Open Label Phase 3b Study to Evaluate the Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia (LIBerate-Kids OLE)

The goal is to assess the long term efficacy (LDL cholesterol reduction) and safety over 3 years of lerodalcibep (Lerochol) SC 300 mg QM administered by auto-injector (AI)/pre-filled pen (PFP) in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins who completed the 24 week placebo controlled base trial.
The main question[s] it aims to answer are:
How effective is Lerochol in maintaining LDL cholesterol reductions over years? How well is it tolerated and are there any safety concerns?
Participants will visit the clinic every month for 3 months and then home dosed with clinic visits every 3 months. They will undergo periodic physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.

Start Date20 Jan 2027

Sponsor / Collaborator

LIB Therapeutics LLC

NCT07102511

/ Enrolling by invitationPhase 3

Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy (LIBerate-Kids)

The goal of this clinical trial is to assess the LDL-Cholesterol reductions at Week 12 and Week 24 with monthly dosing of lerodalcibep (Lerochol) 300 mg administered subcutaneously by auto-injector (AI)/pre-filled pen (PFP) compared to placebo (dummy), in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins.
The main question[s] it aims to answer are:
How effective is Lerochol in reducing LDL cholesterol? How well is it tolerated and are there any safety concerns? Researchers will compare Lerochol to placebo (inert or dummy injection solution).
Participants will visit the clinic every month for months and be asked to fast overnight, but allowed to drink water, before clinic visits. Undergo physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.

Start Date20 Jul 2026

Sponsor / Collaborator

LIB Therapeutics LLC

[+1]

NCT06568471

/ RecruitingPhase 3

A Randomized, Double-blind, Placebo-controlled Phase 3 Clinical Study to Evaluate the Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia

This randomized study is to assess LDL-C reductions at Week 12 with monthly (Q4W [≤31 days]) dosing of HST101 (lerodalcibep) 300 mg administered subcutaneously (SC) compared to placebo in patients with atherosclerotic cardiovascular disease (ASCVD) or very-high/high risk for ASCVD including Heterozygous familial hypercholesterolemia (HeFH) on a stable diet and oral LDL-C lowering drug therapy, followed by 36-week open-label treatment with subsequent 4-week follow-up for total 52-week long-term safety and efficacy evaluation.

Start Date16 Nov 2024

Sponsor / Collaborator

Hasten Biopharmaceutical Co., Ltd.

100 Clinical Results associated with Lerodalcibep

100 Translational Medicine associated with Lerodalcibep

100 Patents (Medical) associated with Lerodalcibep

Literatures (Medical) associated with Lerodalcibep

01 Dec 2026·International journal of cardiology. Cardiovascular risk and prevention

Comment on “Lipoprotein(a) reduction with inclisiran, alirocumab, evolocumab, enlicitide, and lerodalcibep: A systematic review and meta-analysis of randomized controlled trials”

Letter

Author: Paul, Jeffrin Reneus ; Shridevi, Kotina ; Ratnakar, Parag J ; Gupta, Vatsala ; Gaurav

01 Jun 2026·JAMA Cardiology

Response to PCSK9 Inhibition Based on LDL Receptor Function in Familial Hypercholesterolemia

Article

Author: Scott, Russell ; Basson, Matthys M. De Vries ; Stein, Evan A. ; Kayikcioglu, Meral ; Roth, Mendel ; Blom, Dirk J. ; Raal, Frederick J. ; Fourie, Nyda ; Vest, Jeffrey ; Kallend, David

Importance:

In individuals with heterozygous familial hypercholesterolemia (HeFH), it is uncertain whether and to what extent the reduction in low-density lipoprotein cholesterol (LDL-C) with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor therapy is dependent on the residual functional activity of the LDLR gene carrying the pathogenic variant.

Objective:

To evaluate the reduction in LDL-C achieved with PCSK9 inhibition according to FH genotype in a large cohort of patients with HeFH.

Design, Setting, and Participants:

This nonrandomized clinical trial reports on a predefined, pooled subanalysis of participants with HeFH requiring additional lipid-lowering therapy in the open-label worldwide phase 3 study of the PSCK9 inhibitor lerodalcibep. This study included participants randomized to 5 phase 3 studies with the plasma PSCK9 inhibitor lerodalcibep and who participated in the open-label extension study from December 2020 to May 2025. Data were analyzed from March 2025 to February 2026.

Intervention:

All participants received lerodalcibep 300 mg subcutaneously monthly for 72 weeks.

Main Outcome and Measures:

The co–primary efficacy end points were LDL-C reduction at weeks 48 and 72. Secondary and exploratory end points included LDL-C response according to FH genotype and the achievement of currently recommended LDL-C goals.

