SAN DIEGO - Neurocrine (NASDAQ:NBIX) Biosciences, Inc. (NASDAQ:NBIX) announced today that its CAHtalyst™ Adult Phase 3 study has met its primary and key secondary endpoints, indicating potential benefits for adults with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study focused on crinecerfont, an investigational treatment aimed at reducing glucocorticoid doses while maintaining androgen control.
The double-blind, placebo-controlled trial encompassed 182 participants over 24 weeks. Results showed that crinecerfont-treated participants achieved a significant reduction in glucocorticoid doses compared to the placebo group, with 62.7% of participants on crinecerfont reaching a physiologic glucocorticoid dose as opposed to 17.5% on placebo.
Moreover, crinecerfont demonstrated a significant decrease in androstenedione levels, an adrenal androgen, compared to an increase in the placebo group at Week 4. This reduction was maintained throughout the trial, even with the lowered glucocorticoid doses, suggesting a non-glucocorticoid mechanism for androgen control.
The implications of these findings are noteworthy as CAH patients often suffer from conditions related to high glucocorticoid levels, such as osteoporosis and diabetes, due to the standard treatment approach. Crinecerfont's ability to lower glucocorticoid dosing to more physiological levels while controlling androgen excess could potentially mitigate these comorbidities.
Short-term favorable trends were also observed in endpoints like body weight, insulin resistance, and glucose tolerance, alongside an increase in bone turnover markers, indicating a possible relief from glucocorticoid-induced suppression.
Crinecerfont was generally well-tolerated, with fatigue and headache being the most common adverse events. No serious adverse events were attributed to the treatment, and there were few discontinuations due to adverse events.
The results from this study, which have been published in The New England Journal of Medicine, support the submissions of two New Drug Applications to the U.S. Food and Drug Administration in April 2024.
The information in this article is based on a press release.
InvestingPro Insights
As Neurocrine Biosciences (NASDAQ:NBIX) makes strides with its CAHtalyst™ Adult Phase 3 study, investors are watching closely, and the latest metrics from InvestingPro offer a snapshot of the company's financial health. Neurocrine's market capitalization stands at a robust $13.63 billion USD, reflecting investor confidence in the company's growth trajectory. The company is trading at a high earnings multiple, with a P/E ratio of 35.83 and an adjusted P/E ratio for the last twelve months as of Q1 2024 at 32.25, suggesting that investors are anticipating future earnings growth.
The company's strong financial position is further underscored by a revenue growth of 23.99% over the last twelve months as of Q1 2024, pointing to a solid increase in sales. Additionally, Neurocrine has demonstrated an impressive gross profit margin of 68.54% in the same period, indicating efficient management and a strong market position for its products.
InvestingPro Tips highlight that Neurocrine Biosciences is expected to grow its net income this year, aligning with the positive outlook presented by the successful clinical trial results. With 9 analysts having revised their earnings downwards for the upcoming period, it suggests caution, yet the company's fundamentals remain strong. Neurocrine operates with a moderate level of debt and has liquid assets that exceed short-term obligations, which may provide financial flexibility for ongoing research and development activities.
For investors seeking more in-depth analysis, InvestingPro offers additional tips that can provide a comprehensive view of Neurocrine's financial and operational standing. There are 12 more InvestingPro Tips available at https://www.investing.com/pro/NBIX, which can help investors make more informed decisions. Additionally, users can take advantage of a special offer using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, unlocking valuable insights into Neurocrine Biosciences and other investment opportunities.
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