Autosomal dominant polycystic kidney disease (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or kidney transplant. To date, no effective and safe therapies exist for this deadly disease. Tolvaptan (Tol), the only FDA-approved drug for treatment of ADPKD, has some benefit in slowing kidney disease progression, but Tol causes frequent urination and thirst and also injures the liver in a small number of patients. The investigators' goal, therefore, is to develop new strategies to treat ADPKD that are safe and tolerable.
The development of cysts in ADPKD patients results from two main cellular processes. The first is cell growth with an increase in the number of kidney cells that make up the outer surface of the cyst. The second is an increase in fluid secretion into the cysts that develop. The investigators have shown that an enzyme, AMP-activated protein kinase (AMPK), when activated can inhibit both of those processes. Moreover, genetic mutations that cause ADPKD may alter the energy metabolism of the cell, which in turn inhibits AMPK activity. Bempedoic acid (BA), a medication that is FDA-approved for the treatment of individuals with high cholesterol and has a good safety record, activates AMPK. In addition to activating AMPK, BA inhibits a second enzyme called ATP-citrate lyase (ACLY), which is involved in cholesterol synthesis. ACLY has received growing attention as a novel target for cancer treatment. ACLY inhibition blocks increases of cell numbers by inhibiting the lipid synthesis that is required for creation of new cell membranes. This study will test whether targeting these pathways through treatment with BA will help reverse dysfunctional metabolism in individuals with ADPKD and slow disease progression.
The investigators will test this using a phase 2 clinical trial in which 120 individuals with rapidly progressive ADPKD and an estimated glomerular filtration rate of 35 or greater will be treated with either BA or placebo (inactive look-alike pill) for two years. Participants on or off a stable dose of Tol will be included in the study. Participants will be recruited from the U. of Vermont, U. of Maryland, and Tufts University, which have active PKD clinics and are recognized by the PKD Foundation as Centers of Excellence. Through follow-up visits and lab work, the investigators will assess the safety and tolerability of BA in the participants as the primary outcomes. The secondary goals are to assess preliminary efficacy and effects of BA on quality of life in study participants. The growth of cysts results in increased volume or size of the kidneys and liver. Total and cyst volumes of the kidney and liver and visceral abdominal fat content via magnetic resonance imaging (MRI) will be measured to gauge the effectiveness of this drug. The investigators also predict that proteins and small molecules involved in regulating cell energy metabolism, inflammation, and injury, as well as proteins directly involved in AMPK and ACLY function, will be altered in ADPKD patients. Levels of these proteins and small molecules may then subsequently change with BA therapy. Exploratory, mechanistic goals of this study are to identify prognostic and predictive urinary biomarkers in study participants. Successful completion of this study would have a significant impact on individuals with ADPKD by laying the groundwork for a new treatment strategy as well as by providing a new way to help guide treatment decisions.
In summary, the goals of this phase 2 randomized, double-blind, placebo-controlled clinical trial are to test the safety, tolerability and preliminary efficacy of the drug bempedoic acid, FDA-approved to lower cholesterol, when used in ADPKD patients.

Start Date21 Sep 2026

Sponsor / Collaborator

University of Vermont

[+5]

NCT07474649

/ Not yet recruitingPhase 3

Effects of Bempedoic Acid/Ezetimibe/High-intensity Statin on Plaque Regression and Stabilisation of Coronary Atherosclerosis Among Patients Without Cardiovascular Events

The overall objective of the trial is to evaluate the effect of the triple therapy consisting of bempedoic acid (BA), ezetimibe (EZE), and high-intensity atorvastatin or rosuvastatin on changes in coronary plaque burden and plaque morphology in patients with coronary atherosclerosis without significant obstructive coronary artery disease and without prior history of an ischemic vascular event.

Start Date15 Aug 2026

Sponsor / Collaborator

Daiichi Sankyo Co., Ltd.

NCT07623915

/ Not yet recruitingPhase 3

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Bempedoic Acid in Patients With Hyperlipidemia Not Adequately Controlled by Statins

This trial is a multicenter randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy and safety of Bempedoic Acid Tablets in patients with hyperlipidemia not adequately controlled by Statins.

Start Date12 Jun 2026

Sponsor / Collaborator

CSPC Ouyi Pharmaceutical Co., Ltd.

