Alpha- Antitrypsin Deficiency - Pipeline Insight, 2024

This “Alpha-1 antitrypsin deficiency -Pipeline Insight, 2024,” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Alpha-1 antitrypsin deficiency 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 Understanding

Alpha-1 antitrypsin deficiency: Overview

Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease. The signs and symptoms of the condition and the age at which they appear vary among individuals. People with alpha-1 antitrypsin deficiency usually develop the first signs and symptoms of lung disease between ages 20 and 50. Mutations in the SERPINA1 gene cause alpha-1 antitrypsin deficiency. This gene provides instructions for making a protein called alpha-1 antitrypsin, which protects the body from a powerful enzyme called neutrophil elastase. Without enough functional alpha-1 antitrypsin, neutrophil elastase destroys alveoli and causes lung disease. Abnormal alpha-1 antitrypsin can also accumulate in the liver and damage this organ. Environmental factors, such as exposure to tobacco smoke, chemicals, and dust, likely impact the severity of alpha-1 antitrypsin deficiency.

Symptoms
The earliest symptoms of Alpha-1 antitrypsin deficiency are shortness of breath following mild activity, reduced ability to exercise, and wheezing. Other signs and symptoms can include unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Affected individuals often develop emphysema, which is a lung disease caused by damage to the small air sacs in the lungs (alveoli). Characteristic features of emphysema include difficulty breathing, a hacking cough, and a barrel-shaped chest.

Diagnosis
Alpha-1 antitrypsin deficiency only can be detected through blood tests. One type of blood test measures the body’s level of Alpha-1 antitrypsin. If the Alpha-1 antitrypsin level is lower than normal, the healthcare provider may order a genotype or a phenotype blood test. These genetic analysis tests look at the amount and type of Alpha-1 antitrypsin being produced and compare it with normal patterns. One test in a lifetime is sufficient for diagnosis.

Treatment
At this time, there is no cure for Alpha-1 antitrypsin deficiency, but there are treatments that can improve symptoms. The healthcare provider may prescribe medications such as bronchodilators or inhaled steroids to help open the airways. The healthcare provider also may recommend pulmonary rehabilitation to improve breathing. People who have severe emphysema from Alpha-1 antitrypsin deficiency may be candidates for a lung transplant. A treatment called Alpha-1 antitrypsin augmentation therapy, which may slow down or stop the destruction of lung tissue, may also be prescribed.

Alpha-1 antitrypsin deficiency Emerging Drugs Chapters

This segment of the Alpha-1 antitrypsin deficiency 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 Emerging Drugs

VX-814 and VX-864: Vertex Pharmaceuticals Vertex Pharmaceuticals is investigating the use of small molecules, which have the potential to address the underlying cause of disease and impact the lung and liver disease associated with AATD. We are currently investigating two candidate medicines, VX-814 and VX-864, and continue to discover and develop a portfolio of medicines for the potential treatment of AATD. It is currently in phase II stage of development.

ZF874: Z Factor Ltd ZF874 was developed with the help of a proprietary crystal structure solved by the Huntington lab. It is a novel compound that acts as a molecular ‘patch’ for the faulty protein, allowing it to fold correctly, thereby simultaneously relieving the liver burden of polymer accumulation and providing fully-functional Z-A1AT in the circulation to protect the lungs. In mice genetically engineered to express human Z-A1AT in their livers, oral doses of ZF874 were able to substantially increase levels of correctly folded protein in the blood and to completely eliminate accumulation of misfolded protein in the liver. It is currently in phase I stage ofdevelopment.

APB-101: Apic Bio APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. APB-101 has achieved a pre-clinical proof of concept with efficacy demonstrated in vitro and in vivo. It is currently undergoing pre-clinical GLP toxicology studies in non-human primates. Patients living with Alpha 1 lack sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases, and experience the accumulation of mutant AAT polymers in theliver.

Alpha-1 antitrypsin deficiency: Therapeutic Assessment

This segment of the report provides insights about the different Alpha-1 antitrypsin deficiency drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Alpha-1 antitrypsin deficiency

There are approx. 40+ key companies which are developing the therapies for Alpha-1 antitrypsin deficiency. The companies which have their Alpha-1 antitrypsin deficiency drug candidates in the most advanced stage, i.e. phase II include Vertex Pharmaceuticals and others

Phases

This report covers around 40+ products under different phases of clinical development like

• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Alpha-1 antitrypsin deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

Product Type

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

Alpha-1 antitrypsin deficiency: 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 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 drugs.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Alpha-1 antitrypsin deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Alpha-1 antitrypsin deficiency.

• In July 2019, Vertex Pharmaceuticals reported that VX 864 received fast track designation from the US FDA for the treatment of alpha-1 antitrypsin (AAT) deficiency.
• Apic Bio’s Thrive platform both silences a disease-causing gene product and replaces a normal gene product in a single “dual-function” vector.
• Apic was founded as a spinout of University of Massachusetts Medical School by scientific co-founders Robert Brown Jr. and Christian Mueller, who were looking to combine mi RNA silencing with gene therapy. UMass was recently awarded a U.S. patent for r AAV-Based Compositions and Methods, which it licensed toApicBio.

Alpha-1 antitrypsin deficiency Report Insights

• Alpha-1 antitrypsin deficiency Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Alpha-1 antitrypsin deficiency 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 drugs?
• How many Alpha-1 antitrypsin deficiency 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?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Alpha-1 antitrypsin deficiency 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 and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Intellia Therapeutics
• Apic Bio
• Linton Pharm
• Z Factor Limited
• Vertex Pharmaceuticals
• Grifols
• Kamada
• Octapharma
• PPL Therapeutics
• Precigen Inc

Key Products

• APB 101

Research programme: gene editing therapeutics- LP 503

• ZF 874
• VX 864
• Alpha-1-antitrypsin inhalation
• Alpha-1 antitrypsin
• OctaAlpha1
• Alpha-1-antitrypsin subcutaneous

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