Its a tough market for cystic fibrosis these days. Just ask Savara.
Two years ago, the Austin-based biotech designed and launched a trial to test whether their lead GM-CSF drug molgradex can reduce lung infection in adult CF patients. At the time, Vertex only had 3 CF drugs, they only covered a portion of patients, and they werent always incredibly effective. Then last year came Trikafta, the expensive triple-combination that had significant benefits for around 90% of patients.
So where did that leave Savara? Out of luck, it seems. On Friday, the company announced that it was discontinuing the Phase II trial, citing both Vertexs success and the toll Covid-19 took on patient recruitment. The news spells the end of development for CF and shifts further focus on the companys troubled lead program for autoimmune pulmonary alveolar proteinosis.
For Savara, Trikafta and Covid-19 were a double-whammy that hit on two different fronts. First, the Vertex drug became the standard of care for CF patients. That meant 8 out of the 14 patients they recruited for the study started taking Trikafta while they were taking the Savara inhalant, confounding any attempt to tease out whether improvements were a product of the Savara drug or the Vertex one. Then in March, the company stopped enrolling patients after Covid-19 hit the US, overwhelmed hospital systems and disrupted the entire clinical trial system.
CMO Badrul Chowdhury noted 5 patients who were also on Trikafta saw three consecutive tests without infection. But it wasnt clear enough to warrant further investment.
While the results of ENCORE are interesting, he said, additional controlled studies would be required to accurately understand the therapeutic potential of Molgradex in combination with the triple-combination modulator treatment.
The end of the CF program consolidates focus on their lead indication, where the jury remains out. Last year, the company saw their shares crushed after a Phase III study missed its primary endpoint and multiple secondary endpoints. Although the company talked about a high placebo effect, pointing to patient-reported outcomes and later releasing slides showing endpoints that trended toward significance, few investors seemed to believe the story.
But then in December, the FDA handed the company breakthrough status on the drug. They evidently bought some portion of the executives story, though which part and what the path forward is for the company remains unclear.
Social: Rob Neville, Savara CEO
More:
Vertex and Covid-19 spell the end for Savara's CF program - Endpoints News
Recommendation and review posted by G. Smith
