Hyperoxaluria - Pipeline Insight, 2025

This “Hyperoxaluria - Pipeline Insight, 2025” report provides comprehensive insights about 2+ companies and 3+ pipeline drugs in Hyperoxaluria 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.

Hyperoxaluria: Understanding

Hyperoxaluria: Overview

Hyperoxaluria, characterized by elevated urinary oxalate levels, is a significant contributor to kidney stone formation, particularly calcium oxalate stones. It can be classified into primary and secondary forms based on its underlying cause. While both types are associated with kidney stones, they differ in severity, onset, and complications. Effective management requires early detection, often through 24-hour urine testing, and preventive therapies to reduce recurrence and mitigate kidney damage. Hyperoxaluria can also lead to kidney damage even in the absence of stones, causing tubular toxicity and fibrosis, highlighting the importance of timely diagnosis and management.

Nephrolithiasis affects approximately 10% to 15% of individuals in the developed world, with men having a higher incidence than women. In the United States, the prevalence is about 10% in men and 7% in women, and recurrence rates for kidney stones can reach 50% within 10 years. Calcium-based stones, particularly calcium oxalate, make up about 80% of all kidney stones, with recurrence rates around 60% over a decade if preventive measures are not implemented. Secondary hyperoxaluria, a key contributor to recurrent stones, affects 25% to 45% of stone formers and is more prevalent in certain populations, particularly in Asia. Obesity and genetics also play a role in elevated urinary oxalate levels. Primary hyperoxaluria (PH), a rare genetic disorder, primarily affects children and is diagnosed late, often after nephrocalcinosis or end-stage renal disease develops, with a prevalence of 1 to 3 per 1 million in the U.S. and higher rates in inbred and developing populations.

Oxalate is an organic acid primarily found in plant foods, particularly in green leafy vegetables like spinach. While it has no known nutritional value for humans, oxalate can be absorbed in the colon, metabolized in the liver, and excreted in the urine, where it binds with calcium to form crystals. This can lead to the formation of calcium oxalate kidney stones, the most common type of stone. Oxalate is a potent promoter of kidney stones, being 15 to 20 times more effective than urinary calcium in promoting stone formation. Stone formation occurs through a series of steps, including nucleation, supersaturation, crystal growth, and agglomeration, influenced by factors such as urine pH, supersaturation levels, and the presence of crystallization inhibitors. In cases of elevated oxalate levels, such as in hyperoxaluria, calcium oxalate can accumulate in the renal tissues, causing nephrocalcinosis, inflammation, and potential renal damage, leading to chronic kidney disease (CKD) and end-stage renal disease (ESRD). Additionally, oxalate crystals may contribute to kidney fibrosis and damage through inflammation and cellular toxicity.

Treatment of hyperoxaluria involves a combination of conservative medical measures, surgical interventions for nephrolithiasis, and specific therapies aimed at reducing oxalate levels. Conservative management focuses on increasing fluid intake to enhance urine volume and reduce calcium oxalate supersaturation, with a target of at least 3 liters of urine per day. Urinary citrate supplementation with potassium citrate helps prevent crystal aggregation by maintaining a favorable urine pH, and dietary modifications, such as limiting oxalate-rich foods, are recommended. Pyridoxine (vitamin B6) supplementation can significantly reduce hyperoxaluria in patients with type I primary hyperoxaluria (PH), as it enhances the activity of Alanine-Glyoxylate Aminotransferase (AGT). For patients with secondary hyperoxaluria, dietary changes, calcium supplementation, and limiting excessive vitamin C intake are beneficial. Novel treatments include oral Oxalobacter formigenes supplements, anti-inflammatory agents targeting NLRP3 inflammasome activation, and the use of orthophosphates and magnesium supplements to reduce oxalate absorption. Other therapies, such as cholestyramine for enteric hyperoxaluria and pentosan polysulfate for inhibiting crystal aggregation, are also being explored. In cases of larger stones or those that do not pass, surgical intervention may be necessary.

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

Hyperoxaluria Emerging Drugs Chapters

This segment of the Hyperoxaluria 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.

Hyperoxaluria Emerging Drugs

Stiripentol: Biocodex

Stiripentol is an innovative therapeutic agent developed by Biocodex for the treatment of hyperoxaluria, particularly in conditions such as primary hyperoxaluria. This drug functions primarily as an inhibitor of Lactate Dehydrogenase A (LDHA), an enzyme crucial in the metabolic pathway that converts Glyoxylate into Oxalate in the liver. By inhibiting LDHA, stiripentol effectively reduces the synthesis of oxalate, thereby decreasing urinary oxalate excretion and mitigating the risk of calcium oxalate crystal formation in the kidneys. This mechanism not only addresses the underlying cause of hyperoxaluria but also offers a potential therapeutic strategy to protect renal function and improve patient outcomes in those affected. Currently, the drug is in Phase III stage of its development for the treatment of Hyperoxaluria.

META 001 PH: META Pharmaceuticals

META-001-PH is a groundbreaking small molecule drug developed by META for the treatment of primary hyperoxaluria. Preclinical experiments in animal disease models have shown that META-001-PH can significantly reduce urinary oxalate excretion by up to 80%. While existing therapeutic agents are unable to effectively control urinary oxalate levels in the long term, META-001-PH, administered orally daily, can maintain oxalate at normal levels, thus demonstrating the potential for better long-term control of kidney stone formation in patients with PH. META-001-PH has also demonstrated good tolerability and safety in preclinical animal models and is undergoing IND-enabling toxicology studies. META 001 PH has also been granted Rare Pediatric Disease Designation by the FDA. Currently, the drug is in Preclinical stage of its development for the treatment of Hyperoxaluria.

Hyperoxaluria: Therapeutic Assessment

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

Major Players in Hyperoxaluria

• There are approx. 2+ key companies which are developing the therapies for Hyperoxaluria. The companies which have their Hyperoxaluria drug candidates in the most advanced stage, i.e. Phase II include, Biocodex.

Phases

The report covers around 3+ 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

Hyperoxaluria 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.

Hyperoxaluria: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Hyperoxaluria 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 Hyperoxaluria drugs.

Hyperoxaluria Report Insights

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

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

Key Players

• Biocodex
• META Pharmaceuticals
• Arbor Biotechnologies

Key Products

• Stiripentol
• META 001 PH
• ABO-101

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