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 Insight Report
- DelveInsight’s Cystic Fibrosis Pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Cystic Fibrosis.
- 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.
- Promising Cystic Fibrosis Pipeline Therapies such as OligoG, Ensifentrine, MRT5005, CB280, KB407, SPL84231, 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.
- In the report, a detailed description of the drug is given which includes the 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.
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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.
Recent Breakthroughs of 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 Cystic Fibrosis Pipeline landscape @ Cystic Fibrosis Clinical Trials Analysis
Cystic Fibrosis Emerging Drugs
- ELX-02: Eloxx Pharmaceuticals
ELX-02, is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to restore activity. It is currently in phase II stage of development to treat Cystic fibrosis.Eloxx has also begun evaluation of inhaled (nebulizer-based) delivery of the current subcutaneous formulation of ELX-02. Eloxx believes that inhaled delivery has the potential to further improve the activity of ELX-02 as a single agent and in combination with other drugs given potential for increased drug exposure in the lung versus plasma. Prior animal studies have shown a 19-fold increase in ELX-02 exposure at a similar dose when administered as an inhalation agent versus subcutaneously. We expect to submit an Investigational New Drug application in the second half of 2022.
- S1226: SolAeroMed
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 has completed a phase I trial demonstrating S1226 is safe in healthy subjects and a phase II clinical trial showing S1226 is safe and effective in relieving an allergen-induced asthma. SolAeroMed is currently conducting a phase II clinical trial in cystic fibrosis.
- Lenabasum: Corbus Pharmaceuticals
Lenabasum is a novel, oral, small molecule that selectively binds as an agonist to the cannabinoid receptor type 2 (CB2) and resolves inflammation and limits fibrosis in animal and human models of disease. CB2 is preferentially expressed on activated immune cells and on fibroblasts, muscle cells, and endothelial cells. Lenabasum has demonstrated acceptable safety and tolerability profiles and has not been immunosuppressive in clinical studies to date.CF-002 was a multinational Phase 2b study evaluating the efficacy and safety of lenabasum in CF. This was a double-blind, randomized, placebo-controlled study, with dosing of lenabasum at 5 mg twice per day, lenabasum 20 mg twice per day or placebo twice per day for 28 weeks, with 4 weeks safety follow-up off active treatment. The primary efficacy endpoint was the event rate of new PEx per subject per 28 weeks, when the primary definition of new PEx was physician diagnosis of PEx, prescription of new antibiotics for that PEx starting more than 28 days after completion of the last antibiotic course for any previous PEx, with 4 out of 12 Fuch’s criteria present in the subject. The Phase 2b CF study was funded in part by a Therapeutic Development Award for up to $25 Million from the Cystic Fibrosis Foundation.
- Lonodelestat: Santhera Pharmaceuticals
Lonodelestat (previously known as POL6014), a highly potent and selective peptide inhibitor of human neutrophil elastase (hNE), is in development for the treatment of cystic fibrosis. Currently, it is in Phase I/II stage of development. Santhera obtained the worldwide, exclusive rights from Polyphor AG to develop and commercialize lonodelestat in CF and other diseases. In preclinical studies lonodelestat was effective in animal models of neutrophil activation in lung tissue and of acute lung injury (ALI). Currently available clinical data demonstrated that single and multiple doses (Phase 1b) of lonodelestat when administered by inhalation via an optimized eFlow® nebulizer (PARI Pharma GmbH) can lead to high drug concentrations within the lung, resulting in inhibition of hNE in sputum of patients, an enzyme associated with lung tissue inflammation. The Phase 1b study further confirmed the tolerability of lonodelestat after treatment of up to four weeks in patients with CF. Lonodelestat may also show therapeutic benefit for a range of neutrophilic pulmonary diseases with high medical need such as non-CF bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD), chronic obstructive pulmonary disease (COPD), acute respiratory distress syndrome (ARDS) or primary ciliary dyskinesia (PCD). Lonodelestat has EU orphan drug designations (ODD) for the treatment of CF as well as for AATD and PCD in both the EU and US.
DelveInsight’s Cystic Fibrosis Pipeline Report covers around 80+ 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
For further information, refer to the detailed report @ Cystic Fibrosis Pipeline Therapeutics
Scope of the Cystic Fibrosis Pipeline Report
- Coverage- Global
- Cystic Fibrosis Pipeline Assessment by Product Type
- Cystic Fibrosis Pipeline Assessment by Stage and Product Type
- Cystic Fibrosis Pipeline Assessment by Route of Administration
- Cystic Fibrosis Pipeline Assessment by Stage and Route of Administration
- Cystic Fibrosis Pipeline Assessment by Molecule Type
- Cystic Fibrosis Pipeline Assessment by Stage and Molecule Type
- 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
- Cystic Fibrosis Therapies- OligoG, Ensifentrine, MRT5005, CB280, KB407, SPL84231, and others
Table of content
- 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
Dive deep into rich insights for drugs for Cystic Fibrosis treatment, visit @ New Drug for Cystic Fibrosis Treatment
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