DelveInsight’s, “Cystic Fibrosis Pipeline Insight, 2022,” report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in the 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.
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.
- The leading Cystic Fibrosis Companies such as Eloxx Pharmaceuticals, NovaBiotics, Arrowhead Pharmaceuticals, SolAeroMed, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Vertex Pharmaceuticals, AlgiPharma, Corbus Pharmaceuticals, Galapagos NV, Santhera Pharmaceuticals, Calithera Biosciences, Inc, AbbVie, Spyryx Biosciences, Inc., Verona Pharma, Laurent Pharmaceuticals Inc., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Krystal Biotech, Insmed Incorporated, BiomX, Arcturus Therapeutics, and others are developing potential drug candidates to improve the Cystic Fibrosis treatment scenario.
- The promising Cystic Fibrosis therapies in the pipeline included such as BAYQ3939, P-1037, Hypertonic Saline, VX-371 + HS, MP-376, SIMEOX, and others.
- 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.
Latest Breakthroughs and Developments in the Cystic Fibrosis Treatment Landscape
- In December 2021, AlgiPharma has been awarded up to NOK 16 million from the Norwegian Research Councils Innovation Project for the Industrial Sector program for the project “Therapeutic Alginates for Resistant and Recurrent Infections: Generating Eradication Therapies (TARRGET)”. The project grant awarded from the Research Council combined with the Norwegian government tax incentive scheme (SkatteFUNN) results in a total award value for AlgiPharma of about NOK 22 million (about EUR 2.2 MM / USD 2.5 MM).
- AlgiPharma has received more than USD 46 million in research and development grants from EU’s 7th framework program, Horizon 2020, and Eurostars programs, the Norwegian Research Council, Innovate UK, US Army through Congressional Earmark funding, and the Cystic Fibrosis Foundation (CFF).
- In November 2021, Calithera Biosciences shared interim safety and efficacy results from a Phase 1b, randomized, double-blind, placebo-controlled, dose-escalation trial evaluating CB-280, the company’s investigational arginase inhibitor, in adults with cystic fibrosis (CF). The data were shared in a poster presentation at the North American Cystic Fibrosis Foundation Conference (NACFC; Abstract 529).CB-280 demonstrated linear pharmacokinetics with plasma exposure increasing proportionally with dose. Complete and continuous target inhibition in plasma was achieved at the 100 mg dose and above. CB-280 also demonstrated robust pharmacodynamic effects, with rapid and significant dose-proportional increases in plasma arginine, the key driver of NO production.
- In November 2021, Eloxx Pharmaceuticals announced positive topline results from the monotherapy arms of its Phase 2 clinical trial of ELX-02 in Class 1 cystic fibrosis (CF) patients with at least one G542X nonsense allele mutation. ELX-02 was well tolerated and achieved a statistically significant 5.4mmol/L reduction in sweat chloride in patients at the1.5mg/kg/day dose.
- In October 2021, Boehringer Ingelheim, IP Group, the UK Cystic Fibrosis Gene Therapy Consortium (GTC, consisting of researchers from Imperial College London and the Universities of Oxford and Edinburgh) and Oxford Biomedica (OXB), today that Boehringer Ingelheim has exercised its options on intellectual property and know-how from the partners to progress and further accelerate the development of a potential, new treatment option for patients with CF. In the partnership, IP Group, acting on behalf of the three GTC host Universities, is granting exclusive global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development. OXB is adding its leading competence in manufacturing lentiviral vector-based therapies to Boehringer Ingelheim’s expertise in the development of novel breakthrough therapies for respiratory diseases.
- In August 2021, Sanofi entered into a definitive agreement with Translate Bio (NASDAQ: TBIO), a clinical-stage mRNA therapeutics company, under which Sanofi will acquire all outstanding shares of Translate Bio for $38.00 per share in cash, which represents a total equity value of approximately $3.2 billion (on a fully diluted basis). The Sanofi and Translate Bio Boards of Directors unanimously approved the transaction.On the therapeutic side, Translate Bio has an early-stage pipeline in cystic fibrosis and other rare pulmonary diseases. In addition, discovery work is ongoing in diseases that affect the liver, and Translate Bio’s MRTTM platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as oncology. Sanofi’s recent acquisition of Tidal Therapeutics expanded the company’s mRNA research capabilities in both immuno-oncology and inflammatory diseases. The Translate Bio acquisition further accelerates Sanofi’s efforts to develop transformative medicines using mRNA technology.
