Crinetics Pharmaceuticals Receives Orphan Drug Designation for Paltusotine for the Treatment of Acromegaly

Rhea-AI Summary

Crinetics Pharmaceuticals (Nasdaq: CRNX) announced that the FDA has granted Orphan Drug Designation for paltusotine to treat acromegaly, a rare endocrine disease. This designation incentivizes development, potentially exempting the company from user fees and providing seven years of market exclusivity upon approval. The designation highlights the promising Phase 2 clinical trial results for paltusotine. Crinetics plans to report topline results for the ACROBAT trials later this year and initiate a Phase 3 trial in 2021.

Positive

• FDA granted Orphan Drug Designation for paltusotine, enhancing its market potential.
• Paltusotine aims to provide a once-daily oral treatment option for acromegaly patients, addressing a significant treatment gap.

Negative

• None.

SAN DIEGO, July 08, 2020 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced that the U.S. Food and Drug Administration (FDA) has granted paltusotine Orphan Drug Designation for the treatment of acromegaly.

The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies the sponsor of the drug for certain development incentives, that may include exemption from FDA prescription drug user fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. if the treatment is approved.

“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 Michael Monahan, Senior Director of Regulatory Affairs at Crinetics.

“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 Ajay Madan, Ph.D., Chief Development Officer of Crinetics.

About Paltusotine

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 $3.1 billion annually. Non-clinical chronic toxicology studies are complete and no dose limiting toxicity was identified at the maximum feasible doses in rats and dogs. Crinetics previously completed a Phase 1 trial that showed potent suppression of the growth hormone (GH) axis in healthy volunteers, which provided clinical proof-of-concept. In addition, the molecule’s observed plasma half-life of approximately 2 days suggested the potential for paltusotine for once daily oral administration. A subsequent Phase 1 trial showed that paltusotine was 70% orally bioavailable. Paltusotine is an investigational drug currently undergoing two Phase 2 clinical trials for the treatment of acromegaly, the ACROBAT Edge and Evolve trials. Paltusotine has been discovered, characterized, and developed by Crinetics and U.S. patents have been issued covering paltusotine to 2037.

About Acromegaly

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.

About Crinetics Pharmaceuticals

Crinetics Pharmaceuticals is a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine (formerly CRN00808), is an oral, selective nonpeptide somatostatin receptor type 2 biased agonist undergoing two Phase 2 clinical trials for the treatment of acromegaly, an orphan disease affecting more than 25,000 people in the United States. Crinetics plans to advance paltusotine into a Phase 3 trial in acromegaly and a Phase 2 trial for the treatment of carcinoid syndrome associated with neuroendocrine tumors in 2021. The company is also developing an oral nonpeptide somatostatin type 5 agonist for hyperinsulinism, as well as an oral nonpeptide ACTH antagonist for the treatment of Cushing’s disease, congenital adrenal hyperplasia and other diseases of excess ACTH. All of the company’s drug candidates are new chemical entities resulting from in-house drug discovery efforts and are wholly owned by the company. For more information, please visit www.crinetics.com.

Forward-Looking Statements

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 the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of the company’s product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Crinetics may use its available capital resources sooner than it expects; Crinetics’ ability to obtain and maintain intellectual property protection for its product candidates; and other risks described under the heading “Risk Factors” in documents the company files from time to time with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Crinetics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Contacts:
Marc Wilson
Chief Financial Officer
IR@crinetics.com
(858) 450-6464

Robert H. Uhl
Westwicke ICR
robert.uhl@westwicke.com
(858) 356-5932

FAQ

What is the significance of FDA's Orphan Drug Designation for CRNX?

The Orphan Drug Designation for paltusotine enhances its market potential and qualifies the company for certain development incentives.

When are the topline results for the ACROBAT trials expected to be announced?

Topline results for the ACROBAT clinical trials are expected to be announced in the fourth quarter of 2020.

What is paltusotine designed to treat?

Paltusotine is designed to treat acromegaly, a rare endocrine disease caused by a growth hormone-secreting tumor.

What are the market exclusivity benefits of the Orphan Drug Designation for CRNX?

If approved, the Orphan Drug Designation provides seven years of market exclusivity in the U.S. for paltusotine.

What is the expected timeline for the Phase 3 trial of paltusotine?

Crinetics plans to initiate the Phase 3 trial for paltusotine in the first half of 2021.