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Business Wire
14-07-2025
- Health
- Business Wire
U.S. FDA Approves KERENDIA ® (finerenone) to Treat Patients With Heart Failure With Left Ventricular Ejection Fraction ≥40% Following Priority Review
WHIPPANY, N.J.--(BUSINESS WIRE)--Bayer today announced that the U.S. Food and Drug Administration (FDA) approved KERENDIA ® (finerenone) to treat patients with heart failure (HF) with left ventricular ejection fraction (LVEF) ≥40%, 1 following the agency's Priority Review of its supplemental New Drug Application (sNDA). Even with guideline-directed medical treatment, the approximately 3.7 million adults in the U.S. with HF with LVEF ≥40%, also known as mildly reduced ejection fraction (HFmrEF) and preserved ejection fraction (HFpEF), face high rates of hospitalization for HF, impacting survival and placing considerable strain on healthcare systems. 2 Each hospitalization is associated with a more than two-fold increase in patients' risk of cardiovascular (CV) death. 3 The approval was based on results from the Phase III FINEARTS-HF trial, which showed that, on top of standard of care, KERENDIA achieved a 16% relative risk reduction of the composite primary endpoint of CV death and total HF events, defined as hospitalization for HF or an urgent HF visit, compared to placebo on top of standard of care (RR=0.84, 95% CI: 0.74-0.95, p=0.007). 1 The treatment effect was consistent across all prespecified subgroups including with or without SGLT2i use. 1 KERENDIA is a non-steroidal mineralocorticoid receptor antagonist (nsMRA) that selectively and potently blocks mineralocorticoid receptor overactivation in the heart and kidneys, 1,4,5 taking aim at HF with LVEF ≥40% from a different angle. 1 The overall safety profile of KERENDIA was consistent across all studied indications. 1 From FINEARTS-HF, the adverse reactions reported in ≥1% of patients on KERENDIA and more frequently than placebo were hyperkalemia (9.7% vs 4.2%), hypotension (7.6% vs 4.7%), hyponatremia (1.9% vs 0.9%), 6 and events related to worsening renal function (18% vs 12%). 1 Detailed results from the Phase III FINEARTS-HF trial were published in the New England Journal of Medicine. 7 'The FDA's approval of finerenone expands treatment options for patients with heart failure with a left ventricular ejection fraction of ≥40% – a large and growing group of patients with a poor prognosis,' said Scott D. Solomon, MD, Professor of Medicine, Harvard Medical School, Director, Clinical Trials Outcomes Center, Mass General Brigham, and Chair of the Executive Committee for the FINEARTS-HF study. 'Based on the clinical efficacy we saw in the FINEARTS-HF study, finerenone can become a new pillar of comprehensive care.' 'People with heart failure with left ventricular ejection fraction ≥40% face the very real possibilities of hospitalization for heart failure or CV death due to their disease,' 2,3 said Alanna Morris-Simon, MD, MSc, Senior Medical Director of U.S. Medical Affairs at Bayer. 'Even with current treatments, 21% of patients with symptomatic heart failure escalate to hospitalization for heart failure or CV death, 8 and 25% who experience hospitalization are readmitted due to heart failure within one year of discharge. 9 Now, as a core pillar of treatment, KERENDIA can help patients reduce these risks.' Since July 2021, KERENDIA has been approved to reduce the risk of CV death, hospitalization for HF, non-fatal myocardial infarction (MI), sustained estimated glomerular filtration rate (eGFR) decline, and end-stage kidney disease in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D). 1 This new indication expands KERENDIA's established cardiovascular benefit to a new patient population not limited to CKD associated with T2D—adult patients with HF with LVEF ≥40%. 1 About FINEARTS-HF 10 The FINEARTS-HF trial, a randomized, double-blind, placebo-controlled, multicenter, event-driven Phase III trial evaluated the efficacy and safety of KERENDIA for the reduction of risk of CV death and total HF events in patients with a diagnosis of symptomatic HF (New York Heart Association class II-IV) with an LVEF ≥40%, measured by local imaging within the last 12 months as well as receiving diuretic treatment for at least 30 days prior to randomization. The primary endpoint of FINEARTS-HF was the composite of CV death and total (first and recurrent) HF events, defined as hospitalizations for HF or urgent HF visits. Approximately 6,000 participants were randomized to receive either KERENDIA or placebo once daily for up to 42 months in addition to their background HF therapies. The FINEARTS-HF trial is part of KERENDIA's MOONRAKER program, which is expected to be one of the largest HF study programs to date. With more than 15,000 patients worldwide, MOONRAKER aims to establish a comprehensive body of evidence for KERENDIA across a broad spectrum of patients and clinical settings. 