100 Clinical Results associated with Bempedoic acid

100 Translational Medicine associated with Bempedoic acid

100 Patents (Medical) associated with Bempedoic acid

379

Literatures (Medical) associated with Bempedoic acid

31 Dec 2026·RENAL FAILURE

Emerging roles of ATP citrate lyase in kidney diseases: from pathogenic driver to therapeutic target

Review

Author: Yang, Yiya ; Chen, Yinyin ; Tao, Mina ; Wei, Mengjiao ; Li, Guoli ; Tan, Huifang ; Jin, Zhiyuan ; Xiao, Zheng

ATP-citrate lyase (ACLY) is a crucial cytosolic and nuclear enzyme that catalyzes the synthesis of acetyl-CoA from citrate, serving as a central metabolic node linking carbohydrate metabolism to de novo lipogenesis and histone acetylation. Accumulating evidence positions ACLY not merely as a metabolic enzyme but as a key pathogenic driver in various kidney diseases. This review systematically examines the multifaceted roles of ACLY in renal physiology and pathology. During development, ACLY is essential for nephron progenitor cell maintenance and nephrogenesis. In diabetic kidney disease, hyperglycemia upregulates and promotes nuclear translocation of ACLY, fueling histone acetylation and the transcription of pro-fibrotic genes, while also contributing to oxidative stress and lipid peroxidation. In obesity-related kidney injury, ACLY drives renal ectopic lipid accumulation and inflammation by providing substrates for adipogenic enzymes and inducing histone hyperacetylation. ACLY also promotes renal fibrosis in chronic kidney disease via pathways such as AKT/ACLY signaling. In clear cell renal cell carcinoma, ACLY expression is upregulated through HIF-2α/LPCAT1/FBXW7 and VHL/PPARγ axes, promoting lipid synthesis, tumor proliferation, and metastasis. Furthermore, ACLY activity influences hypocitraturia, a key factor in nephrolithiasis, and is upregulated in polycystic kidney disease, where its inhibition attenuates cystic growth. Given its central role across diverse renal pathologies, ACLY emerges as a promising therapeutic target. Several inhibitors, including bempedoic acid, SB-204990, and natural compounds, show potential in modulating ACLY activity. This review consolidates current knowledge on ACLY in kidney diseases, highlighting its mechanistic contributions, and underscoring its significant potential for diagnostic and therapeutic innovation.

01 Sep 2026·Atherosclerosis plus

Systematic literature review on the real-world effectiveness and safety of bempedoic acid

Review

Author: Gautam, Raju ; Ghatak, Aishee ; Anwar, Saeed ; Chhabra, Richa ; Cannon, Christopher P ; Powell, Heather A ; Sarnes, Evelyn ; Cicero, Arrigo Francesco Giuseppe ; Jawla, Shantanu

Background and aims:

Cardiovascular disease remains a significant global health burden. Bempedoic acid is a novel, oral adenosine triphosphate-citrate lyase inhibitor that lowers low-density lipoprotein cholesterol (LDL-C). While randomized controlled trials (RCTs) have established its efficacy and safety, evidence from real-world clinical practice is needed.

Methods:

A systematic literature review (SLR) was conducted to identify studies reporting real-world use of bempedoic acid as monotherapy, in fixed dose combination with ezetimibe, or in combination with any other lipid lowering therapy (LLT). MEDLINE, EMBASE, and Cochrane Library were searched from inception to February 19, 2025. Two reviewers independently screened studies, with discrepancies resolved by a third. Data extraction and validation were conducted, and the risk of bias assessed using the Newcastle-Ottawa Scale.

Results:

Of 269 records, 28 reports on 22 unique studies met the eligibility criteria. Most studies evaluated LDL-C changes from baseline after initiation of bempedoic acid; one pragmatic randomized trial was identified. Bempedoic acid achieved LDL-C reductions of 15% to 39% when added to maximally tolerated statins, 22% to 38% in statin intolerant cohorts, and 18% to 42% in mixed statin intolerant cohorts. Safety data was limited; treatment discontinuation ranged from 9% to 36%, with higher rates in cohorts with greater statin-intolerance.

Discussion:

This SLR shows bempedoic acid, with or without other LLTs, achieves a minimum of approximately 20% LDL-C reductions, consistent with RCTs. Data suggests it may be effective as an adjunct to statins and as an oral alternative for statin-intolerant patients.

