Breakthrough for NF1? Mirdametinib Shows Significant Tumor Reduction in All Ages


Christopher Moertel, MD, discusses mirdametinib's mechanism of action and the implications of the phase 2b ReNeu trial of the agent in NF1-associated plexiform neurofibroma.

Mirdametinib was evaluated in the phase 2b ReNeu trial (NCT03962543) to see if it was effective and safe for treating plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1). Findings were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

The study found that mirdametinib significantly reduced the size of PN tumors in both adults and children. Additionally, mirdametinib led to improvements in pain severity, pain interference, and health-related quality of life. The medication was well-tolerated with a manageable safety profile.

These results suggest that mirdametinib could be a promising new treatment option for patients with NF1 and PN.

Here, Christopher Moertel, MD, professor of pediatrics at the University of Minnesota and presenter of the study at ASCO, discusses mirdametinib's mechanism of action and the implications of these findings.


0:05 | Mirdametinib is an investigational highly selective MEK1/2 inhibitor. It's taken orally, it's CNS-penetrant, and is a MEK inhibitor that's very useful for patients who have RAS-driven tumors. NF1-associated plexiform neurofibroma is a very important problem for people living with NF1.

0:29 | So, having treatment options available available to them is very important. This is the first study that really has provided good data regarding adults with NF1. And currently, there are no approved therapies for adults with NF1. Likewise, having an alternative that may have improved and improved toxicity profile is very important to this patient population.

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