DURHAM, N.C., June 6, 2018 /PRNewswire/ -- Today Spyryx shared positive data for the first cohort of the SPX-101 Phase 2 clinical trial HOPE-1 (Hydration for Optimal Pulmonary Effectiveness), which showed that treatment with SPX-101 in patients with cystic fibrosis (CF) resulted in a meaningful benefit for lung function via a novel method for modulation of ENaC. Notably, the inhaled peptide therapeutic can be used in all patients regardless of their CF mutation.
On Friday June 8 at the European Cystic Fibrosis Society in Belgrade, Serbia, Isabelle Fajac, M.D., Ph.D., Principal Investigator, Vice President and President Elect of the European Cystic Fibrosis Society and Professor of Physiology at Université Paris Descartes, Paris, France will formally present the data in an oral late-breaking presentation. The session – WS20 at 17:30 on June 8 – will be chaired by Harm Tiddens, Professor of Pediatric Pulmonology at Erasmus MC-Sophia Children's Hospital in Rotterdam and Damien Downey, National Coordinating Investigator for the study for the United Kingdom and Clinical Senior Lecturer in Respiratory Medicine at Queen's University Belfast.
"There is urgent need for an effective and safe treatment to promote airway clearance for all patients with CF, regardless of their genetic mutation," said Prof. Fajac. "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."
HOPE-1 (NCT03229252) is an exploratory, randomized, placebo-controlled, 28-day Phase 2 study of Spyryx's lead therapeutic candidate, SPX-101, for the treatment of patients with cystic fibrosis (n=46) independent of CFTR mutation type. Cohort 1 of this study compared a high dose (120mg bid, n=16), a low dose (60mg bid, n=15) and placebo (n=15) in patients with baseline ppFEV1 (percent predicted forced expiratory volume in 1 second) of 40-80%. The drug was administered by nebulization via a silent, rapid and portable nebulizer. ppFEV1 was measured at Day 7, Day 14 and Day 28. The primary efficacy endpoint was the absolute change from baseline in ppFEV1 after 28 days.
Results show that at Day 28, a dose related benefit was seen with the high dose showing a placebo adjusted increase of 5.2% in ppFEV1 (p=0.054). With the high dose, ppFEV1 separation from placebo was seen by Day 7 and persisted through the treatment period. In the subgroup of moderate patients with baseline pp FEV1 > 55% (n=20) the high dose response at Day 28 was 8.3% (p=0.048). SPX-101 was safe and well tolerated with the most common adverse events being cough and sputum production.
"SPX-101 shows the first successful modulation of ENaC surface density resulting in a meaningful benefit on the gold standard regulatory approval endpoint of ppFEV1 in patients with cystic fibrosis," said Alistair Wheeler, M.D., M.F.P.M., Chief Medical Officer at Spyryx. "SPX-101 is agnostic to CFTR mutation with a mechanism that is complementary to CFTR modulators and correctors."
"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," said Tim Shannon, M.D., Chairman of the Spyryx Board, General Partner at Canaan and a pulmonologist. "We're looking forward to completing Phase 2 and beginning preparations for Phase 3 development activities in 2019."
The HOPE-1 study is an exploratory Phase 2 study that uses an adaptive design and will now continue to explore the SPX-101 dose-response, and target patient population to prepare for subsequent pivotal studies. In total, the study is planned to enroll approximately 90 adult subjects with CF and is being conducted globally.
About Spyryx Biosciences
Spyryx Biosciences is a clinical-stage biopharmaceutical company developing innovative therapeutics to address severe pulmonary diseases. Spyryx's lead clinical candidate, SPX-101, is completing a multinational Phase 2 study as a novel treatment for cystic fibrosis. The product has demonstrated a robust ability to restore mucociliary clearance in animal models of the disease. SPX-101 has also been shown to be safe and well tolerated in a completed Phase 1b in adult patients with cystic fibrosis and has the potential to improve lung function in all people living with cystic fibrosis independent of their genotype. Spyryx is funded by a first-tier syndicate of life science investors, including Canaan, 5AM Ventures and Hatteras Venture Partners - as well as the Cystic Fibrosis Foundation. Further information regarding Spyryx Biosciences is available at www.spyryxbio.com.
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