07 Jul 2026·Journal of the American Heart Association

Efficacy and Safety of Bempedoic Acid in Patients Aged ≥75 Years Stratified by Varying Statin Exposure: Results From Phase 3 Studies of Bempedoic Acid

Article

Author: Mukherjee, Debabrata ; Nicholls, Stephen J. ; Catapano, Alberico L. ; Leiter, Lawrence A. ; Duell, P. Barton ; Nissen, Steven E. ; Laufs, Ulrich ; Bloedon, LeAnne ; Powell, Heather A. ; Lincoff, A. Michael ; Broestl, Christine ; Li, Na ; Goldberg, Anne C. ; Mancini, G. B. John ; Herout, Peter M. ; Banach, Maciej

Background:

Lowering low‐density lipoprotein cholesterol (LDL‐C) reduces the risk of major vascular events across all age groups. We analyzed phase 3 studies of bempedoic acid to characterize the safety and efficacy in patients aged ≥75 years with and without a concomitant statin.

Methods:

Post hoc analysis of bempedoic acid in high‐risk patients with cardiovascular disease by age from phase 3 placebo‐controlled, randomized clinical trials: two 52‐week primary hyperlipidemia studies with maximal background statin (“max‐statins pool”), and 1 cardiovascular outcomes trial (“statin‐intolerant pool”). Efficacy (LDL‐C, non–high‐density lipoprotein cholesterol, total cholesterol, high‐sensitivity C‐reactive protein) and safety were evaluated.

Results:

The max‐statins pool included 3009 (2010 bempedoic acid, 999 placebo) patients. The statin‐intolerant pool included 13 970 patients (6992 bempedoic acid, 6978 placebo). In total 7022 patients were aged 18 to <65 years; 7372 were aged 65 to <75 years; 2585 were aged ≥75 years. Placebo‐corrected mean percentage change in LDL‐C at week 12 in the max‐statin pool with bempedoic acid was −18.4% (95% CI, −21.3 to −15.6) for patients aged 18 to <65 years, −18.6% (95% CI, −21.2 to −16.1) for 65 to <75 years, and −18.3% (95% CI, −22.2 to −14.5) for ≥75 years. In the statin‐intolerant pool, LDL‐C change was −21.9% (95% CI, −23.1 to −20.7) for patients aged 18 to <65 years, −22.9% (95% CI, −24.0 to −21.8) for 65 to <75 years, and −24.5% (95% CI, −26.2 to −22.7) for ≥75 years. Frequency of adverse events compared with placebo was similar across the ages, but more frequent among patients ≥75 years regardless of background statin. Cardiovascular event reduction was statistically comparable across all ages.

Conclusions:

Bempedoic acid was well tolerated in patients aged ≥75 years with efficacy and safety comparable with younger subgroups regardless of background statin usage. Bempedoic acid may be considered a viable strategy for managing hypercholesterolemia in adults, regardless of age.

