US-based Corbus Pharmaceuticals Holdings has started patient enrolment in the Phase II clinical trial of its investigational new drug Resunab to treat adult patients with cystic fibrosis (CF).
Resunab is a new oral drug targeting the resolution of inflammation and fibrosis associated with disease progression in CF across all cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations.
It is a new synthetic oral endocannabinoid-mimetic drug that preferentially binds to the CB2 receptor expressed on activated immune cells and fibroblasts.
The company noted that in a pre-clinical CF animal model, Resunab improved inflammation, weight loss, bacterial infection and survival.
In April, the company secured a $5m development award from Cystic Fibrosis Foundation Therapeutics to support this multi-centre, international Phase II trial of Resunab in CF.
In the US, the trial is being led by principal investigator James Chmiel, a specialist in paediatric pulmonary diseases in the Division of Pediatric Pulmonology, Allergy, Immunology and Sleep Medicine and associate director of the LeRoy W Matthews Cystic Fibrosis Center at University Hospitals Rainbow Babies & Children's Hospital.
The trial is also being led in the EU by Northern Ireland's Queen's University Belfast School of Medicine, Dentistry and Biomedical Sciences dean and co-principal investigator Stuart Elborn.
Approximately 70 adults with CF, irrespective of their CFTR mutation, will be enrolled in this double-blind, randomised, placebo-control Phase II trial of Resunab.
In the trial, each patient will be treated daily for a period of 84 days, with a follow-up period of 28 days.
Scheduled to be completed within 18 to 21 months, the trial is designed to evaluate the safety and tolerability of Resunab, as well as its potential impact on clinical outcomes as measured by FEV1, Lung Clearance Index, CFQ-R Respiratory Symptom scale, and lung microbiota.
Additionally, the trial will test the impact of Resunab on biomarkers of inflammation in the sputum and blood.
Corbus chief executive officer Yuval Cohen said: "Resunab is a first-in-class drug that induces the resolution of inflammation.
"The CF study is our third Phase II trial launched so far this year in a rare inflammatory disease, joining the Phase II trials in systemic sclerosis and dermatomyositis launched previously in 2015.
"The systemic sclerosis and CF studies are scheduled to conclude in the fourth quarter of 2016 and we look forward to reporting top-line data at that time."
In pre-clinical and Phase I trials, Resunab is shown to have a favourable safety, tolerability and pharmacokinetic profile, while the drug also showed promising potency in pre-clinical models of inflammation and fibrosis.
In August, the company began enrolling patients in the Phase II trial of Resunab for the treatment of diffuse cutaneous systemic sclerosis (systemic sclerosis).
The double-blind, randomised, placebo-control trial will enrol up to 36 individuals with systemic sclerosis who will be treated for 84 days with a follow up period of 28 days.
Image: 'Clubbing' of the fingers is a classic feature of Cystic Fibrosis. Photo: courtesy of Jerry Nick, M D.