Spyryx Biosciences Announces Successful Completion of Phase 1 Clinical Trial for SPX-101 and Planned Phase 2 Initiation in Cystic Fibrosis During 2017

DURHAM, N.C., Feb. 6, 2017 /PRNewswire/ -- Spyryx Biosciences, Inc., a clinical stage biopharmaceutical company developing innovative therapeutics to address severe lung diseases, today announced the successful completion of a Phase 1 study for its lead compound, SPX-101, in healthy volunteers.

SPX-101 is an inhaled SPLUNC1-derived peptide with a novel biological mechanism for regulating epithelial ion channels in the airway.  The drug is designed to restore a natural pathway in the lung that regulates airway hydration and promotes mucociliary clearance, which is dysfunctional in cystic fibrosis (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 CF patients.

"The data from this study validate the safety of SPX-101's novel mechanism of action; the internalization of epithelial sodium channels with a small, inhaled peptide. We are pleased to confirm the absence of safety or tolerability concerns and the lack of significant systemic exposure following 14-day administration," said Alistair Wheeler, MD, Chief Medical Officer of Spyryx. "Given the absence of dose-limiting adverse effects in healthy adults, supported by the excellent non-clinical safety data and the strong scientific rationale for a meaningful effect on pulmonary function, we are eager to move the program into the CF patient population."

SPX-101 was evaluated in the 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.  SPX-101 was administered in the MAD arm for 14 days.  Importantly, SPX-101 showed no treatment related adverse effects and no dose-limiting safety findings, including the absence of adverse effects on lung function, no shifts in serum or urinary electrolytes and no evidence of hyperkalemia.  As expected, the peptide demonstrated very low systemic exposure and rapid clearance from the circulation.

Based on the strength of these results, Spyryx has begun preparations for a multinational Phase 2 study in CF patients, with the goal of assessing SPX-101's ability to improve lung function, as well as the safety and tolerability of the inhaled drug.  The Phase 2 design will not be limited by patients' CFTR mutation.

Summary of Key Findings

• SPX-101 was safe and well-tolerated in all dose cohorts of the SAD and MAD arms.
• SPX-101 had no adverse effect on lung function.
• SPX-101 demonstrated very low systemic exposure and rapid clearance, with no effect on electrolyte levels or indication of hyperkalemia.

About Cystic Fibrosis
Cystic fibrosis (CF) is an autosomal recessive genetic disorder affecting approximately 75,000 individuals worldwide and is caused by mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The condition affects multiple organ systems including the lungs and respiratory tract, pancreas, intestine, liver and kidneys. However, the highest medical need is in the lungs, where complications from CF-related lung disease accounts for 80% of all CF deaths.

CF lung disease is hallmarked by dehydration of the fluid volume on the airway surface, resulting in reduced clearance of mucus, the lung's principle mechanism for cleaning itself. This mucus becomes thick and sticky, progressively accumulating into obstructions that are chronically colonized by viruses and bacteria, leading to frequent acute lung infections, inflammation and loss of lung function. The long-term result is permanent tissue damage and scarring (fibrosis) that commonly requires lung transplantation and often leads to early death. No cure for cystic fibrosis is known, although several treatments have been approved to address the underlying cause of the disease in some patients.  Despite currently available treatment, the median age of survival for CF patients is still only approximately 37 years of age.

About Spyryx Biosciences
Spyryx Biosciences is a privately held, 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 that is advancing into Phase 2 in CF patients. 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 in cystic fibrosis patients independent of their CFTR 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. Further information regarding Spyryx Biosciences is available at www.spyryxbio.com.

SOURCE Spyryx Biosciences

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