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SAN DIEGO - Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX) announced Thursday that the U.S. Food and Drug Administration has granted Orphan Drug Designation to atumelnant for the proposed treatment of classic congenital adrenal hyperplasia (CAH). The company, which maintains a strong financial position with cash reserves exceeding its debt obligations, continues to advance its drug development pipeline despite not being profitable in the last twelve months.
Atumelnant is described as the first and only small molecule adrenocorticotropic hormone (ACTH) receptor antagonist in clinical development. The once-daily oral medication works by selectively targeting the melanocortin type 2 receptor on the adrenal gland. According to InvestingPro analysis, while Crinetics trades at a high revenue multiple, analysts anticipate sales growth in the current year, suggesting market confidence in the company’s drug development pipeline. For deeper insights into Crinetics’ valuation and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
"Receiving Orphan Drug Designation from the FDA underscores the significant unmet need faced by people living with CAH," said Dana Pizzuti, Chief Medical and Development Officer of Crinetics, in a press release statement. While the company’s liquid assets currently exceed its short-term obligations, InvestingPro data indicates that analysts do not expect profitability this year, highlighting the typical development path of early-stage pharmaceutical companies.
In January, Crinetics reported positive results from its Phase 2 TouCAHn trial of atumelnant in adults with classic CAH. The study demonstrated substantial reductions in key biomarkers, including up to an 80% mean reduction in androstenedione, along with improvements in multiple clinical symptoms.
The company expects to begin randomizing participants in the CALM-CAH Phase 3 study for adults and the BALANCE-CAH Phase 2/3 study for pediatric patients in the second half of 2025.
CAH is caused by genetic mutations that impair cortisol synthesis, leading to high levels of ACTH and overproduction of androgens. Current treatment involves chronic glucocorticoid steroid supplementation, often at supraphysiologic levels, which can cause significant additional medical problems.
The FDA’s Orphan Drug Designation provides benefits that may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. if the treatment receives approval.
In other recent news, Crinetics Pharmaceuticals reported its Q2 2025 earnings, revealing an earnings per share (EPS) of -1.23, which was below the forecasted -1.1. However, the company exceeded revenue expectations, reporting $1 million compared to the forecast of $750,110. Analysts have adjusted their price targets for Crinetics, with Citizens JMP lowering its target to $86 from $90 while maintaining a Market Outperform rating. The adjustment was based on expectations for a modest initial launch of Crinetics’ acromegaly treatment, Palsonify. Stifel also revised its price target for Crinetics to $58 from $60, maintaining a Buy rating following the company’s earnings report. Stifel noted that Crinetics remains well-positioned in the rare endocrinology space, with a diversified pipeline and sufficient capital. The firm also highlighted that the September 2025 PDUFA date for paltusotine in acromegaly remains on track. These developments reflect the company’s ongoing transition into a commercial-stage entity.
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