Pulmonary Arterial Hypertension (PAH) - Drug Pipeline Landscape, 2023

Pulmonary Arterial Hypertension (PAH) - Drug Pipeline Landscape, 2023

Pulmonary Arterial Hypertension (PAH) is a rare, progressive type of pulmonary hypertension characterized by thickening and narrowing of small pulmonary arteries which blocks the blood flow through the lungs which raises the blood pressure in the lungs.

The most common causes of Pulmonary Arterial Hypertension are mutations in the BMPR2 gene, underlying disease conditions such as systemic scleroderma, lupus erythematosus, critical congenital heart disease, or Down syndrome, blood clots in the lungs, injury to the cells that line the blood vessels of the lung.

The most common symptoms of Pulmonary Arterial Hypertension include shortness of breath (dyspnea), difficulty in exercising, dizziness, chest pain, fatigue, fainting episodes, swelling (edema) of the ankles or legs and a rapid heart rate.

Pulmonary Arterial Hypertension can be diagnosed by performing common tests that rule out other diseases are echocardiography, blood tests, pulmonary function tests, X-rays of the chest, lung blood flow scans, electrocardiography (ECG) and the 6-minute walk test, which measures how far an individual can walk in that time period and confirmation by cardiac catheterization with and without vasodilator testing.

The most common treatments for Pulmonary Arterial Hypertension are prostaglandins, endothelin receptor antagonists, phosphodiesterase type 5 inhibitors etc.

Major pharmaceutical companies are involved in the development of potential drug candidates to improve the Pulmonary Arterial Hypertension treatment such as Yutrepia, MK-5475, Ralinepag, Macitentan and Tadalafil FDC, CS1 and others. Key players involved in the development of therapies to treat pulmonary arterial hypertension are Liquidia Technologies Inc, Merck Inc, United Therapeutics, Johnson & Johnson, Cereno Scientific and others. One drug is under late-stage Pre-Registration, ten drugs are under Phase III clinical trials, thirteen drugs are in Phase II clinical trials and some other drugs are under Phase I, Preclinical, Discovery and Unknown stages of development.

In December 2022, Cereno Scientific announced that six clinical sites across the US are activated and actively recruiting patients in the ongoing CS1 Phase 2 study in pulmonary arterial hypertension (PAH) with top-line results expected in mid-2023.

Report Highlights

Global Insight Service's, Pulmonary Arterial Hypertension (PAH) - Drug Pipeline Landscape, 2023 report provides an overview of the Pulmonary Arterial Hypertension (PAH) pipeline drugs. This report covers detailed insights on Pulmonary Arterial Hypertension (PAH) drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Pulmonary Arterial Hypertension (PAH) pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.


The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.


The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.

