Pulmonary Arterial Hypertension (PAH)-Pipeline Insight, 2020

DelveInsights, Pulmonary Arterial Hypertension (PAH) Pipeline Insight, 2020, report provides comprehensive insights about 100+ companies and 100+ pipeline drugs in Pulmonary Arterial Hypertension 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
• Pulmonary Arterial Hypertension Understanding

Pulmonary Arterial Hypertension: Overview

Pulmonary arterial hypertension is a progressive disorder characterized by abnormally high blood pressure (hypertension) in the pulmonary artery, the blood vessel that carries blood from the heart to the lungs. Pulmonary arterial hypertension is one form of a broader condition known as pulmonary hypertension. Pulmonary hypertension occurs when most of the very small arteries throughout the lungs narrow in diameter, which increases the resistance to blood flow through the lungs. To overcome the increased resistance, blood pressure increases in the pulmonary artery and in the right ventricle of the heart, which is the chamber that pumps blood into the pulmonary artery. Ultimately, the increased blood pressure can damage the right ventricle of the heart.

Symptoms

Signs and symptoms of pulmonary arterial hypertension occur when increased blood pressure cannot fully overcome the elevated resistance. As a result, the flow of oxygenated blood from the lungs to the rest of the body is insufficient. Shortness of breath (dyspnea) during exertion and fainting spells are the most common symptoms of pulmonary arterial hypertension. People with this disorder may experience additional symptoms, particularly as the condition worsens. Other symptoms include dizziness, swelling (edema) of the ankles or legs, chest pain, and a rapid heart rate.

Diagnosis

As the symptoms are similar to other common lung diseases, it can often be hard to diagnose PAH. Diagnosis is a process of eliminating other diseases. With the help of lung and heart specialists (pulmonologist and cardiologist) the patient will need to take a number of tests, such as:

• Blood tests: Include HIV, thyroid tests, autoimmune disease panels (test for systemic lupus erythematosus, scleroderma, and rheumatoid arthritis), liver tests and blood chemistry tests.
• Electrocardiogram: Shows the electrical activity of the heart and may help detect abnormalities. Doctors may also be able to detect signs of strain on the right side of the heart.
• Lung function tests (breathing tests): Check for diseases like asthma or emphysema.
• 6-Minute Walk Test: Objectively measures how far you can walk and to see if your oxygen levels drop when one exert yourself.
• Echocardiogram: Ultrasound of the heart to check the size and condition of the chambers of the heart. It can also be used to estimate the blood pressure in the pulmonary arteries.
• Right Heart Catheterization: This test is invasive, so it is not usually performed unless other tests cannot produce a firm diagnosis. It involves inserting a catheter (small tube) into a large vein in either the neck, arm, or groin, and threading it through the right side of the heart and into the pulmonary artery. This allows measurement of the blood pressure in the lungs.

Treatment

• Several medications have been approved by the US Food and Drug Administration (FDA) for the treatment of PAH. These medications can be broadly broken down into four categories described below.
• Prostaglandins: The orphan drug Flolan (epoprostenol sodium for injection or prostacycline) has been approved as a standard long-term treatment of individuals with severe PAH.
• Endothelin Receptor Antagonists: The orphan drug bosentan (Tracleer) has been approved by the FDA for treatment of PAH. The drug allows affected individuals to exert themselves physically without shortness of breath.
• Phosphodiesterase Type 5 Inhibitors: Revatio (sildenafil), a phosphodiesterase type 5 (PDE5) inhibitor is also used to treat PAH.

Pulmonary Arterial Hypertension Emerging Drugs Chapters

• This segment of the Pulmonary Arterial Hypertension 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.
• Pulmonary Arterial Hypertension Emerging Drugs

LIQ861: Liquidia Technologies

• LIQ861 is an inhaled dry powder formulation of treprostinil designed using our PRINT technology to enhance deep-lung delivery using a convenient, palm-sized, disposable dry powder inhaler (DPI) for the treatment of pulmonary arterial hypertension (PAH). We believe LIQ861 can overcome the limitations of current inhaled therapies and has the potential to maximize the therapeutic benefits of treprostinil in treating PAH by safely delivering higher doses into the lungs.

