Zambon Completes Enrollment in Phase 3 Clinical Development Program Evaluating Liposomal Cyclosporine A for Inhalation (L-CsA-i) in Patients with Bronchiolitis Obliterans Syndrome (BOS)
Milan, Italy and Boston, MA, May 1, 2023 —Zambon, a multinational pharmaceutical company focused on innovating cure and care to improve people’s health and the quality of patients’ lives, announced today that the Company has completed enrollment in its two pivotal Phase 3 studies. The trials are designed to evaluate the safety and efficacy of Liposomal Cyclosporine A for Inhalation (L-CsA-i) for the treatment of BOS in adults following single lung (BOSTON-1) or double lung (BOSTON-2) transplantation.
BOS is a rapidly progressive inflammatory rare disease that irreversibly destroys the airways of the lungs and usually leads to respiratory failure and death within two to four years after diagnosis. Unfortunately, approximately 50 percent of lung transplant patients develop BOS within five years post-transplant, the highest rejection rate of any transplanted organ. Currently, there is no approved treatment indicated for BOS.1
“The completion of enrollment in our Phase 3 BOSTON-1 and -2 studies represents a significant milestone for Zambon and the patient community we aim to serve,” said Paola Castellani, chief medical officer and R&D head at Zambon. “On behalf of the Company, we would like to thank the many participants in our Phase 3 clinical program, as well as our study sites and investigators for their commitment. We look forward to reporting top-line results from the program during 2024.
L-CsA-i is a novel, inhaled therapy administered via the optimized investigational eFlow® Technology nebulizer system (PARI Pharma GmbH). The BOSTON-1 and -2 studies enrolled a total of 231 patients in Europe, Israel and the United States and had participation from 42 lung transplant centers globally.
“At Zambon, we are committed to advancing new therapeutic options that have the potential to make lives better for people affected by severe respiratory diseases such as BOS. The completion of enrollment in our pivotal trials brings us one step closer to our goal of bringing a much-needed therapy to the lung transplant recipients worldwide with BOS,” said Ilaria Villa, chief executive officer and interim chief operations officer of Zambon.
Zambon is also advancing one additional clinical study, BOSTON-3 – an extension study that is open to eligible participants who complete the BOSTON-1 and-2 studies.
L-CsA-i has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and European Medicines Agency for the treatment of BOS, reflecting the high unmet need of the disease. The Fast Track designation was also granted by FDA. If approved, L-CsA-i would be the first treatment indicated for BOS.