According to Savara, the FDA has approved an IND the company submitted for Molgradex, an inhaled molgramostim (recombinant human GM-CSF) formulation for the treatment of autoimmune pulmonary alveolar proteinosis (PAP), which the company filed in December 2017. The approval allows the company to enroll patients in the ongoing Phase 3 IMPALA study at sites in the US.
The IMPALA study was approved by Japanese regulators in 2016 prior to Savara’s acquisition of Molgradex developer Serendex later that year. The IND included several modifications to the study in addition to adding US sites to enable the company to use IMPALA as a pivotal study for a US regulatory submission.
Savara CEO Rob Neville said, “Expansion of the IMPALA study enrollment into the US. is an important and exciting step towards a BLA submission of Molgradex in the US. By conducting the interim sample size check we believe we were able to increase the likelihood of success in our sole pivotal study by ensuring a high level of statistical power not only for the primary endpoint, but now also for two key secondary endpoints that the FDA will be focusing on. We have continued strong progress in enrollment, having reached the original target of 90 patients almost two months ahead of schedule, and we expect to complete the revised enrollment target by the third quarter of 2018 and anticipate that top line results will be available in the second quarter of 2019.”
Read the Savara press release.