Results:

Among 703 included participants (mean [range] age, 53.8 [18-80] years; 372 male [52.9%]), 86 (12.2%) were Black, South Asian, or multiracial and 617 (87.8%) were White; 217 participants (72.3%) had atherosclerotic cardiovascular disease (ASCVD) or were at very high risk for ASCVD and 195 participants (27.7%) were at high risk for ASCVD. Despite most participants receiving treatment with statins or ezetimibe, the mean (SD) baseline LDL-C was 144.9 (61.9) mg/dL. Mean (SD) reductions in LDL-C associated with lerodalcibep were 50.3% (28.9%) and 50.3% (28.7%) (mean [SD] absolute change, −72.6 [50.5] mg/dL and −71.8 [48.0] mg/dL) at weeks 48 and 72, respectively. Of 740 participants (92.5%) who underwent genetic testing, monogenic FH-causing variants were found in 455 participants (61.5%), including 432 participants (95.7%) with the LDLR pathogenic variant. LDL-C reduction with lerodalcibep was independent of LDLR variant functional activity. More than 70% of participants achieved both a reduction in LDL-C of at least 50% and their ASCVD risk-based LDL-C goal.

Conclusions and Relevance:

These findings suggest that lerodalcibep was associated with significantly and consistently reduced LDL-C in patients with HeFH, with response found to be independent of LDLR function of the pathogenic variant. These findings support that LDL-C reductions in patients with HeFH with PCSK9 inhibition are predominantly mediated by upregulation of the unaffected wild-type LDLR .

Trial Registration:

ClinicalTrials.gov Identifier: NCT04798430

01 Dec 2025·Current Atherosclerosis Reports

Highlights of Cardiovascular Disease Prevention Studies Presented at the 2024 American Heart Association Scientific Sessions

Review

Author: Virani, Salim S ; Sheikh, Sana ; Khoja, Adeel ; Inam, Maha ; Murthy, Nikitha ; Epstein, Elizabeth ; Vaughan, Elizabeth ; Mehta, Sandeep ; Minhas, Abdul Mannan Khan ; Junaid, Vashma ; Slipczuk, Leandro ; Waldman, Carly ; Chai, Zohar ; Hermel, Melody ; Hinkamp, Colin

PURPOSE OF REVIEW:

Focused review highlighting ten select studies presented at the 2024 American Heart Association (AHA) Scientific Sessions.

RECENT FINDING:

Included studies assessed effects of intensive blood pressure control in patients with type 2 diabetes (BPROAD); decision support system for physicians to optimize early lipid lowering therapies after acute coronary syndrome (ZODIAC); efficacy and safety of zerlasiran, a short interfering RNA targeting lipoprotein(a) (ALPACAR); efficacy and safety of muvalaplin an oral disrupter of the assembly of lipoprotein(a) particles (KRAKEN); safety and efficacy of obicetrapib in patients with heterozygous familial hypercholesterolemia (BROOKLYN); efficacy and safety of lerodalcibep, a third generation PCSK9 inhibitor in heterozygous familial hypercholesterolemia subjects (LIBerate-HeFH_OLE); personalized app-based coaching to improve physical activity in patients with HFpEF compared to standard care (MyoMobile); semaglutide to improve cardiovascular outcomes in patients with a history of coronary artery bypass surgery and overweight or obesity (the SELECT trial); efficacy and safety of plozasiran in adults with genetically or clinically defined familial chylomicronemia syndrome at high risk of acute pancreatitis (PALISADE); and transcriptomic signatures and predictors of evolocumab added to maximum statin therapy based on intra-coronary plaque characteristics (YELLOW III). Research presented at the 2024 AHA Scientific Sessions emphasized innovative strategies in cardiovascular disease prevention and management.

News (Medical) associated with Lerodalcibep

28 Sep 2026

·www.biospace.com

LIB Therapeutics and Sun Pharma Announce Global Licensing, Commercialization and Manufacturing Agreement for Lerodalcibep, a Novel PCSK9 Inhibitor, Ex-US and China; Lerodalcibep (Lyrokaul®) Approved in the EU

