-No dose-limiting adverse effects in 14-day multiple ascending dose study
-The Company to receive up to total of $5 million to support Phase 2 trial
DURHAM, N.C., June 20, 2017 – Spyryx Biosciences, Inc., a clinical stage biopharmaceutical company developing innovative therapeutics to address severe lung diseases, announced today that Cystic Fibrosis Foundation Therapeutics (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation, has agreed to increase the funding provided under the previously announced development award. Under the revised agreement, the Company is eligible to receive up to a total of $5 million. These funds will be used in support of Spyryx’s HOPE-1 study, a multinational Phase 2 trial of SPX-101 in people living with CF.
Additionally, Spyryx presented an E-Poster of data from the recent Phase 1 study for SPX-101 at the European Cystic Fibrosis Society (ECFS) Conference in Seville, Spain entitled:
Safety and Pharmacokinetics of 14-Day Dosing with SPX-101 in Healthy Human Subjects
This study established the clinical safety and tolerability of SPX-101 for up to 14 days in healthy adults age 18-50 with no history of respiratory disease and with normal lung function. Each of the 32 participants received one of four doses (10 mg, 30 mg, 60 mg or 120 mg) of SPX-101 or placebo twice-daily via an investigational eFlow nebulizer system (PARI Pharma, Starnberg, Germany) in a multiple ascending dose design. There were no clinically meaningful changes in lung function as measured by forced expiratory volume in one second (FEV1) in any overall dose group. None of the subjects in this study had plasma concentrations above the limits of quantification or an abnormal serum potassium value at any time. There were no reports of bronchoconstriction and no serious adverse events (SAEs) observed in the study.
SPX-101 is an inhaled peptide with a novel biological mechanism for regulating epithelial ion channel density 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 people living with CF.
“The data from the Phase 1 study validate the safety of SPX-101’s novel mechanism of action via the internalization of epithelial sodium channels with a small, inhaled peptide,” said Alistair Wheeler, MD, Chief Medical Officer of Spyryx. “Our ability to deliver the peptide without significant systemic exposure or any reports of hyperkalemia are key to advancing the program into a meaningful efficacy study. We are thrilled that CFFT has generously provided additional funding to support our Phase 2 study. We remain committed to bringing this new potential treatment to people living with CF and look forward to further evaluating this innovative peptide.”
About Cystic Fibrosis
Cystic Fibrosis (CF) is an autosomal recessive genetic disorder affecting approximately 75,000 individuals worldwide. The disease is caused by mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CF profoundly affects the lungs and respiratory tract and is characterized by dehydration of the airway surface, resulting in reduced mucus clearance, the lung’s principle mechanism for maintaining a clean environment. The mucus becomes thick and sticky, accumulating into obstructions that block airflow and result in recurrent colonization of the airways by viruses and bacteria. These pathogens lead to frequent, acute lung infections and chronic inflammation. The long-term result of the disease, is progressive, permanent tissue damage and scarring (fibrosis) in the lung. No cure for CF is known, although several treatments have been approved to address the underlying cause of the disease for some CF mutations. Despite the positive advances, more work is needed to ensure effective therapies are available for everyone living with CF.
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 (CF) that is advancing into Phase 2 in people living with CF. 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 independent of the disease-causing 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.