Reasons to Buy
Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

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1. Introduction
1.1 Pulmonary Arterial Hypertension - Pipeline Drugs, 2023 - Coverage
2. Disease Overview - Pulmonary Arterial Hypertension
2.1 Causes
2.2 Signs and Symptoms
2.3 Diagnosis
2.4 Disease Management
3. Pulmonary Arterial Hypertension - Pipeline Drugs Development - Overview
3.1 Comparative Analysis by Stage of Development
3.2 Number of Products under Development by Companies, 2023
3.3 Products under Development by Companies, 2023
4. Assessment of Therapeutics
4.1 Assessment by Target
4.2 Assessment by Mechanism of Action
4.3 Assessment by Molecule Type
4.4 Assessment by Route of Administration
5. Drug Profiles
5.1 Late Stage Drugs - Pre-Registration
5.1.1. Yutrepia
5.2 Clinical Stage Drugs - Phase III
5.2.1 Autologous Endothelial Progenitor Cells (EPCs) Transfected With Human Endothelial NO-synthase
5.2.2 AV-101
5.2.3 Inhaled Nitric Oxide
5.2.4 L606
5.2.5 Macitentan (Pediatric)
5.2.6 Macitentan and Tadalafil FDC
5.2.7 MK-5475
5.2.8 Ralinepag
5.2.9 Selexipag
5.2.10 Sotatercept
5.3 Clinical Stage Drugs - Phase II
5.3.1 CS1
5.3.2 LTP001
5.3.3 Olaparib
5.3.4 Pemziviptadil
5.3.5 Rodatristat Ethyl
5.3.6 RT234
5.3.7 Seralutinib
5.3.8 Tacrolimus
5.3.9 TNX-201
5.3.10 TPN171H
5.3.11 Treprostinil Palmitil
5.3.12 Ularitide
5.3.13 Zamicastat
5.4 Clinical Stage Drugs - Phase I
5.4.1 ABI-009
5.4.2 AER-901
5.4.3 Apabetalone
5.4.4 Brilaroxazine
5.4.5 C21
5.4.6 CAM2043
5.4.7 GMA301
5.4.8 KER-012
5.4.9 LAM-001
5.4.10 NTP-42
5.4.11 Prostacyclin Receptor Agonist
5.4.12 RemoPro
5.4.13 SIF-019
5.5 Early Stage Drugs - Preclinical
5.5.1 ALT-100
5.5.2 Antliabio 111
5.5.3 Antliabio 112
5.5.4 Belapectin
5.5.5 BZ371
5.5.6 GMA306
5.5.7 GMA307
5.5.8 GXV-002
5.5.9 INV-200
5.5.10 MFC-1040
5.5.11 MGX292
5.5.12 MN-08
5.5.13 NTP-48
5.5.14 PT001
5.5.15 R-107
5.5.16 RBM-011
5.5.17 REC-1886
5.5.18 SUL-150
5.5.19 TOPV-122
5.5.20 TP-20
5.5.21 Undisclosed Integrin Inhibitor
5.5.22 VRN-13
5.5.23 VTA-04
5.5.24 ZM-A001
5.6 Early Stage Drugs - Discovery
5.6.1 AR001
5.6.2 Nrf-2 Activator
5.7 Unknown Stage Drugs
5.7.1 APT102
6. Key Regulatory Designations
7. Key Deals
8. Key Upcoming Milestones
9. Key Companies Involved
9.1 Aadi Bioscience, Inc.
9.2 Aeon Respire Inc
9.3 Aerami Therapeutics
9.4 Aerovate Therapeutics
9.5 AI Therapeutics
9.6 Altavant Sciences GmbH
9.7 Antlia Bioscience Inc
9.8 Apaxen
9.9 APT Therapeutics Inc
9.10 Aqualung Therapeutics Corp
9.11 AstraZeneca
9.12 ATXA Therapeutics
9.13 Bellerophon
9.14 Bial - Portela C S.A.
9.15 Biozeus Pharmaceutical SA
9.16 C4X Discovery Holdings Plc
9.17 Camurus AB
9.18 Cardiorentis AG
9.19 Centessa Pharmaceuticals
9.20 Cereno Scientific AB
9.21 Claritas Pharmaceuticals
9.22 Galectin Therapeutics Inc
9.23 GEXVal
9.24 Gmax Biopharm LLC
9.25 Gossamer Bio Inc.
9.26 Guangzhou Magpie Pharmaceutical Co Ltd
9.27 Insmed Incorporated
9.28 INVENT Pharmaceuticals Inc
9.29 Johnson & Johnson
9.30 Keros Therapeutics
9.31 Liquidia Technologies, Inc
9.32 Merck Inc
9.33 Morphic Therapeutic Inc
9.34 Northern Therapeutics
9.35 Novartis
9.36 Pharmosa Biopharm Inc
9.37 PhaseBio Pharmaceuticals Inc.
9.38 PulmoSIM Therapeutics
