SFEBES2023 Poster Presentations Neuroendocrinology and Pituitary (74 abstracts)
Paltusotine maintains IGF-1, GH, and symptom control in patients with acromegaly switched from injected octreotide or lanreotide monotherapy: Topline results from PATHFNDR-1, a phase 3, randomized, double-blind, placebo-controlled, multicenter study
Monica R. Gadelha , Alessandra Casagrande , MD , PhD , Christian J. Strasburger MD , Martin Bidlingmaier , Peter Snyder MD , Mirtha Guitelman MD , Cesar Boguszewski MD , Michael Buchfelder MD , Ilan Shimon MD , Gerald Raverot MD , Miklos Toth MD , Emese Mezosi MD , Mirjana Doknic MD , Xiaolin Fan PhD , David Clemmons MD , Michael Keeley PhD , Peter J. Trainer MD , R. Scott Struthers PhD , Alan Krasner MD & Beverly M.K. Biller MD
Neuroendocrinology Research Center/Endocrinology Division-Medical School and Hospital Universitario Clementino Fraga Filho-Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil
Paltusotine is a once-daily, oral, selectively-targeted SST2 agonist in development for the treatment of acromegaly. PATHFNDR-1 (NCT04837040) enrolled patients with acromegaly who had an IGF-1 ≤1xULN on a stable (>12 weeks) dose of lanreotide or octreotide. Patients were randomized 1:1 to receive paltusotine 40 mg/day or placebo for 36 weeks. During the first 24 weeks, the paltusotine dose was titrated (range 20-60 mg) based on IGF-1 and tolerance. Dose changes were not permitted after week 24. IGF-1 was measured centrally using the iSYS immunoassay. Acromegaly symptoms were assessed using the Acromegaly Symptoms Diary (ASD), where higher scores represent greater symptom burden. 58 patients [(paltusotine n=30; placebo n=28), mean age 54.9 (SD 13.7) years, 55% female] were enrolled all of whom were controlled on octreotide (59%) or lanreotide (41%). The primary endpoint was achieved, with a significantly greater proportion of patients maintaining IGF-1 levels at ≤1.0xULN (mean of weeks 34 and 36) after switching from somatostatin receptor ligands (SRLs) to paltusotine compared to placebo (83% vs. 4%, P<0.0001). All three secondary endpoints were achieved. Paltusotine maintained mean IGF-1, GH and ASD scores significantly better than placebo [mean change from baseline in IGF-1: paltusotine +0.04 vs. +0.8xULN placebo, P<0.0001; proportion of patients who maintained GH <1.0 ng/ml (week 34) (paltusotine (87% vs. 28% placebo, P=0.0003) & mean change from baseline in ASD score (paltusotine -0.6 vs. +4.6 placebo, P=0.02)]. Paltusotine was well-tolerated with the most common AEs being consistent with either SRL therapy or acromegaly. One serious TEAE of acute cholecystitis was reported in a placebo patient. One placebo patient discontinued the study due to patient decision. There were no clinically significant changes in pituitary tumor size. In conclusion, once-daily, paltusotine treatment was significantly better than placebo at maintaining IGF-1, GH, and symptom control in patients switched from SRLs, and was well-tolerated.
Volume 94
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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