Autosomal dominant polycystic kidney disease - Pipeline Insight, 2024

This “Autosomal dominant polycystic kidney disease - Pipeline Insight, 2024,” report provides comprehensive insights about 12+ companies and 12+ 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 (ADPKD) is a genetic disorder characterized by the formation of cysts within the kidneys. Symptoms caused by cyst formation in the kidneys include high blood pressure (hypertension), pain on the sides of the body between the last rib and the hip (flank pain), blood in the urine (hematuria) and progressively poor function of the kidneys (kidney insufficiency). In most patients, ADPKD eventually progresses to cause end stage renal disease, requiring renal replacement therapy, either dialysis or renal transplantation. ADPKD is not simply a kidney disorder and other organ systems of the body can potentially be affected (multisystem disorder) by the development of cysts. The specific symptoms present in each person depend upon the specific organ systems involved. The liver, pancreas, a membrane covering the spinal cord and brain (arachnoid membrane), the prostate, and the glands of the male reproductive tract that produce fluid that is part of semen (seminal vesicles) may become involved. Abnormalities affecting the heart and blood vessels (cardiovascular system) may also occur in individuals with ADPKD. ADPKD usually does not become apparent until the fourth or fifth decade and was once known as “adult” polycystic kidney disease. However, it has been reported in children and infants. ADPKD is caused by mutations of one of two genes that create certain proteins essential for the proper health of the kidneys and other parts of the body. Approximately 85 % have ADPKD1, the most aggressive form of the disease; those with ADPKD2 progress to kidney insufficiency about 20 years later.

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

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Autosomal dominant polycystic kidney disease.
• In the coming years, the Autosomal dominant polycystic kidney disease market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics that 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 the disease condition.
• A detailed portfolio of major pharma players who are involved in fueling the Autosomal dominant polycystic kidney disease treatment market. Several potential therapies for Autosomal dominant polycystic kidney disease are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Autosomal dominant polycystic kidney disease market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Autosomal dominant polycystic kidney disease) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

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 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

GLPG2737: Galapagos NVGLPG2737 is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) inhibitor which was observed to be well tolerated by patients in previous clinical trials. It is hypothesized that inhibition of the CFTR channel might reduce cyst growth and enlargement for patients with autosomal dominant polycystic kidney disease (ADPKD). In December 2020, we announced the first dosing with GLPG2737 in the Phase 2 MANGROVE trial in patients with ADPKD and in November 2021 full patient recruitment with topline results anticipated in the first half of 2023.

Bardoxolone methyl: Reata PharmaceuticalsBardoxolone is an investigational, once-daily, orally administered activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA and European Commission have granted Orphan Drug designation to bardoxolone for the treatment of Alport syndrome and autosomal dominant polycystic kidney disease (“ADPKD”). Bardoxolone is currently being studied in FALCON, a Phase 3 study for the treatment of CKD caused by ADPKD, EAGLE, an open-label, extended access trial in patients with CKD caused by Alport syndrome who participated in the CARDINAL trial and patients with ADPKD who participated in the FALCON trial, and AYAME, a Phase 3 study for the treatment of diabetic kidney disease that is being conducted by Kyowa Kirin in Japan.

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. 12+ 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 III include, Reata Pharmaceuticals.

Phases

This report covers around 12+ 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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

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

• Galapagos NV
• Reata Pharmaceuticals
• Kadmon Pharmaceuticals
• Sanofi
• Anakuria Therapeutics
• Regulus Therapeutics
• AceLink Therapeutics
• Healx
• XORTX Therapeutics
• Poxel
• Alebund Pharmaceuticals
• Chinook Therapeutics

Key Products

• GLPG2737
• Bardoxolone methyl
• Tesevatinib
• Venglustat
• AT 20494
• RGLS 8429
• AL 01211
• XRx-008
• PXL770

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