11 About KERENDIA's Clinical Trial Program KERENDIA's clinical trial program—called FINEOVATE—currently comprises 10 Phase III studies with dedicated programs in HF (MOONRAKER) and CKD (THUNDERBALL). The MOONRAKER program includes FINEARTS-HF as well as the ongoing, collaborative, investigator-sponsored studies REDEFINE-HF, 12 CONFIRMATION-HF 13 and FINALITY-HF. 14 The THUNDERBALL CKD program consists of the completed studies FIDELIO-DKD and FIGARO-DKD as well as the ongoing investigational studies FIND-CKD, 15 FIONA, 16 FIONA-OLE, 17 and FINE-ONE. 18 About KERENDIA ® (finerenone) 1 INDICATIONS: KERENDIA (finerenone) is indicated to reduce the risk of: sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D) (10mg, 20mg tablets) cardiovascular death, hospitalization for heart failure, and urgent heart failure visits in adult patients with heart failure with left ventricular ejection fraction (HF LVEF) ≥40% (10mg, 20mg, 40mg tablets) IMPORTANT SAFETY INFORMATION 1 CONTRAINDICATIONS: Hypersensitivity to any component of this product 1 Concomitant use with strong CYP3A4 inhibitors 1 Patients with adrenal insufficiency 1 WARNINGS AND PRECAUTIONS: Hyperkalemia: KERENDIA can cause hyperkalemia. The risk for developing hyperkalemia increases with decreasing kidney function and is greater in patients with higher baseline potassium levels or other risk factors for hyperkalemia. 1 Measure serum potassium and eGFR in all patients before initiation of treatment with KERENDIA and dose accordingly. Do not initiate KERENDIA if serum potassium is >5 mEq/L. Measure serum potassium periodically during treatment with KERENDIA and adjust dose accordingly. More frequent monitoring may be necessary for patients at risk for hyperkalemia, including those on concomitant medications that impair potassium excretion or increase serum potassium. 1 Worsening of Renal Function in Patients with Heart Failure: KERENDIA can cause worsening of renal function in patients with heart failure. Rarely, severe events associated with worsening renal function, including events requiring hospitalization, have been observed. 1 Measure eGFR in all patients before initiation of treatment or with dose titration of KERENDIA and dose accordingly. Initiation of KERENDIA in patients with heart failure and an eGFR <25 mL/min/1.73 m 2 is not recommended. Measure eGFR periodically during maintenance treatment with KERENDIA in patients with heart failure. Consider delaying up-titration or interrupting treatment with KERENDIA in patients who develop clinically significant worsening of renal function. 1 MOST COMMON ADVERSE REACTIONS: CKD associated with T2D: From the pooled data of FIDELIO-DKD and FIGARO-DKD, the adverse reactions reported in ≥1% of patients on KERENDIA and more frequently than placebo were hyperkalemia (14% vs 6.9%), hypotension (4.6% vs 3%), and hyponatremia (1.3% vs 0.7%). 1 HF LVEF ≥40%: From FINEARTS-HF, the adverse reactions reported in ≥1% of patients on KERENDIA and more frequently than placebo were hyperkalemia (9.7% vs 4.2%), hypotension (7.6% vs 4.7%), and hyponatremia (1.9% vs 0.9%). 6 Events related to worsening renal function were reported more frequently in the KERENDIA group (18%) compared with placebo (12%). 1 DRUG INTERACTIONS: Strong CYP3A4 Inhibitors: Concomitant use of KERENDIA with strong CYP3A4 inhibitors is contraindicated. Avoid concomitant intake of grapefruit or grapefruit juice. 1 Moderate and Weak CYP3A4 Inhibitors: Monitor serum potassium during drug initiation or dosage adjustment of either KERENDIA or the moderate or weak CYP3A4 inhibitor, and adjust KERENDIA dosage as appropriate. 1 Strong and Moderate CYP3A4 Inducers: Avoid concomitant use of KERENDIA with strong or moderate CYP3A4 inducers. 1 Sensitive CYP2C8 Substrates at KERENDIA 40mg: Monitor patients more frequently for adverse reactions caused by sensitive CYP2C8 substrates if KERENDIA 40mg is co-administered with such substrates, since minimal concentration changes may lead to serious adverse reactions. 1 USE IN SPECIFIC POPULATIONS: Lactation: Avoid breastfeeding during treatment with KERENDIA and for 1 day after treatment. 1 Hepatic Impairment: Avoid use of KERENDIA in patients with severe hepatic impairment (Child Pugh C) and consider additional serum potassium monitoring with moderate hepatic impairment (Child Pugh B). 1 Please see the Prescribing Information for KERENDIA. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 'Health for all, Hunger for none,' the company's products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to Find more information at Follow us on Facebook: Follow us on X: @BayerPharma Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports, which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. 1 Bayer Pharmaceuticals. Kerendia (finerenone) [package insert]. U.S. Food and Drug Administration. Available at: Accessed July 1, 2025. 2 Bozkurt B, Ahmad T, Alexander K, et al. HF STATS 2024: Heart Failure Epidemiology and Outcomes Statistics An Updated 2024 Report from the Heart Failure Society of America. J Card Fail. 2025;31(1):66-116. doi:10.1016/ 3 Huusko J, et al. ESC Heart Fail. 2020;7(5):2406-2417. doi:10.1002/ehf2.12792. 