Dublin, Nov. 21, 2024 (GLOBE NEWSWIRE) -- The "Niemann-Pick disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.
This "Niemann-Pick disease - Pipeline Insight, 2024," report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Niemann-Pick 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.
The report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann-Pick disease pipeline landscape is provided which includes the disease overview and Niemann-Pick disease treatment guidelines. The assessment part of the report embraces, in depth Niemann-Pick 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, Niemann-Pick 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 Niemann-Pick disease R&D. The therapies under development are focused on novel approaches to treat/improve Niemann-Pick disease.
Niemann-Pick disease Emerging Drugs Chapters
This segment of the Niemann-Pick 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.
Niemann-Pick disease Emerging Drugs
Trappsol Cyclo: Cyclo Therapeutics
Trappsol Cyclo is a proprietary therapeutic formulation developed by Cyclo Therapeutics, Inc., primarily aimed at treating Niemann-Pick Disease Type C1 (NPC1). The drug is composed of 2-hydroxypropyl-beta-cyclodextrin (HP-?-CD), which acts by facilitating the transport of excess cholesterol out of lysosomes. In NPC1, the defective NPC1 protein prevents effective cholesterol transport, leading to toxic buildup.
Trappsol Cyclo mimics this protein's function, helping to restore normal cholesterol homeostasis within cells. The drug is currently in a Phase III trial aimed at evaluating its long-term efficacy and safety in NPC patients. Previous Phase I and Phase II studies have shown promising results, indicating clinical benefits such as stabilization or slowing of disease progression.
Nizubaglustat: Azafaros
Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including Niemann-Pick disease type C (NPC). The results of the Phase II RAINBOW study mark a pivotal moment in the development of nizubaglustat. The dual mode of action sets nizubaglustat apart as a leap forward from other agents. The company plans to initiate a Phase III trial for the treatment of NPC by 2025.
Niemann-Pick disease: Therapeutic Assessment
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Niemann-Pick disease drugs?
- How many Niemann-Pick disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Niemann-Pick disease?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Niemann-Pick 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 Niemann-Pick disease and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Cyclo Therapeutics
- Azafaros
- IntraBio
Key Products
- Trappsol Cyclo
- Nizubaglustat
- Acetylleucine
Major Players in Niemann-Pick disease
There are approx. 10+ key companies which are developing the therapies for Niemann-Pick disease. The companies which have their Niemann-Pick disease drug candidates in the most advanced stage, i.e. Phase III include, Cyclo Therapeutics.
Phases
The 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
Niemann-Pick 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
- Oral
- Parenteral
- intravenous
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Niemann-Pick disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann-Pick 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 Niemann-Pick disease drugs.
Niemann-Pick disease Report Insights
- Niemann-Pick disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Niemann-Pick disease Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/7q1qf2
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