Adrenocortical Adenoma

MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today reported its results for the quarter and year ended December 31, 2023. Financial Results Fourth quarter revenue of $135.4 million, a 31 percent increase over the same period in 2022Full year 2023 revenue of $482.4 million, a 20 percent increase over 2022Reiterated 2024 revenue guidance of $600 – $630 millionFourth quarter net income of $31.4 million, compared to $16.6 million in fourth quarter 20222023 net income of $106.1 million, compared to $101.4 million in 2022 Cash and investments of $425.4 million as of December 31, 2023 “The medical field is increasingly recognizing that Cushing’s syndrome is much more prevalent than was previously assumed. Our strong 2023 commercial results reflect that physicians are more regularly screening for hypercortisolism. The results demonstrate our ability to support these physicians as they manage this complex disease. We are confident these trends will continue and are reiterating our 2024 revenue guidance of $600 – $630 million,” said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. Corcept’s fourth quarter 2023 revenue was $135.4 million, compared to $103.1 million in the fourth quarter of 2022. Revenue for the full year was $482.4 million, compared to $401.9 million in 2022. Diluted net income per common share was $0.28 in the fourth quarter of 2023, compared to $0.14 in the fourth quarter of 2022. For the full year, it was $0.94 compared to $0.87 in 2022. Cash and investments were $425.4 million at December 31, 2023 compared to $436.6 million at December 31, 2022. In 2023, Corcept paid $154.5 million to purchase its common stock in connection with the April 2023 tender offer, the exercise of employee stock options and vesting of restricted stock grants. Clinical Development “Our clinical development programs are advancing rapidly and will reach important milestones this year. We are on-track to submit our NDA for relacorilant in Cushing’s syndrome and will report data from our trials in Cushing’s syndrome (GRACE, GRADIENT and CATALYST), ovarian cancer (ROSELLA) and ALS (DAZALS),” added Dr. Belanoff. Cushing’s Syndrome GRACE – Phase 3 trial of relacorilant in patients with all etiologies of Cushing’s syndrome – results from open label and randomized withdrawal phases expected in the second quarterRelacorilant New Drug Application (NDA) – NDA submission for Cushing’s syndrome expected in the second quarter GRADIENT – Phase 3 trial of relacorilant in patients with Cushing’s syndrome caused by adrenal adenomas – enrollment continues; results expected in the second half of this yearCATALYST – Phase 4 trial examining the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes; patients with hypercortisolism may enter a randomized, double-blind, placebo-controlled study of Korlym – enrollment continues; preliminary prevalence phase results: 24% prevalence rate in the first 700 patients enrolled; final results from prevalence and treatment phases expected by year-end “We are on-track to submit our relacorilant NDA in the second quarter. Relacorilant has demonstrated tremendous promise as a treatment for patients with Cushing’s syndrome and we are eager to make it more broadly available,” said Bill Guyer, PharmD, Corcept’s Chief Development Officer. “Our CATALYST trial is the largest study ever conducted to establish the prevalence of hypercortisolism in patients with difficult-to-control diabetes. We expect CATALYST’s findings to greatly stimulate physicians’ efforts to screen for hypercortisolism in patients with difficult-to-control diabetes and to treat them when hypercortisolism is found. Almost all of these patients currently go undiagnosed. We believe that CATALYST will be a landmark study and look forward to presenting the final results from the prevalence phase at the American Diabetes Association’s 84th Scientific Sessions in June,” added Dr. Guyer. Oncology ROSELLA – Pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer – enrollment continues; results expected by year-endOpen-label, Phase 1b trial of relacorilant plus pembrolizumab in patients with adrenal cancer with cortisol excess – enrollment completed; results expected by mid-yearRandomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer in collaboration with the University of Chicago – enrollment continues “If our ROSELLA trial replicates the results of our Phase 2 trial in patients with platinum-resistant ovarian cancer (results published in The Journal of Clinical Oncology, June 2023), it will constitute a major medical advance and could establish the combination of relacorilant and nab-paclitaxel as a new standard of care for women with this devastating disease. We expect data from ROSELLA by the end of this year,” said Dr. Guyer. Amyotrophic Lateral Sclerosis (ALS) DAZALS – Randomized, double-blind, placebo-controlled, Phase 2 trial of dazucorilant in patients