Path BioAnalytics (PBA), a biotechnology company based in North Carolina, has licensed the rights to further develop cavosonstat (N91115), a targeted therapy for cystic fibrosis (CF), from the Chinese company Laurel Therapeutics.
Cavosonstat is a small molecule designed to increase the stability of the CFTR protein, defects of which cause CF. The CFTR modulator works by blocking the enzyme S-nitrosoglutathione reductase (GSNOR), and preserving the activity of the S-nitrosoglutathione (GSNO) enzyme in cells’ membranes.
This investigational therapy is “the most clinically advanced GSNOR inhibitor in Laurel’s broad portfolio of GSNOR inhibitors,” PBA said in a press release.
Early clinical trials of this new CFTR modulator in patients with at least one F508del mutation demonstrated its safety and tolerability. However, efficacy data failed to meet the pre-established endpoints.
PBA plans to use its technology to help identify those people with CF for whom cavosonstat may be most effective. If this is successful, PBA will file an investigational new drug application with the U.S. Food and Drug Administration. That would allow PBA to further explore the treatment’s impact in these particular CF patients in future clinical trials.
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“At PBA, we have developed an innovative organoid platform using primary cells to support drug development for targeted patient populations,” said John Mellnik, PhD, the CEO of PBA. Organoids are cells grown in dishes, but in a three-dimensional structure that more closely mimics cells in a human body.
“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,” Mellnik added. “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.”
Per the terms of the agreement, PBA will have exclusive rights to develop cavosonstat outside of Asia for CF treatment.
“We are pleased that PBA has selected cavosonstat for IND-enabling studies,” said Tom Liu, CEO 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,” he said.