150

News (Medical) associated with Bempedoic acid

05 Aug 2026

·www.biospace.com

HLS Therapeutics Provides Update on NILEMDO™ Market Access

Reimbursement secured with the largest private payers in Canada, estimated to represent approximately 80% of privately insured Canadians Canada's Drug Agency issues final recommendation, with its expert committee voting unanimously to recommend reimbursement of NILEMDO™ by public drug plans TORONTO , Aug. 5, 2026 /CNW/ -- HLS Therapeutics Inc. ("HLS" or the "Company") (TSX: HLS), a pharmaceutical company focused on addressing unmet needs in the treatment of psychiatric disorders and cardiovascular disease, announces positive developments in both private and public reimbursement coverage for NILEMDO™ (bempedoic acid) in Canada. Private Payer Coverage HLS has now secured reimbursement for NILEMDO with the largest private payers in Canada, representing approximately 80% of privately insured Canadians, the vast majority of whom can now access NILEMDO as a full benefit without restrictions. Several of these agreements are already in effect, with the remainder taking effect during the third quarter of 2026. Public Reimbursement On the public side, Canada's Drug Agency (CDA-AMC) has finalized its reimbursement recommendation for NILEMDO. The Canadian Drug Expert Committee voted unanimously to recommend that NILEMDO be reimbursed by participating public drug plans. This recommendation opens the window for HLS to begin negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) toward product listing agreements. HLS intends to commence those negotiations later this year, keeping it on track for initial provincial listings in the first half of 2027. A link to the CDA-AMC report can be found here: Bempedoic Acid (NILEMDO) "We are pleased with our progress on securing market access for NILEMDO," said Craig Millian, Chief Executive Officer at HLS. "For the many Canadians who are not reaching their LDL cholesterol targets on statins, whether due to intolerance or inadequate response, NILEMDO provides physicians and patients with a much-needed oral treatment option. Broad private coverage without restrictions, together with a clear pathway toward public reimbursement, means more Canadian patients will be able to access this medicine to help manage their cardiovascular risk." ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); ABOUT NILEMDO NILEMDO is indicated to reduce low-density lipoprotein cholesterol ("LDL-C") and the risk of major cardiovascular events in adults who require additional LDL-C lowering due to an inability to reach target levels with currently available therapies or due to statin intolerance. HLS in-licensed the exclusive rights to NILEMDO and NEXLIZET® (bempedoic acid and ezetimibe combination) for the Canadian market from Esperion Therapeutics, Inc. ABOUT HLS THERAPEUTICS INC. Formed in 2015, HLS is a pharmaceutical company focused on the acquisition and commercialization of late-stage development, commercial stage promoted and established branded pharmaceutical products in the North American markets. HLS's focus is on products targeting the treatment of psychiatric disorders and cardiovascular disease. HLS's management team is composed of seasoned pharmaceutical executives with a strong track record of success in these therapeutic areas and at managing products in each of these lifecycle stages. For more information visit: FORWARD LOOKING INFORMATION This release includes forward-looking statements regarding HLS and its business. Such statements are based on the current expectations and views of future events of HLS's management. In some cases the forward-looking statements can be identified by words or phrases such as "may", "will", "expect", "plan", "anticipate", "intend", "potential", "estimate", "believe" or the negative of these terms, or other similar expressions intended to identify forward-looking statements, including, among others, statements with respect to HLS's pursuit of additional product and pipeline opportunities in certain therapeutic markets, statements regarding growth opportunities, expectations regarding financial performance, and the NCIB and ASPP. The forward-looking events and circumstances discussed in this release may not occur and could differ materially as a result of known and unknown risk factors and uncertainties affecting HLS, including risks relating to the specialty pharmaceutical industry, risks related to the regulatory approval process, economic factors and many other factors beyond the control of HLS. Forward-looking statements and information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause HLS's actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statement or information. A discussion of the material risks and assumptions associated with this release can be found in the Company's Annual Information Form dated March 11, 2026, and Management's Discussion and Analysis dated May 14, 2026, both of which have been filed on SEDAR+ and can be accessed at . Accordingly, readers should not place undue reliance on any forward-looking statements or information. Except as required by applicable securities laws, forward-looking statements speak only as of the date on which they are made and HLS undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise. SOURCE HLS Therapeutics Inc.

