Once-daily pill paltusotine shows lasting relief for carcinoid syndrome patients

A phase 2 trial presented at the 2026 NANETS Multidisciplinary NET Medical Symposium shows that once-daily oral paltusotine delivers durable, meaningful symptom relief for patients with carcinoid syndrome — with results holding strong over two years. The study enrolled 36 patients with locally advanced or metastatic grade 1 or 2 neuroendocrine tumors who had active carcinoid syndrome symptoms, including both treatment-naïve patients and those who had washed out of somatostatin receptor ligand (SRL) therapy.

After an 8-week randomized treatment period, 26 patients continued into a 102-week open-label extension. Across the board, patients saw meaningful reductions in daily flushing episodes, bowel movements, and urgent bowel movements — with improvements deepening over time rather than fading.

By the Numbers:

• Mean daily flushing episodes dropped from 2.4 at baseline → 0.3 at week 110
• Mean worst flushing severity score fell from 3.4 → 0.7
• Mean daily bowel movements decreased from 3.6 → 2.1
• Mean urgent bowel movements fell from 1.2 → 0.1
• Investigator-assessed PFS rate: 80% at a median follow-up of 22.3 months

Why it matters: Carcinoid syndrome can be debilitating, and current injectable SRL therapies come with adherence challenges. Paltusotine's once-daily oral format, combined with its strong two-year safety and efficacy profile, could meaningfully improve quality of life for patients — and a phase 3 trial is already underway.