D&D Pharmatech receives FDA nod for phase 2 GLP-1R agonist trial for multiple sclerosis

D&D Pharmatech, a Korean drugmaker, has received U.S. FDA approval to proceed with a phase 2 clinical trial of its experimental drug, NLY01, for multiple sclerosis (MS). 

Following encouraging results from an earlier trial in Parkinson’s disease, the upcoming study, led by researchers at Baltimore-based Johns Hopkins University, will evaluate the drug’s potential to slow neurodegeneration in MS patients. 

The Korean biotech company, which focuses on the development of glucagon-like peptide-1 receptor (GLP-1R) agonists—drugs that mimic glucose-regulating hormones—said on Tuesday that the FDA approved its investigational new drug (IND) application for this trial. NLY01 had previously completed a phase 2 trial for Parkinson’s disease in the U.S. and North America. 

The upcoming phase 2 trial for MS will be led by Professor Ellen Mowry's research team at the Johns Hopkins University School of Medicine’s Multiple Sclerosis Center. This investigator-initiated trial will use NLY01 supplied by D&D Pharmatech, comparing it with a placebo, but the company will not incur any additional costs related to the study.

“The primary objective of this trial is to assess whether NLY01 is associated with a reduction in imaging biomarkers related to neurodegeneration in MS patients,” a D&D Pharmatech official said.

The main outcome measure will be the change in cerebral volume, a standard imaging metric in MS phase 2 trials.

MS is a degenerative brain disease that strikes at the prime of life, typically targeting those between 20 and 40. As autoimmune inflammation and neurodegenerative damage take hold, the disease strips away the protective myelin sheath surrounding nerve fibers in the central nervous system—the brain, spinal cord, and optic nerves—leading to a downward spiral of repeated neuroinflammatory attacks. Each episode leaves the central nervous system more damaged, piling on disabilities and eroding the patient’s quality of life.

NLY01, a GLP-1R agonist designed to inhibit neuroinflammation—a key driver of degenerative brain diseases—has demonstrated promising results in a recent phase 2 clinical trial conducted across the U.S. and North America.

Involving 255 patients, the trial showed its potential as a disease-modifying treatment, particularly for Parkinson’s patients under 60.

In 2021, a research team led by Professor Peter Arthur Calabresi, Director of the Multiple Sclerosis Center at Johns Hopkins University School of Medicine, published findings in the international journal Neurotherapeutics (Impact Factor: 6.9) indicating that NLY01 significantly slowed the progression of MS in animal models by reducing neuroinflammation and protecting nerve cells. 

D&D Pharmatech's newly approved phase 2 clinical trial for MS will involve 240 patients and be conducted over 96 weeks. 

The trial will employ a randomized, double-blind, parallel-group design. Given the excellent therapeutic effects observed in younger patients under 60 during the phase 2 trial for Parkinson's disease, a D&D Pharmatech official said, this study will focus on MS patients aged 18 to 60, raising expectations for positive outcomes.

D&D Pharmatech CEO Lee Seul-ki added that the FDA's approval for the phase 2 trial of NLY01 in MS reflects the “notable disease-modifying effects we observed in younger patients during our phase 2 Parkinson's trial,” which was built on years of preclinical collaboration with Johns Hopkins University School of Medicine. 

The company is currently working with the UK-based nonprofit Cure Parkinson's on research into the therapeutic effects of multiple GLP-1R agonists, including NLY01, in young Parkinson's patients. Based on these results, Lee said the goal of her company is to initiate global licensing efforts to advance the development of Parkinson's treatments.

Additionally, D&D Pharmatech revealed that the International Progressive MS Alliance, a global nonprofit organization that supports research into progressive MS, is considering funding the trial. 

The company also noted that it has been invited to apply for funding through the NeuroNEXT program, a clinical research network led by the National Institutes of Health (NIH) focused on neurological disorders. If the trial is selected for U.S. government or private funding, Mowry's research team could receive broader support, accelerating the clinical trial process.