Hydrocortisone

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat serious endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today reported its results for the quarter ended March 31, 2025. Financial Results “In the first quarter, we had another record number of prescriptions from new and existing prescribers, broadly distributed throughout the country. Growing physician awareness of hypercortisolism has resulted in increased screening and treatment of patients with this devastating disease. Our specialty pharmacy vendor began the quarter unable to fulfill this surge in demand, which negatively affected our first quarter financial results. Pharmacy operations improved substantially in March and April, with each month setting a record for tablets dispensed. We are reiterating our 2025 revenue guidance of $900 – $950 million,” said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. Corcept’s first quarter 2025 revenue was $157.2 million, compared to $146.8 million in the first quarter of 2024. First quarter 2025 operating expenses were $153.8 million, compared to $117.3 million in the same period last year. Net income was $20.5 million in the first quarter of 2025, compared to $27.8 million in the first quarter of 2024. Cash and investments were $570.8 million at March 31, 2025, compared to $603.2 million at December 31, 2024. The balance at March 31, 2025 reflects the acquisition of $43.3 million of common stock in the first quarter pursuant to the company’s stock repurchase program, net exercise of employee stock options and net vesting of restricted stock grants. Clinical Development “Our New Drug Application (NDA) for relacorilant in hypercortisolism is progressing towards approval by the end of this year. We will submit our NDA next quarter for relacorilant in platinum-resistant ovarian cancer. We expect that relacorilant will have a role in helping treat earlier stages of ovarian cancer and other tumors that express the glucocorticoid receptor and have already begun our next clinical trial, BELLA. Meanwhile, we are making progress in understanding the role of cortisol modulation to treat a broad range of other serious disorders, including ALS and Metabolic Dysfunction-Associated Steatohepatitis (MASH),” added Dr. Belanoff. Hypercortisolism (Cushing’s Syndrome) “The positive results from our pivotal GRACE, GRADIENT, long-term extension and Phase 2 studies provide powerful support for the NDA for relacorilant in hypercortisolism. Patients in these studies experienced clinically significant improvements in a wide array of the signs and symptoms of hypercortisolism, without the off-target effects and toxicities that accompany currently available treatments. Relacorilant has the potential to become the new standard of care for patients with hypercortisolism,” said Bill Guyer, PharmD, Corcept’s Chief Development Officer. “CATALYST is a landmark study that will change the way physicians treat some of their sickest patients. Its findings are striking: One in four patients whose diabetes resists treatment with the best available medications have hypercortisolism; hyperglycemia in these patients responds powerfully to treatment with a cortisol modulator. We reached an important milestone with the publication of CATALYST’s prevalence phase results in Diabetes Care and look forward to presenting the full results of the study’s treatment phase at ADA next month,” added Dr. Guyer. “Building on the insights from CATALYST, our MOMENTUM study will deepen physicians' understanding of hypercortisolism as a cause of resistant hypertension.” Oncology “The ROSELLA results are an important advance for patients with platinum-resistant ovarian cancer, a disease with few treatment options. The PFS and OS improvements demonstrated in ROSELLA, with no increase in safety burden, bring us closer to delivering a new standard of care for these patients. We look forward to presenting the full results from ROSELLA in a late-breaker session at ASCO and submitting our NDA next quarter,” said Dr. Guyer. “We are building on the findings from ROSELLA with our BELLA study, which will examine whether combining relacorilant with two medications – nab-paclitaxel and bevacizumab – will offer patients with platinum-resistant ovarian cancer another potent treatment option.” Amyotrophic Lateral Sclerosis (ALS) “ALS is a devastating disease. Patients who received dazucorilant did not show improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R), which was DAZALS’ primary endpoint. An improvement in overall survival, first seen at the six-month mark, was also observed at year one of the study. An exploratory analysis determined that patients who received 300 mg of dazucorilant at the start of the study lived significantly longer than patients who received placebo and did not switch to dazucorilant in the long-term extension study, with a hazard ratio of 0.16 (p-value: 0.0009). This long-term