4 Kolkhof P, Jaisser F, Kim SY, Filippatos G, Nowack C, Pitt B. Steroidal and Novel Non-steroidal Mineralocorticoid Receptor Antagonists in Heart Failure and Cardiorenal Diseases: Comparison at Bench and Bedside. Handb Exp Pharmacol. 2017;243:271-305. doi:10.1007/164_2016_76. Available at Accessed July 1, 2025. 5 Kolkhof P, Joseph A, Kintscher U. Nonsteroidal mineralocorticoid receptor antagonism for cardiovascular and renal disorders - New perspectives for combination therapy. Pharmacol Res. 2021;172:105859. doi:10.1016/ Available at Accessed July 1, 2025. 6 Data on file. 7 Solomon SD, McMurray JJV, Vaduganathan M, et al. Finerenone in Heart Failure with Mildly Reduced or Preserved Ejection Fraction. N Engl J Med. 2024;391(16):1475-1485. doi:10.1056/NEJMoa2407107. 8 Chatur S, Vaduganathan M, Claggett BL, et al. Outpatient Worsening Among Patients With Mildly Reduced and Preserved Ejection Fraction Heart Failure in the DELIVER Trial. Circulation. 2023;148(22):1735-1745. doi:10.1161/CIRCULATIONAHA.123.066506 9 Cheng RK, et al. Am Heart J. 2014;168(5):721-730. doi:10.1016/ 10 Study to Evaluate the Efficacy and Safety of Finerenone on Morbidity & Mortality in Participants With Heart Failure and Left Ventricular Ejection Fraction Greater or Equal to 40% (FINEARTS-HF). Clinical trial registration No. NCT 04435626. Accessed June 26, 2025. 11 Data on file. 12 A Study to Determine the Efficacy and Safety of Finerenone on Morbidity and Mortality Among Hospitalized Heart Failure Patients (REDEFINE-HF). Clinical trial registration No. NCT 06008197. Accessed March 10, 2025. 13 A Study to Determine the Efficacy and Safety of Finerenone and SGLT2i in Combination in Hospitalized Patients with Heart Failure (CONFIRMATION-HF) (CONFIRMATION). Clinical trial registration No. NCT06024746. Accessed March 10, 2025. 14 A Study to Evaluate Finerenone on Clinical Efficacy and Safety in Patients with Heart Failure Who are Intolerant or Not Eligible for Treatment with Steroidal Mineralocorticoid Receptor Antagonists (FINALITY-HF). Clinical trial registration No. NCT06033950. Accessed March 10, 2025. 15 A Trial to Learn How Well Finerenone Works and How Safe it is in Adult Participants With Non-diabetic Chronic Kidney Disease (FIND-CKD). Clinical trial registration No. NCT05047263. Accessed March 10, 2025. 16 A Study to Learn More About How Well the Study Treatment Finerenone Works, How Safe it is, How it Moves Into, Through and Out of the Body, and the Effects it Has on the Body When Taken With an ACE Inhibitor or Angiotensin Receptor Blocker in Children with Chronic Kidney Disease and Proteinuria (FIONA). Clinical trial registration No. NCT05196035. Accessed March 10, 2025. 17 A Study to Learn More About How Safe the Study Treatment Finerenone is in Long-term Use When Taken With an ACE Inhibitor or Angiotensin Receptor Blocker Over 18 Months of Use in Children and Young Adults From 1 to 18 Years of Age With Chronic Kidney Disease and Proteinuria (FIONA OLE). Clinical trial registration No. NCT05457283. Accessed March 10, 2025. 18 A Study to Learn How Well the Study Treatment Finerenone Works and How Safe it is in People With Long-term Decrease in the Kidneys' Ability to Work Properly (Chronic Kidney Disease) Together With Type 1 Diabetes (FINE-ONE). Clinical trial registration No. NCT05901831. Accessed March 10, 2025. Expand
Business Insider
21-06-2025
- Business
- Business Insider
Incyte announces FDA extends review period for Opzelura sNDA
The company states: 'Incyte (INCY) announced that the FDA has extended the review period for the supplemental New Drug Application, sNDA, for ruxolitinib cream, Opzelura, a topical Janus kinase inhibitor, for the treatment of children 2-11 years old with mild to moderate atopic dermatitis. The PDUFA action date has been extended by three months to September 19, 2025. The FDA extended the PDUFA action date to allow time to review additional chemistry, manufacturing and controls data on the 0.75% strength submitted by Incyte in response to a recent FDA information request.' Steven Stein, M.D., Chief Medical Officer, Incyte stated: 'We are confident in the potential of ruxolitinib cream to become an important non-steroidal, topical treatment option for pediatric patients with atopic dermatitis and we will continue to work closely with the FDA to ensure the Agency has all of the information needed to complete its review.' Confident Investing Starts Here:
Yahoo
10-03-2025
- Health
- Yahoo
MannKind Showcases Efficacy, Safety, and Mealtime Control Data from Recent Pediatric and Adult Studies of Afrezza® at the ATTD Conference, March 19-22
