ETD001 / Enterprise Therap - LARVOL DELTA

ETD001, a long-acting inhaled ENaC blocker, is well tolerated in a first human, healthy participant trial. (PubMed, J Cyst Fibros) - "ETD001 at doses up to and including 4.65 mg twice daily for 14 days was well tolerated in healthy people. The drug was slowly absorbed out of the lung and into the systemic circulation, consistent with prolonged retention in the lung, supporting twice daily dosing during further development."

Journal • Atrial Fibrillation • Cardiovascular • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis (clinicaltrials.gov) - P2 | N=57 | Completed | Sponsor: Enterprise Therapeutics Ltd | Active, not recruiting ➔ Completed

Trial completion • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis (clinicaltrials.gov) - P2 | N=57 | Active, not recruiting | Sponsor: Enterprise Therapeutics Ltd | Recruiting ➔ Active, not recruiting

Enrollment closed • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: Enterprise Therapeutics Ltd | Trial completion date: Jul 2025 ➔ Nov 2025 | Trial primary completion date: Jul 2025 ➔ Nov 2025 | N=40 ➔ 60

Enrollment change • Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Optimisation of a novel series of ENaC inhibitors, leading to the selection of the long-acting inhaled clinical candidate ETD001, a potential new treatment for cystic fibrosis. (PubMed, Eur J Med Chem) - "The resulting optimized clinical candidate ETD001 demonstrates potent inhibition of ENaC (59 nM) prolonged retention in the airways of rats (13 % of the delivered dose retained after 6h) following intratracheal administration and a potent and long-acting effect in a sheep model of mucociliary clearance following inhalation (ED100 (4-6h) = 9 μg/kg). ETD001 entered a phase II study in CF patients in July 2024."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

Enterprise Therapeutics Publishes on Medicinal Chemistry of ETD001, a Novel Inhaled ENaC Blocker for Treatment of Cystic Fibrosis (Businesswire) - "Enterprise Therapeutics Ltd...announced the publication of a peer reviewed study in The European Journal of Medicinal Chemistry. The paper describes the medicinal chemistry optimisation of a new series of ENaC blockers, resulting in the invention of ETD001, Enterprise’s lead asset, a potential new inhaled treatment for cystic fibrosis (CF)....The Company commenced and is currently undergoing recruitment for a Phase 2 clinical study of ETD001 in pwCF in summer 2024, expected to complete in 2025, to understand whether 28 days of treatment will improve lung function."

Clinical • Enrollment status • Cystic Fibrosis • Genetic Disorders

Enterprise Therapeutics Granted ‘Rare Pediatric Disease Designation’ in the US for Novel Cystic Fibrosis Investigational Therapy ETD001 (Businesswire) - "ETD001 commenced Phase 2 clinical trials in July 2024, expected to complete in 2025....Enterprise Therapeutic...today announced its novel cystic fibrosis (CF) investigational therapy, ETD001, has been granted ‘rare pediatric disease designation’ (RPD) in the US by the Food and Drug Administration (FDA)."

FDA event • Trial completion • Cystic Fibrosis

ETD001: a well-tolerated inhaled ENaC blocker with extended lung retention in humans (ERS 2024) - No abstract available

Cystic Fibrosis • Fibrosis • Genetic Disorders • Growth Hormone Deficiency (Adult) • Immunology • Pulmonary Disease • Respiratory Diseases

Enterprise Therapeutics Doses First Person with Cystic Fibrosis in Phase 2 Trial for Novel Therapy ETD001 (Businesswire) - "Enterprise Therapeutics Ltd...announced dosing of the first person with cystic fibrosis (pwCF) in its Phase 2a trial of ETD001....The Phase 2a trial aims to deliver clinical proof-of-concept and to assess the safety profile of ETD001 in the 10% of pwCF with the highest unmet medical need."

Trial status • Cystic Fibrosis • Genetic Disorders

ETD001: A novel inhaled ENaC blocker with an extended duration of action in vivo. (PubMed, J Cyst Fibros) - "These data support that the ENaC blocker hypothesis is yet to be appropriately tested in pwCF. ETD001 has a profile that enables dosing at a level sufficient to provide a long duration of action in a Phase 2 clinical study in pwCF scheduled for 2024."

