Vertex's success kills off Savara's experimental cystic fibrosis drug hopes


Texas-based biotech Savara is killing off a midstage test of its investigational cystic fibrosis (CF) hopeful Molgradex after a new winner-takes-all competitor from Vertex.

The biotech in fact halted the test of its phase 2a exploratory test, known as ENCORE, back in March due to the COVID-29 pandemic.

This was set up as a 48-week, open-label, noncontrolled test of its drug Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of nontuberculous mycobacterial (NTM) lung infection in people living with CF.

While most trials that were halted in March have got back up and running, Savara said it will not look to continue the trial.

The biotech pointed to a number of factors for its termination, including COVID-19 recruitment issues (especially for a respiratory-based test), but predominately “the availability of the new triple-combination CFTR modulator, approved during the treatment period of ENCORE, that has become a preferred treatment option for many CF patients.”

While not mentioned by name, this is referring to Vertex’s Trikafta (elexacaftor/ivacaftor/tezacaftor), now FDA approved for patients 12 years and older with CF who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.

“Study recruitment was terminated at the end of March with 14 patients enrolled out of a total target of 30,” the biotech said. “Additionally, nine out of 14 patients were on the triple-combination CFTR modulator. Eight patients started it during the study and one patient was on the triple-combination modulator from baseline.”

The biotech will now squarely focus the development of Molgradex on its lead indication, autoimmune pulmonary alveolar proteinosis, which is in phase 3 (but hit a roadblock last year when it failed a primary endpoint) “and does not plan to conduct further development activities related to Molgradex in NTM.”

The biotech was down 12% premarket Friday morning.