Corbus Pharmaceuticals recently reported that its Phase 2 clinical trial examining multiple doses of anabasum (formerly known as JBT-101 or Resunab) compared to placebo in patients with cystic fibrosis (CF) achieved the trial’s primary endpoint demonstrating an acceptable safety and tolerability profile at all doses, with no serious or severe adverse events.
The study (NCT02465450) included 85 patients who were treated at CF centers in the United States, Belgium, France, Germany, Italy, Poland and the United Kingdom. It compared a range of dose regimens to placebo, with a main focus on anabasum’s safety. The study lasted 84 days with a 28-day follow-up period.
Secondary outcomes of the trial included evaluation of changes in lung function and bacterial composition of sputum.
The company reported that patients treated with anabasum showed dose-dependent reductions in the number of acute pulmonary exacerbations. Compared to placebo-treated patients, those treated with the highest anabasum dose (20 mg orally, twice daily) had a 75 percent reduction in the annualized rate of pulmonary exacerbations.
Treatment with anabasum also led to a reduction in patients’ inflammation-associated sputum cells. Specifically, the treatment reduced leukocytes counts, as well as neutrophils, eosinophils and macrophages. The treatment also reduced interleukin-8, neutrophil elastase and immunoglobulin G in sputum — all known inflammatory mediators.
Patients’ lung function, as measured by forced expiratory volume in one second (FEV1), remained stable.
According to Corbus, which is headquartered in Norwood, Mass., these results demonstrate that anabasum treatment reduces pulmonary exacerbations in CF patients. They also provide clinical evidence of the drug’s biological activity in treating innate immune responses in the lungs.
“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,” lead researcher James Chmiel, MD, MPH, of Case Western Reserve University, 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.”
“We are delighted that in this first-in-CF patient study, anabasum demonstrated an acceptable safety profile and potential clinical benefit in reducing acute pulmonary exacerbations in CF patients and that these findings are supported by biomarker data consistently showing reduction of inflammation in the lungs,” said Corbus CEO Yuval Cohen, PhD. “These positive results coincide with our third anniversary as a company and come on the heels of positive data from our Phase 2 study in systemic sclerosis. We are very grateful to all the patients, investigators and clinical staff who participated in this study and to Cystic Fibrosis Foundation Therapeutics for their support.”
Anabasum is a synthetic, oral endocannabinoid-mimetic drug for inflammation, thought to increase the production of anti-inflammatory mediators while reducing the production of those that increase inflammation. Reducing inflammation helps to prevent permanent tissue damage in the lungs of people with CF.
Anabasum has been designated an orphan drug for treating CF in the United States and Europe. The U.S. Food and Drug Administration has granted Anabasum fast-track status to speed the drug’s development.