Five presentations affirm positive outcomes utilizing inhaled insulin An sNDA filing for Afrezza in pediatric population anticipated in 2025 DANBURY, Conn. and WESTLAKE VILLAGE, Calif., March 10, 2025 (GLOBE NEWSWIRE) -- MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products and delivery devices for patients with endocrine and orphan lung diseases, will showcase data from recent studies of inhaled insulin across five presentations at the 18th International Conference on Advanced Technologies and Treatments for Diabetes to be held March 19-22 in Amsterdam. 'Data from both the INHALE-1 pediatric and INHALE-3 adult studies continue to drive groundbreaking conversations around inhaled insulin,' said Dr. Kevin Kaiserman, Senior Vice President, Therapeutic Area Head, Endocrine Diseases for MannKind Corporation. "Afrezza continues to be an important treatment option for adults living with diabetes, including those utilizing MDI and AID, and we look forward to the study investigators' presentations at the ATTD meeting.' The following oral presentations are on the scientific program at ATTD 2025 to be held virtually and at the RAI Amsterdam Convention Center: What's New in Pulmonary Inhaled Insulin?Thursday, March 20, 2025 – 4:40 PM (CET) in Hall IPresenter: Dr. Irl B. Hirsch Inhaled Insulin in Pediatrics (INHALE-1 Peds Study)Thursday, March 20, 2025 – 4:40 PM (CET) in Hall IPresenter: Dr. Michael J. Haller Sustained Benefit from Use of Inhaled Insulin in the INHALE-3 Extension StudySaturday, March 22, 2025 – 11:05 AM (CET) at Station 4Presenter: Dr. Grazia Aleppo Post-Prandial Glucose Excursion with Inhaled Insulin in Youth Compared with Adults with Type 1 Diabetes (INHALE-1 Peds Study)Saturday, March 22, 2025 – 11:30 AM (CET) in Hall D2Presenter: Dr. Michael J. Haller Comparison of a Regimen of Inhaled Technosphere Insulin Plus Insulin Degludec Versus Usual Care in Adults with Type 1 DiabetesSaturday, March 22, 2025 – 11:30 AM (CET) in Hall D2Presenter: Dr. Anastasios Manessis In addition to the planned presentations, MannKind will host booth #40 in the exhibit hall (Hall I) throughout ATTD. Members of MannKind's Clinical Education Team will be available for scientific exchange in the medical section of the booth. For more information about ATTD programming, and/or to register to attend the conference (virtual or in person), please visit: About the INHALE-1 Pediatric StudyINHALE-1 is a Phase 3, randomized controlled trial in children and teenagers aged 4-17 with type 1 or type 2 diabetes to evaluate the efficacy and safety of inhaled insulin in combination with basal insulin versus multiple daily injections of rapid acting insulin in combination with basal insulin. The 26-week, open-label clinical trial randomized 230 pediatric subjects to one of two groups: Afrezza or multiple daily injections (MDI) of rapid acting insulin analog (RAA) in combination with basal insulin. The primary endpoint was a non-inferior change in HbA1c levels after 26 weeks. A 26-week extension phase in which all remaining MDI patients switched to Afrezza is still ongoing. Six-month results were announced by MannKind in December 2024, and 12-month findings are expected mid-year 2025. About the INHALE-3 Study (Adults)The INHALE-3 study was a 17-week, randomized controlled trial with a 13-week extension conducted across 19 U.S. sites. The study, which enrolled 141 patients (123 randomized), assigned participants over 18 years of age with T1D who are using MDI, an automated insulin delivery system, or a pump without automation to either continue their standard of care or initiate an insulin regimen of a daily basal injection plus Afrezza for boluses (mealtime and corrections). Subjects utilizing Afrezza (inhaled insulin) received a higher initial conversion dose than in the current U.S. product label. Both arms utilized continuous glucose monitoring to assess glucose control. The randomized control trial (RCT) included an inhaled insulin group that began with 62 subjects at randomization and 57 at 17 weeks; the usual care group consisted of 61subjects at randomization and 58 at 17 weeks. The 17-week results shared that the study met its primary efficacy endpoint of a non-inferior change in HbA1c between baseline and week 17 compared to the usual care group. At 17 weeks, those who utilized Afrezza (plus basal insulin) continued with it through the extension phase, and those who were on usual care switched over to Afrezza to week 30. The extension phase started with 45 subjects from the inhaled insulin group and 43 completed the extension; the usual care-to-Afrezza group started with 49 in the extension, with 42 completing. There was no control group in the extension phase. A1c levels were obtained at baseline, 17 and 30-weeks. The 30-week results from the study expanded upon the positive 17-week data and showed that more people living with T1D were able to reach target A1c levels when they remain on Afrezza (plus basal insulin) or switch to Afrezza from usual care. About AfrezzaAfrezza (insulin human) Inhalation Powder is a rapid-acting inhaled human insulin indicated to improve glycemic control in adults with diabetes mellitus. Limitations of Use: Not recommended for the treatment of diabetic ketoacidosis or in patients that smoke or have recently stopped smoking. Important Safety Information WARNING: RISK OF ACUTE BRONCHOSPASM IN PATIENTS WITH CHRONIC LUNG DISEASE Acute bronchospasm has been observed in Afrezza-treated patients with asthma