Niemann-Pick Disease - Pipeline Insight, 2024

This “Niemann-Pick-Disease- Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 20+ 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.

Geography Covered

• Global coverage

Niemann-Pick-Disease: Understanding

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. Niemann-Pick Disease type A is characterized by severe, early CNS deterioration and massive visceral and cerebral sphingomyelin storage. Type B is related to the chronic cause with marked visceral involvement but a sparing of the nervous system. Type C & D are characterized by sub-acute nervous system involvement with a moderate and slower course and a milder visceral storage., NPC has an extremely heterogeneous clinical presentation, but it is characterized by a range of progressive neurological problems that become severe and limiting at a later stage of the disease. Niemann-Pick disease type A occurs in infancy and is characterized by hepatosplenomegaly, jaundice, failure to thrive, and profound neurodegeneration leading to death by age 3 years. Ocular findings include a cherry-red spot in at least 50% of cases, mild corneal clouding, and brown granular discoloration of the anterior lens cortex or capsule. The clinical course is similar to Tay-Sachs disease; however, the visual loss is delayed due to the preservation of ganglion cells resulting in a less well-defined opacification that extends farther into the periphery but persists. Niemann-Pick disease type A occurs more frequently among individuals of Ashkenazi Jewish descent. Niemann-Pick disease type B is a non-neuropathic form that occurs in all populations. Patients tend to have normal vision, hepatosplenomegaly, and usually survive into adulthood. A macular halo is classically observed. Niemann-Pick disease type C is caused by mutations in either the NPC1 (~95%) or NPC2 (~5%) gene. The NPC1 gene produces a protein that is located in membranes inside the cell and is involved in the movement of cholesterol and lipids within cells. A deficiency of this protein leads to the abnormal build-up of lipids and cholesterol within cells. The NPC2 gene produces a protein that binds and transports cholesterol, although its exact function is not fully understood. Type C is characterized by onset in childhood with progressive psychomotor deterioration, moderate visceral and central nervous system involvement, vertical ophthalmoplegia, normal vision, and a macular halo similar to that seen in Type B. Type C is usually fatal by age 20. A presumptive diagnosis can be established in the setting of clinical symptoms, abnormal radiologic findings, and associated hepatosplenomegaly. The diagnosis of NP disease may be confirmed by genetic testing or determination of sphingomyelinase activity in peripheral blood leukocytes or cultured skin fibroblasts. No specific therapy is available for NPD.

Niemann-Pick-Disease- Pipeline Insight, 2024 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

Arimoclomol: Orphazyme Arimoclomol, a small molecule activator of chaperones present in cells under stress is under development for the treatment of Niemann-Pick Disease and Gaucher’s Disease. The drug candidate is an add-on therapy, which is under Phase II/III stage of development for the treatment of NPC. It is an orally administered drug with the bioavailability of 80-90%. It acts by targeting heat shock protein 70 (HSP70).

Trappsol Cyclo: Cyclo Therapeutics Trappsol Cyclo is a proprietary formulation of hydroxypropyl beta cyclodextrin and in multiple clinical studies has shown encouraging results to effectively manage the transportation of cholesterol. Taking the place of the defective NPC1 protein, Trappsol® Cyclo™, with its cyclic structure, facilitates the transport of accumulated cholesterol out of cellular lysosomes so it can be further processed and excreted out of cells. Trappsol Cyclo is currently being evaluated in Phase III clinical trials for the potential treatment of Niemann-Pick Disease Type C1 (NPC), a rare, fatal and progressive genetic disorder and Alzheimer’s disease, an irreversible, progressive neurological disorder, in which high cholesterol is also implicated as a risk factor. Cyclo Therapeutics recently formed a Global Steering Committee (GSC) comprised of Key Opinion Leaders and experts on lysosomal storage diseases to guide the pivotal Phase 3 global clinical development program of Trappsol® Cyclo™ for the treatment of NPC.

VTS-270: Mandos LLCVTS-270 (also known as Kleptose; 2-Hydroxypropyl- beta-cyclodextrin; HP-beta- CD; HPBCD) is under development by Mandos and is under Phase II/III stage of development for the treatment of NPC type 1. VTS-270 is a mixture of 2-hydroxypropyl-B-cyclodextrins with a specific compositional fingerprint for the treatment of NPC1. The therapeutic candidate targets cholesterol and modulates its activity. Thus, it enables the transport of accumulated cholesterol in lysosomes. The therapeutic candidate is administered by intrathecal route.

ESB1609: E-scape Bio ESB1609 is a novel, orally-administered, brain-penetrant and highly-selective sphingosine 1-phosphate 5 (S1P5) receptor agonist. S1P5 receptors are preferentially expressed within the CNS. Agonizing S1P5 receptors upregulate CNS lipid transporters and is a powerful upstream target rectifying lysosomal deficits. Results from preclinical studies have demonstrated the potential of ESB1609 to restore dysfunctional CNS lipid homeostasis and reduce downstream markers of neurodegeneration in animal models of neurodegeneration. Currently it is being evaluated in Phase I stage of development for the treatment of Niemann-Pick disease type C and Parkinson'sdisease.

Niemann-Pick-Disease: Therapeutic Assessment

This segment of the report provides insights about the different Niemann-Pick-Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Niemann-Pick-Disease

There are approx. 20+ 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. Pre-Registration, Orphazyme.

Phases

This report covers around 20+ 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

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

Key Questions

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

• Orphazyme
• Cyclo Therapeutics
• Mandos LLC
• IntraBio
• E-scape Bio
• Scenic Biotech
• Synaptogenix
• Evox Therapeutics
• StrideBio
• SOM Biotech
• ENDECE
• Oraxion Therapeutics
• Polaryx Therapeutics

Key Products

• ESB1609
• IB1001
• VTS-270
• Trappsol Cyclo

Research programme: gene modulating therapeutics- Bryostatin-1: gene modulating therapeutics
Research programme: exosomes based therapeutics (EVX-101)- STRX 210

• SOM 0208
• NDC 1308
• ORX-301
• PLX-100
• PLX-300

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