extension study is on-going. We will immediately seek input from U.S. and European regulatory authorities on the next steps with dazucorilant,” said Dr. Guyer. Metabolic Dysfunction-Associated Steatohepatitis (MASH) “In our Phase 1b study, miricorilant reduced liver fat very rapidly, improved liver health and key metabolic and lipid measures and was well-tolerated. We look forward to building on these promising results in our MONARCH study. First results are expected by the end of next year,” said Dr. Guyer. Conference Call We will hold a conference call on May 5, 2025, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller. A listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of Corcept.com. About Corcept Therapeutics For over 25 years, Corcept has focused on cortisol modulation and its potential to treat patients with a wide variety of serious disorders, leading to the discovery of more than 1,000 proprietary selective cortisol modulators and glucocorticoid receptor antagonists. Corcept is conducting advanced clinical trials in patients with hypercortisolism, solid tumors, ALS and liver disease. In February 2012, the company introduced Korlym, the first medication approved by the U.S. Food and Drug Administration for the treatment of patients with endogenous hypercortisolism. Corcept is headquartered in Redwood City, California. For more information, visit Corcept.com. Forward-Looking Statements Statements in this press release, other than statements of historical fact, are forward-looking statements based on our current plans and expectations and are subject to risks and uncertainties that might cause our actual results to differ materially from those such statements express or imply. These risks and uncertainties include, but are not limited to, those related to our ability to: operate our business; study and develop Korlym, relacorilant, miricorilant, dazucorilant and our other product candidates; our investigational compounds’ clinical attributes, regulatory approvals, mandates, oversight and other requirements; and the scope and protective power of our intellectual property. These risks and uncertainties are set forth in our SEC filings, which are available at our website and the SEC’s website. In this press release, forward-looking statements include those concerning: trends in medical practice, including trends regarding the identification and treatment of patients with hypercortisolism; our 2025 revenue guidance and factors that may affect our revenue and continued revenue growth, such as increased uptake or price reductions in competing medications, including generic versions of Korlym, and the performance of our third-party pharmacy and other vendors; relacorilant as a treatment for patients with hypercortisolism and ovarian and other cancers, dazucorilant as a treatment for patients with ALS, miricorilant as a treatment for patients with MASH; the timing and outcome of relacorilant’s NDAs and planned MAA in hypercortisolism and ovarian cancer; the timing and outcome of our CATALYST, MOMENTUM, ROSELLA, BELLA, DAZALS and MONARCH trials and their impact on patient care and Corcept’s commercial prospects; and the accrual and attributes of our clinical data. We disclaim any intention or duty to update forward-looking statements made in this press release. CORCEPT THERAPEUTICS INCORPORATED CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) March 31, 2025 December 31, 2024(1) (Unaudited) Assets Cash and investments $ 570,804 $ 603,165 Trade receivables, net of allowances 61,819 53,976 Inventory 15,534 15,995 Operating lease right-of-use asset 5,147 5,324 Deferred tax assets, net 144,445 130,914 Other assets 48,706 31,179 Total assets $ 846,455 $ 840,553 Liabilities and Stockholders’ Equity Accounts payable $ 27,947 $ 15,376 Operating lease liabilities 6,835 6,936 Other liabilities 128,388 138,652 Stockholders’ equity 683,285 679,589 Total liabilities and stockholders’ equity $ 846,455 $ 840,553 (1) Derived from audited financial statements at that date CORCEPT THERAPEUTICS INCORPORATED CONDENSED CONSOLIDATED STATEMENTS OF INCOME (In thousands, except per share data) Three Months Ended March 31, 2025 2024 Revenues Product revenue, net $ 157,214 $ 146,808 Operating expenses Cost of sales 2,403 2,535 Research and development 60,735 58,505 Selling, general and administrative 90,660 56,268 Total operating expenses 153,798 117,308 Income from operations 3,416 29,500 Interest and other income 6,202 5,493 Income before income taxes 9,618 34,993 Income tax benefit (expense) 10,929 (7,231 ) Net income $ 20,547 $ 27,762 Net income attributable to common stockholders $ 20,288 $ 27,514 Basic net income per common share $ 0.19 $ 0.27 Diluted net income per common share $ 0.17 $ 0.25 Weighted-average shares outstanding used in computing net income per common share Basic 104,106 102,791 Diluted 119,819 109,915

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.