Corbus Pharmaceuticals announced that the European Medicines Agency (EMA) has designated the company’s synthetic oral endocannabinoid-mimetic, Resunab (JBT-101), an orphan drug as a potential treatment for people with cystic fibrosis (CF).
Resunab, which is currently being evaluated in a Phase 2 clinical trial in CF patients, preferentially binds to the CB2 receptor expressed on activated immune cells and fibroblasts. CB2 activation triggers endogenous pathways that resolve inflammation and stop fibrosis.
Preclinical and early clinical studies demonstrated that Resunab has a favorable safety, tolerability and pharmacokinetic profile. In preclinical models of inflammation and fibrosis, it was seen to stop tissue scarring, essentially ‘turning off’ chronic inflammation and fibrotic processes without causing immunosuppression.
“Receiving Orphan Designation for Resunab in the treatment of CF in the European Union is a noteworthy milestone in our global regulatory strategy for the clinical development of Resunab in CF,” Yuval Cohen, PhD, Corbus’ chief executive officer, said in a press release.
Corbus previously announced that the U.S. Food and Drug Administration (FDA) also granted Resunab orphan drug designation, as well as fast track status, as a possible treatment of CF and systemic sclerosis.
In the current study (NCT02465450), a double-blinded, randomized, and placebo-control clinical trial at sites in the U.S. and Europe, Resunab is being assessed for its safety, tolerability, and potential clinical benefits in 89 CF patients, as well as for its impact on bacterial load in the lungs and on biomarkers of inflammation in the sputum and blood. Top-line safety and efficacy results are expected early in 2017.
The trial is partially supported by a $5 million development award from the Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit drug discovery and development arm of the Cystic Fibrosis Foundation (CFF).
“There are no approved treatments for inflammation in CF, which is the major cause of lung damage in individuals with CF,” said Barbara White, MD, Corbus’ chief medical officer. “Resunab provided benefit in a murine model of lung inflammation in CF and in other pre-clinical models of lung inflammation and lung fibrosis. These data show Resunab’s potential to improve lung inflammation and consequently potentially improve lung function and pulmonary exacerbations in people with CF. This Orphan Designation … was granted based on its potential to provide significant benefit to individuals with CF.”
In Europe, orphan drug designation is reserved for medicines showing the potential to treat rare diseases affecting not more than five per 10,000 people on the continent. It offers financial and regulatory benefits to the drug’s manufacturer, and 10-year market exclusivity should the treatment be approved.
The drug candidate is also being evaluated for its therapeutic potential in systemic sclerosis and dermatomyositis. A fourth trial, in systemic lupus erythematosus (SLE), is planned for early next year.