Approximately 75,000 patients live with Cystic Fibrosis (CF) worldwide, a devastating progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. Thanks to improvements in diagnosis and patient care there has been a significant increase in survival rates the last 2-3 decades. However, there is still no cure, and the median age of survival is approximately 37 years of age.
The hallmark of CF lungs is that they excrete thick and sticky mucus, which clog the airways and make it difficult to breathe. The genetic mutation that result in CF cause a structural change in a regulatory protein in the lung, called SPLUNC1. This makes the epithelial sodium channel hyperabsorbative, upsetting the fluid regulation in the lungs and drying out the mucus.
The North Carolina-based startup Spyryx Biosciences is developing novel therapeutics for severe lung diseases with a special focus on CF. The startup is based on research by Dr Robert Tarran at UNC-Chapel Hill, who discovered a peptide derived from the SPLUNC1 protein that may stop the epithelial sodium channel from getting overactivated, normalizing the excretion of mucus in the lungs. Their lead clinical candidate, SPX-101, is an inhaled peptide replacement therapy that is currently completing Phase 1 safety study. The product has demonstrated robust results in preclinical studies by dramatically increasing survival in animal models, and could improve lung function in all CF patients.
There has been significant progress made in developing treatments for CF in recent years, but the most effective drug currently on the market only works in a small subset of patients. And although the drug demonstrates how impactful precision medicine can be, it highlights the huge opportunity of Spyryx and their drug candidate that could be effective regardless of mutation. The startup has the potential to transform CF treatment, ultimately improving CF care, quality of life and increasing median age of survival.
In 2015, Spyryx Biosciences announced they had secured $18 million in a Series A venture capital financing from Canaan Partners, Hatteras Venture Partners and 5AM Ventures. The startup has also received an award of an undisclosed amount from Cystic Fibrosis Foundation Therapeutics Inc. to support the development of the peptides.