with ALS – enrollment continues; results expected by year-end “ALS is a lethal illness with an urgent need for better treatment. Dazucorilant showed great promise in animal models of ALS – improving motor performance and reducing neuroinflammation and muscular atrophy. Our DAZALS study is investigating dazucorilant’s potential to significantly improve the lives of patients with ALS. We expect data by the end of this year,” said Dr. Guyer. Non-alcoholic Steatohepatitis (NASH) MONARCH – Randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed NASH – enrollment continues “Miricorilant has the potential to greatly benefit the millions of patients with NASH. Our Phase 1b study demonstrated that miricorilant effectively reduces liver fat, improves liver health and key metabolic and lipid measures and is well-tolerated. We look forward to building on these promising results in our MONARCH study,” said Dr. Guyer. Conference Call We will hold a conference call on February 15, 2024, 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. Additionally, a listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of www.corcept.com. About Corcept Therapeutics For over 25 years, Corcept’s focus on cortisol modulation and its potential to treat patients across a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept’s advanced clinical trials are being conducted in patients with hypercortisolism, solid tumors, ALS and NASH. 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 Cushing’s syndrome. Corcept is headquartered in Menlo Park, California. For more information, visit www.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 that 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, our ability to operate our business; generate sufficient revenue to fund our activities; the availability of competing treatments for hypercortisolism, including generic versions of Korlym; our ability to obtain acceptable prices and adequate insurance coverage and reimbursement for Korlym; risks related to the development of relacorilant, dazucorilant, miricorilant and our other product candidates, including their clinical attributes, regulatory approvals, mandates, oversight and other requirements; the timing, cost and outcome of legal disputes and investigations; and the scope and protective power of our intellectual property. These and other risks 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: our continued revenue growth and 2024 revenue guidance; the rates of screening and treatment of hypercortisolism; cortisol modulation’s potential to treat many serious diseases; development of relacorilant as a treatment for Cushing’s syndrome and ovarian, adrenal and prostate cancer; the design, timing and expectations regarding our GRACE trial; the timing and outcome of relacorilant’s NDA in Cushing’s syndrome; the design, timing and expectations regarding our GRADIENT trial; the design, timing and expectations regarding our CATALYST trial; the timing and expectations of our ROSELLA trial and the potential for relacorilant plus nab-paclitaxel to become a standard of care; the timing and expectations of our DAZALS trial of dazucorilant in patients with ALS; the timing and substance of our MONARCH trial in patients with NASH, and the pace of enrollment, study design and timelines, and the accrual and attributes of clinical data, as well as the timing of regulatory submissions with respect to all of our development activities. We disclaim any intention or duty to update forward-looking statements made in this press release. CORCEPT THERAPEUTICS INCORPORATEDCONDENSED CONSOLIDATED BALANCE SHEETS(In thousands) December 31, 2023 December 31, 2022(1) (Unaudited) Assets Cash and investments$425,397 $436,619Trade receivables, net of allowances 41,123 31,057Insurance recovery receivable related to Melucci litigation 14,000 14,000Inventory 15,974 17,031Operating lease right-of-use asset 120 1,143Deferred tax assets, net 90,605 61,465Other assets 34,298 22,115Total assets$621,517 $583,430Liabilities and Stockholders’ Equity Accounts payable$17,396 $11,976Accrued settlement related to Melucci litigation 14,000 14,000Operating lease liabilities 151 1,143Other liabilities 83,265 54,469Stockholders’ equity 506,705 501,842Total liabilities and stockholders’ equity$621,517 $583,430 (1) Derived from audited financial statements at that date CORCEPT THERAPEUTICS INCORPORATEDCONDENSED CONSOLIDATED STATEMENTS OF INCOME(In thousands, except per share data) Three Months Ended Year Ended December 31, December 31, 2023 2022 2023 2022 Revenues Product revenue, net$135,405 $103,056 $482,375 $401,858 Operating expenses Cost of sales 1,876 1,480 6,481 5,385 Research and development 54,707 36,754 184,353 130,991 Selling, general and administrative 47,152 42,323 184,259 152,848 Settlement expense related to Melucci litigation — 14,000 — 14,000 Insurance recovery related to Melucci litigation — (14,000) — (14,000)Total operating expenses 103,735 80,557 375,093 289,224 Income from operations 31,670 22,499 107,282 112,634 Interest and other income 5,139 1,777 17,275 3,557 Income before income taxes 36,809 24,276 124,557 116,191 Income tax expense (5,454) (7,675) (18,417) (14,773)Net income$31,355 $16,601 $106,140 $101,418 Net income attributable to common stockholders$31,138 $16,553 $105,496 $101,288 Basic net income per common share$0.30 $0.15 $1.02 $0.95 Diluted net income per common share$0.28 $0.14 $0.94 $0.87 Weighted-average shares outstanding used in computing net income per common share Basic 102,455 107,700 103,560 106,787 Diluted 110,886 116,328 111,742 115,966 CONTACT: Investor inquiries: ir@corcept.com Media inquiries: Communications@corcept.com www.corcept.com

Initial data from the clinical trial is expected in 2024 PORTLAND, Ore.--(BUSINESS WIRE)-- Sparrow Pharmaceuticals, an emerging, clinical-stage biopharmaceutical company developing novel, targeted therapies to address unmet needs in both rheumatology and endocrinology, today announced that the first patient has been dosed in the Phase 2 ACSpire study (NCT number: NCT05436639) of SPI-62, a potent and selective HSD-1 inhibitor, for the treatment of autonomous cortisol secretion (ACS), a prevalent yet serious condition caused by the overproduction of cortisol from a benign tumor of the adrenal gland. This trial will complement the ongoing Phase 2 RESCUE study (NCT number: NCT05307328) for the treatment of ACTH-dependent Cushing’s syndrome. “ACS is a dangerous condition with high morbidity and risk of death, with patients often having unexplained metabolic disease, cardiovascular disease, or osteopenia, and experiencing a 35% higher rate of mortality over a five-year span,” said Frank Czerwiec, chief medical officer of Sparrow. “However, most patients don’t even know they have ACS and are only diagnosed after an appropriate endocrine workup following the discovery of an adrenal tumor on a CT or MRI scan for unrelated reasons. SPI-62 could represent the first treatment to be approved for this large yet underappreciated and underserved patient population.” The pilot, long-term, open-label study is evaluating the efficacy, safety, and pharmacological effect of SPI-62 in participants with hypercortisolism related to a benign adrenal tumor. The study will examine SPI-62's effect on morbidities of hypercortisolism including diabetes or impaired glucose tolerance, hyperlipidemia, hypertension, and osteopenia. Sparrow is actively enrolling up to 30 participants in ACSpire at sites in the United States, soon to be joined by sites in Romania, France, and the United Kingdom. Criteria for participation include adults with documented benign adrenal lesions with proven ACS, and documentation of treatment for, or evidence of, ongoing metabolic consequences for at least one of the following: hyperglycemia, hypertension, hyperlipidemia, or osteopenia, attributable to clinically significant hypercortisolism. Surgery as first-line therapy should be discussed with all eligible participants, who will be included only if they have failed or rejected available surgical therapy. To learn more about Sparrow Pharmaceuticals and its clinical trials, visit the website at . About Autonomous Cortisol Secretion Autonomous cortisol secretion (ACS) is caused by the overproduction of cortisol from a tumor of the adrenal gland. It is a somewhat milder form of hypercortisolism than Cushing’s syndrome, which is differentiated by a characteristic constellation of signs and symptoms. As patients with ACS lack these outward clinical features of Cushing’s but present with common, chronic conditions (diabetes, hypertension, high cholesterol, weakened bones), it is rarely diagnosed or addressed specifically. ACS is currently only discovered when the tumor shows up on a radiology scan for an unrelated health condition and the patient subsequently receives an adequate endocrine workup. As this infrequently occurs, most patients with ACS are dangerously exposed to excess cortisol for long periods, leading them to face major health complications, significant healthcare costs, and increased risk of early death. The signs and symptoms of ACS vary across a wide range of mental and physical effects – including diabetes, hypertension, bone fractures, and weight gain, as well as mood, cognition, and sleep disorders. About Sparrow Pharmaceuticals Sparrow Pharmaceuticals was founded to spare patients the ravages of steroids. Leveraging underappreciated scientific insights into glucocorticoid biology, the company is working to provide better treatment options for serious disorders of hypercortisolism, and to revolutionize the treatment of autoimmune and inflammatory conditions. Its lead product, SPI-62, is an oral, small molecule, novel therapeutic treatment designed to target the source of active intracellular glucocorticoids in key tissues.

