Las Vegas, USA, Jan. 26, 2022 (GLOBE NEWSWIRE) -- Cystic Fibrosis Pipeline Offers Promising New Options for Treatment | DelveInsight
The Cystic Fibrosis (CF) therapeutic development pipeline contains candidate drugs that address many different aspects of the disease. Drug development for Cystic Fibrosis has become increasingly complex over the last 20 years, and clinical trial endpoints are changing because traditional endpoints are becoming less common events.
DelveInsight's "Cystic Fibrosis Pipeline Insight" report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in the Cystic Fibrosis pipeline landscapes. It comprises Cystic Fibrosis pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Cystic Fibrosis therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Cystic Fibrosis pipeline products.
Some of the key takeaways from the Cystic Fibrosis Pipeline Report
Get an overview of pipeline landscape @ Cystic Fibrosis Clinical Trials Analysis
Cystic Fibrosis is a genetic condition and people with this disease have a faulty protein that affects the body's cells, tissues, and the glands that make mucus and sweat.
Cystic Fibrosis Emerging Drugs
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 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 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 (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.
For further information, refer to the detailed report @ Cystic Fibrosis Pipeline Therapeutics
Scope of Cystic Fibrosis Pipeline Drug Insight
· Cystic Fibrosis Therapies Late-stage (Phase III)
· Cystic Fibrosis Therapies Mid-stage (Phase II)
· Cystic Fibrosis Therapies Early-stage (Phase I)
· Cystic Fibrosis Preclinical stage and Discovery candidates
· Discontinued and Inactive candidates
· Cystic fibrosis transmembrane conductance regulator expression modulators
· Ribosomal protein modulators
· Leukocyte elastase inhibitors
· Cannabinoid receptor CB2 agonists
· Peptides
· Monoclonal antibodies
· Small molecules
· Polymer
· Gene therapy
· Parenteral
· Intravenous
· Oral
· Subcutaneous
· Topical
· Monotherapy
· Combination
· Mono/Combination
Key Questions Regarding Current Cystic Fibrosis Treatment Landscape and Emerging Therapies Answered in the Pipeline Report
Table of Contents
| 1 | Cystic Fibrosis Report Introduction |
| 2 | Cystic Fibrosis Executive Summary |
| 3 | Cystic Fibrosis Overview |
| 4 | Cystic Fibrosis Pipeline Therapeutics |
| 5 | Cystic Fibrosis Therapeutic Assessment |
| 6 | Cystic Fibrosis – DelveInsight's Analytical Perspective In-depth Commercial Assessment |
| 7 | Cystic Fibrosis Late Stage Products (Phase III) |
| 7.1 | Drug name: Company name |
| 8 | Cystic Fibrosis Mid Stage Products (Phase II) |
| 8.1 | ABBV-3067: AbbVie |
| 9 | Cystic Fibrosis Early Stage Products (Phase I) |
| 9.1 | ETD 001: Enterprise Therapeutics Ltd |
| 10 | Cystic Fibrosis Preclinical and Discovery Stage Products |
| 10.1 | SPL84 23 1: SpliSense |
| 11 | Cystic Fibrosis Inactive Products |
| 12 | Cystic Fibrosis Key Companies |
| 13 | Cystic Fibrosis Key Products |
| 14 | Cystic Fibrosis Unmet Needs |
| 15 | Cystic Fibrosis Market Drivers and Barriers |
| 16 | Cystic Fibrosis Future Perspectives and Conclusion |
| 17 | Cystic Fibrosis Analyst Views |
| 18 | Appendix |
| 19 | About DelveInsight |
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