Sept. 26, 2024 -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for paltusotine, the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist in development for the proposed treatment and long-term maintenance therapy of acromegaly.
“This NDA submission brings us one step closer to our goal of delivering a new generation of therapy that can help people living with acromegaly,” said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. “Based on the comprehensive data from the Phase 3 PATHFNDR program, we are excited about the significance of this potential advancement for the acromegaly community, as well as what it represents to Crinetics as a company. Paltusotine is the leading candidate of a deep, innovative pipeline – the first of many therapeutic candidates that have been purposefully designed in-house to transform the lives of people impacted by a wide range of endocrine conditions.”
The NDA is supported by data from 18 clinical trials, including two Phase 3 trials that evaluated paltusotine for the treatment of acromegaly in medically untreated and treated patients. All primary and secondary endpoints were met in both Phase 3 studies. Treatment with paltusotine was well-tolerated and resulted in biochemical control and patient reported symptom control compared to placebo.
Crinetics anticipates receiving notification from the FDA on the status of the NDA submission in December.
Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. It was designed by Crinetics with the goal of providing a once-daily, oral option for reliable and consistent control of acromegaly. In Phase 3 studies, once-daily, oral paltusotine maintained IGF-1 levels and symptom control in patients with acromegaly who were switched from monthly injectable medications (PATHFNDR-1) and rapidly decreased IGF-1 levels and symptom burden in medically untreated acromegaly patients (PATHFNDR-2). IGF-1 is the primary biomarker endocrinologists use to manage acromegaly patients. Results from the Phase 2 study in carcinoid syndrome will provide an opportunity for paltusotine to potentially demonstrate utility in an investigational, Phase 3 trial for another important indication related to the treatment of symptoms in patients with neuroendocrine tumors.
Acromegaly is a serious rare disease generally caused by a pituitary adenoma, a benign tumor in the pituitary that secretes growth hormone (GH). Excess GH secretion causes excess secretion of insulin-like growth factor-1 (IGF-1) from the liver. Prolonged exposure to increased levels of IGF-1 and GH leads to progressive and serious systemic complications, often resulting in bone, joint, cardiovascular, metabolic, cerebrovascular, or respiratory disease. Acromegaly symptoms include headache, joint aches, fatigue, sleep apnea, severe sweating, hyperhidrosis/oily skin, bone and cartilage overgrowth, abnormal growth of hands and feet, enlargement of heart, liver, and other organs and alteration of facial features. Uncontrolled acromegaly results in increased mortality and has a debilitating impact on daily functioning and quality of life.
Surgical removal of pituitary adenomas, if possible, is the preferred initial treatment for most people with acromegaly. Pharmacotherapy is used for people who are not candidates for surgery, or when surgery is unsuccessful in achieving treatment goals. Approximately 50% of people with acromegaly prove to be candidates for pharmacotherapy. Injectable somatostatin receptor ligands are the most common initial pharmacologic treatment; however, these drugs require monthly depot injections with large gauge needles that are commonly associated with pain, injection site reactions, and an increased burden on the lives of patients.
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics is also developing atumelnant (CRN04894), an investigational, first-in-class, oral ACTH antagonist, that is currently completing Phase 2 clinical studies for the treatment of congenital adrenal hyperplasia and Cushing’s disease. All of the company’s drug candidates are orally delivered, small molecule new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease (including thyroid eye disease), diabetes, obesity and GPCR -targeted oncology indications.
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