PRG S&Tech's Trineumin Receives Therapeutic Use Approval in Korea for NF2, the World's First Small Molecule Targeting the Disease Mechanism
核心洞察
South Korea's Ministry of Food and Drug Safety has granted therapeutic use approval for PRG S&Tech (搜索)'s Trineumin, allowing adult NF2 patients aged 19 and older access to the investigational drug.
Trineumin is the world's first small molecule candidate that directly targets the NF2 pathological mechanism by modulating the abnormal TβR1 (搜索)-RKIP (搜索) protein interaction caused by NF2 gene (搜索) mutations.
The drug has received Orphan Drug Designation and Fast Track designation from the U.S. FDA, as well as Orphan Medicinal Product Designation from the EMA.
South Korea's Ministry of Food and Drug Safety (MFDS) has granted approval for use for therapeutic purposes to Trineumin, an investigational treatment for type 2 neurofibromatosis (搜索) (NF2) developed by PRG S&Tech (搜索). Announced on June 22, the regulatory decision enables adult NF2 patients aged 19 and older in Korea to access the clinical-stage drug for actual therapeutic use prior to formal marketing authorization.
The approval for use for therapeutic purposes is a conditional regulatory pathway designed for patients with life-threatening or serious diseases who have no suitable treatment alternatives. This mechanism allows the use of drugs still under clinical investigation when no other viable options exist.
Trineumin represents a notable scientific advance as the world's first small molecule therapeutic candidate that directly targets the underlying pathological mechanism of NF2. The drug is designed to suppress tumor development and growth by modulating the abnormal interaction between TβR1 (搜索) and RKIP (搜索) proteins, a disruption caused by mutations in the NF2 gene (搜索).
Regulatory Milestones and Global Development
Trineumin's regulatory trajectory reflects a concerted global development strategy. In 2024, PRG S&Tech (搜索) obtained approval for a domestic phase 1/2a clinical trial plan (IND) from Korean regulators. The following year, in 2025, the company secured IND approval from the U.S. Food and Drug Administration (FDA). The candidate has also been granted Orphan Drug Designation (ODD) and Fast Track designation by the U.S. FDA, alongside Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA), underscoring its international development competitiveness.
Clinical Trial Progress
A phase 1/2a clinical trial is currently underway at Asan Medical Center in Seoul. According to PRG S&Tech (搜索), administration and evaluation have been completed up to the highest planned dose for the phase 1 portion of the study, which is 150 mg. The company is now conducting additional dose-escalation studies aimed at determining the recommended phase 2 dose (RP2D). Once safety, pharmacokinetics (PK), and initial efficacy data are secured, PRG S&Tech plans to accelerate the development of phase 2 clinical trials.
Executive Commentary
Bumjoon Park, CEO of PRG S&Tech (搜索), emphasized the significance of the regulatory milestone: "This approval for use for therapeutic purposes is significant in that the accumulated scientific and clinical evidence from the Trineumin development process has led to expanded treatment opportunities for actual patients. We will continue to pursue clinical development and a global regulatory strategy to provide new possibilities for NF2 patients with limited treatment options."