MUMBAI, India & CINCINNATI--(BUSINESS WIRE)--Sun Pharmaceutical Industries Limited (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715; together with its subsidiaries and/or associated companies, “Sun Pharma”) and LIB Therapeutics Inc. (“LIB”) today announced: An exclusive licensing agreement granting Sun Pharma rights to commercialize and manufacture lerodalcibep worldwide, excluding the United States and China. Lyrokaul (lerodalcibep), a once-monthly PCSK9 inhibitor for LDL-C lowering, comes with 6-month EU ambient storage; received EU approval on September 21, 2026. Strengthens Sun Pharma’s Innovative Medicines portfolio through access to a US$3.7 billion PCSK9 market Ex-US and China, growing at 38% CAGR; global PCSK9 market approaching US$7 billion in 2026. 1 LIB will receive an upfront and future milestone payments, together with royalties based on net sales in the licensed territories. Lerodalcibep is approved in the EU under the brand name Lyrokaul for the treatment of adult patients with hypercholesterolaemia and mixed dyslipidaemia. In the United States, it is FDA-approved under the brand name LEROCHOL ® as an adjunct to diet and exercise: to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). The partnership helps address a significant global unmet need in cardiovascular care. Many patients at high or very high cardiovascular risk remain above recently updated and more globally harmonized 2026 guideline-recommended LDL-cholesterol (LDL-C) goals despite treatment. In the DA VINCI observational study across 18 European countries, only 33% of patients assessed on stable oral lipid-lowering therapy achieved their risk-based 2019 European Society of Cardiology/European Atherosclerosis Society (ESC/EAS) LDL-C goals. 2 Treatment guidelines now emphasize both additional LDL-C reductions and lower targets for patients with, or at very high or high, cardiovascular disease (CVD) risk, with additional therapies when statins alone are insufficient. PCSK9 inhibitors provide the efficacy and safety to achieve these goals. 3 This gap between treatment goals and real-world control reinforces the opportunity for Sun Pharma and LIB to broaden access to innovative lipid-lowering care across Europe and Sun’s world-wide licensed markets. “Once-monthly lerodalcibep dosing, with robust LDL-C reduction and the added benefits of a small-volume injection and six-month ambient storage, simplifies treatment for patients and offers greater convenience,” said Kirti W Ganorkar, Managing Director of Sun Pharma. “This agreement marks a significant step in strengthening our global Innovative Medicines portfolio, particularly in cardiovascular care. With exclusive rights outside the United States and China, we look forward to expanding access to this important treatment option for patients globally.” “LIB is extremely pleased to partner with Sun Pharma, a well-established global pharmaceutical company with a growing focus on innovative medicines,” said Evan Stein, MD, PhD, Chief Operating and Scientific Officer and co-founder of LIB Therapeutics. “Sun Pharma’s international presence and experience in building global innovative brands make it an ideal partner to bring lerodalcibep to more patients. Together, we aim to expand access for patients with cardiovascular disease, or at high cardiovascular risk, who need substantial additional LDL-cholesterol reductions despite existing treatment.” The agreement establishes a significant new growth platform for Sun Pharma in a large and rapidly expanding market. According to IQVIA, the PCSK9 inhibitor market outside the United States and China reached US$ 3.7 billion in the period ending Q2 2026 (MAT Q2 2026), growing at 38% CAGR over the preceding two years. Europe alone accounted for US$ 2.9 billion PCSK9 inhibitor market in MAT Q2 2026.¹ Agreement terms LIB will receive an upfront and future milestone payments, together with royalties based on net sales in the licensed territories. Other terms of the agreement are confidential. Sun Pharma will be responsible for pursuing regulatory approvals in licensed territories where approval has not yet been obtained. For full prescribing information, please visit . About LEROCHOL ® (lerodalcibep-liga) LEROCHOL is a novel, antibody-mimetic, recombinant-fusion protein PCSK9 inhibitor, and is the only PCSK9 inhibitor with once-monthly, small single-injection dose, self-administered by patients with room temperature storage up to three months in a recently approved innovative autoinjector device. These features make lerodalcibep a unique alternative to other PCSK9 inhibitors. The FDA recently approved an update to the LEROCHOL