Doctors have used a new type of CT scan to light up tiny nodules in a hormone gland and cure high blood pressure by their removal. The nodules are discovered in one-in-twenty people with high blood pressure. Doctors at Queen Mary University of London and Barts Hospital, and Cambridge University Hospital, have led research using a new type of CT scan to light up tiny nodules in a hormone gland and cure high blood pressure by their removal. The nodules are discovered in one-in-twenty people with high blood pressure. Published today in Nature Medicine, the research solves a 60-year problem of how to detect the hormone producing nodules without a difficult catheter study that is available in only a handful of hospitals, and often fails. The research also found that, when combined with a urine test, the scan detects a group of patients who come off all their blood pressure medicines after treatment. 128 people participated in the study of a new scan after doctors found that their Hypertension (high blood pressure) was caused by a steroid hormone, aldosterone. The scan found that in two thirds of patients with elevated aldosterone secretion, this is coming from a benign nodule in just one of the adrenal glands, which can then be safely removed. The scan uses a very short-acting dose of metomidate, a radioactive dye that sticks only to the aldosterone-producing nodule. The scan was as accurate as the old catheter test, but quick, painless and technically successful in every patient. Until now, the catheter test was unable to predict which patients would be completely cured of hypertension by surgical removal of the gland. By contrast, the combination of a 'hot nodule' on the scan and urine steroid test detected 18 of the 24 patients who achieved a normal blood pressure off all their drugs. The research, conducted on patients at Barts Hospital, Cambridge University Hospital, and Guy's and St Thomas's, and Universities of Glasgow and Birmingham, was funded by the National Institute for Health and Care Research (NIHR) and Medical Research Council (MRC) partnership, Barts Charity, and the British Heart Foundation. Professor Morris Brown, co-senior author of the study and Professor of Endocrine Hypertension at Queen Mary University of London, said: "These aldosterone-producing nodules are very small and easily overlooked on a regular CT scan. When they glow for a few minutes after our injection, they are revealed as the obvious cause of Hypertension, which can often then be cured. Until now, 99% are never diagnosed because of the difficulty and unavailability of tests. Hopefully this is about to change." Professor William Drake, co-senior author of the study and Professor of Clinical Endocrinology at Queen Mary University of London, said:"This study was the result of years of hard work and collaboration between centres across the UK. Much of the 'on the ground' energy and drive came from the talented research fellows who, in addition to doing this innovative work, gave selflessly of their time and energy during the national pandemic emergency. The future of research in this area is in very safe hands." In most people with Hypertension (high blood pressure), the cause is unknown, and the condition requires life-long treatment by drugs. Previous research by the group at Queen Mary University discovered that in 5-10% of people with Hypertension the cause is a gene mutation in the adrenal glands, which results in excessive amounts of the steroid hormone, aldosterone, being produced. Aldosterone causes salt to be retained in the body, driving up the blood pressure. Patients with excessive aldosterone levels in the blood are resistant to treatment with the commonly used drugs for Hypertension, and at increased risk of heart attacks and strokes.