Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses data on the efficacy and safety of paltusotine for the treatment of acromegaly.
Acromegaly is an endocrine disorder that results from the overproduction of growth hormone in the pituitary gland. It is most often diagnosed in middle-aged adults, although symptoms can appear at any age. Signs and symptoms include abnormal growth and swelling of the hands and feet; bone changes that; alter various facial features; arthritis; carpal tunnel syndrome; enlargement of body organs; and various other symptoms. The condition is usually caused by benign tumors on the pituitary called adenomas. Rarely, it is caused by tumors of the pancreas, lungs, and other parts of the brain.
Paltusotine is an oral selective somatostatin receptor type 2 agonist currently in development for the treatment of acromegaly. Data on this treatment was recently presented at the 2025 ENDO meeting.
Data from a long-term extension study of two phase 3 studies of paltusotine showed the ability of the treatment to stabilize biochemical control and was well-tolerated. Results from patient experience of symptoms also illustrated good stability of symptom control.
Dr. Krasner stresses the importance of measuring symptoms and the burden they put on patients in analyzing disease control. In a recent study, a new patient-reported outcome tool called the Acromegaly Symptom Diary was used once a day to gain insight into symptom response to platusotine versus placebo. Results from the study indicated statistically significant symptom control in patients treated with paltusotine compared to placebo.
Two posters at ENDO also explored the robustness of the new symptom tool as well as the variability of symptom control in patients. The Acromegaly Symptom Diary tool was observed to be robust across a large range of patient groups regardless of biochemical control of disease. Additionally, results from the tool showed high frequency of symptom exacerbation in patients receiving standard-of-care first line injection therapies. A post-hoc exploratory analysis illustrated that when patients switched from an injection therapy to paltusotine, a reduction in frequency of symptom exacerbations was observed.
Paltusotine is currently under review at the U.S. Food and Drug Administration (FDA) with a PDUFA data of September 25, 2025.
For more information on acromegaly and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/