Spyryx Biosciences Announces Successful Completion of Phase 1 Clinical Trial for SPX-101 and
Planned Phase 2 Initiation in Cystic Fibrosis During 2017
- Study results indicate SPX-101 was well-tolerated with no dose limiting safety concerns
- Company has initiated plans for a Phase 2 study to investigate clinical benefit of SPX-101 in treating
cystic fibrosis-related lung disease, regardless of mutation
DURHAM, N.C., February 6, 2017 – 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 doselimiting
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
CONTACT: Medical Dynamics, Cynthia Sutherland, Media Group Director
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