License out/in

31 Jul 2026

·www.biospace.com

Daiichi Sankyo Reports First Quarter Fiscal Year 2026 Financial Results

Revenue increased 21.1% to 574.7 billion yen year-over-year driven by Enhertu ® and Datroway ® Fiscal year 2026 guidance for revenue and operating profit revised upward Conference call to take place on Friday, July 31 at 5:30 pm Japan Standard Time TOKYO--(BUSINESS WIRE)--Daiichi Sankyo (TSE: 4568) reported financial results for the first quarter of fiscal year 2026, ending June 30, 2026. “Daiichi Sankyo delivered strong revenue growth in the first quarter of fiscal year 2026 as we began execution of our new Five-Year Business Plan,” said Hiroyuki Okuzawa, President and CEO of Daiichi Sankyo. “Three new U.S. approvals for Enhertu and Datroway were received in a single week in May, underscoring the continued strength of our portfolio where we anticipate delivering more than 20 new indications across five medicines by 2030. These results reinforce the durability of our growth trajectory and support our outlook for fiscal 2026 and beyond.” Q1 FY2026 Results Global revenue increased 21.1% year-over-year to 574.7 billion yen, driven by the company’s global oncology products. Operating profit decreased 12.0% year-over-year to 85.1 billion yen, primarily due to EU Specialty Business restructuring expenses. Global revenue from oncology products was driven by Enhertu ® and Datroway ® , the company's flagship antibody drug conjugates (ADCs). Enhertu revenue, including milestone payments, was 239.8 billion yen, a 48.9% increase. Datroway revenue, including milestone payments, was 23.9 billion yen, a 176.4% increase. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Revenue from the Japan Business was 120.0 billion yen, driven by Lixiana ® and Tarlige ® . ASCA (Asia, South and Central America) Business revenue was 68.7 billion yen, driven by Enhertu. EU Specialty Business revenue was 74.7 billion yen, driven by Lixiana and Nilemdo ® /Nustendi ® . Full fiscal year 2026 guidance has been revised upward from the forecast issued in May 2026. Global revenue is anticipated to increase from 2.28 to 2.34 trillion yen, driven by stronger sales anticipated in the U.S. and favorable foreign exchange rates. Operating profit is anticipated to increase from 315.0 to 320.0 billion yen. Further details on financial performance can be found in the Financial Results Presentation Material . Daiichi Sankyo will host an investor and media conference call and webcast to discuss Q1 FY2026 results on Friday, July 31 at 5:30 pm Japan Standard Time. Q4 FY2025 Results Revision Operating profit has increased from 229.1 billion yen to 258.0 billion yen. Following the announcement of the Q4 FY2025 financial results on May 11, 2026, the Company identified an error in the aggregation process for selling, general and administrative (SG&A) expenses. As a result of the decrease in SG&A expenses, operating profit has been revised upward. Portfolio and Pipeline Highlights Daiichi Sankyo continued to advance its portfolio and pipeline with several milestones across its oncology portfolio, demonstrating momentum towards its goal of launching 20 new indications across five medicines by 2030. Three new breast cancer indications were approved within one week in the U.S. for Enhertu and Datroway in May 2026, including simultaneous approval of two new indications for Enhertu in the neoadjuvant and adjuvant HER2 positive early breast cancer setting based on the DESTINY-Breast11 and DESTINY-Breast05 phase 3 trials, followed by one new indication for Datroway in the first-line metastatic triple negative breast cancer (TNBC) setting for patients not candidates for immunotherapy based on the TROPION-Breast02 phase 3 trial. Datroway received approval in the EU for the same TNBC indication and Enhertu received a positive CHMP opinion for the first-line treatment of HER2 positive metastatic breast cancer based on the DESTINY-Breast09 phase 3 trial in July 2026. A Biologics License Application (BLA) was accepted and granted priority review in the U.S. in April 2026 for ifinatamab deruxtecan (I-DXd) for the treatment of adult patients with extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy, with a Prescription Drug User Fee Act (PDUFA) date of October 10, 2026. Additional regulatory approvals were received in June 2026 for medicines in other major countries/regions. Enhertu received approval in the EU for a HER2 positive metastatic tumor agnostic indication and Vanflyta ® was approved in China in the newly diagnosed setting of FLT3 -ITD positive acute myeloid leukemia (AML). Data from more than 25 abstracts across multiple cancers were presented at the 2026 American Society of Clinical Oncology (#ASCO26) in June 2026. Medicine Indication Q1 FY2026 Update Enhertu Breast Enhertu Approved in the U.S. for Two New Indications for Patients with HER2 Positive Early Breast Cancer ( May 2026 ) Enhertu Plus Pertuzumab Recommended for Approval in the EU by CHMP as First-Line Treatment for Patients with HER2 Positive Metastatic Breast Cancer ( July 2026 ) Tumor Agnostic Enhertu Approved in the EU as First Tumor Agnostic HER2 Directed Therapy and Antibody Drug Conjugate for Patients with Previously Treated HER2 Positive Metastatic Solid Tumors ( June 2026 ) Datroway Breast Datroway Approved in the U.S. as First TROP2 Directed Antibody