CXA-10: Complexa

• CXA-10 is an oral nitrated fatty acid compound that is being evaluated in the phase II PRIMEx clinical trial for the treatment of PAH. It acts through a constellation of actions including:
• Upregulation of Nrf2 pathways, a key pro-reparative and metabolic pathway
• Inhibition of NF-Kappa B and TLR4, key drivers of inflammatory molecules and pathways
• Inhibition of Xanthine Oxidoreductase, a key intracellular producer of Reactive Oxygen Species add that result in cellular oxidative stress
• Increasing the expression of heat shock proteins, which act as chaperones during cellular stress to restore cellular homeostatic mechanisms

Sotatercept: Acceleron Pharma

• Sotatercept is an investigational product being studied in patients with pulmonary arterial hypertension Sotatercept is a ligand trap with high selectivity for multiple proteins within the TGF-beta superfamily, including activins, GDFs, and others. With its believed ability to block the TGF-beta superfamily signaling pathway, sotatercept could promote a rebalancing of BMPR-II signaling and, potentially, restore vascular homeostasis.
• GB002: Gossamer Bio
• GB002 is a novel inhaled pdgfr kinase inhibitor, demonstrates efficacy in the su5416 hypoxia rat model of PAH. It is currently in phase I stage of development.
• RBM-011: Ribomic
• RBM-011 is a promising candidate to provide a new treatment of PAH by inhibiting the action of IL-21. IL-21 is known to promote the proliferation of pulmonary artery smooth muscle cells and promote PAH pathology. It is currently in preclinical stage of development.

Further product details are provided in the report..

• Pulmonary Arterial Hypertension: Therapeutic Assessment
• This segment of the report provides insights about the different Pulmonary Arterial Hypertension drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Pulmonary Arterial Hypertension
• There are approx. 100+ key companies which are developing the therapies for Pulmonary Arterial Hypertension. The companies which have their Pulmonary Arterial Hypertension drug candidates in the most advanced stage, i.e. phase III include Liquidia Technologies and others

Phases

• DelveInsights report covers around 100+ 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

Pulmonary Arterial Hypertension 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
• Oral
• Parenteral
• Subcutaneous
• Topical.
• Intravenous
• 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.

Pulmonary Arterial Hypertension: Pipeline Development Activities

• The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Pulmonary Arterial Hypertension 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 Pulmonary Arterial Hypertension drugs.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Pulmonary Arterial Hypertension R&D. The therapies under development are focused on novel approaches to treat/improve Pulmonary Arterial Hypertension.
• In August 2020 Liquidia Announces Notice of Allowance for US Patent Application Covering Methods of Treating Pulmonary Hypertension with Dry Powder Treprostinil.
• In April 2020 Liquidia Technologies announced that the US Food and Drug Administration (FDA) accepted for review the Companys New Drug Application (NDA) seeking marketing approval for LIQ861 for the treatment of pulmonary arterial hypertension (PAH). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a goal date of November 24, 2020.
• In October 2019, The Complexas lead candidate, CXA-10, has received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH).
• In April 2020, Acceleron Pharma announced that the United States Food and Drug Administration (FDA) has granted Breakthrough herapy designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH).

Pulmonary Arterial Hypertension Report Insights

• Pulmonary Arterial Hypertension Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
• Pulmonary Arterial Hypertension 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 Pulmonary Arterial Hypertension drugs?
• How many Pulmonary Arterial Hypertension drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pulmonary Arterial Hypertension?
• What are the key collaborations (IndustryIndustry, IndustryAcademia), Mergers and acquisitions, licensing activities related to the Pulmonary Arterial Hypertension 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 Pulmonary Arterial Hypertension and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Acceleron Pharma/Celgene Corporation
• Liquidia Technologies
• Vigonvita Life Sciences
• Gossamer Bio, Inc.
• Resverlogix Corp
• Merck Sharp & Dohme Corp.
• PhaseBio Pharmaceuticals
• Pharmosa BioPharm
• Complexa, Inc.
• Gmax Biopharm Australia Pty Ltd.
• Ribomic

Key Products

• Sotatercept
• LIQ861
• TPN171H
• GB002
• Apabetalone
• MK-5475
• PB1046
• L606
• CXA-10
• GMA301
• RBM-011