Venatorx hits goal in antibiotic phase 3, clearing path to FDA

Venatorx Pharmaceuticals’ mission to start restocking the thinning cupboard of antibiotics has advanced. With its experimental candidate hitting the primary endpoint in phase 3, Venatorx is preparing to file for FDA approval in patients with urinary tract infections by the end of the year.

The phase 3 trial enrolled 661 adults with complicated urinary tract infections. Two-thirds of participants received a combination of the approved beta-lactam antibiotic cefepime and Venatorx’s beta-lactamase inhibitor taniborbactam. The other one-third of participants got meropenem, an established beta-lactam antibiotic. 

In the microbiological intent-to-treat population, bacterial eradication and symptomatic clinical success was seen in 70% of participants on the cefepime-taniborbactam combination, compared to 58% of their peers in the meropenem cohort, causing the trial to meet its primary noninferiority endpoint. Venatorx then performed a prespecified superiority test that showed cefepime-taniborbactam performed better statistically. The superiority persisted out to follow-up visits that happened on Day 28 to Day 35.

“These data demonstrate that cefepime-taniborbactam may represent a significant improvement over the standard of care and could support global health efforts to combat antibiotic-resistant infections,” Venatorx CEO Christopher Burns said in a statement. “Cefepime-taniborbactam, if approved by the FDA, may offer a new treatment option for patients with infections caused by highly resistant bacteria, even those resistant to widely used carbapenem antibiotics.”

Treatment-emergent adverse events (TEAEs) affected 35.5% of people on cefepime-taniborbactam and 29% of their counterparts who received meropenem. Most of the events were mild to moderate, with the rate of serious TEAEs coming in at around 2% in each arm, but the rate of discontinuations because of TEAEs was higher in the cefepime-taniborbactam arm, 3%, than the control group, 0.9%. There was one death in the cefepime-taniborbactam cohort, but it was deemed to be unrelated to the treatment.

Venatorx, which plans to share full results from the trial at an upcoming scientific meeting, thinks the data support a planned filing for approval in the U.S. The submission of a NDA to the FDA is penciled in for the fourth quarter of 2022.

When Venatorx goes to the FDA, it will benefit from the qualified infectious disease product (QIDP) and fast-track designations it has received from the agency. The potential benefits of QIDP include a further five years of exclusivity. If cefepime-taniborbactam is approved, Venatorx will face the challenge of turning the product into a commercial success, a task that has vexed other companies that have brought antibiotics to market in recent years.