Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences

Spyryx Biosciences Presents Data and Corporate Update at Two Recent Conferences in Support of Clinical Candidate for Cystic Fibrosis

– Provides corporate update at Needham Healthcare Conference

– Highlights positive data & mechanism of action of SPX-101 at
ECFS Basic Science Conference

DURHAM, N.C., April 5, 2017 – Spyryx Biosciences, Inc., a clinical-stage biopharmaceutical company developing innovative therapeutics to address severe lung diseases, recently presented at both the Needham Healthcare Conference in New York and the European Cystic Fibrosis Society Basic Science Conference in Albufeira, Portugal.  These presentations provided updates and data on Spyryx’s progress in advancing SPX-101, the Company’s pipeline therapeutic treatment for cystic fibrosis (CF).

SPX-101 is an inhaled peptide with a novel mechanism for regulating epithelial sodium channel density in the airway.  The drug is designed to restore a cellular pathway in the lung that modulates 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, which makes it a potential therapy for all CF patients.

Needham Healthcare Conference, New York
John Taylor, President and CEO of Spyryx, provided an update to the financial community on the Company’s clinical activities, study results and anticipated milestones for SPX-101, including successful Phase 1 results and plans for Phase 1b and Phase 2 clinical trials in CF patients to begin by mid-2017.

European Cystic Fibrosis Society’s 14th Basic Science Conference, Albufeira, Portugal
Spyryx’s scientific team presented two abstracts at this meeting, which described the mechanism of action and highlight the potential clinical benefit for SPX-101.

The abstracts presented were:

1.    “ENac Internalization By SPX-101 Is A Novel CF Therapy For All CFTR Mutations”
o   SPX-101 binds selectively to ENaC and promotes intracellular internalization of the alpha, beta, and gamma subunits, reducing the cell membrane density of ENaC and decreasing the amiloride-sensitive current in primary human bronchial epithelial cells (HBEC) from healthy and CF donors.
o   Once-daily intranasal dosing of SPX-101 in transgenic βENaC mice, a mouse model with CF-like lung disease, reduced neutrophil and eosinophil infiltration in broncho-alveolar lavage fluids and increased survival to more than 90%.

2.    “SPX-101 Is A Novel ENaC-Targeted Therapeutic For Cystic Fibrosis That Restores Mucus Transport”
o   SPX-101 significantly increased velocity and directionality of mucus transport in an ex vivo mouse model using transgenic βENaC mice trachea.  Additionally, in a large animal in vivo model of CF lung disease using sheep, the SPX-101 treatment showed a durable, dose responsive restoration of tracheal mucus velocity (TMV).  This effect of SPX-101 lasted at least 8 hours with a single dose and fully restored TMV to baseline levels with the top dose tested.
o   Nebulized SPX-101 was tested in GLP toxicology studies in two species for 28 days.  The testing showed no local or systemic toxicity, including no adverse effects on serum or urinary electrolytes and no observed indication of diuretic or hyperkalemic effects, up to and including the maximum deliverable doses.

“We believe the results shared in these studies, combined with the absence of dose-limiting adverse effects in our toxicology and clinical testing, provide a strong scientific rationale for a meaningful benefit of SPX-101 in treating all CF patients, regardless of their CFTR mutation,” said Alistair Wheeler, MD, Chief Medical Officer of Spyryx.  “The protocols for our pending clinical studies in CF patients have completed review by the clinical community and have been well received. We are excited to have the opportunity to investigate the therapeutic potential of SPX-101 in treating CF-related lung disease.  It is our desire to deliver a powerful therapy to the patients suffering from this devastating disease.”

About 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 hallmarked by dehydration of the fluid volume on the airway surface, resulting in reduced clearance of mucus, the lung’s principle mechanism for maintaining a clean environment. The mucus becomes thick and sticky, progressively accumulating into obstructions that block oxygen exchange and are recurrently colonized by inhaled viruses and bacteria.  These pathogens lead to frequent, acute lung infections, chronic inflammation and impaired lung function. The long-term result of the disease, is progressive, permanent tissue damage and scarring (fibrosis) in the lung.  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 approximately 40 years of age.

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 that has successfully completed a Phase 1 study in healthy volunteers and 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 all 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.

2 thoughts on “Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences”

  1. Thank you so much for all that your organization does for
    all of us who have CF!

    Marion J Rojas

    (rojasmarionj79@gmail.com)

  2. I so value each of the organizations that contributes to
    CF organization!

    Appreciatively,
    Marion J Rojas, Oakland, California 94609

    (rojasmarionj79@gmail.com)

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