Crinetics Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for CRN04777 for the Treatment of Congenital Hyperinsulinism
Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) announced that the FDA has granted rare pediatric disease designation for CRN04777, an investigational SST5 agonist aimed at treating congenital hyperinsulinism (HI). This serious condition causes life-threatening hypoglycemia in infants. The RPD designation may provide a priority review voucher upon approval. Dr. Christine Ferrara-Cook emphasized the critical nature of this condition, while CEO Scott Struthers highlighted the urgent need for new treatments. Crinetics plans a Phase 1 clinical study for CRN04777 in early 2021.
- FDA granted rare pediatric disease designation to CRN04777, indicating significant unmet medical need.
- Potential to receive a priority review voucher, enhancing future drug approval prospects.
- CRN04777 is the first oral SST5 agonist specifically designed to treat congenital HI.
- No guarantee of receiving a priority review voucher; the designation does not ensure approval.
- Clinical trial timelines are subject to delays, including those caused by COVID-19.
- Dependence on regulatory feedback for advancement into clinical trials presents uncertainty.
SAN DIEGO, Sept. 21, 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 rare pediatric disease designation (“RPD”) for CRN04777, an investigational, orally available, nonpeptide somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital hyperinsulinism (HI). Congenital HI is a devastating rare disease in which infants are born with life threatening hypoglycemia (low blood glucose) due to excess insulin secretion.
“Congenital HI is a serious condition of neonates, infants, and children. Failure to recognize and effectively treat this disease can lead to seizures, permanent neurological sequelae, and even death,” explained Christine Ferrara-Cook, M.D., Medical Director at Crinetics. “CRN04777 is the first oral, selective nonpeptide SST5 receptor agonist designed to reduce insulin secretion and is intended to be a universal treatment for all patients with congenital HI.” Dr. Ferrara-Cook is a pediatric endocrinologist and key member of the CRN04777 development team with an expertise in congenital HI that she gained through a decade in medicine and life sciences drug development.
A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act to include a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. In an effort to address the challenges drug companies face when developing treatments for these unique patient populations, the FDA developed the Rare Pediatric Disease Priority Review Voucher (“PRV”) Program. Under this program, companies are eligible to receive a priority review voucher following approval of a product with an RPD designation if the marketing application submitted for the product satisfies certain additional conditions*. If issued, a sponsor may redeem a PRV for priority review of a subsequent marketing application for a different product candidate, or the PRV could be sold or transferred to another sponsor.
“We are encouraged by the positive and collaborative interaction we had with the FDA regarding the clinical development of CRN04777 for congenital HI,” added Scott Struthers, Ph.D., founder and Chief Executive Officer of Crinetics. “The receipt of this RPD designation points to the seriousness of the disease for which there is an enormous need for new treatment options. Inspired by the bravery of the patients with congenital HI and their families, we are working diligently to ready CRN04777 for a Phase 1 clinical study in early 2021.”
About Congenital Hyperinsulinism
Hyperinsulinism is a heterogeneous condition in which dangerously low blood sugar levels are caused by increased insulin secretion from pancreatic ß-cells. Congenital HI is a severe form of hyperinsulinism driven by genetic mutations in certain genes involved in regulating insulin secretion. The incidence of congenital HI is approximately 1 in 30,000 to 50,000 new births in the United States. While this is a rare disease, congenital HI is a leading cause of persistent hypoglycemia in infants and children. Early diagnosis is vital to prevent neurological complications due to recurrent low blood sugar, which can result in apnea, seizures, developmental delays, learning disabilities, epilepsy, and even death.
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 NETs in 2021. The company is also developing CRN04777, an oral nonpeptide somatostatin receptor type 5 (SST5) 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.
*NOTE: Unless modified by Congress, the RPD Priority Review Voucher Program is currently expected to expire on September 30, 2020. A drug that has been granted RPD designation prior to this date is still eligible to receive the PRV as long as it receives final FDA approval before September 30, 2022.
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: Crinetics’ eligibility to receive a priority review voucher following approval of CRN04777 for the treatment of congenital HI; the potential of CRN04777 to have a meaningful impact on patient care; the potential to initiate Phase 1 trials of CRN04777 and the timing thereof; the potential of CRN04777 to be an effective treatment option for congenital HI patients; the potential for any of our ongoing clinical trials to show safety or efficacy; and the potential to initiate a pivotal Phase 3 trial of paltusotine in acromegaly or a Phase 2 trial for the treatment of carcinoid syndrome and the timing thereof. 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: Crinetics may not meet the eligibility criteria for a priority review voucher; potential delays in the commencement, enrollment and completion of clinical trials and the reporting of data therefrom; advancement of paltusotine into a Phase 3 trial for acromegaly or a Phase 2 trial for carcinoid syndrome and CRN04777 into a Phase 1 trial for congenital HI are dependent on and subject to the receipt of further feedback from the FDA and foreign regulatory agencies; 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; unexpected adverse side effects or inadequate efficacy of the company’s product candidates that may limit their development, regulatory approval and/or commercialization; 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 | Media: Aline Sherwood Scienta Communications asherwood@scientapr.com (312) 238-8957 |
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