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The investigational treatment NEO100, a highly purified form of perillyl alcohol, showed encouraging preliminary results in a Phase IIa trial for patients with recurrent or progressive IDH1-mutant grade 3 or 4 gliomas. The NEO100 treatment is self-administered by patients through the nose four times a day, using a nasal mask and nebulizer, with the intranasal delivery intended to help overcome the blood-brain barrier. In the single-arm study, 24 patients received treatment in 28-day cycles; researchers compared the results with historical results for patients receiving treatment for recurrent high-grade glioma.
The study met its primary endpoint, with 49% of patients progression-free at six months (compared with an expected 20% based on the study's historical benchmark). Median overall survival was reported as 26 months, with 87% of patients alive at six months, 61% at one year, and 54% at two years. Five patients were still receiving treatment when the preliminary results were reported, including one who had remained progression-free for 19 months, and the company reported several radiographic responses, including one patient whose enhancing tumor largely resolved after prolonged treatment. NEO100 was reportedly well tolerated, with the company describing no significant toxicity even with prolonged treatment, although the full safety and efficacy data have not yet been published in a peer-reviewed journal.
It is important to note that this was a small, non-randomized trial, and our understanding of the prognosis of recurrent IDH1-mutant tumors continues to evolve (for example, recent research suggests that acquired RAS-MAPK mutations may be associated with shorter survival). Despite these caveats, the results are encouraging for a disease with no approved targeted therapies and very few salvage options in the recurrent setting. The company plans to meet with the FDA to discuss the next steps and a potential registrational trial.