A Phase 2a trial usually looks at a drug’s effectiveness in a small number of patients, while the goal of a Phase 2b study is generally to find an optimal dose to test on a larger group.
Corbus will seek trial-design guidance from the U.S. Food and Drug Administration and the European Medicines for the new study. It will also collaborate on the design with the Cystic Fibrosis Foundation Therapeutics Development Network.
Corbus also said it plans to submit a Pediatric Investigational Plan to the European Medicines Agency. The aim of the plan is to ensure that Corbus collects enough information about anabasum’s effect on children to support European regulators approving the medicine for youngsters.
The therapy targets immune cells and fibroblasts, or cells involved in scarring. It promotes the release of substances that help resolve inflammation and halt fibrosis.
Anabasum reduced the rate of pulmonary exacerbations requiring antibiotics and lowered levels of several inflammation biomarkers, according to results of the Phase 2 trial (NCT02465450). It also had an acceptable safety and tolerability profile.
Corbus presented the results at the European Cystic Fibrosis Society conference in Seville, Spain, in June. It will present additional results at the North American Cystic Fibrosis Conference in Indianapolis, Indiana, in November.
“We have made significant progress on the corporate, operational and clinical fronts, enabling the company to move forward with a strong balance sheet and plans to execute on multiple potentially impactful milestones coming up in the second half of this year,” Dr.Yuval Cohen, Corbus’ CEO, said in a press release.
The FDA gave anabasum orphan drug and fast track status as a CF treatment in 2015, and European regulators gave it orphan drug status in 2016. Corbus is also testing it as therapy for several other conditions, including systemic sclerosis, dermatomyositis, and systemic lupus erythematosus.