Results of the safety, stability, and effectiveness of Spyryx Biosciences‘ drug candidate SPX-101 for cystic fibrosis (CF) will be presented at the North American Cystic Fibrosis Conference (NACFC) Thursday through Saturday in Orlando, Florida.
SPX-101 mimics the function of the protein SPLUNC1, which plays an important role in controlling airway surface hydration and mucus clearance, a mechanism impaired in CF patients that results in the accumulation of mucus and in bacterial lung infections. By replacing SPLUNC1 activity, SPX-101 restores mucociliary clearance and removes bacteria and foreign particles from the lungs.
The drug’s mechanism of action is independent of the genetic mutations that cause cystic fibrosis, which is why SPX-101 is considered a potential disease-modifying therapy for all CF patients.
“Patients with cystic fibrosis have difficulty clearing mucus from their lungs, leaving them prone to frequent chest infections, which leads to progressive and irreversible damage to their lungs,” John Taylor, president and CEO of Spyryx, said in a press release.
“SPX-101 represents the first therapeutic opportunity to leverage a natural, biological mechanism that is important to the maintenance of normal mucus clearance that also has the potential to provide meaningful clinical benefit to all CF patients,” he said.
The results will be presented at NACFC 2016 in three posters (numbers 222, 257, and 289) to show that SPX-101:
- can regulate airway surface liquid height in human airway cells and restore tracheal mucus velocity in a large animal model of CF;
- acts independently of the mutation that caused the development of the disease;
- is a safe and tolerable therapy, both as a single dose and multiple dose;
- remains stable under long-term and accelerated manufacturing conditions.
“The data to be presented at this meeting have supported an early entry into the clinic and we are pleased to have completed the single-dose phase of our Phase 1 study without any dose-limiting adverse effects in the lung and with no evidence of significant systemic bioavailability,” said Alistair Wheeler, MD, the company’s chief medical officer.
The Cystic Fibrosis Foundation awarded a development prize to Spyryx in 2015 to support SPX-101 as a therapy for CF.