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 8+ companies and 10+ 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) is a common inherited cause of liver disease, with the most severe mutation found in 1:3500 live births and currently 180,000 individuals worldwide. In children, A1ATD is the most frequent genetic etiology for pediatric liver disease and transplantation. The diagnosis of A1ATD is confirmed with laboratory analysis. Serum levels of A1AT can result in a false positive result because it is also an acute phase reactant and therefore elevated in the setting of inflammation. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Phenotypic expression varies within and between families. In adults, smoking is the major factor in accelerating the development of COPD; nonsmokers may have a normal life span, but can also develop lung and/or liver disease. Although reported, emphysema in children with AATD is extremely rare. AATD-associated liver disease, which is present in only a small portion of affected children, manifests as neonatal cholestasis. The incidence of liver disease increases with age. Liver disease in adults (manifesting as cirrhosis and fibrosis) may occur in the absence of a history of neonatal or childhood liver disease. The risk for hepatocellular carcinoma (HCC) is increased in individuals with AATD.

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

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.
• A detailed portfolio of major pharma players who are involved in fueling the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease treatment market. Several potential therapies for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
• In July 2021, Arrowhead Pharmaceuticals announced that it was granted Breakthrough Therapy designation (BTD) by the US FDA for ARO-AAT, also known as TAK-999, being co-developed with Takeda Pharmaceutical Company as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
• In July 2021, Dicerna Pharmaceuticals announced interim results from the four completed active-treatment dose cohorts (0.1, 1.0, 3.0 and 6.0 mg/kg) of its Phase I double-blind, placebo-controlled, randomized trial of belcesiran, an investigational Gal XC™ RNAi therapeutic in development for the treatment of alpha-1 antitrypsin deficiency-associated liver disease(AATLD).
• In October 2020, Arrowhead and Takeda Pharmaceutical Company announced a collaboration and licensing agreement to develop ARO-AAT. Under the terms of the agreement, Arrowhead and Takeda will co-develop ARO-AAT which, if approved, will be co-commercialized in the U.S. under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global commercialization strategy and receive an exclusive license to commercialize ARO-AAT with Arrowhead eligible to receive tiered royalties of 20-25% on net sales. Arrowhead will receive an upfront payment of $300 million and is eligible to receive potential development, regulatory and commercial milestones up to $740 million.

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 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.

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

ARO-AAT: Arrowhead Pharmaceuticals ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited (Takeda”) as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).

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. 8+ 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 II/III include, Arrowhead Pharmaceuticals.

Phases

This report covers around 10+ 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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

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

• Centessa Pharmaceuticals
• Dicerna Pharmaceuticals
• PH Pharmaceuticals
• Arrowhead Pharmaceuticals
• Alnylam Pharmaceuticals
• LogicBio Therapeutics
• WaVe life Sciences
• Wuhan Healthgen Biotechnology

Key Products

• ZF874
• Belcesiran
• PHP 303
• ARO-AAT
• ALN-AAT02
• LB 201
• WVE 006
• Recombinant human alpha-1 antitrypsin

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