Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease - Pipeline Insight, 2024

This “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Understanding

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease, is an inherited metabolic disorder in which this protein is absent or defective. The deficiency is caused by mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Alpha-1 antitrypsin is a large, 52‐kDa serum glycoprotein and a serine protease inhibitor. AAT is primarily produced in the liver and plays a crucial role in protecting the lungs from inflammation caused by infection and irritants such as tobacco smoke.

Alpha-1 antitrypsin deficiency occurs worldwide, but its prevalence varies by population. This disorder affects about 1 in 1,500 to 3,500 individuals with European ancestry. It is uncommon in people of Asian descent. Many individuals with alpha-1 antitrypsin deficiency are likely undiagnosed, particularly people with a lung condition called chronic obstructive pulmonary disease (COPD). COPD can be caused by alpha-1 antitrypsin deficiency; however, the alpha-1 antitrypsin deficiency is often never diagnosed. Some people with alpha-1 antitrypsin deficiency are misdiagnosed with asthma.

Some people with A1ATD experience no symptoms at all. But in some people it can lead to the development of lung conditions and liver disease. Symptoms of A1ATD Liver disease includes breathlessness during exercise or physical activity, cough that lasts a long time, production of phlegm, wheezing, repeated chest infections, prolonged jaundice, hepatomegaly, cholestasis, muscle wasting and abdominal pain.

In children, A1ATD Liver Disease is the most frequent genetic etiology for pediatric liver disease and transplantation. It is caused by a mutation in chromosome 14, the disease is inherited in an autosomal recessive manner with codominant expression. There are over 100 variants of the alpha-1 antitrypsin (A1AT) gene (SERPINA1), which can be influenced by other genetic and environmental factors, leading to marked variation in the onset and severity of disease. The liver disease associated with A1ATD is a gain-of-toxic function mechanism.

The diagnosis of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is confirmed with laboratory analysis. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Although liver biopsy is not required for diagnosis, it may be helpful in difficult cases and/or for prognostication. Anti-human A1AT antibody can also confirm the presence of ATZ globules. Electron microscopy studies can identify multiple globular inclusions within dilated, congested rough ER cisterns, adjacent to secondary lysosomes.

Treatment involves smoking cessation, preventive vaccinations, bronchodilators, supplemental oxygen when indicated, and physical rehabilitation in a program similar to that designed for patients with smoking-related COPD. Augmentation therapy may help slow lung damage. Evolving therapies under research studies for the treatment of A1ATD Liver Disease management includes: Chaperones, Autophagy-Enhancing Agents, Bile Acids as Small Molecule Therapy, Inhibiting Polymerization of ATZ, Gene Therapy and Editing, and Cell Therapy.

"Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline landscape is provided which includes the disease overview and Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease treatment guidelines. The assessment part of the report embraces, in depth Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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, Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease R&D. The therapies under development are focused on novel approaches to treat/improve Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs Chapters

This segment of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs

Fazirsiran: Arrowhead Pharmaceuticals

Fazirsiran (ARO-AAT) is a subcutaneous RNAi-based therapeutics, being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD). ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. Reducing production of the inflammatory Z-AAT protein is expected to halt the progression of liver disease and potentially allow it to regenerate and repair. The product candidate is being co-developed in partnership with Takeda. Currently, the drug is in the Phase III stage of its development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.

Alvelestat: Mereo BioPharma

Alvelestat (MPH966) is an oral drug that is being investigated for the treatment of Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD). Alvelestat acts to inhibit the neutrophil elastase enzyme and Mereo believes that it has the potential to help protect people with AATD by slowing progressive lung damage. While AATD may affect both the lungs and the liver, alvelestat’s mechanism of action only addresses lung disease, which is the most common effect of AATD in adults. Other companies are researching the effects of AATD on the liver. Currently, the drug is in the Phase II stage of its development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Therapeutic Assessment

This segment of the report provides insights about the different Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease

There are approx. 10+ key companies which are developing the therapies for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease. The companies which have their Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drug candidates in the most advanced stage, i.e. Phase III include, Arrowhead Pharmaceuticals.

Phases

The report covers around 15+ 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

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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:

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as:

• Monoclonal antibody
• Small molecule
• Peptide

Product Type

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

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Report Insights

• Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs?
• How many Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Arrowhead Pharmaceuticals
• Kamada Pharmaceuticals
• Novo Nordisk
• Peak Bio Inc
• Wave Life Sciences

Key Products

• Fazirsiran
• Inhaled AAT
• Belcesiran (DCR-A1AT)
• PHP 303
• WVE-006

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