and COPD Afrezza is contraindicated in patients with chronic lung disease such as asthma or COPD Before initiating Afrezza, perform a detailed medical history, physical examination, and spirometry (FEV1) to identify potential lung disease in all patients. Most common adverse reactions are hypoglycemia, cough, and throat pain or irritation. Please see additional Important Safety Information, Full Prescribing Information, including BOXED WARNING, available on About MannKindMannKind Corporation (Nasdaq: MNKD) focuses on the development and commercialization of innovative inhaled therapeutic products and devices to address serious unmet medical needs for those living with endocrine and orphan lung diseases. We are committed to using our formulation capabilities and device engineering prowess to lessen the burden of diseases such as diabetes, nontuberculous mycobacterial (NTM) lung disease, pulmonary fibrosis, and pulmonary hypertension. Our signature technologies – dry-powder formulations and inhalation devices – offer rapid and convenient delivery of medicines to the deep lung where they can exert an effect locally or enter the systemic circulation, depending on the target indication. With a passionate team of Mannitarians collaborating nationwide, we are on a mission to give people control of their health and the freedom to live life. Please visit to learn more, and follow us on LinkedIn, Facebook, X or Instagram. Forward-Looking StatementsThis press release contains forward-looking statements about planned presentations at a scientific conference and a potential sNDA submission for Afrezza that involves risks and uncertainties. Words such as 'believes', 'anticipates', 'plans', 'expects', 'intends', 'will', 'goal', 'potential' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon MannKind's current expectations. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risk that issues that develop in the review by the FDA may subject us to unanticipated delays or prevent us from obtaining the expanded indication as well as other risks detailed in MannKind's filings with the Securities and Exchange Commission, including under the 'Risk Factors' heading of its Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent periodic reports on Form 10-Q and current reports on Form 8-K. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. All forward-looking statements are qualified in their entirety by this cautionary statement, and MannKind undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this press release. AFREZZA and MANNKIND are registered trademarks of MannKind Corporation. CONTACT: For MannKind: Media Relations Christie Iacangelo, (818) 292-3500 Email: media@ Investor Relations Ana Kapor, (818) 661-5000 Email: ir@ in to access your portfolio
Yahoo
24-02-2025
- Business
- Yahoo
Travere Therapeutics price target raised to $30 from $24 at TD Cowen
TD Cowen raised the firm's price target on Travere Therapeutics (TVTX) to $30 from $24 and keeps a Buy rating on the shares. The firm said Q4 Filspari results were in line with the preannouncement with impressive 40% quarter-over-quarter sales growth and a 37% quarter-over quarter increase in PSFs. Filspari growth in IgAN should continue in 2025 as there are multiple tailwinds and the FSGS sNDA submission should occur by the end of Q1 setting up a potential approval by year-end. See what stocks are receiving strong buy ratings from top-rated analysts. Filter, analyze, and streamline your search for investment opportunities with TipRanks' Stock Screener. Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on TVTX: Travere Therapeutics Posts Strong 2024 Financial Results Strong Growth Potential and Strategic Advancements Drive Buy Rating for Travere Therapeutics Closing Bell Movers: Booking gains 3%, Akamai down 8% on earnings Is TVTX a Buy, Before Earnings? Travere Therapeutics price target raised to $47 from $45 at Guggenheim Sign in to access your portfolio
Associated Press
21-02-2025
- Business
- Associated Press
Intra-Cellular Therapies Reports Fourth Quarter and Full-Year 2024 Financial Results
Fourth quarter 2024 CAPLYTA net product sales grew to $199.2 million, representing a 51% increase over the same period in 2023 Full year 2024 CAPLYTA net product sales were $680.5 million, representing year-over-year growth of 47% The U.S. Food and Drug Administration (FDA) accepted for review the lumateperone supplemental New Drug Application (sNDA) submission for adjunctive treatment of major depressive disorder (MDD) BEDMINSTER, N.J., Feb. 21, 2025 (GLOBE NEWSWIRE) -- Intra-Cellular Therapies, Inc. (Nasdaq: ITCI), a biopharmaceutical company focused on the development and commercialization of therapeutics for central nervous system (CNS) disorders, today announced its financial results for the fourth quarter ended December 31, 2024 and provided a corporate update. Financial Highlights Net product sales of CAPLYTA were $680.5 million for the full year 2024. This represents an increase of 47% compared to 2023. Net product sales of CAPLYTA were $199.2 