Drug Conjugate for First-Line Treatment of Patients with Metastatic Triple Negative Breast Cancer Who Are Not PD-1/PD-L1 Inhibitor Candidates ( May 2026 ) Datroway Approved in the EU as Only TROP2 Directed Medicine with Overall Survival Benefit for the First-Line Treatment of Patients with Metastatic TNBC Who Are Not Candidates for Immunotherapy ( July 2026 ) I-DXd Lung Ifinatamab Deruxtecan Granted Priority Review in the U.S. for Adult Patients with Previously Treated Extensive-Stage Small Cell Lung Cancer who Experienced Disease Progression on or After Platinum-Based Chemotherapy ( April 2026 ) Vanflyta AML Vanflyta Approved in China as First and Only FLT3 Inhibitor for Patients with Newly Diagnosed FLT3 -ITD Positive AML ( June 2026 ) Pipeline Various Daiichi Sankyo Showcases Progress Across Industry-Leading Oncology Portfolio with Latest Research Updates at ASCO ( May 2026 ) Corporate Highlights Daiichi Sankyo announced its next Five-Year Business Plan (FY2026 - FY2030) in May 2026 outlining how the company plans to deliver more than 2.3 trillion yen in oncology revenue by 2030 and be a global top five oncology company by 2035 as part of its larger 2035 Vision to be recognized as a “trusted healthcare innovator transforming the lives of people through our science and technology.” As part of the company’s continued transformation into a global oncology company, Daiichi Sankyo announced in April 2026 that it will transfer in stages all of its shares of Daiichi Sankyo Healthcare Co., Ltd, its over-the-counter drug business in Japan, to Suntory Holdings Limited. The deal is estimated to be completed by June 2029. About Daiichi Sankyo Daiichi Sankyo (TSE: 4568) is a global healthcare company committed to becoming a trusted healthcare innovator, transforming the lives of people through its strength in science and technology. The company discovers and develops new standards of care to address diverse medical needs to fulfill its purpose of contributing to the enrichment of quality of life around the world. With a strategic focus on oncology, Daiichi Sankyo is advancing an industry-leading antibody drug conjugate portfolio along with identifying new breakthrough generating technologies to deliver practice-changing medicines to patients, healthcare professionals and society. For more information, please visit . Forward-Looking Statements Management strategies and plans, financial forecasts, future projections and policies, and R&D information that Daiichi Sankyo discloses in this material are all classified as Daiichi Sankyo’s future prospects. These forward-looking statements, including words such as “anticipate,” “estimated,” “favorable,” “goal,” “momentum,” “outlook,” “trajectory,” were determined by Daiichi Sankyo based on information obtained as of today with certain assumptions, premises and future forecasts, and thus, there are various inherent risks as well as uncertainties involved. As such, please note that actual results of Daiichi Sankyo may diverge materially from the outlook of Daiichi Sankyo or the content of this material. Furthermore, there is no assurance that any forward-looking statements in this material will be realized. Regardless of the actual results or facts, Daiichi Sankyo is not obliged and does not have in its policy the duty to update the content of this material from the date of this material onward. Some of the compounds under discussion are investigational agents and are not approved by the FDA or any other regulatory agency worldwide as a treatment for indications under investigation. Efficacy and safety have not been established in areas under investigation. There are no guarantees that these compounds will become commercially available in indications under investigation. Daiichi Sankyo takes reasonable care to ensure the accuracy of the content of this material but shall not be obliged to guarantee the absolute accuracy, appropriateness, completeness and feasibility, etc. of the information described in this material. Furthermore, any information regarding companies, organizations or any other matters outside the Daiichi Sankyo Group that is described within this material has been compiled or cited using publicly available information or other information, and Daiichi Sankyo has not performed in-house inspection of the accuracy, appropriateness, completeness and feasibility, etc. of such information, and does not guarantee the accuracy thereof. The information described in this material may be changed hereafter without notice. Accordingly, this material or the information described herein should be used at your own judgment, together with any other information you may otherwise obtain. This material does not constitute a solicitation of application to acquire or an offer to sell any security in the United States, Japan or elsewhere. This material disclosed here is for reference purposes only. Final investment decisions should be made at your own discretion. Daiichi Sankyo assumes no responsibility for any damages resulting from the use of this material or its content, including without limitation damages related to the use of erroneous information. Contacts MEDIA CONTACTS: Global: Jennifer Brennan jennifer.brennan@daiichisankyo.com +1 908 900 3183 (mobile) INVESTOR RELATIONS CONTACT: DaiichiSankyoIR_jp@daiichisankyo.com Japan: DS-PR_jp@daiichisankyo.com

