Executive Summary / Key Takeaways

• Corcept Therapeutics is poised for significant growth, transitioning from a single-product company to a multi-asset biopharma leveraging its proprietary selective cortisol modulation platform across endocrinology and oncology.
• The core hypercortisolism business is experiencing rapid growth, driven by increasing physician awareness of the condition's high prevalence in difficult-to-control diabetes (1 in 4 patients per CATALYST study) and resistant hypertension, supported by an expanding sales force and educational initiatives.
• Relacorilant, a next-generation selective cortisol modulator, is under FDA review for hypercortisolism with a PDUFA date of December 30, 2025, offering a potentially superior safety profile compared to existing treatments, including Korlym, positioning it as a potential new standard of care.
• Pivotal Phase 3 ROSELLA trial success in platinum-resistant ovarian cancer demonstrated significant improvements in progression-free survival (30% risk reduction, HR 0.70) and overall survival (median OS 16 months vs. 11.5 months, HR 0.69) with relacorilant plus nab-paclitaxel, opening a major new oncology market opportunity with an NDA planned for Q2 2025.
• While facing generic competition for Korlym and operational challenges in Q1 2025, the company maintains strong 2025 revenue guidance of $900 million to $950 million, anticipating volume growth to offset price impacts and projecting relacorilant could generate $3 billion to $5 billion annually in hypercortisolism alone within 3-5 years.

The Cortisol Conundrum: A Platform for Growth

Corcept Therapeutics, founded in 1998, has built its foundation on understanding and modulating the effects of the hormone cortisol. Initially establishing a commercial presence with Korlym (mifepristone), approved by the FDA in 2012 for hyperglycemia secondary to hypercortisolism in specific adult patients, the company has since relied on this product's revenue to fuel its ambitious research and development efforts. This journey has led to the discovery of a proprietary library of over 1000 selective cortisol modulators, forming a platform aimed at addressing a range of severe endocrinologic, oncologic, metabolic, and neurologic disorders.

The market for hypercortisolism treatment, while historically considered niche, is undergoing a significant re-evaluation. Recent data, particularly from Corcept's own CATALYST study, reveal a much higher prevalence of the condition than previously assumed. The prevalence phase of CATALYST found that a striking 23.8% of patients with difficult-to-control type 2 diabetes also have hypercortisolism. This finding, coupled with ongoing investigation into its prevalence in resistant hypertension through the MOMENTUM trial, is fundamentally expanding the perceived treatable patient population and driving increased screening and diagnosis among physicians.

Corcept's core strategy is to leverage its deep expertise in cortisol biology and its extensive library of selective modulators to develop differentiated therapies. This approach is exemplified by relacorilant, a next-generation compound designed to selectively target the glucocorticoid receptor (GR) while avoiding the progesterone receptor (PR) affinity associated with Korlym. This technological distinction is critical, as it aims to provide the therapeutic benefits of cortisol modulation without the PR-mediated side effects seen with Korlym, such as endometrial thickening and vaginal bleeding. Clinical data for relacorilant also indicate it does not cause hypokalemia, adrenal insufficiency, or QT prolongation, adverse events that can occur with Korlym and other existing treatments for Cushing's syndrome. These quantifiable safety advantages position relacorilant as a potentially superior treatment option. The company's R&D efforts continue to explore new modulators with distinct properties, including tissue specificity and brain penetration, with the goal of advancing the most promising candidates to the clinic and further expanding the platform's reach.