- In August 2019, Path BioAnalytics Inc. (PBA) announced it had licensed rights to cavosonstat from Laurel Therapeutics. Cavosonstat is a novel CFTR modulator designed to correct a subset of CFTR mutations by increasing stability of the CFTR protein in the cell membrane through inhibition of S-nitrosoglutathione reductase (GSNOR) and preservation of S-nitrosoglutathione (GSNO).
Get an overview of the pipeline landscape @ Cystic Fibrosis Clinical Trials Analysis
Cystic Fibrosis Overview
Cystic fibrosis is a progressive, genetic disease that causes long-lasting lung infections and limits the ability to breathe over time. More than 30,000 children and adults in the United States have CF (70,000 worldwide) and CF affects people of every racial and ethnic group. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s unable to help move chloride — a component of salt — to the cell surface. Without the chloride to attract water to the cell surface, the mucus in various organs becomes thick and sticky. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. For this reason, avoiding germs is a top concern for people with CF. In men, CF can affect their ability to have children. People with CF can have a variety of symptoms, including: Very salty-tasting skin, persistent coughing, at times with phlegm, frequent lung infections including pneumonia or bronchitis, wheezing or shortness of breath, poor growth or weight gain in spite of a good appetite, frequent greasy, bulky stools or difficulty with bowel movements, nasal polyps, chronic sinus infections and many more. Cystic fibrosis is a complex disease. The types of symptoms and how severe they are can differ widely from person to person.
Cystic Fibrosis Emerging Drugs
- OligoG: Algi Pharma
- Ensifentrine: Verona Pharma
- MRT5005: Translate Bio
- CB280: Calithera Biosciences
- KB407: Krystal Biotech
- SPL84231: SpliSense
Learn more about the novel and emerging Cystic Fibrosis pipeline therapies @ Cystic Fibrosis Pipeline Outlook
Cystic Fibrosis Pipeline Therapeutics Analysis
There are approx. 75+ 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, Algi Pharma.
Cystic Fibrosis Pipeline Report
- The report provides detailed insights about emerging therapies for Cystic Fibrosis treatment and the aggregate therapies developed by major pharma companies.
- It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cystic Fibrosis Treatment.
- It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- The Cystic Fibrosis Pipeline Report evaluates the drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Cystic Fibrosis market.
The Cystic Fibrosis Pipeline Report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third party sources etc.
Scope of the Cystic Fibrosis Pipeline Report
- Coverage- Global
- Cystic Fibrosis Companies- Eloxx Pharmaceuticals, NovaBiotics, Arrowhead Pharmaceuticals, SolAeroMed, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Vertex Pharmaceuticals, AlgiPharma, Corbus Pharmaceuticals, Galapagos NV, Santhera Pharmaceuticals, Calithera Biosciences, Inc, AbbVie, Spyryx Biosciences, Inc., Verona Pharma, Laurent Pharmaceuticals Inc., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Krystal Biotech, Insmed Incorporated, BiomX, Arcturus Therapeutics, and others are developing potential drug candidates to improve the Cystic Fibrosis treatment scenario.
- Cystic Fibrosis Therapies- BAYQ3939, P-1037, Hypertonic Saline, VX-371 + HS, MP-376, SIMEOX, and others.
- Cystic Fibrosis Therapeutic Assessment by Product Type
- Cystic Fibrosis Therapeutic Assessment by Stage and Product Type
- Cystic Fibrosis Therapeutic Assessment by Route of Administration
- Cystic Fibrosis Therapeutic Assessment by Stage and Route of Administration
- Cystic Fibrosis Therapeutic Assessment by Molecule Type
- Cystic Fibrosis Therapeutic Assessment by Stage and Molecule Type
- Cystic Fibrosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends
- Cystic Fibrosis – Market Drivers and Barriers
- Cystic Fibrosis – Future Perspectives and Conclusion
Table of Content
- Introduction
- Executive Summary
- Cystic Fibrosis: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Cystic Fibrosis – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Drug name: Company Name
- Mid Stage Products (Phase II)
- OligoG : Algi pharma
- Early Stage Products (Phase I)
- CB280: Calithera Biosciences
- Preclinical and Discovery Stage Products
- SPL84231: Spli Sense
- Inactive Products
- Cystic Fibrosis -Key Companies
- Cystic Fibrosis -Key Products
- Cystic Fibrosis – Unmet Needs
- Cystic Fibrosis – Market Drivers and Barriers
- Cystic Fibrosis – Future Perspectives and Conclusion
- Cystic Fibrosis -Analyst Views
- Cystic Fibrosis- Key Companies
- Appendix
For further information on the Cystic Fibrosis Pipeline therapeutics, reach out to Cystic Fibrosis Drugs
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