Cystic Fibrosis - Pipeline Insight, 2024

This “Cystic Fibrosis - Pipeline Insight, 2024,” report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Cystic Fibrosis 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

Cystic Fibrosis Understanding

Cystic Fibrosis: Overview

Cystic fibrosis (CF) is a genetic condition that affects a protein in the body. People who have cystic fibrosis have a faulty protein that affects the body’s cells, tissues, and the glands that make mucus and sweat. Mucus is normally slippery and protects the linings of the airways, digestive tract, and other organs and tissues. People who have cystic fibrosis make thick, sticky mucus that can build up and lead to blockages, damage, or infections in the affected organs. Inflammation also causes damage to organs such as the lungs and pancreas. Some people who have cystic fibrosis have few or no signs or symptoms, while others experience severe symptoms or life-threatening complications. Symptoms of cystic fibrosis depend on which organs are affected and the severity of the condition. The most serious and common complications of cystic fibrosis are problems with the lungs, also known as pulmonary or respiratory problems, which may include serious lung infections. People who have cystic fibrosis often also have problems maintaining good nutrition, because they have a hard time absorbing the nutrients from food. This is a problem that can delay growth.

Cystic Fibrosis - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cystic Fibrosis pipeline landscape is provided which includes the disease overview and Cystic Fibrosis treatment guidelines. The assessment part of the report embraces, in depth Cystic Fibrosis 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, Cystic Fibrosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Cystic Fibrosis R&D. The therapies under development are focused on novel approaches to treat/improve Cystic Fibrosis.

Cystic Fibrosis Emerging Drugs Chapters

This segment of the Cystic Fibrosis 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.

Cystic Fibrosis Emerging Drugs

VX-121: Vertex PharmaceuticalsVX-121 and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. VX-561 (deutivacaftor) is a potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. The triple combination of VX-121/tezacaftor/VX-561 is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene. It is currently being investigated in Phase III stage of development.

Brensocatib: AstraZenecaBrensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. The drug is currently being evaluated in Phase II clinical trial to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug.

ELX-02: Eloxx PharmaceuticalsELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. Structurally, ELX-02 is an aminoglycoside analogue that induces read-through of nonsense mutations through interaction with the ribosome, resulting in the production of full-length functional proteins. The therapy was designated an orphan drug in the US, an orphan medicinal product in Europe, and given fast track designation in the US. ELX-02 is being tested in CF patients carrying least one G542X mutation in two parallel open-label Phase II clinical trials.

S-1226: SolAeroMed Inc.S1226 is SolAeroMed’s lead therapy. S1226 is formulated to rapidly reopen constricted, mucus plugged airways, and should increase the effectiveness of respiratory drug delivery. The S1226 formulation consists of aerosolized carbon dioxide (CO2) and nebulized perflubron; which is delivered into the lung. The delivery of this formulation results in an immediate relaxant effect on the patient’s constricted airways, supported by a lowering of surface tension in inflamed areas (resulting in enhanced bronchial dilation) and possible clearing of mucus plugs of blocked airways. SolAeroMed is currently conducrting a phase II clinical trial in cystic fibrosis.

Cystic Fibrosis: Therapeutic Assessment

This segment of the report provides insights about the different Cystic Fibrosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Cystic Fibrosis

There are approx. 60+ key companies which are developing the therapies for Cystic Fibrosis. The companies which have their Cystic Fibrosis drug candidates in the most advanced stage, i.e. phase II include, Eloxx Pharmaceuticals.

Phases

This report covers around 60+ 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

Cystic Fibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Cystic Fibrosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cystic Fibrosis 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 Cystic Fibrosis drugs.

Cystic Fibrosis Report Insights

• Cystic Fibrosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cystic Fibrosis 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 Cystic Fibrosis drugs?
• How many Cystic Fibrosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cystic Fibrosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cystic Fibrosis 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 Cystic Fibrosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Eloxx Pharmaceuticals
• Arrowhead Pharmaceuticals
• SolAeroMed
• Parion Sciences
• Translate Bio, Inc.
• Path BioAnalytics
• Aridis Pharmaceuticals
• Novartis
• Vertex Pharmaceuticals
• AlgiPharma
• Proteostasis Therapeutics, Inc.
• Galapagos NV
• Santhera Pharmaceuticals
• Calithera Biosciences, Inc
• 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
• AlgiPharma AS
• Sound Pharmaceuticals
• Spirovant
• 4D Molecular Therapeutics
• Santhera Pharmaceuticals
• Armata Pharmaceuticals

Key Products

• 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
• LAU-7b
• IONIS-ENaCRx
• CHF 6333
• Theradux
• PRX 110
• LAU-7b
• LUNAR-CF
• ETD001
• 4D-710
• AP-PA02

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