Niemann-Pick disease - Pipeline Insight, 2024

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.

Geography Covered

• Global coverage

Niemann-Pick disease Understanding

Niemann-Pick disease: Overview

Niemann-Pick disease (NPD) is a group of inherited metabolic disorders characterized by the abnormal accumulation of lipids in various tissues of the body. This accumulation leads to the progressive deterioration of cells and organs, including the liver, spleen, lungs, bone marrow, and brain. There are different types of Niemann-Pick disease, classified primarily into Types A, B, and C, each distinguished by genetic cause, clinical features, and progression. The disease is caused by mutations in genes responsible for lipid metabolism, particularly the SMPD1 gene (Types A and B) and NPC1 or NPC2 genes (Type C). These mutations disrupt normal lipid processing and trafficking, leading to lipid buildup in cells.

Type A Niemann-Pick disease is the most severe form and typically presents in infancy. It is characterized by rapidly progressive neurological decline, hepatosplenomegaly (enlarged liver and spleen), and failure to thrive. Unfortunately, children with Type A often do not survive past early childhood due to severe neurodegeneration. Type B, also known as the non-neuropathic form, has a later onset and is generally milder. Individuals with Type B may have a more normal lifespan but still experience symptoms such as enlarged organs and respiratory complications. Neurological involvement is absent or mild in this form.

Niemann-Pick Type C (NPC) is distinct from Types A and B in its genetic origin and pathophysiology. NPC is caused by mutations in the NPC1 or NPC2 genes, which affect cholesterol transport within cells. This leads to a buildup of cholesterol and other lipids within the lysosomes. NPC has a highly variable onset, from infancy to adulthood, and presents with a wide range of symptoms, including difficulty in coordinating movement (ataxia), cognitive decline, and psychiatric symptoms. In some cases, vertical gaze palsy, a difficulty in moving the eyes up and down, is a key diagnostic feature.

Diagnosis of Niemann-Pick disease involves genetic testing to identify mutations in the SMPD1, NPC1, or NPC2 genes, along with biochemical tests such as sphingomyelinase enzyme assays (for Types A and B) and tests for cholesterol storage and trafficking in NPC. Imaging studies and biopsies of affected tissues may also support the diagnosis. Early diagnosis is critical, especially for Type C, as emerging treatments may help slow disease progression.

Currently, treatment options are limited and mainly supportive. For Niemann-Pick Types A and B, enzyme replacement therapies are being investigated, while in Type C, medications like miglustat are used to reduce lipid accumulation. However, these therapies are not curative, and managing symptoms remains the primary approach. Researchers are exploring gene therapy, substrate reduction therapy, and other innovative approaches to address the underlying causes of Niemann-Pick disease.
"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

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-β-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

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. 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

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

• Cyclo Therapeutics
• Azafaros
• IntraBio

Key Products

• Trappsol Cyclo
• Nizubaglustat
• Acetylleucine

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