Gaithersburg’s Neuraly Announces Completion of Enrollment in Phase 2 Clinical Trial of NLY01 in Patients with Parkinson’s Disease

Earlier this week Neuraly, headquartered at 704 Quince Orchard Rd.,  announced the completion of patient enrollment in its Phase 2 study of NLY01 for Parkinson’s disease (PD). NLY01, Neuraly’s lead development asset, is a glucagon-like peptide 1 receptor (GLP-1R) agonist that has been demonstrated to inhibit pathological activation of microglia by binding upregulated GLP-1R in animal models of neurodegenerative diseases such as PD. In doing so, NLY01 has demonstrated the ability to inhibit neuroinflammation and prevent neuronal cell death, offering a potentially first-of-its-kind, disease-modifying therapy capable of slowing the progression of PD.

Per the press release:

The Phase 2 study has enrolled 255 patients across 60 clinical sites in the United States and Canada. The randomized, double-blind, placebo-controlled trial is designed to assess the safety, tolerability, and efficacy of NLY01 in subjects with early PD. Enrolled patients have been randomized to receive either weekly injections of NLY01 (2.5 mg or 5.0 mg) or placebo for 36 weeks. Following 36 weeks of treatment, investigators will evaluate the impact of treatment with NLY01, as compared to placebo, using the Unified Parkinson’s Disease Rating Scale assessment (MDS-UPDRS). Top-line study results are expected in the first half of 2023.

“We are pleased to announce the completion of enrollment in this important study as it moves us another step closer to the highly anticipated readout of data from the trial. 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. We are eager to see if the findings of this large Phase 2 study validate that belief and provide support for the ongoing advancement of NLY01 as a potentially disease-modifying agent for Parkinson’s patients,” said Seulki Lee, Ph.D., president and chief executive officer of Neuraly.

“NLY01 has been well-tolerated, and patients have managed the injections without difficulty,” said Andrew Siderowf, M.D., MSCE, Hurtig-Stern professor of neurology and chief of the movement disorders division in the University of Pennsylvania Perelman School of Medicine. “With the close of enrollment for the NLY01-PD-1 trial, I am looking forward to the completion of the study, which evaluates a treatment that addresses a potential patho-mechanism in PD.”

Results from a previously completed a Phase 1 clinical trial demonstrated NLY01 to be well-tolerated and capable of providing near continuous exposure to target therapeutic levels of the drug with once-weekly dosing. In that study, the compound demonstrated an impressive pharmacokinetic profile, highlighted by a half-life of 12.5 days, which provided increased exposure to the drug as compared to currently approved GLP-1 agonists. This is particularly critical for treating conditions such as PD and Alzheimer’s disease (AD) as it allows for increased delivery of the drug into the brain. Furthermore, preclinical studies of NLY01 have consistently demonstrated impressive efficacy in models of PD, AD, multiple sclerosis, glaucoma and retinal diseases.

“Today, there remains a critical unmet need in the area of Parkinson’s disease treatment. Current therapies are severely limited by their ability to only treat symptoms of the disease and do so in a transient manner. Patients who are battling the continuous decline in function that is associated with Parkinson’s disease require new treatments that are able to prevent the neuronal cell death that drives its progression,” said Viktor Roschke, Ph.D., 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. With this in mind, we are excited to complete the ongoing Phase 2 trial and review the top-line data that are expected to be available in the first half of 2023.”

More information about the ongoing Phase 2 study of NLY01 in PD is available at www.clinicaltrials.gov under the identifier NCT04154072.

About NLY01

NLY01 is a proprietary long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor (GLP-1R) agonist. NLY01 penetrates the blood-brain barrier (BBB) in animal models and its receptor (GLP-1R) is highly expressed on glial cells. NLY01 is being developed as a disease-modifying agent for neurodegenerative disorders including Parkinson’s and Alzheimer’s disease. In clinically relevant animal models, NLY01 was found to prevent neuronal cell death by inhibition of microglial activation and formation of A1 neurotoxic astroglial cells. Treatment with NLY01 slowed disease progression, improved motor and cognitive functions and extended the lifespan in mice with Parkinson’s disease.