Journal • Preclinical • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis (clinicaltrials.gov) - P2 | N=40 | Recruiting | Sponsor: Enterprise Therapeutics Ltd

New P2 trial • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Enterprise Therapeutics Publishes Preclinical Profile of ETD001, a Novel Inhaled ENaC Blocker (Businesswire) - "Enterprise Therapeutics...today announced the publication of a peer reviewed study in the Journal of Cystic Fibrosis. The paper describes low doses of the ENaC blocker ETD001, Enterprise’s lead asset, enhancing airway mucus clearance with an exceptionally long duration of action in a sheep model....ETD001 is scheduled to commence a Phase 2 clinical study in pwCF in summer 2024, to understand whether 28 days of treatment will improve lung function."

New P2 trial • Preclinical • Cystic Fibrosis

Enterprise Therapeutics Closes £26 million ($33.1 million) Series B Follow-on Financing (Businesswire) - "Enterprise Therapeutics...today announced that it has closed a £26 million ($33.1 million USD) Series B follow-on financing round, led by Panakes Partners....The investment will fund the Phase 2a clinical trial of the Company’s lead programme, ETD001, to deliver clinical proof of concept to treat cystic fibrosis."

Financing • Cystic Fibrosis

Tolerance of ETD001, a long-acting inhaled epithelial sodium channel blocker, in humans (NACFC 2022) - P1 | "ETD001 was well tolerated at single doses up to 10.8 mg andmultiple doses of 3.1 mg QD for 7 days and 4.65 mg BID for 14 days. Thewide safety margin is predicted to enable doses capable of durable targetengagement in the lung, which are expected to enhance mucociliaryclearance in people with cystic fibrosis."

Atrial Fibrillation • Cardiovascular • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases

ETD001: A Long-Acting Inhaled ENaC Blocker, is Well Tolerated in Humans. (NACFC 2022) - No abstract available

Late Breaking Abstract - ETD001: A long-acting inhaled ENaC blocker, is well tolerated in humans (ERS 2022) - "ETD001 was well tolerated at SAD up to 10.8mg and MAD up to 4.65mg BID for 14 days. The wide safety margin is predicted to enable doses capable of durable target engagement in the lung, which are expected to enhance mucociliary clearance in people with cystic fibrosis."

Late-breaking abstract • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ETD001: a long-acting inhaled ENaC blocker is well tolerated in humans (ECFS 2022) - "ETD001 was well tolerated at SAD up to 7.2 mg and MAD of 1.55 mg BID and 3.1 mg QD for 7 days. The wide safety margin is predicted to enable doses capable of durable target engagement in the lung, which are expected to enhance mucociliary clearance in people with cystic fibrosis."

Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ET-ENAC-01: A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects (clinicaltrials.gov) - P1 | N=98 | Completed | Sponsor: Enterprise Therapeutics Ltd | Active, not recruiting ➔ Completed

Trial completion • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ET-ENAC-01: A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects (clinicaltrials.gov) - P1 | N=98 | Active, not recruiting | Sponsor: Enterprise Therapeutics Ltd | Recruiting ➔ Active, not recruiting

Enrollment closed • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ET-ENAC-01: A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects (clinicaltrials.gov) - P1; N=104; Recruiting; Sponsor: Enterprise Therapeutics Ltd; Trial completion date: Oct 2021 ➔ Mar 2022; Trial primary completion date: Oct 2021 ➔ Mar 2022

Clinical • Trial completion date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects (clinicaltrials.gov) - P1; N=88; Recruiting; Sponsor: Enterprise Therapeutics Ltd

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Enterprise Therapeutics doses first subjects in Phase I trial for novel cystic fibrosis therapy ETD001 (Businesswire) - "Enterprise Therapeutics...announced it has successfully dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis (CF) therapy, ETD001. The first-in-man safety study is being conducted in healthy participants...ETD001 could significantly improve quality of life for people living with cystic fibrosis and other respiratory diseases linked to mucus obstruction."

Trial status • Cystic Fibrosis • Genetic Disorders

ETD001: a long-acting and safely inhaled ENaC blocker to enhance mucociliary clearance (ECFS 2020) - "The preclinical profile of ETD001 is consistent with a candidate that can be tested for efficacy in CF patients. The wide safety margin observed with ETD001 is predicted to enable dosing up to a threshold required to observe a long-lasting enhancement of MCC in CF patients."