SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the European Commission (EC) has approved PALSONIFY® (paltusotine), the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist, for the treatment of adult patients with acromegaly.
“The European Commission’s decision to approve Palsonify reflects the strength of the clinical data and marks a pivotal step toward bringing this important therapy to even more people living with acromegaly,” said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. “This approval represents another exciting milestone for Palsonify as it accelerates to become the new standard in acromegaly care in the U.S., and soon abroad. This is also a notable achievement for Crinetics in pursuit of our vision to become the global leader in endocrinology.”
The EC approval is supported by positive results from the pivotal data from the PATHFNDR-1 and PATHFNDR-2 Phase 3 trials, which evaluated PALSONIFY’s safety and efficacy in previously treated and medically untreated adults with acromegaly. Across both trials, PALSONIFY consistently demonstrated rapid onset, reliable biochemical control, and sustained efficacy. PALSONIFY also has Orphan Drug Designation in the EU.
Participants also reported significant reductions in signs and symptoms associated with acromegaly – including headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness/tingling – as measured by the Acromegaly Symptom Diary (ASD), a validated patient-reported outcome tool developed to capture the symptoms that matter to people living with acromegaly.
Treatment with PALSONIFY was generally well-tolerated, with no serious adverse events reported in the randomized controlled portion of the trial. The most frequently reported adverse reactions with paltusotine were diarrhea, abdominal pain, nausea, and abdominal discomfort.
The approval by the EC is valid in all 27 member states of the EU and three European Economic Area (EEA) countries. Crinetics is currently planning initial commercialization efforts in Germany and Austria.
PALSONIFY is approved by the U.S. Food and Drug Administration (FDA) for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. Crinetics is also in partnership with Sanwa Kagaku Kenkyusho Co., Ltd (SKK) to develop and commercialize PALSONIFY for acromegaly in Japan, where the Ministry of Health, Labour and Welfare recently granted an orphan drug designation. SKK recently submitted a new drug application (NDA) in Japan for paltusotine for the treatment of acromegaly. In Brazil, Crinetics recently submitted a Marketing Authorization Application (MAA) to Brazil’s National Health Surveillance Agency (ANVISA) for PALSONIFY for the proposed treatment of acromegaly in adults.
About PALSONIFY® (Paltusotine)
PALSONIFY, a selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist, is the first and only once-daily, oral therapy approved for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. In Phase 3 studies, once-daily, oral PALSONIFY 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. Paltusotine is also in Phase 3 clinical development for carcinoid syndrome associated with neuroendocrine tumors (CAREFNDR). Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel movement frequency, which are the most common symptoms of carcinoid syndrome.
PALSONIFY is approved in the U.S. for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. It is also approved for use in the EU for the medical treatment of adult patients with acromegaly.
Important Safety Information
The full European Summary of Product Characteristics (SmPC) for PALSONIFY will be available on the European Medicines Agency website at www.ema.europa.eu.
About Crinetics Pharmaceuticals
Crinetics Pharmaceuticals is a global pharmaceutical company committed to transforming the treatment of endocrine diseases and endocrine-related tumors through science rooted in patient needs. Crinetics is focused on discovering, developing, and commercializing novel therapies, with a core expertise in targeting G-protein coupled receptors (GPCRs) with small molecules that have specifically tailored pharmacology and properties.
Crinetics’ lead product, PALSONIFY® (paltusotine), is the first once-daily, oral treatment approved by healthcare regulatory authorities in U.S. and European Union for the treatment of adults with acromegaly. Paltusotine is also in clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics’ deep pipeline of 10+ disclosed programs includes late-stage investigational candidate atumelnant, which is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and CRN09682, a nonpeptide drug conjugate candidate that is being developed to treat SST2 expressing neuroendocrine tumors and other SST2 expressing solid tumors. Additional discovery programs address a variety of endocrine conditions such as neuroendocrine tumors, Graves’ disease (including Graves’ hyperthyroidism and Graves’ orbitopathy, or thyroid eye disease), polycystic kidney disease, hyperparathyroidism, diabetes, obesity, and GPCR-targeted oncology indications. Crinetics is headquartered in San Diego, with European commercialization operations based in Zug, Switzerland. Please visit www.crinetics.com for more information.
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