Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. (“Crinetics”) shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial. Within the trial, researchers are evaluating paltusotine for patients with acromegaly. Currently, the standard-of-care in acromegaly treatment is injectable. If paltusotine is safe, effective, and becomes approved for treatment, it would offer an oral, non-invasive therapeutic option for patients.
Paltusotine
To begin, what is paltusotine? According to Crinetics, paltusotine:
establishes 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, or GH, from the pituitary gland.
This investigational therapy fits into Crinetics’ goal of advancing therapeutic options for patients with rare endocrine conditions. Altogether, 52 patients will enroll within the Phase 3 PATHFNDR-1 clinical trial. During the trial, researchers will begin by evaluating IGF-1 and GH levels. Next, patients will receive either paltusotine or a placebo for a 9-month period. Following the 9 months, patients may continue within an open-label extension study. Altogether, trial results should be available by 2023.
Acromegaly
An estimated 60 out of every 1 million people have acromegaly, a hormonal disorder resulting from excess growth hormone (GH). Normally, the pituitary glands produce GH. However, patients with acromegaly produce too much GH, often due to benign adenomas (tumors) on the pituitary gland. As a result, the liver also excretes too much IGF-1, another hormone. Typically, acromegaly occurs in middle-aged adults. Hormone overproduction then leads to a variety of symptoms and physical characteristics, such as:
- Osteoarthritis
- Malocclusion (separated and misaligned teeth)
- Muscle and joint pain
- Abnormal hand and foot growth
- Protruding brow and lower jaw
- Enlarged nasal bone
- Fatigue
- Deepening voice
- Darkened or thickened skin
- Hirsutism (excess hair growth)
- Organ enlargement
- Oily skin
- Excessive sweating
- Sleep apnea
Learn more about acromegaly.