Drug ApprovalImmunotherapyPhase 3ASCOFinancial Statement

16 Jul 2026

·www.biospace.com

Merck dives into cholesterol with first FDA approval for oral PCSK9 inhibitor

iStock, NatuskaDPI Merck’s once-daily pill is the first oral PCSK9 inhibitor to hit the market for high cholesterol, beating AstraZeneca in the race to develop more accessible treatment options. Merck has secured the first FDA approval for a once-daily oral PCSK9 inhibitor, a drug designed to lower “bad” LDL cholesterol without the need for an injection. The FDA greenlight is for enlicitide, to be sold as Lipfendra for adults with high levels of LDL cholesterol and paired with diet and exercise. With Lipfendra, Merck has beat AstraZeneca to market. The U.K. pharma giant is developing its own oral PCSK9 candidate, dubbed AZD0780. A macrocyclic peptide, Lipfendra was approved under the FDA’s Commissioner’s National Priority Voucher (CNPV) program, an initiative launched under former commissioner Marty Makary that is designed to speed up reviews for medicines and companies aligned with certain U.S. government priorities. Lipfendra represents the FDA’s ninth CNPV approval, per BioSpace tracking. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); The approval was supported by a pair of Phase 3 trials from Merck’s CORALreef program, one of which included patients with heterozygous familial hypercholesterolemia (HeFH), a genetic disorder that causes high LDL levels. In that trial, Lipfendra significantly reduced LDL cholesterol by a placebo-adjusted 59% at 24 weeks, while lowering LDL cholesterol by 56% in a trial of adults with hypercholesterolemia. For patients without the genetic condition, the drug’s safety pro similar to that of placebo, while the most common adverse event for those with HeFH was diarrhea. “LIPFENDRA’s label is the cleanest in the PCSK9 class, carrying zero contraindications or hypersensitivity warnings, unlike currently approved injectable competitors (Repatha, Praluent, and Leqvio), which are each contraindicated in patients with prior serious hypersensitivity reactions,” RBC Capital Markets wrote in a Thursday note to investors. Cardiovascular disease Merck’s Cholesterol Pill Delivers ‘Antibody-Like Efficacy’ After Earning FDA Voucher Merck is eyeing a quick review for its lipid-lowering drug candidate enlicitide, which in December was awarded a Commissioner’s National Priority Voucher. March 31, 2026 · 2 min read · Tristan Manalac Read more In an add-on trial, Lipfendra significantly outperformed other oral non-statin drugs Nexletol and Zetia in patients with hypercholesterolemia. “This is a pivotal moment as we bring the first U.S. FDA-approved oral PCSK9 inhibitor to adults with high LDL-C, offering patients an important new option,” Dean Li, Merck’s R&D head, said in a Thursday announcement. “We’re proud of our work with regulators on this rigorous and efficient review process.” RBC Capital Markets estimate peak sales of around $5 billion by 2034 for Lipfendra. Merck is launching the drug for $315 per month, representing a “meaningful discount” to injectable PCSK9 inhibitors, which range from $500–$600 a month. Merck is also evaluating Lipfendra for its effect on cardiovascular morbidity and mortality.

Drug ApprovalPhase 3Clinical Result

100 Deals associated with Bempedoic acid

External Link

KEGG	Wiki	ATC	Drug Bank
D10691	Bempedoic acid	C10AX15	DB11936

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyper LDL cholesterolaemia	Canada	HLS Therapeutics, Inc.	05 Mar 2026
Hypercholesterolemia	Japan	大塚製薬株式会社	19 Sep 2025
Cardiovascular Diseases	United States	Esperion Therapeutics, Inc.	22 Mar 2024
Myocardial Infarction	United States	Esperion Therapeutics, Inc.	22 Mar 2024
Primary Hyperlipidemia	United States	Esperion Therapeutics, Inc.	22 Mar 2024
Dyslipidemias	European Union	Daiichi Sankyo Europe GmbH	01 Apr 2020
Dyslipidemias	Iceland	Daiichi Sankyo Europe GmbH	01 Apr 2020
Dyslipidemias	Liechtenstein	Daiichi Sankyo Europe GmbH	01 Apr 2020
Dyslipidemias	Norway	Daiichi Sankyo Europe GmbH	01 Apr 2020
Primary hypercholesterolemia	European Union	Daiichi Sankyo Europe GmbH	01 Apr 2020
Primary hypercholesterolemia	Iceland	Daiichi Sankyo Europe GmbH	01 Apr 2020
Primary hypercholesterolemia	Liechtenstein	Daiichi Sankyo Europe GmbH	01 Apr 2020
Primary hypercholesterolemia	Norway	Daiichi Sankyo Europe GmbH	01 Apr 2020
Atherosclerosis	United States	Esperion Therapeutics, Inc.	21 Feb 2020
Heterozygous familial hypercholesterolemia	United States	Esperion Therapeutics, Inc.	21 Feb 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hyperlipidemias	Phase 3	United States	Esperion Therapeutics, Inc.	23 Oct 2017
Cerebrovascular Disorders	Phase 3	United States	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Argentina	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Australia	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Austria	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Belgium	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Brazil	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Bulgaria	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Canada	Esperion Therapeutics, Inc.	22 Dec 2016
Cerebrovascular Disorders	Phase 3	Chile	Esperion Therapeutics, Inc.	22 Dec 2016

