Path BioAnalytics In-licenses Potential Cystic Fibrosis Therapy

Chapel Hill’s Path BioAnalytics (PBA), a precision-medicine company focused on new treatments for respiratory disease, has licensed rights to a cystic fibrosis drug candidate from a Chinese biopharmaceutical company.

PBA acquired the rights to cavosonstat from Laurel Therapeutics, a global clinical-stage company based in Hangzhou, China. 

PBA said it would use its proprietary technology to identify subpopulations of patients in which the drug candidate is effective. If successful, it will file an Investigational New Drug (IND) application to conduct clinical trials.

Under the terms of the license, PBA has exclusive rights outside Asia to develop cavosonstat for cystic fibrosis.

“At PBA, we have developed an innovative organoid platform using primary cells to support drug development for targeted patient populations,” said John Mellnik, Ph.D., chief executive officer of PBA. “We are excited to be able to apply this technology to cavosonstat and advance it through our pipeline and into the clinic for cystic fibrosis.”

Added Mellnik: “This program advances our strategy of developing novel candidates with proven clinical safety and identifying subpopulations in which they will be most effective.  We believe that we have found an excellent partner in Laurel Therapeutics, a company that shares our focus on addressing the high burden of respiratory diseases world-wide.”

CF is a fatal genetic disorder affecting 70,000+

Cystic fibrosis is a fatal genetic disease affecting more than 70,000 people worldwide. It is caused by genetic mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes the CFTR protein.

Over 2,000 mutations in the CFTR gene with varying degrees of clinical relevance have been identified, according to the company.

Cystic fibrosis affects multiple organ systems in the body, but its impact on the lungs, where it causes frequent lung infections and shortness of breath, is the primary driver of mortality. There is no known cure for the genetic disease.

“We are pleased that PBA has selected cavosonstat for IND-enabling studies,” said Tom Liu, chief executive officer of Laurel Therapeutics. “Precision medicine is often thought of in terms of the doctor-patient relationship and personalizing the care that a patient receives. PBA has taken this several steps further by identifying a target patient population and optimizing the compound specifically for them, even before the compound enters the clinic. We feel this approach will be highly impactful for cystic fibrosis, especially for patients with rare CFTR mutations.”

Cavosonstat is a novel CFTR modulator designed to correct a subset of CFTR mutations by increasing stability of the CFTR protein in the cell membrane through inhibition of S-nitrosoglutathione reductase (GSNOR) and preservation of S-nitrosoglutathione.

Earlier clinical trials didn't target potential beneficiaries

Late-stage clinical trials for cavosonstat in a broad population of cystic fibrosis patients with at least one particular CFTR mutation previously demonstrated safety and tolerability but did not reach statistical significance for efficacy. 

Cavosonstat is the most clinically advanced GSNOR inhibitor in Laurel’s portfolio of GSNOR inhibitors.

PBA couples a personalized 3D cell culture system with in-house clinical datasets from a broad cross-section of individuals to provide a platform for drug development. The company is using its technology to advance internal and external drug programs for a range of diseases.

PBA’s internal development programs are focused on cystic fibrosis, chronic obstructive pulmonary disease and pulmonary fibrosis.

The company said its patient-centric approach to drug development allows it to “de-risk R&D decisions, improve the efficiency of clinical testing, and ultimately produce more effective medications for targeted patient populations.”

PBA was founded in 2014 and has six employees in Chapel Hill and three at a lab in Durham, Mellnik said. The company has been financed primarily through SBIR and STTR grants from the National Institutes of Health.

Laurel Therapeutics is a fully integrated clinical stage biopharmaceutical company focused on respiratory disease. It was founded by Laurel Venture Capital to pursue the development of cavosonstat for chronic obstructive pulmonary disease and asthma. 

Laurel Venture Capital is a life science-focused venture capital firm that invests in early- and mid-stage therapeutic assets of life science companies in China, Europe and the United States.