SpringWorks Conducting Several Studies with MEK Inhibitor, Mirdametinib

Author: SpringWorks Therapeutics

Published On: 09/14/2021

NF1-Associated Plexiform Neurofibroma

SpringWorks Therapeutics is conducting a clinical trial to study an investigational MEK inhibitor called mirdametinib in people with a plexiform neurofibroma (PN) associated with Neurofibromatosis type 1 (NF1). The study, called the ReNeu trial, is currently enrolling people ages 2 to 17. The purpose is to determine the effectiveness of mirdametinib at improving a PN that is causing significant issues in people with NF1. Mirdametinib is offered as an oral capsule or in a pediatric formulation for children who cannot swallow a capsule.  Participants will receive mirdametinib, which can be taken without regard to food, dosed at approximately 2mg/m2/dose (up to a maximum of 4mg twice a day) on 3 weeks on, 1 week off intermittent dosing schedule.  For more information and to find out if you or your loved one is eligible, please visit the ReNeu study website by clicking here.  

Low-Grade Glioma

Another study is underway to evaluate mirdametinib in children, adolescents, and young adults with low-grade glioma (LGG). LGG is the most common central nervous system tumor in children, and NF1 related LGGs can potentially impact close to a third of NF1 patients. Recently, it has been recognized that most cases of pediatric LGG have genetic alterations that upregulate the MAPK pathway. Mirdametinib is designed to inhibit MEK1 and MEK2, which are proteins that occupy pivotal positions in the MAPK pathway. This Phase 1/2 clinical trial is sponsored by St. Jude Children’s Research Hospital. More information on the trial can be found on www.clinicaltrials.gov under the identifier NCT04923126.

ER+ Metastatic Breast Cancer

In August, SpringWorks also announced a study to evaluate mirdametinib as a combination therapy with fulvestrant, a selective estrogen receptor degrader (SERD) in patients with estrogen receptor positive (ER+) metastatic breast cancer (mBC) with MAPK alterations, particularly inactivating mutations in NF1. In women under age 50, breast cancer can be up to 5 times more common in NF1 patients compared to other women. This study is sponsored by Memorial Sloan Kettering Cancer Center and is expected to begin enrolling patients in the coming months.

Advances in research for the NF community are made possible by clinical trials. We thank the people who choose to participate in clinical trials and the researchers who conduct them. If you or a loved one is interested in participating in NF research, please read our clinical trials brochure and learn more about the clinical trial process and what that entails.  If you are interested in learning more about NF1-related clinical trials, please speak to your doctor.