indication statement in its prescribing information reflecting the reduction of risk for major adverse cardiovascular events (MACE) achieved in adults via LDL-C lowering with statins or with monoclonal antibody PCSK9 inhibitors used as an add-on to statin therapy. About the LIBerate Clinical Trial Program ® The FDA approval of LEROCHOL was based on data from the comprehensive global Phase 3 LIBerate Clinical Trial Program, which enrolled a diverse population of over 2,900 patients with CVD, without CVD at very high and high risk for CVD, including heterozygous and homozygous familial hypercholesterolemia. Lerodalcibep was dosed once monthly for up to 52 weeks in these key registration-enabling, placebo-controlled trials, and over 2,400 patients continued in the 72-week open-label extension trial. In clinical trials, LEROCHOL demonstrated sustained LDL-C reductions of ≥60% in patients with, or at very high or high risk of, cardiovascular disease and ≥59% in those with HeFH who have more severe LDL-C elevations. LEROCHOL was generally well tolerated across the LIBerate Clinical Trial Program, with no treatment-related serious adverse events reported in the long-term extension studies. Commercial Availability In the USA, LIB Therapeutics is committed to working with payers, healthcare providers, and patient advocacy organizations to ensure broad access to LEROCHOL for patients who need it. LIB plans to make the LEROCHOL autoinjector available in the United States by January 2027. Insurance coverage of LEROCHOL is expected to expand gradually over the course of 2027, and when launched, the autoinjector of LEROCHOL will sit alongside the pre-filled syringe in the company’s direct-to-patient cash pay program for $199 per month. * The excluded China territory comprises mainland China, Hong Kong, Macau and Taiwan. # Worldwide rights exclude the United States and China. Certain other excluded territories, limited in number, will be covered through separate agreements between Sun and LIB. ¹ Source: IQVIA, PCSK9 inhibitor sales at manufacturer prices in US dollars, MAT Q2 2026; two-year CAGR from MAT Q2 2024 to MAT Q2 2026. Market analysis excludes the United States, Puerto Rico and China. Figures are rounded. 2 Ray KK, Molemans B, Schoonen WM, et al. EU-Wide Cross-Sectional Observational Study of Lipid-Modifying Therapy Use in Secondary and Primary Care: the DA VINCI study. Eur J Prev Cardiol. 2021;28(11):1279-1289. doi:10.1093/eurjpc/zwaa047. The 2019 goals were applied retrospectively to data collected in 2017-2018; the 33% figure relates to patients assessed on stable therapy. 3 Mach F, Koskinas KC, Roeters van Lennep JE, et al. 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias. Eur Heart J. 2025;46(42):4359-4378. doi:10.1093/eurheartj/ehaf190. About LIB Therapeutics Inc. LIB Therapeutics is a privately-held, commercial-stage biopharmaceutical company dedicated to bringing novel, highly effective, and safe therapies to help the millions of patients with cardiovascular disease and familial hypercholesterolemia finally achieve their LDL-C goals. In Greater China, a Biologics License Application has been submitted by LIB’s licensing partner Everest Medicines with potential approval in 2027. For more information, please visit: . LIB was advised and assisted by Greenhill & Co investment bank UK About Sun Pharmaceutical Industries Limited (CIN - L24230GJ1993PLC019050) Sun Pharma is a leading global pharmaceutical company with a presence in Innovative Medicines, Generics and Consumer Healthcare products. It is the largest pharmaceutical company in India and is a leading generic company in the US as well as Global Emerging Markets. Sun’s high growth Global Innovative Medicines portfolio spans innovative products in dermatology, ophthalmology, and onco-dermatology and accounts for about 22% of company sales. The company’s vertically integrated operations deliver high-quality medicines, trusted by physicians and consumers in over 100 countries. Its manufacturing facilities are spread across five continents. Sun Pharma is proud of its multi-cultural workforce drawn from over 50 nations. For further information, please visit and follow them on LinkedIn & X (formerly Twitter). Contacts LIB Therapeutics For media inquiries: Carly Scaduto Carly@carlyscadutoconsulting.com For non-media inquiries: info@libtherapeutics.com Sun Pharma Investors Dr. Abhishek Sharma Tel: +91 22 4324 4324, Ext 2929 Tel Direct: +91 22 4324 2929 Mobile: +91 98196 86016 Email: abhi.sharma@sunpharma.com Media Gaurav Chugh Tel: +91 22 4324 4324, Ext 5373 Tel Direct: +91 22 4324 5373 Mobile: +91 98104 71414 Email: gaurav.chugh@sunpharma.com