million for the fourth quarter of 2024, compared to $131.5 million for the same period in 2023, representing 51% growth. Selling, general and administrative (SG&A) expenses were $504.5 million for the year ended December 31, 2024, compared to $409.9 million for the same period in 2023. This increase is primarily due to an increase in commercialization, marketing and infrastructure costs. Research and development (R&D) expenses were $236.1 million for the year ended December 31, 2024, compared to $180.1 million for the same period in 2023. This increase is primarily due to higher lumateperone and non-lumateperone project costs, including the ITI-1284, ITI-214, and ITI-1500 programs. Cash, cash equivalents, investment securities, and restricted cash totaled $1.0 billion on December 31, 2024, compared to $499.7 million at December 31, 2023. Commercial and Clinical Highlights In the first quarter of 2025, we commenced a field sales force expansion in anticipation of the potential approval of CAPLYTA for the adjunctive treatment of MDD. The FDA has accepted for review the sNDA for lumateperone, an investigational agent for the treatment of MDD as adjunctive therapy. Two positive Phase 3 global placebo-controlled studies, Study 501 and Study 502, as well as the long term open-label safety Study 503, form the basis of the sNDA. Advancing our pipeline: In 2024, we initiated 10 late-stage clinical trials including six Phase 3 lumateperone clinical trials and four ITI-1284 clinical trials. Lumateperone: In our pediatric program, in the fourth quarter of 2024, we commenced patient enrollment in two Phase 3 studies in pediatric patients for the treatment of irritability associated with autism spectrum disorder. Patient enrollment is ongoing in our double-blind, placebo-controlled study in bipolar depression and in our open-label safety study in schizophrenia and bipolar disorder in pediatric patients. Patient enrollment is ongoing in our two Phase 3 studies evaluating lumateperone in adults in the acute treatment of manic or mixed episodes associated with bipolar I disorder (bipolar mania). ITI-1284-ODT-SL program: Patient enrollment is ongoing in two Phase 2 clinical studies evaluating ITI-1284 in patients with generalized anxiety disorder (GAD). Our first study evaluates ITI-1284 as an adjunctive therapy to approved GAD medications while a second study evaluates ITI-1284 as monotherapy. Patient enrollment continues in a Phase 2 clinical study evaluating ITI-1284 in patients with psychosis associated with Alzheimer's disease (AD) and in our Phase 2 program in agitation associated with AD. Other pipeline programs: Phosphodiesterase type I inhibitor (PDE1) program: Patient enrollment in our lenrispodun (ITI-214) Phase 2 Study in Parkinson's disease (PD) is ongoing. Our second PDE1 inhibitor, ITI-1020 being developed in oncology indications, continues its Phase 1 single ascending dose study in healthy volunteers. ITI-1500 non-hallucinogenic neuroplastogen program: ITI-1549 is advancing IND enabling studies. Important Safety Information Boxed Warnings: Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. CAPLYTA is not approved for the treatment of patients with dementia-related psychosis. Antidepressants increased the risk of suicidal thoughts and behaviors in pediatric and young adults in short-term studies. All antidepressant-treated patients should be closely monitored for clinical worsening, and for emergence of suicidal thoughts and behaviors. The safety and effectiveness of CAPLYTA have not been established in pediatric patients. Contraindications: CAPLYTA is contraindicated in patients with known hypersensitivity to lumateperone or any components of CAPLYTA. Reactions have included pruritus, rash (e.g., allergic dermatitis, papular rash, and generalized rash), and urticaria. Warnings & Precautions: Antipsychotic drugs have been reported to cause: Cerebrovascular Adverse Reactions in Elderly Patients with Dementia-Related Psychosis, including stroke and transient ischemic attack. See Boxed Warning above. Neuroleptic Malignant Syndrome (NMS), which is a potentially fatal reaction. Signs and symptoms include: high fever, stiff muscles, confusion, changes in breathing, heart rate, and blood pressure, elevated creatinine phosphokinase, myoglobinuria (and/or rhabdomyolysis), and acute renal failure. Patients who experience signs and symptoms of NMS should immediately contact their doctor or go to the emergency room. Tardive Dyskinesia, a syndrome of uncontrolled body movements in the face, tongue, or other body parts, which may increase with duration of treatment and total cumulative dose. TD may not go away, even if CAPLYTA is discontinued. It can also occur after CAPLYTA is discontinued. Metabolic Changes, including hyperglycemia, diabetes mellitus, dyslipidemia, and weight gain. Hyperglycemia, in some cases extreme and associated with ketoacidosis, hyperosmolar coma or death, has been reported in patients treated with antipsychotics. Measure weight and assess fasting plasma glucose and lipids