By Tamer Abouras
There is big news pertaining to Cystic Fibrosis (CF) coming out of the 2016 North American Cystic Fibrosis Conference (NACFC), where three posters presented by Spyryx Biosciences provided data demonstrating the safety, stability and efficacy of SPX-101 in animal models and supporting further clinical development, according to a press release from the company.
SPX-101 is a SPLUNC1-derived peptide which restores a pathway (dysfunctional in patients with CF) in the human lung. It plays a key role in regulating airway surface liquid and mucociliary clearance, especially for patients with CF, who have difficulty clearing mucus from their lungs. This dysfunction causes CF patients to be prone to frequent chest infections, leading to progressive and irreversible damage.
“We are excited to share these positive results with the community of researchers and physicians who are all dedicated to finding breakthrough treatments for cystic fibrosis,” said John Taylor, president and CEO of Spyryx Biosciences. “We believe that SPX-101 represents the first therapeutic opportunity to leverage a natural, biological mechanism that is important to the maintenance of normal mucus clearance, with the potential to provide meaningful clinical benefit to all CF patients, regardless of mutation.”
A few highlights summarizing Spyryx’s findings in relation to SPX-101 include:
· SPX-101 is a potent promoter of ENaC internalization, capable of regulating mucus hydration in vitro and in vivo
· The scale-up and productization of SPX-101 for preclinical inhaled toxicology
· SPX-101 demonstrates safety as an ENaC-affecting therapy for the treatment of cystic fibrosis
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Further summarizing the posters, the release stated that, “In GLP toxicology studies in two species, daily inhalation of SPX-101 at delivered doses up to 19.6 mg/kg/day for 28 days, no SPX-101 related systemic or local respiratory tract toxicity or adverse clinical signs were observed. No SPX-101 related effects on body weights, food consumption, urinalysis, blood chemistry, or circulating electrolytes were observed. Importantly, SPX-101 did not increase serum potassium levels in these studies, demonstrating the potential of inhaled SPX-101 to improve outcomes for CF patients with a favorable safety profile. Given the doses administered, these findings provide adequate safety margins for the proposed clinical development plan.”
“These studies provide important data demonstrating low toxicity and supporting the proposed clinical doses, with no dose-limiting toxicity in the lung or systemically, and with no significant systemic bioavailability,” said Alistair Wheeler, MD, chief medical officer at Spyryx. “We believe these non-clinical data, combined with the exciting results from the single-ascending dose portion of our Phase 1 safety study, and the pending data from the multiple-ascending dose portion of the Phase 1, supports our intention to move forward with early entry into the clinic in our Phase 2 clinical development plan.”
For more information regarding Spyryx Biosciences and SPX-101, visit spyryxbio.com