Crinetics Pharmaceuticals Receives Orphan Drug Designation for Paltusotine for the Treatment of Acromegaly
“The orphan drug designation by the FDA for paltusotine to treat acromegaly validates the encouraging clinical data we have seen so far in the Phase 2 ACROBAT Edge clinical trial, and underscores the need for a once daily oral treatment option for patients suffering from this rare disease,” said
“Receiving orphan drug designation is another important milestone for the paltusotine program. We look forward to announcing topline results for our ACROBAT clinical program in the fourth quarter of this year and to the initiation of our planned Phase 3 trial for paltusotine in the first half of 2021,” said
Paltusotine (formerly CRN00808) is an orally available nonpeptide biased agonist that is designed to be highly selective for the somatostatin type 2 receptor. It was designed by the Crinetics discovery team to provide a once daily oral option for patients with acromegaly and neuroendocrine tumors that are currently treated by injected therapies that sell approximately
Acromegaly is a serious disease generally caused by a benign growth hormone secreting tumor in the pituitary. Excess GH secretion causes excess secretion of insulin-like growth factor-1 (IGF-1) from the liver, which causes bone and cartilage overgrowth, organ enlargement, and changes in glucose and lipid metabolism. The symptoms of acromegaly include abnormal growth of hands and feet and changes in shape of the bone and cartilage that result in alteration of facial features. Overgrowth of bone and cartilage and thickening of tissue leads to arthritis, carpal tunnel syndrome, joint aches, deepening of voice due to enlarged vocal cords, sleep apnea due to obstruction of airways, and enlargement of lips, nose, tongue, heart, liver, and other organs.
Surgical removal of pituitary adenomas, if possible, is the preferred initial treatment for most acromegaly patients. Pharmacological treatments are used for patients that are not candidates for surgery, or when surgery is unsuccessful in achieving treatment goals. Approximately 50% of patients with acromegaly prove to be candidates for pharmacological treatment. Long-acting somatostatin receptor ligands (SRLs) are usually the initial pharmacologic treatment, however these drugs require monthly injections and are commonly associated with pain, injection site reactions, and increased burden in the lives of patients. Although over 90% of patients have demonstrable responses to SRLs (Annals of Internal Medicine. 1992; 117:711-718) only 20-40% of patients achieve normalization of IGF-1 (J Clin Endocrinol Metab 99: 791–799, 2014). Additional pharmacological treatment options include dopamine agonists or GH receptor antagonists that may be used in combination with SRLs.
Crinetics cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the potential for any of our ongoing clinical trials to show safety or efficacy; the potential benefits of paltusotine for acromegaly patients; the potential to initiate a pivotal Phase 3 trial of paltusotine in acromegaly based on interim results obtained to date and the timing thereof; the planned expansion of the paltusotine development program to include the treatment of carcinoid syndrome in patients with neuroendocrine tumors and the expected timing thereof, including initiation of a Phase 2 trial in these patients; and the anticipated timing of topline data for Edge and Evolve. The inclusion of forward-looking statements should not be regarded as a representation by Crinetics that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Crinetics’ business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; advancement of paltusotine into a Phase 3 trial is dependent on and subject to the receipt of further feedback from the FDA; the COVID-19 pandemic may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical trials and preclinical studies, manufacturing and supply chain, or impairing employee productivity; the company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; the success of Crinetics’ clinical trials and nonclinical studies for paltusotine and its other development candidates; regulatory developments in
Chief Financial Officer
Source: Crinetics Pharmaceuticals, Inc.