Hypercholesterolemia Pipeline Summary

DelveInsight’s, “Hypercholesterolemia - Pipeline Insight, 2026” report provides comprehensive insights about 30+ companies and 32+ pipeline drugs in Hypercholesterolemia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Hypercholesterolemia: Understanding

Hypercholesterolemia: Overview

Hypercholesterolemia is a condition characterized by abnormally elevated blood cholesterol, particularly low-density lipoprotein cholesterol (LDL-C), which is a major modifiable risk factor for atherosclerotic cardiovascular disease (ASCVD). It may occur as primary hypercholesterolemia, including genetically determined familial hypercholesterolemia (FH), or secondary to other diseases, medications, or lifestyle factors. FH causes lifelong elevation of LDL-C and substantially increases the risk of premature coronary artery disease. Despite being relatively common, FH remains considerably underdiagnosed and undertreated.

Hypercholesterolemia is often asymptomatic, particularly during the early stages, and many individuals are identified through routine lipid testing. With prolonged exposure to high LDL-C, cholesterol can accumulate in tissues and blood vessels, contributing to atherosclerosis and cardiovascular complications such as coronary artery disease, myocardial infarction and stroke. Patients with severe or familial disease may develop tendon xanthomas, skin xanthomas or corneal arcus, although the absence of these findings does not exclude FH. Premature cardiovascular disease or a strong family history may provide an important clinical clue.

The causes range from genetic abnormalities affecting LDL metabolism to acquired secondary factors. Familial hypercholesterolemia most commonly involves pathogenic variants affecting the LDL receptor (LDLR), apolipoprotein B (APOB) or PCSK9, resulting in impaired hepatic clearance of circulating LDL particles. Consequently, LDL-C remains elevated from an early age, promoting progressive cholesterol deposition within arterial walls. Secondary hypercholesterolemia can result from conditions such as hypothyroidism, diabetes, kidney or liver disease, as well as certain medications and dietary or lifestyle factors.

Diagnosis is primarily based on a lipid profile, including LDL-C, total cholesterol, HDL-C and triglycerides, with repeated LDL-C measurements recommended when evaluating suspected FH. Clinical assessment incorporates personal and family history of premature ASCVD, physical findings and validated criteria such as the Dutch Lipid Clinic Network or Simon Broome criteria; genetic testing can identify pathogenic FH variants and facilitate cascade screening. Treatment aims to achieve substantial and sustained LDL-C reduction through a heart-healthy lifestyle and pharmacotherapy, with high-intensity statins generally serving as first-line therapy. Ezetimibe, PCSK9 inhibitors, bempedoic acid and other agents can be added when LDL-C remains above target, while severe homozygous FH may require specialized therapies such as evinacumab or lipoprotein apheresis.

"Hypercholesterolemia- Pipeline Insight, 2026" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hypercholesterolemia pipeline landscape is provided which includes the disease overview and Hypercholesterolemia treatment guidelines. The assessment part of the report embraces, in depth Hypercholesterolemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hypercholesterolemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Hypercholesterolemia R&D. The therapies under development are focused on novel approaches to treat/improve Hypercholesterolemia.

Hypercholesterolemia Emerging Drugs Chapters

This segment of the Hypercholesterolemia report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hypercholesterolemia Emerging Drugs

Zodasiran: Arrowhead Pharmaceuticals

Zodasiran is an investigational, liver-targeted RNA interference (RNAi) therapeutic designed to silence ANGPTL3, a key regulator of lipid metabolism that inhibits lipoprotein and endothelial lipases. By reducing ANGPTL3 production, zodasiran is intended to lower LDL-C and other atherogenic lipoproteins, with potential utility across severe forms of hypercholesterolemia, including homozygous familial hypercholesterolemia (HoFH). It is currently being evaluated in Phase III, including the YOSEMITE study in HoFH, with quarterly subcutaneous dosing and LDL-C reduction as the primary efficacy endpoint. The mechanism is particularly relevant to HoFH because ANGPTL3 inhibition can reduce atherogenic lipoproteins through pathways that are relatively independent of LDL-receptor function.

DNV001: Hangzhou Dinovate Biotech

DNV001 is an investigational small-interfering RNA (siRNA) therapy targeting PCSK9, designed to reduce hepatic PCSK9 production and thereby enhance LDL-receptor activity and clearance of circulating LDL-C. It is being evaluated for primary hypercholesterolemia and mixed hyperlipidemia in patients whose LDL-C remains inadequately controlled despite statin therapy. The ongoing Phase II study is assessing multiple subcutaneous dose regimens, with the primary endpoint being the percentage change in LDL-C from baseline at Week 24. DNV001 therefore represents a potentially longer-acting PCSK9-lowering approach within the emerging RNAi-based treatment landscape for hypercholesterolemia.

STX-1150: Scribe Therapeutics

STX-1150 is an investigational genetic therapy designed to lower LDL-C by epigenetically silencing the PCSK9 gene in the liver without altering the underlying DNA sequence. It combines mRNA and guide RNA (gRNA) encapsulated in lipid nanoparticles (LNPs) for intravenous delivery, enabling targeted modulation of hepatic PCSK9 expression. Reduced PCSK9 levels are expected to increase LDL-receptor availability on hepatocytes and enhance clearance of circulating LDL-C. STX-1150 is currently being evaluated in a Phase I study in adults with elevated LDL-C/high cholesterol, with its potential for durable LDL-C reduction being assessed.

Further product details are provided in the report……..

Hypercholesterolemia: Therapeutic Assessment

This segment of the report provides insights about the different Hypercholesterolemia drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Hypercholesterolemia
• There are approx. 30+ key companies which are developing the therapies for Hypercholesterolemia. The companies which have their Hypercholesterolemia drug candidates in the most advanced stage, i.e. Phase III include, Arrowhead Pharmaceuticals.

• Phases

DelveInsight’s report covers around 32+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

• Route of Administration

Hypercholesterolemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hypercholesterolemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Hypercholesterolemia therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hypercholesterolemia drugs.

Hypercholesterolemia Report Insights

• Hypercholesterolemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hypercholesterolemia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hypercholesterolemia drugs?
• How many Hypercholesterolemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hypercholesterolemia?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hypercholesterolemia therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hypercholesterolemia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Arrowhead Pharmaceuticals
• Hangzhou Dinovate Biotech
• Scribe Therapeutics
• Addpharma
• Jiangsu Hengrui Pharmaceuticals
• Qilu Pharmaceutical
• Novartis
• Eddingpharm Co., Ltd.
• Verve Therapeutics
• CRISPR Therapeutics
• Accuredit Therapeutics
• Emendo biotherapeutics

Key Products

• Zodasiran
• DNV001
• STX-1150
• AD-2281
• SHR-1918
• QLC7401
• QCZ484
• EDP167
• VERVE-201
• CTX310
• ART002g1
• EMD-301