9.39 Recursion Pharmaceuticals Inc
9.40 Respira Therapeutics, Inc.
9.41 Resverlogix Corp
9.42 Reviva Pharmaceuticals
9.43 Ribomic
9.44 Sulfateq BV
9.45 Systimmune Inc
9.46 Tenax Therapeutics, Inc.
9.47 Topadur Pharma AG
9.48 United Therapeutics
9.49 VasThera Co Ltd
9.50 Vicore Pharma
9.51 Vigonvita Life Sciences
9.52 Vivus LLC
9.53 Voronoi Group
9.55 Zymedi
10. Dormant Drugs
10.1 Inactive Drugs
10.2 Discontinued Drugs
11. Appendix
List of Tables
Table 1.1 Number of Products under Development by Pulmonary Arterial Hypertension
Table 1.2 Number of Products under Development by Companies
Table 1.3 Products under Development by Companies
Table 1.4 Products by Targets
Table 1.5 Products by Mechanism of Action
Table 1.6 Products by Molecule Type
Table 1.7 Products by Route of Administration
Table 2.1 Clinical Trial Details - Autologous Endothelial Progenitor Cells (EPCs) Transfected With Human Endothelial NO-synthase/Northern Therapeutics
Table 2.2 Clinical Trial Details - AV-101/Aerovate Therapeutics
Table 2.3 Clinical Trial Details - Inhaled Nitric Oxide/Bellerophon
Table 2.4 Clinical Trial Details - L606/Pharmosa Biopharm Inc
Table 2.5 Clinical Trial Details - Macitentan (Pediatric)/Johnson & Johnson
Table 2.6 Clinical Trial Details - Macitentan and Tadalafil FDC/Johnson & Johnson
Table 2.7 Clinical Trial Details - MK-5475/Merck
Table 2.8 Clinical Trial Details - Ralinepag/United Therapeutics
Table 2.9 Clinical Trial Details - Selexipag/Johnson & Johnson
Table 2.10 Clinical Trial Details - Sotatercept/Merck
Table 2.11 Clinical Trial Details - CS1/Cereno Scientific AB
Table 2.12 Clinical Trial Details - LTP001/Novartis
Table 2.13 Clinical Trial Details - Olaparib/AstraZeneca
Table 2.14 Clinical Trial Details - Pemziviptadil/PhaseBio Pharmaceuticals Inc.
Table 2.15 Clinical Trial Details - Rodatristat Ethyl/Altavant Sciences GmbH
Table 2.16 Clinical Trial Details - RT234/Respira Therapeutics, Inc.
Table 2.17 Clinical Trial Details - Seralutinib/Gossamer Bio Inc.
Table 2.18 Clinical Trial Details - TPN171H/Vigonvita Life Sciences
Table 2.19 Clinical Trial Details - Treprostinil Palmitil/Insmed Incorporated
Table 2.20 Clinical Trial Details - Ularitide/Cardiorentis AG
Table 2.21 Clinical Trial Details - Zamicastat/Bial - Portela C S.A.
Table 2.22 Clinical Trial Details - ABI-009/Aadi Bioscience, Inc.
Table 2.23 Clinical Trial Details - Apabetalone/Resverlogix Corp
Table 2.24 Clinical Trial Details - NTP-42/ATXA Therapeutics
Table 2.25 Clinical Trial Details - Prostacyclin Receptor Agonist/Johnson & Johnson
Table 2.26 Clinical Trial Details - SIF-019/Systimmune Inc
Table 3.1 Regulatory Designations
Table 4.1 Inactive Drugs
Table 4.2 Discontinued Drugs
List of Figures
Figure 1.1 Number of Products under Development for Pulmonary Arterial Hypertension, 2023
Figure 1.2 Products by Top 5 Targets and Stage of Development for Pulmonary Arterial Hypertension, 2023
Figure 1.3 Products by Top 5 Mechanism of Action for Pulmonary Arterial Hypertension, 2023
Figure 1.4 Products by Top 5 Molecule Type for Pulmonary Arterial Hypertension, 2023
Figure 1.5 Products by Top 5 Route of Administration for Pulmonary Arterial Hypertension, 2023

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