In the competitive landscape for hypercortisolism, Corcept's Korlym competes with other approved medications like Recordati (RCDA)'s Signifor and Isturisa, Xeris (XERS)' Recorlev, and off-label generic ketoconazole. The recent launch of a generic mifepristone by Teva (TEVA), and the potential for others from companies like Sun and Hikma, presents a direct competitive challenge to Korlym's market share and pricing. Corcept has responded by making its own authorized generic available, a strategic move to maintain patient access and support services, although it contributes to a lower average net price. Despite this, the underlying market expansion driven by increased awareness and screening, coupled with Corcept's expanding sales force (targeting 175 clinical specialists by year-end 2025, up from 60 at the start of 2024), is expected to drive significant volume growth. While larger pharmaceutical companies like Pfizer (PFE), AbbVie (ABBV), and Novartis (NVS) have broader portfolios and greater scale, Corcept's focused expertise in cortisol modulation provides a competitive edge in this specific biological pathway, allowing it to develop potentially more targeted and safer therapies like relacorilant. Teva, primarily a generics player, competes on cost, posing a pricing pressure risk, but lacks the innovative pipeline in this specific area.

Performance Reflecting Strategic Investment and Market Expansion

Corcept's financial performance in the first quarter of 2025 reflects both the underlying strength of its growing business and temporary operational hurdles. Net product revenue increased to $157.2 million, up from $146.8 million in the first quarter of 2024. This growth was fueled by a substantial 23.5% increase in sales volume year-over-year, demonstrating the impact of increased screening and patient identification. However, this volume growth was partially offset by a 13.3% decrease in the average price per tablet, primarily due to the expected shift towards the authorized generic version of Korlym, which carries a lower net price.

Operating expenses saw significant increases as the company invested heavily in its future. Research and development expenses rose to $60.7 million in Q1 2025 from $58.5 million in Q1 2024, reflecting the advancement of its diverse clinical pipeline. Selling, general and administrative expenses saw a more pronounced jump, reaching $90.7 million compared to $56.3 million in the prior year period. This increase is directly attributable to the strategic build-out of the commercial infrastructure, including the expansion of the sales force, in anticipation of continued Korlym growth and the potential launch of relacorilant. The company's profitability metrics, such as operating and net margins, were impacted by these increased investments and the lower average selling price in the quarter.

Liquidity remains robust, with cash, cash equivalents, and marketable securities totaling $570.8 million as of March 31, 2025. While net cash provided by operating activities decreased in Q1 2025 compared to the prior year, reflecting the higher operating expenses, the company's strong cash position is sufficient to fund planned operations and R&D activities for the next 12 months and beyond without needing to raise additional capital. Financing activities in the quarter included significant cash used for share repurchases under the stock repurchase program and tax withholding related to equity awards, demonstrating a commitment to returning value to shareholders. Operationally, the rapid growth in the Korlym business in late 2024 and early 2025 temporarily strained the capacity of the company's specialty pharmacy vendor, leading to dispensing delays in January and February. Management addressed this issue through increased staffing, resulting in substantial improvements and record tablet dispensing in March and April, mitigating the operational challenge.

A Pipeline Poised for Transformation and a Bullish Outlook

Corcept stands at a pivotal juncture, with multiple late-stage pipeline assets nearing potential commercialization. The most immediate catalyst is relacorilant for endogenous hypercortisolism. Following positive data from the pivotal GRACE trial and supportive evidence from GRADIENT and the long-term extension study, the company submitted an NDA in December 2024, which the FDA accepted for filing in March 2025, setting a PDUFA date of December 30, 2025. The clinical data highlight relacorilant's ability to achieve clinically meaningful and statistically significant improvements across key manifestations of Cushing's syndrome, including hypertension, hyperglycemia, and weight, with a safety profile that avoids common and serious side effects associated with existing therapies. Management is highly confident in relacorilant's potential to become the new standard of care and projects it could generate substantial annual revenue of $3 billion to $5 billion in the hypercortisolism market alone within the next three to five years.