Clinical Result

Indication

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05694260 (CLEAR Path 1, CTgov)	Phase 2	Heterozygous familial hypercholesterolemia	31	Bempedoic Acid (Cohort 1 (16 to <30 Kg) Bempedoic Acid 60 mg)	ltlsghpzmx(gfkbczqsjo) = hkzzmhpfoh xajckcnrfr (egxehvbbho, 39.6) View more	-	17 Jul 2026
				Bempedoic Acid (Cohort 1 (16 to <30 Kg) Bempedoic Acid 90 mg)	ltlsghpzmx(gfkbczqsjo) = buwxuppcsn xajckcnrfr (egxehvbbho, 20.4) View more
NCT02993406 (CLEAR Outcomes, Pubmed \| JAMA Cardiol)	Phase 3	Venous Thromboembolism	13,970	Bempedoic acid	wqxldjvhzp(fslfutlbpm) = bqlbitpbox djystxwzuw (blthdxkuoi )	Positive	27 May 2026
				Placebo	wqxldjvhzp(fslfutlbpm) = rojspiuzgc djystxwzuw (blthdxkuoi )
NCT06925100 (CLEAR Taiwan, Pubmed \| J Am Heart Assoc)	Phase 4	Hypercholesterolemia	180	Bempedoic acid	sjgczeprni(vemsmnprih) = colzpxwzrj nouidcwlgp (luebrkrbzs )	Positive	19 May 2026
NCT05687071 (CTgov)	Phase 3	Hyper LDL cholesterolaemia	130	ETC-1002	ccylkbxkdh = mfyxfvxqnk ibsghbotkg (wdxfkbvqej, nympnoixfj - uwpsckcozf) View more	-	20 Feb 2026
NCT02993406 (CLEAR Outcomes, Pubmed \| Am J Cardiovasc Drugs)	Phase 3	Hypercholesterolemia	13,970	Bempedoic acid	rncenkmffs(gwygjjwbhz) = trynjavjxn mlxqmfbbbl (usnoeidgtf )	Positive	20 Aug 2025
				Placebo	rncenkmffs(gwygjjwbhz) = kegkefhjrl mlxqmfbbbl (usnoeidgtf )
NCT05683340 (CTgov)	Phase 3	Hyper LDL cholesterolaemia	96	Placebo	polcybkgsn(chfzymsypu) = oeosijzafh frdwdcgmti (ouunnwmfuy, 1.901) View more	-	13 Mar 2025
NCT02993406 (CLEAR Outcomes, Pubmed \| J Am Heart Assoc)	Phase 3	Obesity	6,177	Bempedoic Acid 180 mg	jriuilurkm(qrozaaahxl) = kvogawaupt iwwumyyxen (xzvsxajadf, 0.76 [0.63 - 0.92]) View more	Positive	18 Feb 2025
				Placebo	-
NCT05683340 (Company_Website) Manual	Phase 3	Hypercholesterolemia	96	Bempedoic acid 180 mg	vlljgamskn(miktytuwjs) = ruxanyweaa bqebhzaakx (ofenlcpbpb ) Met	Positive	20 May 2024
				Placebo	vlljgamskn(miktytuwjs) = qleyazmfsg bqebhzaakx (ofenlcpbpb ) Met
NCT04784442 (CTgov)	Phase 2	Hypercholesterolemia	188	Placebo	bqtjchmooi(xiscikayec) = krzkmksvsi wtvutbrfya (vuwkobzjzl, 2.117) View more	-	04 Mar 2024
NCT02993406 (CLEAR Outcomes, CTgov)	Phase 3	Cardiovascular Diseases \| Coronary Artery Disease \| Cerebrovascular Disorders ... View more	13,970	Bempedoic acid 180 mg tablet (Bempedoic Acid 180 mg)	jfycovdtwf = zimlvhnlff qmakwucxxi (covdoloklq, upktblxsnx - rlfenhvfaz) View more	-	03 Jan 2024
				Matching placebo tablet (Placebo Comparator)	jfycovdtwf = pnrubtmfif qmakwucxxi (covdoloklq, dkeoxoxovh - vwmpuaaohp) View more

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