DelveInsight Evaluates a Robust Cystic Fibrosis Pipeline as 75+ Influential Pharma Players to Set Foot in the Domain

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Jan 03, 2023, 10:30 ET

Cystic fibrosis is quickly becoming the most prevalent genetic illness in every region of the world. According to the Cystic Fibrosis Patient Registry, more than 30,000 persons in the United States are currently afflicted with this ailment, and it is projected that approximately 70,000 people throughout the world are afflicted with this condition. The introduction of potential drugs is on the new horizon, which is bolstering market growth. Several companies are involved in evaluating novel therapies in different stages of development.

LAS VEGAS, Jan. 3, 2023 /PRNewswire/ -- DelveInsight's 'Cystic Fibrosis Pipeline Insight – 2022' report provides comprehensive global coverage of available, marketed, and pipeline cystic fibrosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the cystic fibrosis pipeline domain.

Key Takeaways from the Cystic Fibrosis Pipeline Report

DelveInsight's cystic fibrosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for cystic fibrosis treatment.
Key cystic fibrosis companies such as Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, AbbVie, Krystal Biotech, BiomX, Inc., Moderna Therapeutics, ReCode Therapeutics, Aeon Respire, Kither Biotech, Carbon Biosciences, SpliSense, Sionna Therapeutics, Lung Therapeutics, Tavanta Therapeutics, Branca Bunús Ltd, and others are evaluating new cystic fibrosis drugs to improve the treatment landscape.
Promising cystic fibrosis pipeline therapies in various stages of development include ELX-02, NM002, ARO ENaC, S-1226, P-1037, MRT5005, Cavosonstat, AR-501, QBW276, VX-121, OligoG, PTI-808, JBT-101, GLPG1837, POL6014, CB-280, Galicaftor, SPX-101, RPL554, IONIS-ENaCRx, CHF 6333, Theradux, PRX 110, LAU-7b, LUNAR-CF, ETD001, 4D-710, AP-PA02,Deutivacaftor/tezacaftor/vanzacaftor, Brensocatib, ABBV-576, KB 407, BX004-A, VXc-522, SP-101, mRNA-based therapeutic program, AR-001, KIT-2014, LTI-05, TAVT-135, BrB102, and others.
In December 2022, Vertex Pharmaceuticals announced that the US Food and Drug Administration had cleared its Investigational New Drug (IND) application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung disease for the approximately 5,000 people with CF who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF.
In December 2022, First Wave BioPharma, Inc. announced that it has selected the initial clinical trial sites for its planned Phase II clinical trial to evaluate an enhanced enteric microgranule delivery formulation of adrulipase as a treatment for exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). Based on anticipated regulatory and clinical trial activities, First Wave BioPharma expects to report topline data from the Phase 2 trial by mid-2023.
In December 2022, Sionna Therapeutics, announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for SION-638, a novel small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The first subject has been dosed in a Phase I study to evaluate the safety and pharmacokinetics of SION-638 in healthy volunteers.
In December 2022, Armata Pharmaceuticals, Inc. announced that the last subject has completed the company's Phase I/II 'SWARM-P.a.' clinical trial of its lead candidate, AP-PA02, in cystic fibrosis (CF) subjects with chronic pulmonary Pseudomonas aeruginosa infection. In March 2020, Armata announced that it had been awarded up to $5 million in a therapeutic development award from the CF Foundation to advance development of AP-PA02. In October 2021, the Foundation subsequently made an equity investment of $3 million in Armata to further support this program.
In November 2022, Aridis Pharmaceuticals, Inc. announced the closing of patient enrollment in the multiple ascending dose (MAD) and dose-ranging cohorts in the Phase IIa clinical trial of AR-501 in cystic fibrosis (CF) patients. The Company is on track to complete database lock, data analyses, and disclose top-line data in the first quarter of 2023.
In September 2022, Eloxx Pharmaceuticals, Inc. announced topline results from the Phase II clinical trial of ELX-02 in combination with ivacaftor in Class 1 cystic fibrosis (CF) patients with at least one nonsense mutation. The combination trial of ELX-02 with ivacaftor was well tolerated but did not achieve statistical significance for efficacy endpoints, including changes from baseline in sweat chloride concentration (SCC) and percent forced expiratory volume (FEV1).

Request a sample and discover the recent advances in cystic fibrosis drug treatment @Cystic Fibrosis Pipeline Report

The cystic fibrosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage cystic fibrosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the cystic fibrosis clinical trial landscape.

Cystic Fibrosis Overview

Cystic fibrosis is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. F508del is the most common variation reported globally, but there are over 2000 others, not all of which cause the disease. Cystic fibrosis symptoms typically appear 6-8 months after birth, though this varies greatly from person to person. Cystic fibrosis symptoms vary with age and can affect different body parts. Two tests for cystic fibrosis diagnosis are newborn screening, a genetic test to look for a gene defect, and a blood test to look for pancreatic problems. A sweat test can be performed after initial testing to confirm the cystic fibrosis diagnosis. Currently available cystic fibrosis medications aim at symptom relief, complication prevention, and, more recently, protein rectifiers to correct underlying structural and functional abnormalities.

