News : Latest News, Press Releases & Events

Spyryx Biosciences Marks Rare Disease Day With an Update on Clinical Trials for SPX-101

Spyryx Biosciences Marks Rare Disease Day
With an Update on Clinical Trials for SPX-101

DURHAM, N.C., February 28, 2017 – Spyryx Biosciences, Inc., a clinical stage biopharmaceutical company
developing innovative therapeutics to address severe lung diseases, today announced the details of an
upcoming Phase 1b study to evaluate the safety of SPX-101 in patients with cystic fibrosis (CF) has been
published on clinicaltrials.gov (NCT03056989).

Additionally, as announced earlier this month, Spyryx is also advancing plans for a Phase 2 study of SPX-
101 in CF patients.

SPX-101 is an inhaled SPLUNC1-derived peptide with a novel biological mechanism for regulating epithelial
ion channels 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 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 CF patients.

“This Phase 1b study is an important final step as we move into a full-fledged Phase 2 study with cystic
fibrosis patients,” said Alistair Wheeler, MD, Chief Medical Officer of Spyryx. “Having completed the study
in healthy subjects and found SPX-101 to be safe and well tolerated, we look forward to advancing into
patients and further evaluating this novel and exciting peptide. It is fitting on Rare Disease Day that we
are able to share our progress in bringing a new potential treatment to these patients.”

The announcement comes on the heels of the successful Phase 1 safety, tolerability and pharmacokinetic
study in both single ascending dose (SAD) and multiple ascending dose (MAD) arms. The drug was welltolerated
in all 64 subjects tested, with all subjects completing the study protocol. SPX-101 was
administered in the MAD arm for 14 days. Importantly, SPX-101 showed no dose-limiting safety findings.
Notably there were no adverse effects on lung function, no shifts in serum or urinary electrolytes and no
evidence of hyperkalemia. As expected, the peptide demonstrated very low systemic exposure and rapid
clearance from the circulation.

“With the absence of dose-limiting adverse effects in healthy adults, we are anticipating a similar result
in cystic fibrosis patients,” said Dr. Wheeler. “Combined with the strong scientific rationale for a
meaningful effect on pulmonary function, we are eager to move the program into the CF patient
population in Phase 2.”

The Phase 2 study will be an innovative, multinational, 28-day, adaptive design in up to 80 CF patients.
This adaptive design allows for testing of the efficacy, tolerability and safety of SPX-101, as well as effects
on quality of life, at multiple SPX-101 dose levels and will study patients regardless of their genetic
mutation.
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 acute lung infections, inflammation and loss of lung function. The long-term
result is permanent tissue damage and scarring (fibrosis) that commonly 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. Despite currently available
treatment, the median age of survival for CF patients is still 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 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 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.

CONTACT:
John Gillespie
Medical Dynamics
646-599-8626
jgillespie@rxmedyn.com