Drug ApprovalPhase 3License out/inClinical Result

17 Aug 2026

·www.businesswire.com

U.S. Food and Drug Administration Approves an Autoinjector Version of LEROCHOL ® (lerodalcibep-liga) and Updated Indication

CINCINNATI--(BUSINESS WIRE)--LIB Therapeutics, Inc. (LIB), a privately held biopharmaceutical company with a singular focus on helping high-risk patients achieve their cholesterol goals, today announced the U.S. Food and Drug Administration (FDA) has approved an autoinjector for LEROCHOL® (lerodalcibep-liga) Injection 300 mg/1.2 mL, a third-generation Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) inhibitor that combines robust and sustained efficacy with once-monthly, single injection self-administration to support long-term adherence. The FDA also approved an update to the LEROCHOL indication statement in its prescribing information reflecting the reduction of risk for major adverse cardiovascular events (MACE) achieved in adults via LDL-C lowering with statins or with monoclonal antibody PCSK9 inhibitors used as an add-on to statin therapy. “Numerous clinical trials over nearly 40 years have repeatedly established that lowering LDL cholesterol with statins and, more recently, with PCSK9 inhibitors when added to statins, further lowers the risk of major cardiovascular events, and we’re pleased that FDA has added labeling across the PCSK9 class converging around that body of evidence,” said Evan Stein, M.D., Ph.D., Founder, CEO and Chief Scientific Officer of LIB Therapeutics. “These studies have resulted in worldwide consensus and alignment of guidelines on new lower targets, and greater additional reductions in LDL-C, for the millions of patients who cannot reach guideline-directed LDL-C goals on maximally tolerated statin therapy. The challenge now, with this newest class of very effective LDL-C lowering agents, is how to make them simple and affordable enough for patients, who must maintain them for what is life-long treatment. LEROCHOL was designed with a patient perspective in mind to achieve exactly that: a robust and sustained LDL-C reduction from a single, small monthly injection that patients self-administer, with room temperature storage of up to 90 days and now in a modern autoinjector device.” The FDA-approved autoinjector is an innovative, single-use device designed for quick, straightforward patient administration. It is pressure-activated with a single motion and delivers a complete dose in seconds. LEROCHOL in a pre-filled syringe remains available, giving patients and prescribers access to additional administration options. LEROCHOL is the first and only PCSK9 inhibitor that patients self-administer as a monthly, single injection, that can be stored at room temperature conditions for up to 90 days making it easily carried with patients on vacation or business travel. “Innovation in PCSK9 inhibition cannot stop at the molecule, because how a medicine for life-long therapy fits into a patient’s life is what determines whether they stick with it over the long haul,” said Kristen Miller, Vice President, Brand Communications, LIB Therapeutics. “The monthly dosing by autoinjector is completed in seconds, does not interfere with any oral medications, require overnight fasting, or limit the timing of food and beverage consumption. These features, along with the extended room temperature storage, mean LEROCHOL fits into patients’ lives and not the other way around.” “PCSK9 inhibitors have been one of the most important advances in cardiovascular medicine of the last 20 years. However, a patient only gets benefit from the lower and sustained LDL-C levels these medications provide if they stay on treatment,” said Dean J. Kereiakes, M.D., FACC, MSCAI, Chairman of The Christ Hospital Heart and Vascular Institute and Professor of Medicine at the University of Cincinnati. “As much as patients expect highly efficacious and safe therapies, they need therapies that fit into their busy lifestyles, and LEROCHOL provides a compelling option on both counts. LEROCHOL provides substantial LDL-C reduction that achieves the 2026 lipid guidelines in the vast majority of my patients with minimal inconvenience. My patients really appreciate having only once-monthly dosing with LEROCHOL, and now the availability of the autoinjector further simplifies this important treatment option.” LIB plans to make the LEROCHOL autoinjector available in the United States by January 2027. Insurance coverage of LEROCHOL is expected to expand