Pompe Disease - Pipeline Insight, 2024

This “Pompe Disease - Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Pompe 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

Pompe Disease: Understanding

Pompe Disease: Overview

Pompe disease also known as Glycogen Storage Disease Type II belongs to the 'lysosomal storage disorders' (LSDs) group of diseases. Within the cells, lysosomes are tiny compartments where all sorts of substances are recycled. By the action of digestive enzymes, the compounds are degraded. There are currently more than 50 different LSDs that are considered to be caused by the deficiency of one of these enzymes. One such enzyme is acid alpha-glucosidase (GAA) and is responsible for glycogen's lysosomal degradation. Glycogen accumulates inside the lysosomes due to a lack or dysfunction of GAA, which eventually leads to cellular failure, cellular damage, tissue damage, and ultimately organ dysfunction.

The dysfunction of the organ in Pompe disease is primarily expressed by muscle weakness and muscle wasting. Common symptoms of hypertrophic cardiomyopathy (HCM) include irregular thickening of the heart walls, particularly in the left ventricle and the septum separating the left and right chambers, which can lead to reduced cardiac activity. Affected individuals may experience rapidly progressive muscle weakness, often referred to as "floppy babies" in infants, along with reduced muscle tone (hypotonia) and respiratory deficiencies. Additionally, HCM is characterized by symptoms such as chest pain, shortness of breath, fatigue, and episodes of fainting, especially during physical exertion. These manifestations can be serious, as they may indicate underlying heart disease associated with HCM, making early diagnosis and management crucial for affected individuals.

Although a precise mechanism has not been described, lysosomal glycogen accumulation leads to impairment of striated muscular cells. Initially, affected areas involve active voluntary muscles (extremities), progressing to cardiac muscle, and lastly, the diaphragm. The diagnosis of Pompe disease is ultimately confirmed with enzyme assays and genetic testing. Clinical history, exam, muscle enzymes, and electromyography (EMG) are the core of the initial workup and are used to help determine which patient should undergo further testing specific to Pompe disease, particularly in the setting of LOPD.

The treatment of Pompe disease is disease-specific, symptomatic and supportive. Alglucosidase alfa (Myozyme/Lumizyme, Sanofi Genzyme) enzyme replacement therapy (ERT) using recombinant human GAA (rhGAA), is currently the only approved treatment for Pompe disease. Although, the ERT appears to slow disease progression but has not been shown to halt or reverse disease for the majority of patients, and thus, significant unmet medical needs remain. Supportive Therapies include Physical therapy that are helpful to strengthen respiratory muscles.

Some patients may need respiratory assistance through mechanical ventilation (i.e. Bipap or volume ventilators) during the night and/or periods of the day or during respiratory tract infections. Mechanical ventilation support can be through noninvasive or invasive techniques. Physiotherapy is recommended to improve strength and physical ability. Occupational therapy, including the use of canes or walkers, may be necessary.

"Pompe Disease- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Pompe Disease pipeline landscape is provided which includes the disease overview and Pompe Disease treatment guidelines. The assessment part of the report embraces, in depth Pompe 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, Pompe 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 Pompe Disease R&D. The therapies under development are focused on novel approaches to treat/improve Pompe Disease.

Pompe Disease Emerging Drugs Chapters

This segment of the Pompe Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

Pompe Disease Emerging Drugs

GC301: GeneCradle Therapeutics

GC301 injection is an AAV gene therapy drug designed and developed by Genecradle Therapeutics for the treatment of Pompe disease. It adopts a one-time intravenous injection strategy for widespread systemic expression, aiming to directly compensate for the GAA gene deficiency in tissues such as the liver, heart, skeletal muscles, and central nervous system. In previous clinical trials conducted (IIT+IND), IOPD subjects successfully discontinued enzyme replacement therapy after gene therapy, with varying degrees of improvement in motor ability. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Pompe disease.

MZE001: Maze Therapeutics

MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. GYS1 is an enzyme responsible for glycogen production. MZE001 is currently being evaluated as a potential oral treatment for patients with Pompe disease, as well as other glycogen storage disorders. Currently, the drug is in Phase I stage of its clinical trial for the treatment of pompe disease.

Pompe Disease: Therapeutic Assessment

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

Major Players in Pompe Disease

• There are approx. 15+ key companies which are developing the therapies for Pompe Disease. The companies which have their Pompe Disease drug candidates in the most advanced stage, i.e. Phase I/II include, GeneCradle Therapeutics.

Phases

The 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

Pompe 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Pompe Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Pompe 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 Pompe Disease drugs.

Pompe Disease Report Insights

• Pompe Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Pompe 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 Pompe Disease drugs?
• How many Pompe Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pompe Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pompe 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 Pompe Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Asklepios Biopharmaceutical
• GeneCradle Therapeutics
• Astellas Gene Therapies
• Maze Therapeutics
• M6P Therapeutics
• Entrada Therapeutics

Key Products

• AAV2/8LSPhGAA
• GC301
• AT845
• MZE 001
• M 021
• ERT/Oligonucleotide

This product will be delivered within 1-3 business days.