Overview
- An interim analysis in the open-label STARBORN-1 trial included 12 children receiving up to four intracystic injections roughly six weeks apart.
- All eight patients who reached the eight-week assessment met the trial’s clinical success criteria, with seven responding after one or two doses and one large macrocystic case achieving a complete response after four doses.
- Among macrocystic cases, 83% (5 of 6) had a 90–100% reduction in lesion volume, the remaining patient showed a 60–<90% reduction, and the sole mixed cystic patient had a complete response.
- Tolerability was favorable with no serious adverse events reported; most side effects were mild to moderate, and one patient discontinued due to Grade 2 fatigue.
- Interpretation carries caveats including a later-identified misdiagnosis of cancer with no response, a case later determined to be a ranula, and a withdrawal after marked resolution; two patients remain disease-free at 32 weeks, and the company reviewed the data on a Nov. 19 webcast.