Financial Data and Key Metrics Changes - The company's revenue for 2025 was $761 million, an increase from $675 million in the previous year, with a revenue guidance for 2026 set at $900 million to $1 billion [3][4] - Net income for 2025 was $99.7 million, down from $141.2 million in the prior year [3] Business Line Data and Key Metrics Changes - The Cushing's syndrome business saw a 37% increase in tablets delivered compared to 2024, with a record number of new prescriptions and first-time prescribers [11][12] - Despite a 61% increase in new prescriptions, the gap between new prescriptions and tablets sold indicates capacity issues with the pharmacy vendor [11] Market Data and Key Metrics Changes - The transition to a new specialty pharmacy began in October 2025, aimed at addressing capacity issues and improving service delivery [12][55] - The company expects its Cushing's syndrome business to grow to at least $2 billion in annual revenue by the end of the decade, driven by increased recognition of hypercortisolism [14][26] Company Strategy and Development Direction - The company plans to appeal the FDA's decision regarding relacorilant and aims to make it available to patients as quickly as possible [10][26] - The focus remains on cortisol modulation to treat serious diseases, with ongoing research in oncology and metabolic conditions [15][21] Management's Comments on Operating Environment and Future Outlook - Management expressed surprise at the FDA's decision not to approve relacorilant, citing strong clinical trial data and alignment with FDA guidance during development [9][10] - The company is optimistic about the future growth of its Cushing's syndrome business and the potential for relacorilant to enhance treatment options [14][26] Other Important Information - The company reported cash and investments of $532 million as of December 31, 2025, reflecting stock repurchase activities [4] - The ROSELLA trial demonstrated a 35% reduction in risk of death for patients receiving relacorilant in combination with nab-paclitaxel, with a median overall survival increase of 4.1 months [19][20] Q&A Session Summary Question: Thoughts on the CRL and FDA's decision - Management believes the FDA may have shifted its expectations and plans to seek clarification in an upcoming meeting [34][38] Question: Guidance assumptions regarding Korlym - The net price for Korlym through the AG is about a 30% discount, with expectations for pricing pressures in 2026 [40] Question: Likelihood of running a randomized trial for relacorilant - Management indicated that the identification and treatment of hypercortisolism is crucial, but the path forward in clinical development remains uncertain [41][42] Question: Confidence in resolving supply chain issues - The transition to a new pharmacy partner has shown promising results, with improvements in service delivery metrics [55][56] Question: Safety profile of relacorilant - The safety profile remains consistent with previous analyses, showing no significant increases in ALT levels compared to nab-paclitaxel alone [60][62] Question: Impact of KEYTRUDA on treatment options - Management noted that KEYTRUDA's approval is limited by biomarker expression, but there is interest in maintaining treatment options for patients [64]

Financial Data and Key Metrics Changes - The company's revenue for 2025 was $761 million, an increase from $675 million in the previous year, with a revenue guidance for 2026 set between $900 million and $1 billion [3][4] - Net income for 2025 was $99.7 million, down from $141.2 million in the prior year [3] Business Line Data and Key Metrics Changes - The Cushing's syndrome business saw a record number of new prescriptions and a 37% increase in tablets delivered compared to 2024, despite a 61% increase in new prescriptions indicating capacity issues with the pharmacy vendor [11][12] - The transition to a new specialty pharmacy began in October 2025, which is expected to resolve previous capacity issues and improve performance [12] Market Data and Key Metrics Changes - The company anticipates its Cushing's syndrome business to grow to at least $2 billion in annual revenue by the end of the decade, driven by increased recognition of hypercortisolism and the approval of relacorilant [14][25] Company Strategy and Development Direction - The company is focused on cortisol modulation to treat serious diseases, with ongoing research in oncology and metabolic conditions [15][16] - The ROSELLA trial demonstrated significant benefits for patients with platinum-resistant ovarian cancer, leading to expectations of relacorilant becoming a new standard of care [19][20] - The company is exploring the use of glucocorticoid receptor antagonism in combination with other therapies, including immunotherapy and androgen deprivation therapy [21][22] Management's Comments on Operating Environment and Future Outlook - Management expressed surprise at the FDA's decision not to approve relacorilant, citing strong clinical data and alignment with FDA guidelines during the development process [10][33] - The company plans to meet with the FDA to understand the reasoning behind the decision and explore options for moving forward [10][37] Other Important Information - The company has completed the MOMENTUM trial, which examines hypercortisolism in patients with resistant hypertension, with results expected to be announced soon [17] - The company is also advancing its proprietary compounds for various conditions, including metabolic dysfunction-associated steatohepatitis (MASH) and amyotrophic lateral sclerosis (ALS) [22][24] Q&A Session Summary Question: Thoughts on the CRL and potential randomized trials for relacorilant - Management believes the FDA may have shifted its criteria for