gradually over the course of 2027, and when launched, the autoinjector of LEROCHOL will sit alongside the pre-filled syringe in the company’s direct-to-patient cash pay program for $199 per month. LIB will share additional details on the timing of autoinjector availability, access options, and patient support programs as those plans are finalized. LEROCHOL requires a prescription from a licensed healthcare provider. Patients and healthcare providers interested in LEROCHOL and the LEROCHOL DirectTM cash pay program are encouraged to visit www.LEROCHOL.com to learn more. IMPORTANT SAFETY INFORMATION Adverse Reactions The most commonly occurring adverse reactions in clinical trials in primary hyperlipidemia in adults (including heterozygous familial hypercholesterolemia [HeFH]) (≥2% of patients treated with LEROCHOL ® (lerodalcibep-liga) and occurring more frequently than with placebo) were nasopharyngitis (15% and 14% versus placebo), local injection site reactions (12% and 5% versus placebo) and peripheral edema (2% and <1% versus placebo). The most commonly occurring adverse reactions in clinical trials in HeFH (≥2% of patients treated with LEROCHOL and occurring more frequently than with placebo) were injection site reactions (18% and 3% versus placebo), nasopharyngitis (13% and 9% versus placebo), diarrhea (3% and 1% versus placebo), nausea (2% and 0% versus placebo) and peripheral edema (2% and <1% versus placebo). The most frequent adverse reaction leading to treatment discontinuation in trials in primary hypercholesterolemia in adults was injection site reactions, with a higher frequency in the LEROCHOL-treated group compared to placebo-treated patients (1% vs. 0%). Immunogenicity LEROCHOL is a recombinant fusion protein. As with all therapeutic proteins, there is potential for immunogenicity with LEROCHOL. INDICATIONS LEROCHOL® (lerodalcibep-liga) is indicated as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). Cardiovascular outcomes trials have demonstrated that reducing LDL‑C lowers the risk for major adverse cardiovascular events (MACE) in adults at increased risk when treated with statins or monoclonal antibody PCSK9 inhibitors as an add-on to statin therapy. For full prescribing information, please visit www.LEROCHOL.com. About LEROCHOL® (lerodalcibep-liga) LEROCHOL is a novel, small protein-binding, third-generation PCSK9 inhibitor, and has been developed as a convenient, once-monthly, self-administered, single small-volume, subcutaneous injection with extended room temperature stability up to three months. These features make LEROCHOL a unique alternative to other PCSK9 inhibitors. The anti-PCSK9 binding domain of LEROCHOL is an 11-kDa polypeptide called an adnectin, engineered for high-affinity subnanomolar binding to human PCSK9, and fused to human serum albumin to enhance plasma half-life. About the LIBerate Clinical Trial Program™ The FDA approval of LEROCHOL was based on data from the comprehensive global Phase 3 LIBerate Clinical Trial Program, which enrolled a diverse population of over 2,900 patients with cardiovascular disease (CVD), without CVD at very high and high risk for CVD, including heterozygous and homozygous familial hypercholesterolemia (HeFH). LEROCHOL was dosed once monthly for up to 52 weeks in these key registration-enabling, placebo-controlled trials, and over 2,400 patients continued in the 72-week open-label extension trial. In clinical trials, LEROCHOL demonstrated sustained LDL-C reductions of ≥60% in patients with, or at very high or high risk of, cardiovascular disease and ≥59% in those with HeFH who have more severe LDL-C elevations. LEROCHOL was generally well tolerated across the LIBerate Clinical Trial Program, with no serious treatment-related adverse events reported in the long-term extension studies. About LIB Therapeutics, Inc. LIB Therapeutics is a privately held, commercial-stage biopharmaceutical company dedicated to bringing novel, highly effective, and safe therapies to help the millions of patients with cardiovascular disease and familial hypercholesterolemia finally achieve their LDL-C goals. The company has also submitted a Marketing Authorization Application to the European Medicines Agency, with anticipated approval in 2H 2026. In Greater China, a Biologics License Application has been submitted, with potential approval in 2027. LIB is pursuing additional regulatory submissions in other markets worldwide. For more information, please visit: www.libtherapeutics.com.

