Abionyx hails success of bid to resurrect failed cholesterol drug—without dishing data to back up claims

Abionyx Pharma has hailed the success of a phase 2a clinical trial designed to resurrect a cholesterol drug that first flamed out almost a decade ago. But with the French biotech yet to share numbers from the study, it is unclear whether the mimic of HDL cholesterol has a future in the treatment of septic patients.

Cerenis Therapeutics originally developed the drug candidate, CER-001, as a treatment for cardiovascular diseases. The wheels came off between 2014 and 2018 when the prospect failed to move the needle in phase 2 and 3 trials of patients with acute coronary disease, post-acute coronary syndrome and genetic HDL deficiency. Cerenis rebranded as Abionyx in 2019 and shifted its focus to other indications. 

A phase 2a clinical trial in septic patients at high risk of developing acute kidney injury is an early test of the pivot. Abionyx said CER-001 cleared the test—but the data-devoid statement published by the biotech makes it impossible for outsiders to fully gauge how the candidate performed in the trial.

According to Abionyx, the study met its primary objective. The EU clinical trial registry lists the co-primary endpoints of the trial as determination of the optimal dose and the onset and severity of acute kidney injury. The study’s lead investigator noted the “trend observed in reducing renal damage” in a statement to disclose the results. 

Abionyx also noted “positive results” on secondary endpoints. The biotech reported a “significant effect” on endotoxin removal and “consequent reduction in the inflammatory cascade,” plus a “significant protective effect … on endothelial functionality” and trends toward fewer ICU days, lower requirement for organ support and improved 30-day survival. 

Safety was reportedly in line with the results of a COVID-19 trial that read out last year. Abionyx is yet to say when it will present the full data set but did outline plans to discuss the results with regulators, first in Europe and later in the U.S., “to design an appropriate clinical and regulatory development strategy for this disease state.”

Abionyx will need to secure funding for further development. The biotech ended September with just 3.8 million euros ($4.1 million) to its name. Shares in Abionyx rose around 15% in early trading in Paris, climbing to 2.22 euros per share.