Savara announces update on Molgradex, new CF study
Savara provided an update on the clinical development and commercial preparatory efforts for its lead product candidate Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor, or GM-CSF. In addition, Savara is expanding its Molgradex nontuberculous mycobacteria, or NTM program with a new study in the U.S. in cystic fibrosis, or CF affected individuals with chronic NTM lung infection. The company also announced its acquisition of the assets of Cardeas Pharma Corporation as part of its launch of an exploratory product pipeline comprising pre-proof-of-concept, high-potential programs focused on difficult-to-treat lung diseases. Savara's pivotal Phase 3 IMPALA study is evaluating Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis, or aPAP. Completion of patient enrollment remains on track for the third quarter of 2018 and topline data are anticipated in Q2 of 2019. The IMPALA study includes a six-month placebo-controlled period with 2:1 randomization on Molgradex or placebo, followed by a six-month open-label period with all subjects receiving Molgradex. Earlier this year, the company launched the IMPALA-X study, an open-label, multicenter study designed to evaluate the long-term use of Molgradex in patients with aPAP. The IMPALA-X study offers patients completing the IMPALA study the opportunity to continue treatment with Molgradex for up to three additional years. Driven by its confidence in the outcome of the IMPALA study, the company will expedite its preparation for potential commercial launch by starting investments into core commercial staff and external activities required for timely launch, subject to product approval by the FDA. The company believes that robust results in the IMPALA study would make Molgradex eligible for designation as a breakthrough therapy and facilitate the submission of the Molgradex Biologic License Application, or BLA, in the first half of 2020, with an anticipated commercial launch in late 2020 or early 2021. The company is now preparing to initiate a new study in the U.S. in CF affected individuals with chronic NTM infection. This Phase 2a open-label study will enroll 30 subjects with CF and chronic NTM lung infection, and is planned to begin in Q1 of 2019. The study will enroll subjects with chronic Mycobacterium abscessus or Mycobacterium avium complex infection. The study will comprise a 48-week treatment period and a 24-week follow-up period, and three subgroups of subjects will be recruited into the study. Group 1 will consist of subjects who have been on antimycobacterial treatment for at least six months prior to the baseline visit. Group 2 will consist of subjects who have stopped antimycobacterial treatment due to intolerance or lack of effect prior to the baseline visit. Group 3 will consist of subjects who have not been treated with antimycobacterial treatment but are clinically stable prior to the baseline visit. The primary endpoint in the study will be NTM sputum culture conversion to negative. As part of its growth strategy, Savara has launched an exploratory product pipeline comprising pre-proof-of-concept, high-potential programs focused on difficult-to-treat lung diseases. As the first new product in the exploratory pipeline, Savara acquired the rights to amikacin/fosfomycin inhalation solution, a Phase 2 ready product candidate that had been in development by Cardeas Pharma Corporation for the treatment of ventilator associated pneumonia.