According to DelveInsight, the Alport Syndrome pipeline features over 4 key pharmaceutical companies actively engaged in developing more than 6 therapeutic candidates aimed at treating this rare disorder.
Alport Syndrome Overview:
Alport syndrome is a rare genetic disorder that affects roughly 1 in 50,000 individuals and is primarily characterized by microscopic hematuria and progressive chronic kidney disease (CKD), often accompanied by hearing and ocular abnormalities.
The disease results from mutations in the COL4A3, COL4A4, and COL4A5 genes, which impair the normal formation of the α3–α4–α5 collagen IV network—a crucial structural component of the basement membranes in the kidneys, inner ear, and eyes.
Early clinical signs, including hematuria, proteinuria, and edema, are often nonspecific, leading to frequent misdiagnosis with other kidney disorders. As the disease progresses, renal function deteriorates, leading to CKD or end-stage renal disease (ESRD).
Beyond the kidneys, sensorineural hearing loss commonly develops in childhood or adolescence and worsens over time. Ocular abnormalities such as anterior lenticonus and retinal changes are also observed, though these typically cause milder visual impairment.
At the molecular level, the disruption of type IV collagen synthesis weakens the structural integrity of basement membranes, resulting in thinning, splitting, and scarring that impair filtration in the kidneys and disrupt cochlear and ocular function—underlying the triad of renal, auditory, and ocular symptoms seen in Alport syndrome.
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DelveInsight’s “Alport Syndrome Pipeline Insight 2025” report offers an in-depth evaluation of the current clinical development landscape and future growth opportunities within the Alport Syndrome therapeutics market.
Key Takeaways from the Alport Syndrome Pipeline Report
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DelveInsight’s Alport Syndrome pipeline report highlights an active landscape with over four companies engaged in developing six or more therapeutic candidates for the treatment of Alport Syndrome.
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In January 2024, Enyo Pharma obtained FDA clearance for its Investigational New Drug (IND) application to initiate a Phase II clinical trial (ALPESTRIA-1) evaluating Vonafexor in patients with Alport Syndrome.
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Leading companies such as Enyo Pharma, Bayer, and others are advancing innovative therapies aimed at enhancing the Alport Syndrome treatment landscape.
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Notable candidates currently in development include Vonafexor, BAY 3401016, and additional promising therapies.
Alport Syndrome Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Alport Syndrome Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Alport Syndrome treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Alport Syndrome market.
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Alport Syndrome Emerging Drugs
Vonafexor – Enyo Pharma
Vonafexor (EYP001), developed by Enyo Pharma, is an investigational treatment targeting kidney disorders such as Alport syndrome and chronic kidney disease (CKD). It is a synthetic, non-steroidal, non-bile acid farnesoid X receptor (FXR) agonist with exceptional selectivity for FXR and no activity on the bile acid receptor TGR5. Distinct from other FXR agonists, Vonafexor’s unique molecular structure induces a differentiated gene expression profile influenced by its ligand-binding behavior. The therapy is currently being evaluated in Phase II clinical trials for 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 and inhibits semaphorin 3A (SEMA3A), a protein that plays a key role in neuronal signaling and immune system regulation. The therapy is currently in Phase I clinical development for the treatment of Alport syndrome.
Alport Syndrome Companies
Around four or more major companies are actively engaged in developing therapies for Alport Syndrome. Among these, Enyo Pharma leads with a drug candidate that has progressed to the most advanced stage—Phase II clinical development.
DelveInsight’s report covers around 75+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Alport Syndrome Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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Alport Syndrome Pipeline Therapeutic Assessment
• Alport Syndrome Assessment by Product Type
• Alport Syndrome By Stage
• Alport Syndrome Assessment by Route of Administration
• Alport Syndrome Assessment by Molecule Type
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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