CAHtalyst™ Met Primary Endpoint Demonstrating a Statistically Significant Decrease from Baseline Daily Glucocorticoid Dose with Androgen Control
Key Secondary Endpoint Achieved Statistically Significant Decrease in Androstenedione at Week 4 versus Placebo
Key Secondary Endpoint Demonstrated a Statistically Significant Number of Patients on Crinecerfont Achieved a Reduction to a Physiologic Glucocorticoid Dose versus Placebo
Crinecerfont Was Generally Well-Tolerated
The Phase 3 study met its primary endpoint at Week 24, demonstrating that treatment with crinecerfont resulted in a statistically significant percent reduction in daily glucocorticoid (GC) dose versus placebo while maintaining androgen control (p-value <0.0001).
The study also met important key secondary endpoints, with a statistically significant decrease in androstenedione at Week 4 versus placebo (p-value <0.0001). At Week 24, approximately 63% of patients on crinecerfont achieved a reduction to a physiologic GC dose versus approximately 18% on placebo (p-value <0.0001).
Crinecerfont was generally well tolerated. The most common adverse events during the double-blind, placebo-controlled period of the trial were fatigue, headache, and coronavirus infection. There were few serious adverse events, with none assessed as related to crinecerfont.
"I am gratified to see the extremely positive and clinically meaningful results from this study, the largest ever interventional trial conducted in this rare disease. It required a global effort, and the top-line results confirm our confidence in crinecerfont as a potential first-in-class medication and first-ever non-glucocorticoid treatment option for patients living with CAH," said
"CAH patients suffer from a number of debilitating symptoms and have had suboptimal treatment options with existing standard of care for their whole lives. These data, along with data from the open label treatment period, will allow us to proceed with our regulatory submissions to the FDA in 2024 and
"CAH is a difficult disorder to live with for patients and their caregivers, taking a huge toll physically and mentally," said Eiry Roberts, M.D., Chief Medical Officer at
Additional information regarding the results from the Phase 3 CAHtalyst study will be discussed at the Morgan Stanley 21st Annual Global Healthcare Conference at
Data from the Phase 3 CAHtalyst Pediatric Study will be available, as planned, in early Q4 2023.
About Classic Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) refers to a group of genetic conditions that result in an enzyme deficiency that alters the production of adrenal hormones which are essential for life. Approximately 95% of CAH cases are caused by a mutation that leads to deficiency of the enzyme 21-hydroxylase (21-OHD). In classic CAH, severe deficiency of this enzyme leads to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, classic CAH can result in salt wasting, dehydration, and even death.
There are currently no non-glucocorticoid treatments approved by the
Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenal androgens through a steroid-independent mechanism for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD). Antagonism of CRF type 1 receptors in the pituitary has been shown to decrease adrenocorticotropic hormone (ACTH) levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with classic CAH. Our data demonstrates that lowering androgen levels enables lower, more physiologic dosing of glucocorticoids and thus could potentially reduce the complications associated with exposure to greater than normal glucocorticoid doses in patients with classic CAH.
To learn more about crinecerfont, click here.
About the CAHtalyst™ Phase 3 Study in Adults
The CAHtalyst™ Phase 3 global registrational study was designed to evaluate the safety, efficacy, and tolerability of crinecerfont in adults (18 years of age and older) with classic congenital adrenal hyperplasia (CAH) due to 21-OHD. The study enrolled 182 female and male patients with CAH and consisted of a 24-week randomized, double-blind, placebo-controlled period followed by one-year of open-label crinecerfont treatment and optional open-label extension. The study started in
For more information about the CAHtalyst Phase 3 study in adults, please visit Clinical TrialsAdult.gov.
For more information about the CAHtalyst Pediatric Phase 3 study, please visit ClinicalTrialsPediatric.gov.
(*in collaboration with AbbVie)
NEUROCRINE, CAHtalyst, and the Neurocrine logo are registered trademarks of
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the clinical results from, and our future development plans with respect to, crinecerfont, as well as the therapeutic potential and clinical benefits or safety profile of crinecerfont. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements include: top-line data that we report may change following a more comprehensive review of the data related to the clinical studies and such data may not accurately reflect the complete results of the clinical study; risks that regulatory submissions for our products and/or product candidates may not occur or be submitted in a timely manner; our products and/or product candidates may not obtain regulatory approvals; or that the
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