Corcept Plunges 47% After FDA Rejects Key Cushing's Syndrome Drug
Regulator issues a Complete Response Letter for relacorilant, demanding more data despite the drug meeting its main goals in a pivotal Phase 3 trial.
Shares of Corcept Therapeutics (NASDAQ: CORT) were nearly cut in half on Wednesday, plummeting 46.6% after the U.S. Food and Drug Administration unexpectedly rejected the company's application for its closely watched drug, relacorilant.
The stock closed at $37.46, erasing approximately $4 billion in market value and falling sharply below its 52-week low. The sell-off came after Corcept announced it had received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for relacorilant, an oral medication intended to treat hypercortisolism, commonly known as Cushing's syndrome.
The regulator's decision deals a significant blow to the Redwood City-based biopharmaceutical company, as relacorilant was positioned as the cornerstone of its future growth and a next-generation successor to its sole revenue-generating product, Korlym.
In a move that surprised investors and the company alike, the FDA determined that the application could not be approved in its present form. According to a company press release, the agency stated it could not reach a favorable benefit-risk assessment and required additional efficacy data before it would reconsider the drug for approval.
The rejection was particularly jarring given that Corcept’s pivotal Phase 3 GRACE trial had successfully met its primary endpoints. The trial demonstrated statistically significant improvements in both blood pressure and glucose metabolism—two common and dangerous consequences of Cushing's syndrome. The FDA acknowledged the trial's success on these endpoints but still concluded the evidence was insufficient.
“We are surprised and disappointed by this outcome,” said Joseph K. Belanoff, Corcept’s Chief Executive Officer, in a statement. “We believe our NDA, which includes positive results from two Phase 3 trials, provides compelling evidence of relacorilant’s benefits.”
Corcept's entire business model has so far relied on Korlym, another cortisol modulator that is expected to generate between $800 million and $850 million in revenue for the 2025 fiscal year. Relacorilant was critical to Corcept's strategy to diversify its revenue and defend against future competition. Without a clear timeline for relacorilant's approval, the company's pipeline now appears barren, with no other late-stage drug candidates expected to reach the market in the near future for this indication, as reported by analysts.
The severe market reaction reflects the high stakes involved. Prior to the announcement, Wall Street had been largely bullish, with a consensus analyst price target of $127.20. Following the CRL, analysts are scrambling to reassess their models. UBS, for instance, has already issued a "Neutral" rating, signaling deep uncertainty about the company's path forward.
Cushing’s syndrome is a rare and serious endocrine disorder caused by prolonged exposure to high levels of the hormone cortisol, leading to weight gain, metabolic issues, and high blood pressure. Relacorilant was designed as a selective glucocorticoid receptor antagonist to mitigate these effects without the side effects associated with Korlym.
Corcept Therapeutics stated it plans to request a meeting with the FDA as soon as possible to discuss the issues raised in the CRL and determine the next steps to advance relacorilant. However, the path to approval is now substantially longer and more costly, likely requiring at least one additional clinical study, a process that could take years to complete.