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.

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  

• Major 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. 
• 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 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: 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.

For further information, refer to the detailed report @ Cystic Fibrosis Pipeline Therapeutics 

Scope of Cystic Fibrosis Pipeline Drug Insight    

• Coverage: Global 
• Major Players: 80+ Key Players
• Prominent Players: 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 many others.   
• Key Drugs Profiles: 80+ Products
• Phases:  

·       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  

• Mechanism of Action:

·      Cystic fibrosis transmembrane conductance regulator expression modulators
·      Ribosomal protein modulators
·      Leukocyte elastase inhibitors
·      Cannabinoid receptor CB2 agonists

• Molecule Types:   

·     Peptides
·     Monoclonal antibodies
·    Small molecules 
·    Polymer 
·    Gene therapy

• Route of Administration:

·     Parenteral 
·      Intravenous 
·      Oral
·      Subcutaneous
·      Topical

• Product Types:

·       Monotherapy
·       Combination
·       Mono/Combination 

Key Questions Regarding Current Cystic Fibrosis Treatment Landscape and Emerging Therapies Answered in the Pipeline Report  

• What are the current options for Cystic Fibrosis treatment?
• How many companies are developing therapies for the treatment of Cystic Fibrosis? 
• How many are Cystic Fibrosis emerging therapies in the early-stage, mid-stage, and late development stages to treat Cystic Fibrosis? 
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Cystic Fibrosis market? 
• Which are the dormant and discontinued products and the reasons for the same?
• What is the unmet need for current therapies for the treatment of Cystic Fibrosis?  
• What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Cystic Fibrosis therapies? 
• What are the critical designations that have been granted for the emerging therapies for Cystic Fibrosis? 
• How many patents are granted and pending for the emerging therapies to treat Cystic Fibrosis?   

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

Get a customized pipeline report @ Cystic Fibrosis Drugs Pipeline Report  

Other Reports  

• Cystic fibrosis Market Insight
• Cystic fibrosis - Epidemiology 
• Non-Cystic Fibrosis Bronchiectasis (NCFB)- Market 

Browse Blog Posts   

• Read Cystic Fibrosis Market: A Rapidly Expanding Playing Field for Mid-Cap Pharma
• Cystic Fibrosis Drug Market: Emerging drugs that may improve lung infections

About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.