when initiating CAPLYTA and monitor periodically during long-term treatment. Leukopenia, Neutropenia, and Agranulocytosis (including fatal cases). Complete blood counts should be performed in patients with pre-existing low white blood cell count (WBC) or history of leukopenia or neutropenia. CAPLYTA should be discontinued if clinically significant decline in WBC occurs in absence of other causative factors. Decreased Blood Pressure & Dizziness. Patients may feel lightheaded, dizzy or faint when they rise too quickly from a sitting or lying position (orthostatic hypotension). Heart rate and blood pressure should be monitored and patients should be warned with known cardiovascular or cerebrovascular disease. Orthostatic vital signs should be monitored in patients who are vulnerable to hypotension. Falls. CAPLYTA may cause sleepiness or dizziness and can slow thinking and motor skills, which may lead to falls and, consequently, fractures and other injuries. Patients should be assessed for risk when using CAPLYTA. Seizures. CAPLYTA should be used cautiously in patients with a history of seizures or with conditions that lower seizure threshold. Potential for Cognitive and Motor Impairment. Patients should use caution when operating machinery or motor vehicles until they know how CAPLYTA affects them. Body Temperature Dysregulation. CAPLYTA should be used with caution in patients who may experience conditions that may increase core body temperature such as strenuous exercise, extreme heat, dehydration, or concomitant anticholinergics. Dysphagia. CAPLYTA should be used with caution in patients at risk for aspiration. Drug Interactions: CAPLYTA should not be used with CYP3A4 inducers. Dose reduction is recommended for concomitant use with strong CYP3A4 inhibitors or moderate CYP3A4 inhibitors. Special Populations: Newborn infants exposed to antipsychotic drugs during the third trimester of pregnancy are at risk for extrapyramidal and/or withdrawal symptoms following delivery. Dose reduction is recommended for patients with moderate or severe hepatic impairment. Adverse Reactions: The most common adverse reactions in clinical trials with CAPLYTA vs. placebo were somnolence/sedation, dizziness, nausea, and dry mouth. CAPLYTA is available in 10.5 mg, 21 mg, and 42 mg capsules. About CAPLYTA (lumateperone) CAPLYTA 42 mg is an oral, once daily atypical antipsychotic approved in adults for the treatment of schizophrenia and the treatment of depressive episodes associated with bipolar I or II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate. While the mechanism of action of CAPLYTA is unknown, the efficacy of CAPLYTA could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity at central dopamine D2 receptors. Lumateperone is being studied for the treatment of major depressive disorder, and other psychiatric and neurological disorders. Lumateperone is not FDA-approved for these disorders. About Intra-Cellular Therapies Intra-Cellular Therapies is a biopharmaceutical company founded on Nobel prize-winning research that allows us to understand how therapies affect the inner-workings of cells in the body. The company leverages this intracellular approach to develop innovative treatments for people living with complex psychiatric and neurologic diseases. For more information, please visit Forward-Looking Statements This news release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the potential approval of CAPLYTA (lumateperone) for the treatment of major depressive disorder as adjunctive therapy; our financial and operating performance, including our future revenues and expenses; our expectations regarding the commercialization of CAPLYTA; our plans to expand our sales force; our plans to conduct clinical or non-clinical trials and the timing of developments with respect to those trials, including enrollment, initiation or completion of clinical conduct, or the availability or reporting of results; whether clinical trial results will be predictive of future real-world results; whether CAPLYTA will serve an unmet need; the goals of our development programs; our beliefs about the potential utility of our product candidates; and development efforts and plans under the caption 'About Intra-Cellular Therapies.' All such forward-looking statements are based on management's present expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, the following: there is no guarantee we will complete the pending transaction with Johnson & Johnson within the timeframe we anticipate or at all; there are no guarantees that CAPLYTA will be commercially successful; we may encounter issues, delays or other challenges in commercializing CAPLYTA; whether CAPLYTA receives adequate reimbursement from third-party payors; the degree to which CAPLYTA receives acceptance from patients and physicians for its approved indications; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; results achieved in CAPLYTA in the treatment of schizophrenia and bipolar depression following commercial launch of the product may be