The Children's Tumor Foundation
02/26/2026 | Press release | Distributed by Public on 02/26/2026 13:32
Inside a New Clinical Trial for NF2-SWN: Early Progress on Trineumin (PRG-N-01)
The Children's Tumor Foundation recently hosted an NF Knowledge Series webinar on a CTF-funded clinical trial in Korea evaluating Trineumin (PRG-N-01), an investigational therapy being studied in people living with NF2-related schwannomatosis (NF2-SWN).
The recording of this webinar is available here.
The webinar featured Dr. Minju Kim, Director of Clinical Development at PRG S&T, Inc, who walked the community through the science behind the drug, the structure of the ongoing trial, and early signals emerging from the first patient cohorts. The session opened with remarks from John Morris, a member of CTF's Board of Directors and the father of an NF2 Hero, underscoring why advancing new options for NF2-SWN remains so urgent.
What this means for the NF2-SWN community:
Early findings from the Phase 1 study suggest that Trineumin is generally well tolerated so far, with preliminary signs of tumor stability or reduction in some patients. While it is still too early to draw firm conclusions, these results support continued study and help determine the safest and most effective dose moving forward.
What Trineumin is designed to do:
Dr. Kim explained that Trineumin is intended to work through a specific biological pathway involving TGF-beta receptor signaling and a protein called RKIP. In the webinar's framing:
Under healthy conditions, the protein RKIP acts as a "brake" that can prevent abnormal Schwann cell growth.
In NF2-related conditions, that balance can be disrupted, and RKIP can be lost through a degradation process.
Trineumin is designed to selectively bind to TGF-beta receptor 1, helping prevent RKIP degradation and potentially slowing tumor growth.
Dr. Kim also noted that the team tested whether the drug can reach the central nervous system, sharing that Trineumin was detected in both brain tissue and spinal fluid in testing-an important point given where NF2-SWN tumors develop.
What the nonclinical work suggests:
Before moving into human trials, PRG S&T Inc. presented a range of nonclinical (pre-human) findings, including:
Evidence of reduced tumor formation in NF2 model mice in preventive studies
Evidence of therapeutic effects when treatment began later (suggesting possible impact on existing disease)
A broad safety testing program (including respiratory, cardiovascular, and central nervous system assessments)
Additional studies are underway, including longer-term toxicity work and juvenile toxicity studies intended to support future consideration in children
Where the clinical trial stands now:
Dr. Kim shared updates on the active Trineumin clinical trial that began in June 2024 in Korea:
Trial type: Phase 1, Part 2a (open-label, dose-finding)
Location: Asan Medical Center (Korea)
Planned Phase 1 enrollment: 16 patients
Main goals of Phase 1: Evaluate safety and tolerability, determine maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), and assess pharmacokinetics (how the drug is absorbed, distributed, and cleared), alongside early signals of activity
The trial uses multiple dose levels and includes a dose-limiting toxicity (DLT) assessment after 12 weeks of treatment for safety monitoring. Dr. Kim shared that the highest dose cohort (150 mg) began dosing in October, and the team expects DLT evaluation for that cohort by early February. After reviewing results, the team plans to meet in a Safety Review Committee (SRC) to decide next steps.
Is the clinical trial going well so far?
The trial appears to be progressing smoothly from a safety standpoint, with early signals that justify continued study, while efficacy remains preliminary and variable across tumor types and individuals.
Safety (encouraging early profile):
Some patients have been on Trineumin for over 18 months (across the study experience shared).
Dr. Kim reported 10 adverse events across 7 patients, with only 1 (fatigue) judged related to Trineumin and graded as Grade 1 (mild).
No serious adverse events were reported in the data shared.
Early efficacy signals (promising, but too early to conclude):
The team is tracking tumor volume changes by MRI, along with hearing measures and quality-of-life surveys.
In the first cohort, Dr. Kim highlighted a meningioma decrease of nearly 20% within three months at an initial dose level and a sustained reduction of over 28% before imaging was affected by a cochlear implant.
In other patients, tumors were described as stable within a range or showing mixed changes, depending on tumor type.
Importantly, the highest-dose cohort's first MRI results were expected late January to February, meaning the strongest "dose-dependent" question is still unfolding.
In Summary:
As one way to think about it: this isn't a home run yet-but it's a solid double. Enough progress to advance the program, and enough unanswered questions to keep going.
What comes next:
The research team is completing the highest-dose cohort now and preparing to move into Phase 2, where they will look more closely at:
the optimal dose,
how different tumor types respond,
whether longer treatment leads to stronger effects, and
which patients may benefit most.
The team also shared plans to expand clinical testing beyond Korea, including discussions about potential U.S.-based trials and other international sites.
CTF's role: accelerating promising options for the NF2-SWN community
This webinar reflects what CTF's mission looks like in action: advancing strong science so potential therapies can reach patients faster. CTF is supporting this trial, including funding for clinical MRI, which is central to assessing whether a therapy affects tumor growth over time. We are grateful to the NF2-SWN community-patients, families, clinicians, and researchers-whose participation and partnership make progress possible.
The Children's Tumor Foundation published this content on February 26, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on February 26, 2026 at 19:32 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]