GNT Pharma, a late-stage biopharmaceutical company with operations in Seoul and New York, announced that South Korea's Ministry of Food and Drug Safety (MFDS) has approved its IND application to commence global Phase 3 clinical trials for Nelonemdaz, a first-in-class dual-action neuroprotectant designed to reduce death and long-term disability following ischemic stroke. This regulatory milestone represents a significant step toward global regulatory approvals and commercialization for the breakthrough therapy.
Novel Dual-Action Mechanism
Nelonemdaz stands as the world's first stroke therapy to simultaneously inhibit NR2B-selective NMDA receptors and scavenge free radicals—two critical drivers of brain cell death following ischemic stroke. The drug is designed to complement existing revascularization therapies and overcome their clinical limitations, representing a novel approach to neuroprotection that targets both acute and diffuse neuronal damage.
Global Phase 3 Trial Design
The upcoming Phase 3 trial will enroll 378 patients with severe ischemic stroke who are eligible for thrombectomy within 12 hours of symptom onset. The protocol requires intravenous administration of Nelonemdaz within 60 minutes of emergency room arrival, with treatment repeated 10 times over a 5-day period. Thrombectomy procedures will be performed within 90 minutes of hospital admission.
The primary efficacy endpoint focuses on functional independence in daily living at 12 weeks post-treatment. The trial will be conducted across more than 20 world-leading stroke centers spanning Korea, the United States, and Australia.
"Nelonemdaz offers a transformative approach to stroke treatment by combining neuroprotection with revascularization," stated Dr. BJ Gwag, CEO of GNT Pharma. "We are excited to partner with world-renowned stroke experts to expand its global reach."
Promising Clinical Data
Nelonemdaz has demonstrated strong clinical promise in previous Phase 2 and 3 trials involving 704 stroke patients in Korea. When administered within 60 minutes of emergency room arrival, the drug improved disability outcomes by a factor of 4.3 compared to placebo. Even when administered within 70 minutes, the therapy showed a 2.22-fold improvement in outcomes. These results were published in the Journal of Stroke in May 2025, Volume 27(2).
Development Background
Developed with support from the Korean Ministry of Science, ICT, and other government agencies, Nelonemdaz represents a paradigm shift in stroke therapy. GNT Pharma, founded in 1998, focuses on developing innovative drugs for neurological, inflammatory, and respiratory diseases. The company's pipeline includes other novel drug candidates such as Crisdesalazine for Alzheimer's disease, and it operates a veterinary medicine division with commercially available treatments for Canine Cognitive Dysfunction Syndrome.
The approval to proceed with global Phase 3 trials marks a crucial advancement for a therapy that could potentially transform the treatment landscape for ischemic stroke, a condition where rapid intervention is critical for patient outcomes.
