Nowe dane Spx-101 jako potencjalna przyszła terapia dla pacjentów CF

by Marek | 6 czerwca 2017 04:14

New Data Supports SPX-101 as Potential Future Therapy for CF Patients[7]

Spyryx Biosciences[8] presented two new studies supporting the therapeutic potential of investigative treatment SPX-101[9] for all types of cystic fibrosis (CF).

The data was presented at the American Thoracic Society (ATS) 2017 International Conference[10] May 19-24 in Washington, D.C. Results of the studies will support the start of a Phase 2 study with CF patients.

“The strong clinical and preclinical data presented at ATS helps support the potential of SPX-101[9] to safely promote airway hydration and mucociliary clearance,” John Taylor, president and CEO of Spyryx Biosciences, said in a news release[11]. “We will be thoroughly investigating the clinical potential of SPX-101 in our HOPE-1 study, a Phase 2 trial in CF patients, which we anticipate getting underway around the middle of this year.”[7]

SPX-101 is an inhaled medication that substitutes for the action of SPLUNC-1, a protein that plays an important role in controlling airway surface hydration and mucus clearance. In CF, this mechanism does not work properly, leading to the accumulation of mucus which, in turn, favors the growth of bacteria in the lungs.

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 causing CF, which makes SPX-101 a potential disease-modifying therapy for all types of CF.

The company presented two posters at ATS 2017. One, titled “SPX-101 Is a Novel ENaC-Targeted Therapeutic for Cystic Fibrosis that Restores Mucus Transport[12],” showed preclinical evidence of the drug’s mechanism of action. Researchers showed that SPX-101 binds specifically to a protein called ENaC, thereby regulating airway surface liquid height in human airway cells and restoring mucus transport in animal models of CF.

The other poster, titled “Safety and Pharmacokinetics of SPX-101 in Healthy Human Subjects[13],” showed the results of a study in 32 healthy, non-smoking individuals ages 18 to 50 with no history of respiratory disease and a FEV1 (forced expiratory volume in one second; a measure of lung function) of at least 80 percent or above of predicted normal at the study’s start and free of respiratory infection for more than 14 days.

Participants received four doses of SPX-101 (20, 60, 120, or 240 mg) or a placebo.

Results showed that SPX-101 was safe and well-tolerated. The rate of adverse of adverse events decreased with the dose used (66.7 percent in the 20 mg group and 16.7 percent in the 240 mg group), but no cases of serious side effects, such as bronchoconstriction, were reported.

“We believe the results shared in the preclinical studies, combined with the absence of dose-limiting adverse effects in our 28-day toxicology and Phase 1 clinical testing, provide a strong scientific rationale for the investigation of SPX-101 in a randomized controlled clinical trial of all CF patients, regardless of their CFTR mutation,” concluded Alistair Wheeler, MD, Spyryx Biosciences chief medical officer.


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  8. Spyryx Biosciences:
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  12. SPX-101 Is a Novel ENaC-Targeted Therapeutic for Cystic Fibrosis that Restores Mucus Transport:
  13. Safety and Pharmacokinetics of SPX-101 in Healthy Human Subjects:
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