Alport Syndrome - Pipeline Insight, 2025

DelveInsight’s, “Alport Syndrome - Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 6+ pipeline drugs in Alport Syndrome 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

Alport Syndrome: Understanding

Alport Syndrome: Overview

Alport Syndrome is a rare, genetic disorder that affects the kidneys, eyes, and hearing. It is primarily caused by mutations in genes responsible for producing type IV collagen, a vital protein for the structural integrity of kidney glomeruli, the inner ear, and the eye's lens. In individuals with Alport Syndrome, these mutations lead to progressive kidney damage, often resulting in chronic kidney disease and, ultimately, end-stage renal disease (ESRD) by early adulthood in severe cases. Hearing loss typically begins in childhood or adolescence, while eye abnormalities may cause vision issues but are generally less severe.

The signs and symptoms of Alport Syndrome primarily involve the kidneys, hearing, and vision. Kidney symptoms typically begin with blood in the urine (hematuria), which may be visible or detected microscopically, and may progress to proteinuria as kidney function declines, often leading to chronic kidney disease or end-stage renal disease. Hearing loss, usually sensorineural, commonly begins in childhood or adolescence and gradually worsens over time. Vision changes may include abnormalities in the lens (such as anterior lenticonus) and the retina, potentially affecting vision but typically not as severely as kidney and hearing issues. The pathology of Alport Syndrome centers on mutations in genes that code for type IV collagen, a key structural protein in the basement membranes of the kidneys, inner ear, and eyes. These mutations—most commonly in the COL4A3, COL4A4, or COL4A5 genes—impair the formation of collagen networks that support cellular structure and filtration functions. In the kidneys, these defects disrupt the glomerular basement membrane, causing it to thin, split, and eventually scar, which compromises its filtration ability and leads to blood and protein leakage in urine, ultimately resulting in kidney disease. In the inner ear, faulty collagen affects the cochlea, leading to sensorineural hearing loss.

Diagnosing Alport Syndrome involves a combination of clinical assessments, family history, genetic testing, and specialized examinations of the kidneys, eyes, and hearing. Initial clues often include persistent blood in the urine (hematuria), progressive kidney disease, or hearing loss, particularly when these symptoms appear in childhood. A kidney biopsy can reveal characteristic changes in the glomerular basement membrane, such as thinning or splitting, which are typical of Alport Syndrome. Eye exams may detect lens abnormalities like anterior lenticonus or retinal flecks, while hearing tests can confirm high-frequency sensorineural hearing loss.

""Alport Syndrome- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alport Syndrome pipeline landscape is provided which includes the disease overview and Alport Syndrome treatment guidelines. The assessment part of the report embraces, in depth Alport Syndrome 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, Alport Syndrome 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 Alport Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Alport Syndrome.

Alport Syndrome Emerging Drugs Chapters

This segment of the Alport Syndrome 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.

Alport Syndrome Emerging Drugs

• Vonafexor: Enyo Pharma

Vonafexor (EYP001) is a promising therapeutic candidate developed by Enyo Pharma, specifically designed to treat various kidney diseases, including Alport syndrome and chronic kidney disease (CKD). Vonafexor is a synthetic non-steroidal, non-bile acid farnesoid X receptor (FXR) agonist. It activates FXR with a high selectivity compared to other nuclear receptors and does not display any activity on bile acid receptor TGR5. This small molecule has a different structure compared to other FXR agonists and induces a differential set of target genes based on ligand binding patterns. Currently, the drug is in the Phase II stage of its development for the treatment of Alport Syndrome.

• BAY 3401016: Bayer

BAY 3401016, also known as SEMA 3A, is a monoclonal antibody developed by Bayer in collaboration with Evotec SE. It targets semaphorin 3A (SEMA3A), a protein implicated in various biological processes, including neuronal guidance and immune responses. Currently, the drug is in the Phase I stage of development to treat Alport Syndrome.

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

Alport Syndrome: Therapeutic Assessment

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

• Major Players in Alport Syndrome
• There are approx. 4+ key companies which are developing the therapies for Alport Syndrome. The companies which have their Alport Syndrome drug candidates in the most advanced stage, i.e. Phase II include, Enyo Pharma.
• Phases

DelveInsight’s report covers around 6+ 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

Alport Syndrome 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.

Alport Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Alport Syndrome 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 Alport Syndrome drugs.

Alport Syndrome Report Insights

• Alport Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Alport Syndrome 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 Alport Syndrome drugs?
• How many Alport Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alport Syndrome?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alport Syndrome 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 Alport Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Enyo Pharma
• Bayer
• ZyVersa Therapeutics

Key Products

• Vonafexor
• BAY 3401016
• VAR 200-02