Autosomal Dominant Polycystic Kidney Disease - Pipeline Insight, 2024

This “Autosomal Dominant Polycystic Kidney Disease - Pipeline Insight, 2024” report provides comprehensive insights about 13+ companies and 14+ pipeline drugs in Autosomal Dominant Polycystic Kidney Disease 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

Autosomal Dominant Polycystic Kidney Disease: Understanding

Autosomal Dominant Polycystic Kidney Disease: Overview

Autosomal Dominant Polycystic Kidney Disease, an inherited kidney disease, is characterized by the relentless development and growth of cysts, causing progressive kidney enlargement associated with hypertension, abdominal fullness and pain, episodes of cyst hemorrhage, gross hematuria, nephrolithiasis, cyst infections, and reduced quality of life (QOL). Despite continuous destruction of renal parenchyma, compensatory hyperfiltration in surviving glomeruli maintains renal function within the normal range for decades. Only when the majority of nephrons have been destroyed, renal function declines, typically after the fourth decade of life, and ESRD eventually ensues. ADPKD is a systemic disorder affecting other organs with potentially serious complications such as massive hepatomegaly and intracranial aneurysm (ICA) rupture. Mutations in the PKD1 and PKD2 genes account for the overwhelming majority of ADPKD cases. There is no convincing evidence for the existence of a third PKD gene. Compared to PKD1, subjects affected with PKD2 mutations have milder renal disease with fewer renal cysts, delayed onset of hypertension and ESRD by almost two decades and longer patient survival. More recent studies have delineated a significant allelic effect in PKD1 with milder disease associated with non-truncating compared to truncating mutations. ADPKD is an inherited disorder characterized by progressive growth of kidney cysts, leading to gradual loss of kidney function and eventual kidney failure, typically after the fourth decade of life. The disease is caused by mutations in the PKD1 or PKD2 genes, with PKD2 mutations generally resulting in milder disease. Cysts arise from any tubular segment, disconnect from the tubule of origin, and continue to accumulate fluid within the lumen.

ADPKD is diagnosed on the basis of imaging. Given the low cost, safety, and availability of ultrasonography, it is a logical first choice for confirming a suspected ADPKD diagnosis. There are useful age-dependent ultrasound criteria for both diagnosis and disease exclusion when a family history of ADPKD has been established. In probands with PKD1 and PKD2 family history, the diagnosis is established by the presence of 3 or more kidney cysts (unilateral or bilateral) for at-risk individuals 15 to 39 years old, 2 (≥2 cysts in each kidney) for individuals 40 to 59 years old, and 8 (≥4 cysts in each) for individuals 60 years or older. If the genotype is unknown, the presence of at least 3 (unilateral or bilateral) kidney cysts, 2 cysts in each kidney, and 4 or more cysts in each kidney can be regarded as sufficient for the diagnosis of at-risk individuals aged 15 to 39, 40 to 59, and 60 years or older, respectively. Magnetic resonance imaging (MRI) and high-resolution ultrasound represent more advanced imaging techniques that may help with disease exclusion in at-risk individuals.

"Autosomal Dominant Polycystic Kidney Disease - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Autosomal Dominant Polycystic Kidney Disease pipeline landscape is provided which includes the disease overview and Autosomal Dominant Polycystic Kidney Disease treatment guidelines. The assessment part of the report embraces, in depth Autosomal Dominant Polycystic Kidney Disease 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, Autosomal Dominant Polycystic Kidney Disease 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 Autosomal Dominant Polycystic Kidney Disease R&D. The therapies under development are focused on novel approaches to treat/improve Autosomal Dominant Polycystic Kidney Disease.

Autosomal Dominant Polycystic Kidney Disease Emerging Drugs Chapters

This segment of the Autosomal Dominant Polycystic Kidney Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II/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.

Autosomal Dominant Polycystic Kidney Disease Emerging Drugs

RGLS8429: Regulus Therapeutics

RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound. The drug is currently being evaluated under Phase I clinical trial for the treatment of patients with Autosomal Dominant Polycystic Kidney Disease.

AL1311: AceLink Therapeutics

AL1311 is a highly potent NRF2 activator that is being developed for Autosomal Dominant Polycystic Kidney disease (ADPKD). AL1311 is different from other electrophilic and reactive NRF2 activators because it works by disrupting the protein-protein interaction between NRF2 and its regulatory protein Keap1. Non-reactive NRF2 activators have the potential to have better specificity for NRF2 activation and therefore a better safety profile. The drug is in the preclinical phase for the treatment of patients with Autosomal Dominant Polycystic Kidney Disease.

Autosomal Dominant Polycystic Kidney Disease: Therapeutic Assessment

This segment of the report provides insights about the different Autosomal Dominant Polycystic Kidney Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Autosomal Dominant Polycystic Kidney Disease

There are approx. 13+ key companies which are developing the therapies for Autosomal Dominant Polycystic Kidney Disease. The companies which have their Autosomal Dominant Polycystic Kidney Disease drug candidates in the most advanced stage, i.e. Phase I include, Regulus Therapeutics.

Phases

This report covers around 14+ 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

Autosomal Dominant Polycystic Kidney Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

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

Autosomal Dominant Polycystic Kidney Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Autosomal Dominant Polycystic Kidney Disease 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 Autosomal Dominant Polycystic Kidney Disease drugs.

Autosomal Dominant Polycystic Kidney Disease Report Insights

• Autosomal Dominant Polycystic Kidney Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Poxel
• Regulus Therapeutics
• AceLink Therapeutics
• DiNAQOR AG
• Shanghai Alebund Pharmaceuticals
• Rege Nephro

Key Products

• PXL770
• RGLS8429
• AL-1311
• DINAK-001
• AP-304
• RN-014

Please note: The report will be dispatched within 2-3 days