MUNICH and MENLO PARK, Calif., March 26, 2019 /PRNewswire/ -- Breath Therapeutics, a private company developing advanced inhaled therapeutics in severe pulmonary orphan diseases, announced today the initiation of the Phase 3 BOSTON clinical studies of L‑CsA‑i for the treatment of Bronchiolitis Obliterans Syndrome (BOS). Breath Therapeutics' lead drug candidate, L‑CsA‑i, is a proprietary liposomal formulation of cyclosporine A for inhalation administered via a drug-specific investigational eFlow® nebulizer (PARI Pharma). L‑CsA‑i has received orphan drug designation for the treatment of BOS from the Food and Drug Administration and European Medicines Agency.
The harmonized global randomized, controlled studies will evaluate the efficacy and safety of L‑CsA‑i in individuals with BOS following a single lung (BOSTON-1) or double lung (BOSTON-2) transplantation. The primary endpoint is mean change in FEV1 (mL) from baseline to week 48 and the key secondary endpoints are mean change in FEV1/FVC and time to progression of BOS. Each study will enroll 110 participants at leading lung transplant specialty centers in eight countries. Upon completion of the 48-week studies, all participants will be eligible to continue in the planned BOSTON-3, an open-label extension trial. Additional information is available at ClinicalTrials.gov. (BOSTON-1 NCT03657342) (BOSTON-2 NCT03656926). To view experts discuss their clinical experience with BOS and the BOSTON-1 and BOSTON-2 studies, please visit here.
Jens Stegemann, Chief Executive Officer of Breath Therapeutics, said, "The initiation of the BOSTON pivotal trials represents a major milestone for Breath Therapeutics and is indicative of the outstanding progress we have achieved in the past 24 months. We have accomplished full commercial scale production capability of both the drug and the drug-specific inhalation device, and have also assembled an exceptional leadership team to execute on our clinical development programs and global commercialization strategy. Together with the enthusiasm and commitment of our renowned investigators and their teams from more than 35 of the leading lung transplant centers world-wide, we are well positioned to succeed in our goal of bringing an effective and safe treatment to people with BOS."
Joseph M. Pilewski, MD, Associate Chief for Clinical Affairs and lung transplant physician in the Division of Pulmonary, Allergy & Critical Care Medicine at the University of Pittsburgh Medical Center, stated, "Although lung transplant survival has improved with the advancement of surgical techniques and perioperative management, and more individuals are undergoing lung transplantation, survival has not improved to reach the rate of other organ transplantations. One key factor impacting survival in lung recipients is rejection, particularly bronchiolitis obliterans syndrome (BOS), the most common form of chronic lung allograft dysfunction. The BOSTON trials are timely and important to lung recipients and the broader lung transplant community because inhaled cyclosporine may provide the first safe and effective treatment for BOS."
Antonio Román, MD, Medical Coordinator of the Lung Transplantation and Director of PAH Unit at Hospital Vall d´Hebrón, Barcelona, said, "We are pleased the BOSTON trials have started and are appreciative for the opportunity to contribute to this important global program. Bronchiolitis obliterans syndrome is a devastating disease for patients and their loved ones and there is an urgent need for a suitable therapy for this underserved community."
Bronchiolitis Obliterans Syndrome (BOS)
Bronchiolitis Obliterans Syndrome (BOS), also known as obliterative bronchiolitis (OB) or chronic lung allograft rejection, is caused by T-cell mediated inflammation that leads to blockage of bronchioles, the small and medium airways in the lungs, resulting in respiratory failure and death. BOS most commonly affects people who have received lung or stem cell transplantation, although it is also associated with autoimmune disease and exposure to environmental contaminants. Over 30,000 lung transplantation and allogeneic hematopoietic stem cell transplantation recipients worldwide are currently affected by BOS. Based on company research, lung and allogeneic stem cell transplantations increased between 3% to 5% in recent years, depending on the geographical region, translating to a similar growth in BOS cases.
Liposomal Cyclosporine A for Inhalation (L-CsA-i)
Breath Therapeutics' lead candidate, L‑CsA‑i, is a novel liposomal formulation of cyclosporine A designed for inhaled delivery to the lungs. Calcineurin inhibitors (CNIs), like cyclosporine A, are highly potent immunosuppressive drugs and a cornerstone of lung transplant medicine. Due to a narrow therapeutic window, mainly caused by nephrotoxicity, the systemic administration of CNIs cannot achieve sufficient drug levels in the bronchiolar tissue needed to treat BOS. L‑CsA‑i is administered via a customized drug-specific investigational eFlow® nebulizer. The drug-device combination is designed to deliver L-CsA-i to the site of disease in the lung while minimizing systemic exposure.
BOSTON Development Program
L-CsA-i is being developed for the treatment of BOS in patients age six and older. Five clinical trials are currently planned or underway. BOSTON-1 and BOSTON-2 are pivotal Phase 3 studies of patients with BOS following lung transplantation. BOSTON-3 is an open-label extension study for all study participants who complete BOSTON-1 or BOSTON-2. BOSTON-4 is a Phase 2 study and will be the first trial of L‑CsA‑i in adults with BOS following allogeneic hematopoietic stem cell transplant. BOSTON-5 is a Phase 2 study in pediatric patients with BOS.
Founded in 2016, Breath Therapeutics is a private, venture-backed clinical stage biopharmaceutical company specializing in advanced inhaled therapeutics for severe respiratory diseases with high unmet medical need. The company's proprietary drug formulations have been specifically designed for inhaled administration with exclusively licensed, high performance nebulizer technology. L‑CsA‑i, Breath Therapeutics' lead asset, is advancing as the first therapy for BOS, a rare and devastating lung disease with no currently available treatment. Breath Therapeutics has offices in Munich, Germany and Menlo Park, CA. For more information, please visit www.breath-therapeutics.com.
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