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Spyryx Biosciences Presents Data at NACFC on SPX-101 Demonstrating Safety, Stability And Efficacy in Animal Models

Spyryx Biosciences Presents Data at NACFC on SPX-101
Demonstrating Efficacy, Safety, and Stability in Animal Models
Three posters presented at NACFC 2016

DURHAM, N.C. – November 2, 2016. Three posters presented by Spyryx Biosciences at the North
American Cystic Fibrosis Conference provided data that demonstrates the safety, stability and efficacy of
SPX-101 in animal models and supports further clinical development.
Patients with cystic fibrosis (CF) have difficulty clearing mucus from their lungs, leaving them prone to
frequent chest infections, which leads to progressive and irreversible damage. SPX-101 is a SPLUNC1-
derived peptide which restores a natural pathway in the human lung that plays an important role in
regulating airway surface liquid and mucociliary clearance. This pathway is dysfunctional in patients with
CF and is critical for mobilizing mucus to ensure the removal of bacteria and foreign particles out of the

“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.”

The posters that were presented summarized Spyryx’s findings in relation to SPX-101, including:

  • SPX-101 is a potent promoter of ENaC internalization, capable of regulating mucus hydration in
    vitro and in vivo (Abstract #257)
    Spyryx has demonstrated effective delivery of SPX-101 by nebulization, which results in a dose
    dependent improvement in survival and mucus clearance in animal models of CF. In these models,
    operating through a novel mechanism of action that promotes the intracellular internalization of
    epithelial sodium channels, SPX-101 reestablishes the pathway that naturally controls the
    concentration of these channels and facilitates appropriate fluid levels on the airway surface to
    restore mucociliary clearance.
  • The scale-up and productization of SPX-101 for preclinical inhaled toxicology (Abstract #222)
    Spyryx presented findings related to the scale-up and compound characterization of SPX-101,
    including no observed changes in the stability of SPX-101 with regards to pH and that it retained
    purity over 6 months in long-term and accelerated storage conditions. In order to achieve
    toxicology coverage in an inhalation exposure study, SPX-101 was produced under cGMP
    conditions with impressive yields at multi-kilogram batch scale.
  • SPX-101 demonstrates safety as an ENaC-affecting therapy for the treatment of cystic fibrosis
    (Abstract # 289)
    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.”

About Cystic Fibrosis
Cystic fibrosis (CF) is an autosomal recessive genetic disorder affecting approximately 75,000 individuals
worldwide and is caused by mutations in the gene for the cystic fibrosis transmembrane conductance
regulator (CFTR) protein. The condition affects multiple organ systems including the lungs and respiratory
tract, pancreas, intestine, liver and kidneys. However, the highest medical need is in the lungs, where
complications from CF-related lung disease accounts for 80% of all CF deaths.
CF lung disease is hallmarked by dehydration of the fluid volume on the airway surface, resulting in
reduced clearance of mucus, the lung’s principle mechanism for cleaning itself. This mucus becomes thick
and sticky, progressively accumulating into obstructions that are chronically colonized by viruses and
bacteria, leading to frequent lung infections, inflammation and loss of lung function. The long-term result
is permanent tissue damage and scarring (fibrosis) that frequently requires lung transplantation and often
leads to early death. No cure for cystic fibrosis is known, although several treatments have been approved
to address the underlying cause of the disease in some patients. Even with these therapies, the median
age of survival for CF patients is only approximately 37 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 is currently completing a Phase 1 safety study. 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 cystic fibrosis patients independent of their genotype. 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