Interim safety and exploratory efficacy results of a phase 2, randomised, parallel-group study of oral Paltusotine treatment in subjects with carcinoid syndrome

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Introduction: Carcinoid Syndrome (CS) is the most common functional NET syndrome. It is characterized by watery diarrhea or cutaneous flushing. Paltusotine is an investigational once-daily, oral, selective SST2 agonist in development for the treatment of acromegaly and CS. Paltusotine has been shown to maintain IGF-1 control at levels similar to injected depot SRLs.

Aim(s): This ongoing open-label, multicenter study examines the safety, tolerability, pharmacokinetics, and exploratory efficacy of paltusotine in the treatment of CS.

Materials and methods: Eligible participants had documented, well-differentiated NETs with CS and were either naïve to therapy with SRLs or had symptomatic control on SRLs. Participants were were randomly assigned to 40 mg or 80 mg of once-daily oral paltusotine for 8 weeks. In addition to the safety and tolerability assessments biomarkers were collected, and exploratory efficacy was assessed using a daily electronic diary.

Conference:

Presenting Author:

Authors: Usiskin K, Chauhan A, Mui C, Dillon J, Fan X,

Keywords: oral, paltusotine, carcinoid syndrome, phase II,