Beyond endocrinology, Corcept's cortisol modulation platform has yielded a significant breakthrough in oncology. The pivotal Phase 3 ROSELLA trial evaluating relacorilant in combination with nab-paclitaxel for platinum-resistant ovarian cancer met its dual primary endpoints in March 2025. The trial demonstrated a 30% reduction in the risk of disease progression (HR 0.70, p=0.0080) and a significant improvement in overall survival, with a median OS of 16 months compared to 11.5 months for nab-paclitaxel alone (HR 0.69, p=0.01). Importantly, the combination was well-tolerated with no new safety signals observed. These compelling results, consistent with prior Phase 2 data, pave the way for an NDA submission in platinum-resistant ovarian cancer planned for the second quarter of 2025, opening a major new therapeutic area and revenue stream for the company. Corcept has already established a standalone oncology division in anticipation of this launch and is exploring relacorilant in earlier lines of ovarian cancer (BELLA trial) and other solid tumors.

The pipeline also includes dazucorilant, a brain-penetrant cortisol modulator, which showed a statistically significant overall survival signal in an exploratory analysis of the Phase 2 DAZALS trial in ALS, despite not meeting the primary functional endpoint. The company is seeking regulatory guidance on next steps for this program. Miricorilant is advancing in the Phase 2b MONARCH trial for MASH, building on Phase 1b data showing reductions in liver fat and improvements in metabolic markers.

Corcept is reiterating its 2025 revenue guidance of $900 million to $950 million, reflecting confidence in the continued growth of the hypercortisolism business. Management anticipates revenue growth will accelerate in the second half of the year, driven by the expanding market, commercial initiatives, and the full impact of the CATALYST study data dissemination. Expected increases in R&D and SG&A expenses in 2025 underscore the ongoing investment in advancing the pipeline and preparing for potential new product launches.

Risks and Considerations

Despite the promising outlook, several risks warrant investor attention. The ongoing patent litigation with Teva regarding generic Korlym introduces uncertainty; while Corcept is appealing an unfavorable trial court ruling, a loss could significantly impact Korlym revenue. Even if successful, the costs and distraction of litigation are material. The increasing availability of generic mifepristone, including Corcept's own authorized generic, will continue to pressure the average net price of the hypercortisolism product, requiring sustained volume growth to meet revenue targets.

Regulatory risks are inherent in the biopharmaceutical industry. While relacorilant's NDA for hypercortisolism has been accepted, there is no guarantee of approval by the PDUFA date. Similarly, the planned oncology NDA and other pipeline assets face the risk of regulatory delays or rejections based on efficacy or safety findings. The Inflation Reduction Act poses a long-term risk by potentially limiting Medicare revenue and profitability starting in 2026 through price negotiation and inflation rebates. Increased scrutiny of pricing, reimbursement, and marketing practices, including potential allegations of off-label promotion or violations of fraud and abuse laws, could result in significant penalties or restrictions.

Operational risks, as highlighted by the recent pharmacy vendor issues, can impact product supply and revenue recognition. Dependence on third-party manufacturers and distributors also introduces potential vulnerabilities. Furthermore, the success of pipeline assets depends on favorable clinical trial outcomes, which are inherently uncertain. Data privacy regulations are becoming increasingly stringent globally, requiring costly compliance efforts and posing risks of significant fines or litigation in case of breaches.

Conclusion

Corcept Therapeutics is undergoing a significant transformation, leveraging its unique expertise and proprietary platform in selective cortisol modulation to target multiple high-need medical conditions. The core hypercortisolism business is demonstrating robust growth driven by increasing market awareness, while the late-stage pipeline, particularly relacorilant in both Cushing's syndrome and platinum-resistant ovarian cancer, presents substantial potential value drivers. The positive pivotal data in oncology is a game-changer, opening a major new market opportunity.

While challenges remain, including generic competition for Korlym and the inherent risks of drug development and regulatory approval, the company's strong cash position and clear strategic focus on differentiated therapies position it for potential long-term success. Investors should closely monitor the upcoming FDA decision for relacorilant in hypercortisolism, the planned oncology NDA submission and subsequent launch preparation, and the outcome of the generic litigation, as these events will be critical in shaping Corcept's trajectory and realizing the full potential of its cortisol modulation platform.