Spyryx Biosciences Announces First Patient Enrolled in Phase 2 Clinical Trial of SPX-101 for the Treatment of Cystic Fibrosis

DURHAM, N.C., Oct. 18, 2017 /PRNewswire/ -- Spyryx Biosciences, Inc., a clinical stage biopharmaceutical company developing innovative therapeutics to address severe respiratory diseases, today announced the first patient was dosed in the HOPE-1 study, a multinational Phase 2 clinical trial for its lead compound, SPX-101 in cystic fibrosis (CF).

HOPE-1 is a 28-day study using an adaptive design testing two doses of SPX-101 against placebo in each of two serial cohorts of patients.  In total, the study is planned to enroll up to 78 subjects with CF and is being conducted in select clinics in Canada and Western Europe. The adaptive trial design allows for efficient testing of the efficacy, safety and tolerability of SPX-101 across multiple dose levels.

"We are delighted to have treated the first patient in our Phase 2 study of SPX-101," said Alistair Wheeler MD MFPM, Chief Medical Officer of Spyryx Biosciences. "This study will provide data relating to efficacy and safety at the earliest opportunity. We are particularly proud that patients who were part of the protocol review process have provided positive feedback, reflecting that we have kept the investment of their valuable time to a minimum. The study has also been embraced by many widely respected researchers in the CF clinical community and we are grateful for their engagement."

SPX-101 is an inhaled peptide with a novel, cellular mechanism that durably reduces sodium absorption in the airway by internalizing epithelial sodium channels (ENaC) from the apical surface of the epithelium.  The therapeutic effect of the drug is to enhance airway and mucus hydration and promote mucociliary clearance, which is dysfunctional in CF.  The mechanism of action of SPX-101 is independent of the genetic mutations that cause CF and has the potential to provide disease-modifying therapy to all people living with this disease. "For several decades, the scientific community has focused on the therapeutic potential of small molecule inhibitors of ENaC in the treatment of cystic fibrosis," said Professor Isabelle Fajac, Professor of Physiology at Université Sorbonne Paris Cité, President-Elect of the European Cystic Fibrosis Society (ECFS) and former head of the Clinical Trial Network of the ECFS. "This novel mechanism, the internalization of sodium channels, represents an important scientific advance in the approach to ENaC as a potential treatment of cystic fibrosis for all patients and it is important that this novel peptide is evaluated in our patient population."

"I am pleased to have treated the first patient in this important study," said Dr. Nicholas Simmonds, Consultant Respiratory Physician and Associate Director of the Adult CF Centre at the Royal Brompton Hospital, London, and a Principal Investigator in the study. "As a potential treatment for the respiratory dysfunction of cystic fibrosis in all patients regardless of their genetic mutation, this study is of great interest to our patient population and we look forward to its successful completion."

"The Clinical Trial Network was pleased to review this important protocol and to approve it for inclusion in the CTN with its highest priority rating," said Dr. Tim Lee, Director of the Clinical Trials Network (CTN) of the European Cystic Fibrosis Society. "We look forward to working with Spyryx Biosciences to continue the development of this important potential treatment."

SPX-101 was evaluated in a Phase 1 study for safety, tolerability and pharmacokinetics in both single ascending dose (SAD) and multiple ascending dose (MAD) arms. The drug was well-tolerated in all 64 subjects tested, with all subjects completing the study protocol. Importantly, SPX-101 showed no dose-limiting safety findings, including the absence of adverse effects on lung function and no shifts in serum or urinary electrolytes. As expected, the peptide was associated with very low systemic exposure and rapid clearance from the circulation.

About Cystic Fibrosis
Cystic Fibrosis (CF) is an autosomal recessive genetic disorder affecting approximately 75,000 individuals worldwide.  The disease is caused by mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CF profoundly affects the lungs and respiratory tract and is characterized by dehydration of the airway surface, resulting in reduced mucus clearance, the lung's principle mechanism for maintaining a clean environment. The reduction in clearance results in frequent, acute lung infections and chronic inflammation. The long-term result of the disease is progressive, permanent tissue damage and pulmonary scarring (fibrosis) with reduced lung function.  No cure for CF is known, although several treatments have been approved to address the underlying cause of the disease for some CF mutations. Despite the positive advances, more work is needed to ensure effective therapies are available for everyone living with CF.

About Spyryx Biosciences
Spyryx Biosciences is a clinical-stage biopharmaceutical company developing innovative therapeutics to address severe lung diseases. Spyryx's lead clinical candidate, SPX-101, is a novel treatment for cystic fibrosis (CF) that is currently in Phase 2 clinical testing in people with CF. The product has demonstrated a robust ability to restore mucociliary clearance in animal models of the disease and has the potential to improve lung function for all patients, independent of their disease-causing mutation. The Spyryx leadership team and scientific staff have extensive experience in the development of respiratory medicines and work closely with a broad group of clinical and scientific experts in the pulmonary field. Spyryx is funded by a first tier syndicate of life science investors, including Canaan Partners, 5AM Ventures and Hatteras Venture Partners, as well as a development award from CFFT, the development arm of the Cystic Fibrosis Foundation. Further information regarding Spyryx Biosciences is available at www.spyryxbio.com.

SOURCE Spyryx Biosciences, Inc.

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