Spyryx Biosciences has announced that data from the first cohort of the Phase 2 HOPE-1 (Hydration for Optimal Pulmonary Effectiveness) study, which was initiated in October 2017, demonstrate improvement in lung function for cystic fibrosis patients independent of type of CF mutation after treatment with SPX-101, an inhaled SPLUNC1-derived peptide.
The 46 patients in this cohort received twice daily 120 mg or 60 mg doses of nebulized SPX-101 or placebo. The primary endpoint of the study is absolute change from baseline ppFEV1 at 28 days, and ppFEV1 was measured at days 7, 14, and 28. For the higher dose, SPX-101 began showing a benefit to lung function compared to placebo at day 7. At day 28, the placebo adjusted increase was 5.5%, with patients having a baseline ppFEV1 greater than 55% having an average increase of 8.3%.
The HOPE-1 study is continuing and is expected to enroll approximately 90 adult CF patients in total.
Principle investigator Isabelle Fajac of Université Paris Descartes commented, “There is urgent need for an effective and safe treatment to promote airway clearance for all patients with CF, regardless of their genetic mutation. These data show that with this novel mechanism – internalization of ENaC – it is possible to inhibit ENaC in the airways without impacting potassium concentrations in the blood, and this has been a goal in research for the past 30 years. These data are also very promising in terms of respiratory benefit gained from ENaC inhibition. I look forward to continuing the study.”
Spyryx board chairman Tim Shannon said, “This encouraging data for the first study of SPX-101 in patients gives hope to those of us who have grappled with CF as patients or providers for decades. These results make us optimistic that we have turned another corner in care for patients with cystic fibrosis. We’re looking forward to completing Phase 2 and beginning preparations for Phase 3 development activities in 2019.”
Read the Spyryx Biosciences press release.
