James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, discusses the  ReNeu trial, which is designed to evaluate the safety, effectiveness, and tolerability of mirdametinib in neurofibromatosis type 1 (NF1) patients with inoperable plexiform neurofibromas.

Neurofibromatosis type 1 is a genetic disorder due to mutations in the NF1 gene that leads to a plethora of symptoms including cutaneous neurofibromas, ‘cafe au lait’ spots, and plexiform neurofibromas, which can cause pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction, and disfigurement. Learning difficulties, visual impairment, twisting and curvature of the spine, hypertension, and epilepsy are also common in NF1 patients.

The ReNeu trial is an ongoing phase 2b clinical trial evaluating mirdametinib as a treatment of inoperable NF1-associated plexiform neurofibromas. Mirdametinib, unlike selumetinib which was approved for the same designation in 2019, does not need to be taken on an empty stomach. This may be significant for younger patients especially who have a difficult time with the fasting required to take selumetinib.

The adult cohort for the ReNeu trial has been fully recruited but the children cohort is still looking to recruit a few more patients. Results from this trial are expected late 2022/early 2023.

To learn more about the ReNeu trial and registration, visit https://www.springworkstx.com/nf1-plexiform-neurofibroma-trial/ 

For more information about NF1 and other rare tumors, visit checkrare.com/cancers/