Drug ApprovalPhase 3Clinical Result

24 Jul 2026

·firstwordpharma.com

J&J's Icotyde, Roche's Susvimo among key CHMP nods in July decisions

The EMA's drug advisory panel backed a dozen medicines for approval at its July meeting, including Johnson & Johnson's IL-23 antagonist Icotyde (icotrokinra) for plaque psoriasis, Roche's ocular implant Susvimo (rabinizumab) for wet age-related macular degeneration (wAMD), and several cholesterol-lowering therapies.Among the more closely watched decisions, the Committee for Medicinal Products for Human Use (CHMP) recommended Icotyde for adults and adolescents aged 12 years and older with moderate-to-severe plaque psoriasis. If approved by the European Commission, Icotyde would be the first oral medicine targeting the Il-23 receptor available in the region, and expand a class that is dominated by injectables at the moment.The drug was approved in the US in March, with J&J recently saying its product is off to a strong commercial start. For related analysis, see Physician Views Results: J&J's Icotyde may have an edge on Takeda's TYK2 hopeful and Spotlight On: As Stelara fades, J&J's IL-23 tag team steps up.The CHMP recommendation was supported by four Phase III trials involving roughly 2500 patients. Across three studies, between 50% and 57% of patients achieved at least a 90% reduction in Psoriasis Area and Severity Index (PASI 90) scores after 16 weeks, versus between 1% and 4% of placebo recipients. Additionally, 65% to 71% of those given Icotyde achieved clear or almost clear skin, compared with 8% to 11% for placebo.Twice-yearly refillsMeanwhile, the Susvimo recommendation covers its use in adults with wAMD whose disease has stabilised following intravitreal anti-VEGF therapy. The treatment combines ranibizumab, which blocks VEGF, with a surgically implanted refillable ocular device designed to continuously release the drug.The implant is refilled every six months and would reduce the burden of monthly intravitreal injections that have been the standard for ranibizumab-treated patients since the drug was first approved in Europe nearly 20 years ago. If ultimately cleared by the European Commission, Susvimo would become the first implant of its kind approved in the EU.The recommendation was based on a Phase III study of 415 patients showing that Susvimo maintained visual and anatomical outcomes comparable to monthly ranibizumab injections. Common side effects included iritis, conjunctival bleb leak, cataract, vitreous floaters, conjunctival haemorrhage, conjunctival hyperaemia, eye pain and headache.Originally approved in the US for wAMD in 2021, Susvimo has since had its label expanded there to include diabetic macular oedema and diabetic retinopathy.Trio of anti-cholesterol drugsCHMP also backed approval of several cholesterol treatments for adults, including NewAmsterdam Pharma and Menarini's Ubeslo, a once-daily oral non-statin therapy containing low-dose CETP inhibitor obicetrapib, as well as Evlarco, a fixed-dose combination containing obicetrapib plus ezetimibe.Those positive opinions recommend the drugs for patients with primary hypercholesterolemia, both heterozygous familial and non-familial or mixed dyslipidemia based on Phase III data from the BROADWAY, BROOKLYN and TANDEM trials, which demonstrated significant LDL-C reductions reaching up to 40% with Ubeslo monotherapy and about 50% with Evlarco, when compared to placebo (see – KOL Views Q&A: NewAmsterdam's obicetrapib will elbow into LDL space pending two unknowns, says leading cardiologist).CHMP also adopted a positive opinion for LIB Therapeutics' Lyrokaul (lerodalcibep), an injection that patients can self-administer monthly. The third-generation PCSK9 inhibitor was approved by the FDA in December as Lerochol, based on data from the Phase III LIBerate programme showing sustained LDL-C reductions of 60% or more in patients with or at high risk of cardiovascular disease, and 50% or more in those with heterozygous familial hypercholesterolemia who have more severe LDL-C elevations.A first for cALDAmong medicines for rare diseases, CHMP recommended approval under exceptional circumstances for Minoryx Therapeutics and Neuraxpharm's Nezglyal (leriglitazone) to treat cerebral adrenoleukodystrophy (cALD) in patients aged two to 12 years with gadolinium-negative brain lesions.The opinion was based on the Phase II/III NEXUS study, where Nezglyal achieved the primary endpoint of clinical and radiological arrested disease at week 96 or at a visit prior to haematopoietic stem cell transplant, as well as additional real-world evidence from compassionate-use programmes.If regulators give the green light, expected in September, Nezglyal would become the first pharmacological treatment for cALD in the EU.Rare disease snubThe committee also issued negative opinions for three applications, including for Zevra Therapeutics' Meplyffa (arimoclomol citrate), which the company had been seeking approval for to treat Niemann-Pick disease type C in patients aged two years and older in combination with miglustat. "Due to the way the data had been handled and the results analysed, there were uncertainties about the reliability and robustness of the results. Moreover, no benefit was seen in terms of ambulation and cognition," the EMA said.The agency noted that data for a subgroup of patients who were also taking miglustat showed a treatment effect favouring Meplyffa, though this "was not considered robust since effectiveness had not been demonstrated in the overall population."Zevra, whose shares tumbled nearly 24% on Friday, said it is going to request a re-examination of the CHMP opinion.The oral heat shock protein amplifier was approved by the FDA in 2024 under the brand Miplyffa, after a bumpy regulatory journey. That decision was based on data from a 50-patient, placebo-controlled Phase II/III study where patients who received thrice-daily Miplyffa had slower disease progression than those on placebo, as measured by the 4-domain NPC Clinical Severity Scale (R4DNPCCSS).

Clinical ResultPhase 3Drug Approval

100 Deals associated with Lerodalcibep

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R&D Status

Approved

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Indication	Country/Location	Organization	Date
Heterozygous familial hypercholesterolemia	United States	LIB Therapeutics LLC	12 Dec 2025
Hypercholesterolemia	United States	LIB Therapeutics LLC	12 Dec 2025

