Crinetics Scores Another Phase III Victory in Acromegaly, Aims for FDA Approval

Crinetics Pharmaceuticals has achieved a significant milestone with the successful completion of a Phase III trial for its investigational oral drug, paltusotine, which is being developed to treat acromegaly—a rare hormonal disorder characterized by an overproduction of growth hormone due to a benign tumor in the pituitary gland. The trial results were so promising that the company's stock prices surged by more than 10% in the morning following the announcement.

The clinical trial demonstrated that 56% of the participants who received paltusotine met the primary endpoint, which was the normalization of insulin-like growth factor 1 (IGF-1) levels, a key indicator of the disease's activity. This was a stark contrast to the 5% of patients on a placebo. Furthermore, the drug was also effective in alleviating the disease's symptoms, which was a secondary goal of the study. Importantly, no serious side effects were reported during the trial.

Acromegaly, which can lead to the enlargement of extremities and various severe health complications such as type 2 diabetes, hypertension, and heart disease if left untreated, affects over 25,000 individuals in the U.S. The standard treatment involves surgical removal of the tumor, but this is not always feasible. Current medical treatments are limited to monthly injections, which can be painful, or a daily injection of a medication from Pfizer.

Crinetics' Phase III success follows positive results from a previous study conducted in September 2023, where 83% of patients who switched to paltusotine from their existing injections maintained normal IGF-1 levels, compared to just 4% of those on a placebo.

Armed with these encouraging data, Crinetics is preparing to submit a New Drug Application to the FDA later this year, with an anticipated market launch in 2025. Analysts from Jefferies estimate that the potential market for the drug could be approximately $300 million.

In addition to acromegaly, Crinetics is also exploring the use of paltusotine for carcinoid syndrome, with positive Phase II results reported recently.

Crinetics Pharmaceuticals' advancements in developing a once-daily oral treatment for acromegaly could revolutionize the treatment landscape for this rare disorder, offering patients a more convenient and potentially less invasive therapeutic option.