sends messages between neurons. A lack of dopamine leads to the disease’s characteristic motor and non-motor symptoms.

NLY01 is designed to suppress the activation of two types of cells — microglia and toxic astrocytes — that participate in the initial inflammatory response that triggers cell death. The therapy, which is similar to approved diabetic medications, is also being investigated in Alzheimer’s disease.

A variety of treatments help to ease Parkinson’s symptoms and improve quality of life by increasing the amount and/or activity of dopamine in the brain, but their effectiveness wanes with long-term use.

“Current therapies are severely limited by their ability to only treat symptoms of the disease and do so in a transient manner,” said Viktor Roschke, PhD, chief scientific officer of Neuraly. “By specifically inhibiting microglial activation and the formation of neurotoxic astroglial cells that are known to trigger neuroinflammation and neuronal cell death, we believe that NLY01 has the potential to provide these patients with the type of disease-modifying therapy that can truly make a meaningful difference in their lives.”

In animal models, NLY01 was found to prevent nerve cell death, which slowed disease progression, aided the animals’ motor and cognitive functions, and extended their lifespan.

A previous Phase 1 clinical trial (NCT03672604), in which NLY01 was administered weekly as an under-the-skin (subcutaneous) injection, demonstrated the treatment was well-tolerated and long-lasting in 96 healthy adults.

The ongoing Phase 2 trial (NCT04154072), dubbed NLY01-PD-1, is assessing the safety, efficacy, and tolerability of NLY01 in early-stage Parkinson’s patients, ages 30–80, not regularly using common disease therapies. Participants were randomly assigned to either weekly injections of NLY01 (2.5 mg or 5.0 mg) or a placebo for 36 weeks (nine months).

Researchers will evaluate NLY01’s efficacy by looking at changes in treated patients, compared to placebo and scores at the study’s start, using parts 2 and 3 of the Unified Parkinson’s Disease Rating Scale, a standardized tool to measure disease motor symptoms and how they affect daily life activities.

“NLY01 has been well-tolerated, and patients have managed the injections without difficulty,” said Andrew Siderowf, MD, a professor of neurology and chief of the movement disorders division at the University of Pennsylvania’s Perelman School of Medicine. “I am looking forward to the completion of the study, which evaluates a treatment that addresses a potential patho-mechanism in [the disease].”

Patient dosing in this Phase 2 trial began in March 2020.