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Complex dyslipidemia	NDA/BLA	European Union	Everest Medicines Ltd.	23 Jul 2026
Primary hypercholesterolemia	NDA/BLA	European Union	Everest Medicines Ltd.	23 Jul 2026
Atherosclerosis	NDA/BLA	United States	LIB Therapeutics LLC	16 Dec 2024
Homozygous familial hypercholesterolemia	NDA/BLA	United States	LIB Therapeutics LLC	16 Dec 2024
Primary Hyperlipidemia	NDA/BLA	United States	LIB Therapeutics LLC	16 Dec 2024
Hyper LDL cholesterolaemia	Phase 3	United States	LIB Therapeutics LLC	22 Apr 2021
Hyperlipidemia Type IIa	Phase 3	United States	LIB Therapeutics LLC	22 Apr 2021
Hyperlipidemia Type IIa	Phase 3	Israel	LIB Therapeutics LLC	22 Apr 2021
Hyperlipidemia Type IIa	Phase 3	Norway	LIB Therapeutics LLC	22 Apr 2021
Hyperlipidemia Type IIa	Phase 3	South Africa	LIB Therapeutics LLC	22 Apr 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04797104 (LIBerate-FH, CTgov)	Phase 3	Hyperlipidemia Type IIa \| Heterozygous familial hypercholesterolemia	478	lerodalcibep (LIB003 (Lerodalcibep))	kuzafnwdjl(nzcmevaxvs) = hytxywywft vpbadsezhn (bnjnuafujw, 1.866) View more	-	28 Sep 2026
				lerodalcibep (Placebo)	kuzafnwdjl(nzcmevaxvs) = bqprwmknai vpbadsezhn (bnjnuafujw, 2.609) View more
CTR20242095 (NEWS) Manual	Phase 3	Hypercholesterolemia	213	HST101	ebbstlkdyg(wvovqkpubk) = HST101 300 mg 每月一次皮下注射给药后，达到了第12周血清 LDL-C降低的主要终点 oiolyjxffm (apvgchnqnl ) Met View more	Positive	25 Aug 2025
				Placebo
NCT04034485 (LIBerate-HoFH, Pubmed) Manual	Phase 3	Homozygous familial hypercholesterolemia	66	Lerodalcibep 300 mg	conjehievo(szdyoybgyd) = qsablpnbkp uihgsgjgrc (vugowpkjoh, 3.5) View more	Negative	01 Mar 2025
				EvolocumabEvolocumab 420 mg	conjehievo(szdyoybgyd) = lllclbpvvx uihgsgjgrc (vugowpkjoh, 3.5) View more
NCT04798430 (LIBerate-OLE, Biospace) Manual	Phase 3	Cardiovascular Diseases \| Homozygous familial hypercholesterolemia \| Heterozygous familial hypercholesterolemia ... View more	1,468	Lerodalcibep	tfiufxuipl(kjegflgnad) = kxaqxvnqhe fjenvcngcb (iunwwthfjy ) View more	Positive	18 Dec 2024
NCT04806893 (Pubmed \| JAMA Cardiol) Manual	Phase 3	Cardiovascular Diseases	922	LerodalcibepLerodalcibep 300 mg	jkihnsuuuq(cldqsuotxi) = xrvjxwyxno adhknehtdf (mzabntcudn ) View more	Positive	01 Sep 2024
				Placebo	jkihnsuuuq(cldqsuotxi) = ognrkjtvzw adhknehtdf (mzabntcudn )
NCT04797247 (LIBerate-CVD, BusinessWire) Manual	Phase 3	Atherosclerosis	922	Lerodalcibep	zrgbownmkt(jpdzvdgjuc) = qledzewnrv rjuyiuhonb (mjpdweavjo ) Met View more	Positive	29 May 2024
				Placebo	lrjjzarogj(xrsmabimsi) = rjwomugtcv tsyouabshc (qovuccggta )
NCT05004675 (LIBerate-VI, BusinessWire) Manual	Phase 3	Atherosclerosis	166	Lerodalcibep 300 mg monthly	cifxreipxn(biciylaxmj) = bkfofhfxdz kxaujxwymu (adgfkbkrih ) View more	Positive	29 May 2024
				Inclisiran 284 mg on Day 1 and 90	cifxreipxn(biciylaxmj) = zpvdoukudj kxaujxwymu (adgfkbkrih ) View more
NCT04806893 (LIBerate-HeFH, Biospace) Manual	Phase 3	Cardiovascular Diseases PCSK9	922	Lerodalcibep 300 mg	jguepeoggb(vxwhynytfm) = jafiawkykq sjqxjmqiyt (glcqkxmhrn ) Met View more	Positive	08 Apr 2024
NCT04798430 (LIBerate-OLE, Biospace) Manual	Phase 3	Cardiovascular Diseases	421	Lerodalcibep	yogcidecsr(xosiszkvok) = kbdbtniejl mpyqczxnds (qstioaaiee )	Positive	07 Apr 2024
NCT04797104 (LIBerate-FH, Literature) Manual	Phase 3	Heterozygous familial hypercholesterolemia	478	Lerodalcibep	pdqpqcufyd(kodujlknhi) = gvrowpodww pmsugfpezj (qlobtdjdxk ) View more	Positive	28 Aug 2023
				placebo	pdqpqcufyd(kodujlknhi) = ksvwebffgd pmsugfpezj (qlobtdjdxk ) View more

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