Topline data from a Phase 2 clinical trial evaluating multiple doses of anabasum compared to placebo shows promising results in the treatment of pulmonary exacerbations in patients with cystic fibrosis (CF), Corbus Pharmaceuticals recently announced.
Anabasum (formerly known as Resunab or JBT-101) is a first-in-class, synthetic oral drug that mimics the effects of endocannabinoids. The drug was designed to trigger the production of specialized pro-resolving lipid mediators that activate an endogenous cascade responsible for resolving inflammation and halting fibrosis, while reducing production of inflammatory mediators without causing immunosuppression.
In the U.S., anabasum was granted both Orphan Drug and Fast Track designations as a potential CF treatment by the U.S. Food and Drug Administration (FDA) in 2015, and Orphan Drug status by the European Medicines Agency (EMA) in 2016.
The 16-week Phase 2 trial (NCT02465450) evaluating anabasum was included in the clinical program developing the therapy. The study dosed 85 adult CF patients with baseline forced expiratory volume in one second (FEV1; a measure of lung function) predicted at equal or above 40%. Patients were enrolled regardless of their mutations in the CF transmembrane conductance regulator gene (CFTR; the gene defective in CF patients).
The trial has now achieved its primary objective by demonstrating positive safety and tolerability profiles with no serious side effects.
Anabasum showed a dose-dependent reduction of acute pulmonary exacerbations, defined as those requiring intravenous antibiotics, compared to placebo. Patients in the highest dose cohort (20 mg orally twice daily) had a 75% reduction in annual pulmonary exacerbations (compared to the group receiving placebo).
The drug candidate was also shown to consistently reduce the number of multiple inflammatory cell types in sputum, as well as inflammatory mediators.
“The reduction in acute pulmonary exacerbations along with reductions in inflammatory cells and inflammatory mediators in sputum demonstrate the potential for anabasum as a new inflammation-targeting therapeutic in cystic fibrosis that can broadly target patients without regard to their specific CFTR mutations,” James Chmiel, MD, principal investigator of the study, said in a press release.
“The outcomes of this 16-week study indicate that anabasum has the potential to address the important unmet need for treatments that target inflammation in CF,” Chmiel added.
The Phase 2 trial was an international, multi-center, double-blind, randomized, placebo-controlled study supported by a $5 million Development Award from the Cystic Fibrosis Foundation Therapeutics.
Corbus will now begin to evaluate the data and design the next clinical trial included in the anabasum development program.
“We are delighted that anabasum demonstrated a safety profile that was well tolerated by the CF patients in this study, especially given the challenges in safely targeting inflammation in CF,” said Barbara White, MD, chief medical officer of Corbus. “In a study of just 12 weeks of active dosing, we are especially encouraged by the consistency in data that couple clinical benefit in pulmonary exacerbations with improvement in the inflammatory response in the lungs.
“We believe these findings reflect the underlying mechanism of action of anabasum in activating resolution of innate immune responses without immunosuppression,” she added.
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