Medical Breakthrough: Crinetics Triumphs in Phase III with a Once-Daily Pill for Rare Disease Acromegaly

In a groundbreaking announcement, Crinetics Pharmaceuticals revealed that its oral investigational wonder drug, paltusotine, has not only met but exceeded expectations in the Phase III PATHFINDR-1 study involving patients with the rare and challenging condition of acromegaly. This California-based pharmaceutical company has reason to celebrate as they unveiled these remarkable findings on a Sunday.

The results are nothing short of astonishing. A staggering 83% of patients treated with paltusotine successfully maintained their insulin-like growth factor 1 (IGF-1) levels within the normal range, a feat achieved by only a mere 4% of those on a placebo. The statistical significance of this achievement is so striking that the p-value is less than 0.0001, according to Crinetics’ announcement.

But that’s not all – paltusotine demonstrated its prowess by triumphing in all key secondary endpoints, including significant improvements in acromegaly symptoms and a notable proportion of participants maintaining growth hormone (GH) levels below 1.0 ng/mL.

Crinetics CEO Scott Struthers hailed these results as a potential game-changer for acromegaly patients, particularly those looking to transition from the standard injectable treatment. He emphasized that if paltusotine gains approval, it could fill a crucial gap by offering a simple, once-daily oral therapy, marking a significant milestone as Crinetics’ first product to reach the market.

Acromegaly, a rare and debilitating hormonal disorder often linked to pituitary adenoma, has long plagued patients with symptoms ranging from headaches and joint pains to severe sweating and organ enlargement. Surgery to remove the pituitary adenoma is the initial treatment, but around 50% of patients cannot undergo surgery and are left with monthly painful injections of depot somatostatin.

Paltusotine, a targeted somatostatin receptor type 2 (SST2) agonist, presents a groundbreaking alternative. By selectively activating SST2 in pituitary tumors, this drug candidate curbs the secretion of GH and IGF-1. Crinetics has aptly named this endeavor the PATHFINDR clinical development program, which includes PATHFINDR-1. While a full analysis of this groundbreaking study is underway, the results will soon be unveiled at upcoming medical meetings.

In an exciting twist, a second study known as PATHFINDR-2 is already in full swing. This study is set to explore paltusotine in treatment-naïve acromegaly patients who have not yet undergone any medical therapy. With topline data expected in the first quarter of 2024, Crinetics is gearing up to seek regulatory approval as swiftly as possible once PATHFINDR-2 wraps up early next year. The future looks promising for acromegaly patients in need of a simpler and more effective solution.

Leave a Comment