approval, expressing confidence in the data supporting relacorilant [33][36] - The likelihood of running a randomized trial for relacorilant in type 2 diabetes or hypertensive patients is uncertain, but management emphasizes the importance of identifying and treating hypercortisolism [40][41] Question: Guidance assumptions regarding Korlym and pricing - The net price for Korlym through the AG is approximately a 30% discount, with expectations for pricing pressures in 2026 [39] Question: Supply chain issues and confidence in new pharmacy partner - Management is confident in the new pharmacy's capabilities, citing their expertise and ability to scale operations effectively [54][55] Question: Safety profile of relacorilant and comparison with nab-paclitaxel - The safety profile of relacorilant in the ROSELLA trial is consistent with previous analyses, showing no significant added toxicity compared to nab-paclitaxel [60][61] Question: Impact of KEYTRUDA on treatment options - Management notes that KEYTRUDA's approval is limited to specific patient populations, and there is interest among oncologists in maintaining treatment options that include relacorilant [63]

Financial Data and Key Metrics Changes - Revenue for Q1 2025 was $157.2 million, an increase from $146.8 million in Q1 2024, representing a year-over-year growth of approximately 9% [5] - Net income decreased to $20.5 million in Q1 2025 from $27.8 million in Q1 2024 [5] - Cash and investments as of March 31, 2025, totaled $570.8 million [5] - The company reiterated its 2025 revenue guidance of $900 million to $950 million [5] Business Line Data and Key Metrics Changes - The prescriber base for hypercortisolism treatments has grown at a record rate for five consecutive quarters, with the number of prescriptions nearly doubling compared to the same period last year [7] - The average price per tablet decreased by 13% due to a transition from branded Korlym to an authorized generic, which has a lower net price [10] Market Data and Key Metrics Changes - The company has seen a substantial improvement in pharmacy operations in March and April 2025, leading to a record number of tablets dispensed [10] - The transition to authorized generic tablets is expected to continue, but the decrease in price will be offset by an increase in the number of tablets shipped [11] Company Strategy and Development Direction - The company is focused on increasing physician awareness and understanding of hypercortisolism, with plans to expand its sales force from 125 to 175 clinical specialists by year-end [8] - The new drug application for relacorilant in hypercortisolism is under FDA review, with an action date set for December 30, 2025 [15] - The company is also exploring the use of relacorilant in combination with other therapies for various cancers, including ovarian and prostate cancers [25][26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth prospects for hypercortisolism treatments, citing the CATALYST study's findings that one in four patients with difficult-to-control diabetes has hypercortisolism [18] - The company anticipates that the publication of full CATALYST treatment results will stimulate further growth in the second half of the year [43] - Management emphasized the potential for relacorilant to become the new standard of care for hypercortisolism patients [33] Other Important Information - The company is actively pursuing regulatory discussions regarding the next steps for its ALS treatment program following mixed results in a Phase II trial [27][66] - The company is preparing to submit relacorilant's NDA for platinum-resistant ovarian cancer in the next quarter [24] Q&A Session Summary Question: How does relacorilant fit into the treatment paradigm compared to Elaher? - Management indicated that relacorilant plus nab-paclitaxel is poised to become a new standard of care in platinum-resistant ovarian cancer, with potential use in multiple lines of therapy [40] Question: What is the expected revenue impact from the recent pharmacy operational improvements? - Management expects growth to continue through the second quarter and accelerate in the second half of the year, driven by strong fundamentals and initiatives [43][44] Question: Can you clarify the mix between brand and authorized generic for Korlym? - Currently, a little over half of patients are on the authorized generic, which is expected to increase, but volume growth is anticipated to outweigh any price changes [50][74] Question: Will there be an advisory committee for the December PDUFA? - Management does not expect an advisory committee for relacorilant, similar to previous approvals in hypercortisolism [51] Question: What are the next steps for relacorilant in solid tumors? - The company plans to rapidly expand studies for relacorilant in combination with other therapies in solid tumors, with a focus on earlier lines of therapy [53]

Financial Data and Key Metrics Changes - Revenue for Q1 2025 was $157.2 million, an increase from $146.8 million in Q1 2024, with a revenue guidance for 2025 set between $900 million and $950 million [5] - Net income decreased to $20.5 million in Q1 2025 from $27.8 million in Q1 2024 [5] - Cash and investments as of March 31, 2025, totaled $570.8 million [5] Business Line Data and Key Metrics Changes - The prescriber base and patient base for hypercortisolism treatments are growing rapidly, with new prescriber growth at a record rate for five consecutive quarters [7] - The number of prescriptions for Korlym in Q1 2025 was nearly double that of the same period last year [7] - The average price per tablet decreased by 13% due to a transition from branded Korlym to an authorized generic [11] Market Data and Key Metrics Changes - The increase in screening for