D&D Pharmatech Secures U.S. Phase 2 Clinical Trial Funding for NLY01, Investigational Therapy for Degenerative Brain Diseases

D&D Pharmatech announced on December 19 that the Johns Hopkins University School of Medicine research team’s Phase 2 clinical trial of NLY01, D&D Pharmatech’s investigational therapy for degenerative brain diseases, has been selected as a final recipient of clinical research funding from the International Progressive MS Alliance (IPMSA) in the field of multiple sclerosis (MS).

The IPMSA is the world’s largest global nonprofit organization dedicated to multiple sclerosis. It is an international alliance comprising MS organizations from 19 countries, including the United States, hundreds of key opinion leaders (KOLs), and nine global pharmaceutical companies. The alliance was established to support the development of therapies for progressive multiple sclerosis through global collaborative research.

This Phase 2 clinical trial will be a researcher-initiated study led by Professor Ellen Mowry, Director of the MS Center at Johns Hopkins University School of Medicine, who has extensive experience in global clinical trials. The study will be conducted at both Johns Hopkins University School of Medicine and Mount Sinai School of Medicine. The Johns Hopkins research team received IND (Investigational New Drug) approval for the Phase 2 trial from the U.S. FDA in September 2024. In addition to the IPMSA research grant, the team plans to secure further funding to carry out the trial. D&D Pharmatech will provide the NLY01 investigational drug free of charge for this clinical study and stated that it will not bear any additional costs related to the trial.

Multiple sclerosis is a chronic neurological disorder that primarily affects young adults in their 20s to 40s. Due to immune system abnormalities, inflammation occurs in the myelin-the protective sheath of the central nervous system, which includes the brain, spinal cord, and optic nerves-resulting in impaired nerve signal transmission. While symptoms can be managed with medication in the early stages, progressive multiple sclerosis, which involves gradual deterioration of nerve function, follows a course similar to that of degenerative brain diseases, and effective treatments remain extremely limited to date.

The Johns Hopkins MS Center research team, through years of collaboration with D&D Pharmatech, has demonstrated that NLY01, as previously observed in Parkinson’s and Alzheimer’s disease models, also reduces myelin damage and protects neurons in multiple sclerosis animal models by inhibiting neuroinflammation, significantly alleviating disease progression and recurrence. The team also identified a novel mechanism by which NLY01 suppresses the passage of activated immune cells that cause neural damage across the blood-brain barrier (BBB) into the central nervous system. This dual inhibition of inflammation and neural loss in both the peripheral and central nervous systems is expected to maximize the therapeutic effect in multiple sclerosis. This research achievement was also selected as one of the U.S. Department of Defense’s research highlights of 2024.

Meanwhile, in February 2023, D&D Pharmatech’s Phase 2 clinical trial of NLY01 for Parkinson’s disease did not achieve statistical significance in the overall patient population. However, among younger patients under 60 years old (95 subjects), a statistically significant and clear therapeutic effect was observed compared to placebo. Since the main onset age for progressive multiple sclerosis-the indication for this investigator-initiated trial-is around the 40s, there is anticipation that the effective therapeutic outcomes seen in younger Parkinson’s patients may be replicated.

Notably, this clinical trial addresses the limitations of the previous NLY01 Parkinson’s Phase 2 study, which involved a low dosage (5mg) and short treatment duration (36 weeks), by increasing the dose to 10mg and extending the treatment period to 96 weeks. By ensuring sufficient drug exposure and long-term treatment in younger patients, the likelihood of clinical success is expected to increase.

The trial is scheduled to begin as early as the first quarter of 2026 and will enroll a total of 120 patients with progressive multiple sclerosis. It will be conducted over 96 weeks as a multicenter, randomized, double-blind, parallel-group study. The primary endpoint will objectively assess the progression of neurodegeneration and damage by measuring changes in brain parenchymal volume using MRI imaging, thereby evaluating how effectively NLY01 can slow disease progression in MS patients.

Lee Seulgi, CEO of D&D Pharmatech, stated, “Being selected for clinical research funding by IPMSA, the world’s largest multiple sclerosis alliance, validates the innovative mechanism and therapeutic potential of NLY01 as recognized by global experts. Considering the therapeutic effects observed in younger Parkinson’s patients and the high-dose, long-term treatment design of this study, we expect to see meaningful clinical results that could offer a new treatment option for multiple sclerosis patients who develop the disease at a relatively young age.”