different than observed in clinical trials, and may vary among patients; challenges associated with supply and manufacturing activities, which in each case could limit our sales and the availability of our product; risks associated with our current and planned clinical trials; we may encounter unexpected safety or tolerability issues with CAPLYTA following commercial launch for the treatment of schizophrenia or bipolar depression or in ongoing or future trials and other development activities; there is no guarantee that a generic equivalent of CAPLYTA will not be approved and enter the market before the expiration of our patents; there is no guarantee that our sNDA for the adjunctive treatment of MDD will be approved, if at all, on the timeline that we expect; our other product candidates may not be successful or may take longer and be more costly than anticipated; product candidates that appeared promising in earlier research and clinical trials may not demonstrate safety and/or efficacy in larger-scale or later clinical trials or in clinical trials for other indications; our proposals with respect to the regulatory path for our product candidates may not be acceptable to the FDA; our reliance on collaborative partners and other third parties for development of our product candidates; impacts on our business, including on the commercialization of CAPLYTA and our clinical trials, as a result of the COVID-19 pandemic, the conflicts in Ukraine, Russia and the Middle East, global economic uncertainty, inflation, higher interest rates or market disruptions; and the other risk factors detailed in our public filings with the Securities and Exchange Commission. All statements contained in this press release are made only as of the date of this press release, and we do not intend to update this information unless required by law. Contact: Intra-Cellular Therapies, Inc. Juan Sanchez, M.D. 646-440-9333 Burns McClellan, Inc. Cameron Radinovic 212-213-0006 INTRA-CELLULAR THERAPIES, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands except share and per share amounts) (Unaudited) (1)(2) Three Months Ended December 31, Twelve Months Ended December 31, 2024 2023 2024 2023 Revenues Product sales, net $ 199,223 $ 131,507 $ 680,501 $ 462,175 Grant revenue — 593 351 2,195 Total revenues, net 199,223 132,100 680,852 464,370 Operating expenses: Cost of product sales 20,405 10,703 56,963 33,745 Selling, general and administrative 137,729 104,720 504,489 409,864 Research and development 70,286 50,773 236,121 180,142 Total operating expenses 228,420 166,196 797,573 623,751 Loss from operations (29,197) (34,096) (116,721) (159,381) Interest income 11,995 6,242 42,518 20,343 Loss before provision for income taxes (17,202) (27,854) (74,203) (139,038) Income tax expense 317 (450) (473) (636) Net loss $ (16,885) $ (28,304) $ (74,676) $ (139,674) Net loss per common share: Basic & Diluted $ (0.16) $ (0.29) $ (0.72) $ (1.46) Weighted average number of common shares: Basic & Diluted 106,095,836 96,285,558 103,131,017 95,881,729 (1) The condensed consolidated statements of operations for the three and twelve months ended December 31, 2024 and 2023 have been derived from the financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements. (2) Some amounts in this statement may not add due to rounding. All percentages have been calculated using unrounded amounts. INTRA-CELLULAR THERAPIES, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands except share and per share amounts) (Unaudited) December 31, 2024 December 31, 2023 Assets Current assets: Cash and cash equivalents $ 306,948 $ 147,767 Investment securities, available-for-sale 694,118 350,174 Restricted cash 1,750 1,750 Accounts receivable, net 166,500 114,018 Inventory 26,283 11,647 Prepaid expenses and other current assets 111,765 42,443 Total current assets 1,307,364 667,799 Property and equipment, net 1,468 1,654 Right of use assets, net 13,428 12,928 Inventory, non-current 38,890 38,621 Other assets 5,762 7,293 Total assets $ 1,366,912 $ 728,295 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 26,074 $ 11,452 Accrued and other current liabilities 65,215 27,944 Accrued customer programs 75,408 53,173 Accrued employee benefits 34,774 27,364 Operating lease liabilities 4,233 3,612 Total current liabilities 205,704 123,545 Operating lease liabilities, non-current 12,748 13,326 Total liabilities 218,452 136,871 Stockholders' equity: Common stock, $0.0001 par value: 175,000,000 shares authorized at December 31, 2024 and December 31, 2023, respectively; 106,240,009 and 96,379,811 shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively 11 10 Additional paid-in capital 2,840,094 2,208,470 Accumulated deficit (1,691,836) (1,617,160) Accumulated comprehensive income 191 104 Total stockholders' equity 1,148,460 591,424 Total liabilities and stockholders' equity $ 1,366,912 $ 728,295 The condensed consolidated balance sheets at December 31, 2024 and December 31, 2023 have been derived from the financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.
