Additionally, as announced earlier this month, Spyryx is also advancing plans for a Phase 2 study of SPX-101 in CF patients.
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 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.
"This Phase 1b study is an important final step as we move into a full-fledged Phase 2 study with cystic fibrosis patients," said Alistair Wheeler, MD, Chief Medical Officer of Spyryx. "Having completed the study in healthy subjects and found SPX-101 to be safe and well tolerated, we look forward to advancing into patients and further evaluating this novel and exciting peptide. It is fitting on Rare Disease Day that we are able to share our progress in bringing a new potential treatment to these patients."
The announcement comes on the heels of the successful Phase 1 safety, tolerability and pharmacokinetic study 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 dose-limiting safety findings. Notably there were no 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.
"With the absence of dose-limiting adverse effects in healthy adults, we are anticipating a similar result in cystic fibrosis patients," said Dr. Wheeler. "Combined with the strong scientific rationale for a meaningful effect on pulmonary function, we are eager to move the program into the CF patient population in Phase 2."
The Phase 2 study will be an innovative, multinational, 28-day, adaptive design in up to 80 CF patients. This adaptive design allows for testing of the efficacy, tolerability and safety of SPX-101, as well as effects on quality of life, at multiple SPX-101 dose levels and will study patients regardless of their genetic mutation.
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 BiosciencesSpyryx 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.
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SOURCE Spyryx Biosciences
