Polycystic Kidney Diseases - Pipeline Insight, 2024

This “Polycystic Kidney Diseases - Pipeline Insight, 2024” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Polycystic Kidney Diseases 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

Polycystic Kidney Diseases: Understanding

Polycystic Kidney Diseases: Overview

Polycystic Kidney Disease (PKD) is a genetic disorder characterized by the development of multiple fluid-filled cysts in the kidneys. These cysts grow over time, leading to an enlargement of the kidneys and progressive loss of kidney function. PKD is primarily categorized into two types: autosomal dominant (ADPKD), which is the more common form and typically manifests in adulthood, and autosomal recessive (ARPKD), which is rarer and often presents symptoms in infancy or childhood. In both forms, the cysts disrupt normal kidney tissue, eventually leading to chronic kidney disease (CKD) or kidney failure.

In patients with polycystic kidney diseases (PKDs), the kidneys contain multiple fluid-filled cysts, although other organs may also be affected. Although PKD is inherited monogenically, it is phenotypically, genically and allelically heterogeneous. Autosomal dominant PKD (ADPKD) is the most common form of PKD and is generally an adult-onset, multisystem disorder that is characterized by gradually growing renal cysts that start to develop in utero and can originate from all areas of the kidneys, although cysts usually form in the distal regions of the nephron and the collecting duct. Progressive fibrocystic renal disease in ADPKD is often accompanied by hepatobiliary changes or other extrarenal abnormalities, such as intracranial arterial aneurysms.

To diagnose polycystic kidney disease and assess the size, number of cysts, and the health of kidney tissue, medical professionals commonly employ imaging techniques such as ultrasound, CT scans, and MRI scans. Ultrasound involves using a transducer emitting sound waves to create images of the kidneys, akin to sonar mapping. CT scans utilize X-ray beams to produce cross-sectional images, providing detailed views of the kidneys. MRI scans, employing magnetic fields and radio waves, generate high-resolution cross-sectional views, aiding in comprehensive evaluation and diagnosis of the condition.

The treatment of PKD focuses on managing symptoms and slowing the progression of kidney damage, as there is no cure. Blood pressure control, pain management, and lifestyle modifications, such as dietary changes and hydration, are key components of treatment. In recent years, new therapeutic approaches, such as the use of vasopressin receptor antagonists like tolvaptan, have shown promise in slowing cyst growth and preserving kidney function in ADPKD patients. Early diagnosis and regular monitoring are essential to improving the quality of life for individuals with PKD and delaying the onset of kidney failure.

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

Polycystic Kidney Diseases Emerging Drugs Chapters

This segment of the Polycystic Kidney Diseases report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Polycystic Kidney Diseases Emerging Drugs

Tamibarotene: Rege Nephro Co., Ltd.

Tamibarotene, developed by Rege Nephro Co., Ltd., is a retinoic acid receptor (RAR) agonist currently undergoing clinical trials for the treatment of autosomal dominant polycystic kidney disease (ADPKD). This drug, also known by its investigational code RN-014, is designed to inhibit cyst formation and improve renal function in patients suffering from this hereditary condition. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Autosomal dominant polycystic kidney disease (ADPKD).

RGLS8429: Regulus Therapeutics Inc.

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 data in preclinical models, where 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, RGLS4326. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Polycystic Kidney Diseases.

AL1311: AceLink Therapeutics, Inc.

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. Currently, the drug is in Preclinical stage of development for the treatment of Polycystic Kidney Diseases.

Polycystic Kidney Diseases: Therapeutic Assessment

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

Major Players in Polycystic Kidney Diseases

• There are approx. 8+ key companies which are developing the therapies for Polycystic Kidney Diseases. The companies which have their Polycystic Kidney Diseases drug candidates in the most advanced stage, i.e. Phase II include, Rege Nephro Co., Ltd.

Phases

The report covers around 10+ 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

Polycystic Kidney Diseases 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.

Polycystic Kidney Diseases: Pipeline Development Activities

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

Polycystic Kidney Diseases Report Insights

• Polycystic Kidney Diseases Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Rege Nephro Co., Ltd.
• Regulus Therapeutics Inc.
• Vertex Pharmaceuticals Incorporated
• AceLink Therapeutics, Inc.

Key Products

• Tamibarotene
• RGLS8429
• VX-407
• AL1311

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