Al's Comment:

 We recently reported on NEO100, an unusual treatment that delivers highly purified perillyl alcohol directly through the nose, allowing patients to self-administer the drug at home. Earlier results were intriguing, particularly because some of the longest survivors had IDH1-mutant tumors.

 

These new Phase 2a results strengthen that story. In 24 patients with recurrent or progressive IDH1-mutant grade 3 or 4 glioma, the 6-month progression-free survival rate was 48.9%—more than double the study's prespecified 20% benchmark—and median overall survival was an impressive 26.1 months. The treatment was also reported to be generally well tolerated, with no major toxicities.

 

There are important limitations. This was a small, single-arm study, so the results need to be confirmed in a larger trial with an appropriate control group. The objective response rate was only 8.3%, reminding us that prolonged disease control does not necessarily show up as dramatic tumor shrinkage on MRI.

 

Still, a median survival of more than two years in patients with recurrent high-grade IDH1-mutant glioma deserves attention. Perhaps just as important, NEO100 appears relatively easy for patients to use compared with many experimental brain tumor treatments. The company now plans to meet with the FDA to discuss a possible registrational pathway.

 

We will be watching closely. If these results hold up in a larger study, NEO100 could become an important new option for this molecularly defined group of brain tumor patients.

 

www.hmpgloballearningnetwork.com/site/onc/news/neo100-01-meets-primary-end-point-supporting-further-development-idh1-mutant-glioma