DelveInsight’s, “Niemann-Pick-Disease Pipeline Insight 2023” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in the Niemann-Pick-Disease pipeline landscape. It covers the Niemann-Pick-Disease 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.
Key Takeaways from the Niemann-Pick-Disease Pipeline Report
- DelveInsight’s Niemann-Pick-Disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Niemann-Pick-Disease treatment.
- The leading companies working in the Niemann-Pick-Disease Market include Orphazyme, Cyclo Therapeutics, Mandos LLC, IntraBio, E-scape Bio, Scenic Biotech, Synaptogenix, Evox Therapeutics, StrideBio, SOM Biotech, ENDECE, Oraxion Therapeutics, Polaryx Therapeutics, and others.
- Promising Niemann-Pick-Disease Pipeline Therapies in the various stages of development include N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, arimoclomol, IB1001, and others.
- September 2023: IntraBio Inc. announced a study of phase 3 clinical trials for N-Acetyl-L-Leucine. This is a multinational, randomized, placebo-controlled, double-blinded, cross-over Phase III study that will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) versus Placebo for the treatment of Niemann-Pick type C disease (NPC).
- September 2023: Genzyme, a Sanofi Company announced a study of phase 2 clinical trials for GZ402665. The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa.
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In the Niemann-Pick-Disease pipeline 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.
Niemann-Pick-Disease Overview
Niemann-Pick Diseases (NPD) include heterogeneous groups of autosomal recessive lysosomal lipid storage disorders, with common features of hepatosplenomegaly and sphingomyelin storage in reticuloendothelial and parenchymal tissues, with or without neurological involvement. Niemann-Pick Disease (NPD) is classified as Niemann-Pick Disease type A, Niemann-Pick Disease type B, and Niemann-Pick Disease type C.
Find out more about Niemann-Pick-Disease Therapeutics Assessment @ Niemann-Pick-Disease Preclinical and Discovery Stage Products
Niemann-Pick-Disease Emerging Drugs Profile
- Arimoclomol: Orphazyme
- Trappsol Cyclo: Cyclo Therapeutics
- VTS-270: Mandos LLC
- ESB1609: E-scape Bio
Niemann-Pick-Disease Pipeline Therapeutics Assessment
There are approx. 20+ key companies which are developing the Niemann-Pick-Disease therapies. The Niemann-Pick-Disease companies which have their Niemann-Pick-Disease drug candidates in the most advanced stage, i.e. Pre-Registration, Orphazyme.
Learn more about the emerging Niemann-Pick-Disease Pipeline Therapies @ Niemann-Pick-Disease Clinical Trials Assessment
Scope of the Niemann-Pick-Disease Pipeline Report
- Coverage- Global
- Niemann-Pick-Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Niemann-Pick-Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Niemann-Pick-Disease Companies- Orphazyme, Cyclo Therapeutics, Mandos LLC, IntraBio, E-scape Bio, Scenic Biotech, Synaptogenix, Evox Therapeutics, StrideBio, SOM Biotech, ENDECE, Oraxion Therapeutics, Polaryx Therapeutics, and others.
- Niemann-Pick-Disease Pipeline Therapies- N-Acetyl-L-Leucine, Hydroxypropyl-beta-cyclodextrin, arimoclomol, IB1001, and others.
Dive deep into rich insights for new drugs for Niemann-Pick-Disease treatment, Visit @ Niemann-Pick-Disease Market Drivers and Barriers, and Future Perspective
Table of Content
- Introduction
- Executive Summary
- Niemann-Pick-Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Niemann-Pick-Disease– DelveInsight’s Analytical Perspective
- Late Stage Products (Pre-Registration)
- Arimoclomol: Orphazyme
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Drug name : Company name
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- ESB1609: E-scape Bio
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name : Company name
- Drug profiles in the detailed report…..
- Inactive Products
- Niemann-Pick-Disease Key Companies
- Niemann-Pick-Disease Key Products
- Niemann-Pick-Disease- Unmet Needs
- Niemann-Pick-Disease- Market Drivers and Barriers
- Niemann-Pick-Disease- Future Perspectives and Conclusion
- Niemann-Pick-Disease Analyst Views
- Niemann-Pick-Disease Key Companies
- Appendix
For further information on the Niemann-Pick-Disease pipeline therapeutics, reach out to Niemann-Pick-Disease Unmet Needs and Analyst Views
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