hypercortisolism is driving rapid patient growth, with expectations for continued acceleration [8] - The transition to authorized generic tablets is expected to grow, but the decrease in price will be offset by an increase in the number of tablets shipped [12] Company Strategy and Development Direction - The company is focused on increasing physician awareness of hypercortisolism and expanding its sales force to support growth [8] - The new drug application for relacorilant in hypercortisolism is under FDA review, with an action date set for December 30, 2025 [16] - The company is exploring the use of cortisol modulation in various cancers, including ovarian and prostate cancers, and is preparing for potential regulatory submissions [22][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth prospects for hypercortisolism treatments, citing the CATALYST study's findings as a catalyst for increased screening and treatment [21] - The company anticipates that the operational improvements at the pharmacy vendor will support continued growth throughout 2025 [61] - Management emphasized the potential for relacorilant to become the new standard of care for hypercortisolism patients [33] Other Important Information - The company has made substantial progress in its oncology portfolio, with positive results from the ROSELA trial indicating potential for broader applications of cortisol modulation [22][33] - The company is actively exploring the potential of proprietary compounds for treating neurologic and hepatic diseases [28][30] Q&A Session Summary Question: Clarification on oncology program and treatment paradigm - Management indicated that relacorilant plus nab-paclitaxel is poised to become a new standard of care in platinum-resistant ovarian cancer, with potential positioning before or after biomarker-driven agents [40][41] Question: Revenue expectations for upcoming quarters - Management expects growth to continue through the second quarter and accelerate in the second half of the year, driven by strong fundamentals and the publication of full CATALYST data [45][46] Question: Mix between brand and authorized generic for Korlym - Currently, a little over half of patients are on the authorized generic, which is expected to increase, but volume growth is anticipated to outweigh any price changes [51][75] Question: Nature of corrective measures taken by pharmacy vendor - The pharmacy vendor has improved staffing and operations to meet increased demand, with March and April showing record dispensing numbers [60][61] Question: Expectations for ALS data and potential approval - Management plans to discuss further studies with regulatory authorities to determine the next steps for the ALS program [66]

Financial Data and Key Metrics Changes - The company's revenue for 2024 was $675 million, representing a 40% increase compared to the previous year [4] - Net income for 2024 was $141 million, an increase of 33% year-over-year [5] - Cash and investments at the end of 2024 were $603 million, up from $425 million at the end of the previous year [5] - The company provided revenue guidance for 2025 of $900 million to $950 million [5] Business Line Data and Key Metrics Changes - The number of new Korlym prescribers and patients receiving Korlym reached record levels throughout 2024 [10] - The CATALYST study indicated that hypercortisolism is more prevalent than previously assumed, with 1 in 4 patients with difficult-to-control type 2 diabetes affected [20] - In the CATALYST treatment phase, patients receiving Korlym showed a significant reduction in hemoglobin A1c by 1.47% compared to a 0.15% decrease in the placebo group [21] Market Data and Key Metrics Changes - The company noted that the prevalence of hypercortisolism is higher than historical estimates, with the FDA agreeing on the increased patient population [80] - The company anticipates that the market for hypercortisolism treatments could grow from $3 billion to $5 billion in annual revenues within 3 to 5 years [81] Company Strategy and Development Direction - The company is focused on increasing physician awareness of hypercortisolism and expanding its treatment offerings, including relacorilant [20][40] - A new drug application for relacorilant was submitted, based on positive results from clinical studies [12][41] - The company is also exploring relacorilant's potential in oncology, particularly in ovarian cancer and prostate cancer [24][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued revenue growth and the effectiveness of their treatments for hypercortisolism [5][39] - The operational challenges faced by their pharmacy partner were acknowledged, but management is optimistic about resolving these issues [78] - The company expects to see a significant impact from the CATALYST study data in the second half of the year and beyond [68] Other Important Information - The company is conducting multiple studies, including the ROSELLA study for ovarian cancer and the MOMENTUM study for resistant hypertension [27][23] - The safety profile of relacorilant was highlighted, with no serious adverse events reported [19] Q&A Session Summary Question: When should the company expect NDA acceptance for relacorilant? - The NDA was submitted on December 30, and the FDA has 60 days to review it for acceptance, with routine correspondence ongoing [49][50] Question: What is the status of the ROSELLA trial and the change in endpoints? - The change to dual primary endpoints was made in collaboration with the FDA, allowing for two chances for a positive outcome [74] Question: How is the CATALYST study impacting patient screening and treatment? - Initial impacts are being observed, with expectations for more significant changes in medical practice as more data is published [68]