New Data Available on Paltusotine for Acromegaly


On September 9, 2022, researchers presented data at the European Neuroendocrine Association (ENEA) meeting. According to Yahoo! Finance, one such presentation centered around new safety and efficacy data from the ACROBAT Advance open-label extension study evaluating paltusotine for individuals with acromegaly.

Pharmaceutical company Crinetics Pharmaceuticals developed paltusotine as a potential therapeutic option for individuals with carcinoid syndrome, acromegaly, or neuroendocrine tumors. Formly known as CRN00808, paltusotine is described as part of:

a new class of oral, selective, nonpeptide, somatostatin receptor type 2 (SST2) agonists designed for the treatment of acromegaly. Somatostatin is a neuropeptide hormone that broadly inhibits the secretion of other hormones, including growth hormone (GH) from the pituitary gland.

This therapy, orally administered once each day, has so far shown a suppression of the growth hormone axis in trials. The ACROBAT Advance open-label extension study allowed patients from the Phase 2 ACROBAT Evolve and Edge trials to continue treatment.

Altogether, the treatment was relatively safe and well-tolerated. Its safety profile is considered relatively close to that of injectable therapies, particularly when used with adjunctive treatments. Paltusotine was able to lower insulin-like growth factor-1 (IGF-1) at the same rate of other standards-of-care. After the open-label extension, 89% of those enrolled decided to continue using paltusotine as their therapy. Paltusotine, then, could offer a safe, effective, and less invasive treatment option than what is currently available for patients.

What is Acromegaly?

Acromegaly is a rare hormonal disorder in which the body (particularly, benign adenomas on the pituitary gland) produces too much growth hormone (GH). This then prompts the liver to over-secrete IGF-1. Together, this excess GH and IGF-1 causes the symptoms and characteristics associated with acromegaly. An estimated 60 in every 1 million people has acromegaly. This condition should not be confused with gigantism. Because acromegaly progresses slowly, many people may not recognize signs for years. This is also a condition which is often misdiagnosed. Symptoms, characteristics, and complications can (but do not always) include:

  • Enlarged hands and feet (which may cause, rings, shoes, etc. to fit more snugly)
  • An enlarged nasal bone and protruding brow and lower jaw
  • Persistent or severe headaches
  • Menstrual irregularities
  • Skin tags
  • Fatigue
  • Sleep apnea and severe snoring
  • High blood pressure
  • Thick, oily skin that is prone to excessive sweating
  • Enlarged facial features
  • Joint or muscle pain/weakness
  • Enlarged heart, liver, and organs
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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