Find out more about drugs for cystic fibrosis @New Cystic Fibrosis Drugs

A snapshot of the Cystic Fibrosis Pipeline Drugs mentioned in the report:

Drugs	Company	Phase	MoA	RoA
VX-121/TEZ/D-IVA	Vertex Pharmaceuticals	Phase III	Cystic fibrosis transmembrane conductance regulator modulators	Oral
Brensocatib	Insmed Corporated	Phase II	Dipeptidyl peptidase I inhibitors	Oral
ABBV-576	AbbVie	Phase II	Cystic fibrosis transmembrane conductance regulator modulators	Oral
S-1226	SolAeroMed Inc.	Phase II	Cell membrane permeability enhancers	Inhalation
AR-501	Aridis Pharmaceuticals	Phase II	Bacterial growth inhibitor	Inhalation
4D-710	4D Molecular Therapeutics	Phase I/II	Gene transference	Inhalation
Lefamulin	Nabriva Therapeutics	Phase I	Immunomodulators; Peptidyltransferase modulators; Protein 50S ribosomal subunit modulators	Oral
KB407	Krystal Biotech	Phase I	Cystic fibrosis transmembrane conductance regulator replacements; Gene transference	Inhalation
ETD-001	Enterprise Therapeutics	Phase I	Epithelial sodium channel antagonists	Inhalation
LUNAR-CF	Arcturus Therapeutics, Inc	Preclinical	Cystic fibrosis transmembrane conductance regulator stimulants	Inhalation
SPL16	SpliSense	Preclinical	Cystic fibrosis transmembrane conductance regulator expression modulators; RNA interference	NA
Gene editing program	Affinia Therapeutics	Discovery	NA	NA

Learn more about the emerging cystic fibrosis pipeline therapies @Cystic Fibrosis Clinical Trials

Cystic Fibrosis Therapeutics Assessment

The cystic fibrosis pipeline report proffers an integral view of cystic fibrosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Cystic Fibrosis Pipeline Report

Coverage: Global
Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical
Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
Therapeutics Assessment By Mechanism of Action: Cystic fibrosis transmembrane conductance regulator modulators, Dipeptidyl peptidase I inhibitors, Cystic fibrosis transmembrane conductance regulator modulators, Cell membrane permeability enhancers, Bacterial growth inhibitor, Gene transference, Immunomodulators; Peptidyltransferase modulators; Protein 50S ribosomal subunit modulators, Cystic fibrosis transmembrane conductance regulator replacements; Gene transference, Epithelial sodium channel antagonists, Cystic fibrosis transmembrane conductance regulator stimulants, Cystic fibrosis transmembrane conductance regulator expression modulators; RNA interference
Key Cystic Fibrosis Companies: Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, AbbVie, Krystal Biotech, BiomX, Inc., Moderna Therapeutics, ReCode Therapeutics, Aeon Respire, Kither Biotech, Carbon Biosciences, SpliSense, Sionna Therapeutics, Lung Therapeutics, Tavanta Therapeutics, Branca Bunús Ltd, and others.
Key Cystic Fibrosis Pipeline Therapies: ELX-02, NM002, ARO ENaC, S-1226, P-1037, MRT5005, Cavosonstat, AR-501, QBW276, VX-121, OligoG, PTI-808, JBT-101, GLPG1837, POL6014, CB-280, Galicaftor, SPX-101, RPL554, IONIS-ENaCRx, CHF 6333, Theradux, PRX 110, LAU-7b, LUNAR-CF, ETD001, 4D-710, AP-PA02,Deutivacaftor/tezacaftor/vanzacaftor, Brensocatib, ABBV-576, KB 407, BX004-A, VXc-522, SP-101, mRNA-based therapeutic program, AR-001, KIT-2014, LTI-05, TAVT-135, BrB102, and others.

Dive deep into rich insights for new drugs for cystic fibrosis treatment; visit @Cystic Fibrosis Medications

Table of Contents

1.	Cystic Fibrosis Pipeline Report Introduction
2.	Cystic Fibrosis Pipeline Report Executive Summary
3.	Cystic Fibrosis Pipeline: Overview
4.	Analytical Perspective In-depth Commercial Assessment
5.	Cystic Fibrosis Clinical Trial Therapeutics
6.	Cystic Fibrosis Pipeline: Late Stage Products (Pre-registration)
7.	Cystic Fibrosis Pipeline: Late Stage Products (Phase III)
7.1	VX-121/TEZ/D-IVA: Vertex Pharmaceuticals
8.	Cystic Fibrosis Pipeline: Mid Stage Products (Phase II)
8.1	Brensocatib: Insmed Corporated
9.	Cystic Fibrosis Pipeline: Early Stage Products (Phase I)
9.1	KB407: Krystal Biotech
10.	Cystic Fibrosis Pipeline Therapeutics Assessment
11.	Inactive Products in the Cystic Fibrosis Pipeline
12.	Company-University Collaborations (Licensing/Partnering) Analysis
13.	Key Companies
14.	Key Products in the Cystic Fibrosis Pipeline
15.	Unmet Needs
16.	Market Drivers and Barriers
17.	Future Perspectives and Conclusion
18.	Analyst Views
19.	Appendix

For further information on the cystic fibrosis pipeline therapeutics, reach out @Cystic Fibrosis Drug Treatment

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