Data shows that NST-628, a new “molecular glue” drug targeting the RAF/MEK pathway, may offer a promising treatment for patients with NRAS and BRAF class II/III mutant melanoma, who currently have few targeted therapy options.
In an early phase clinical trial involving heavily pretreated patients, NST-628 showed strong anti-cancer activity. In melanoma patients, it achieved a 38% response rate and an 85% disease control rate. The drug also showed activity in other cancers, including ovarian, cervical, and colorectal cancers with KRAS mutations.
A notable finding was its ability to cross the blood-brain barrier. One patient with a brain tumor experienced a 70% reduction in tumor size, suggesting potential for treating cancers that spread to the brain.
Side effects were generally mild, such as rash and diarrhea, and few patients stopped treatment due to toxicity. Researchers say the drug works by locking two key proteins in the cancer growth pathway into an inactive state, preventing tumor resistance.
The study is now expanding to test NST-628 in more patients and combination therapies.