Sabizabulin use tied to 55% relative reduction in deaths in COVID-19 patients

MIAMI: Positive efficacy and safety results have been announced from a planned interim analysis of the double-blind, randomized, placebo-controlled Phase 3 COVID-19 clinical trial evaluating oral sabizabulin 9 mg versus placebo in 150 hospitalized COVID-19 patients at high risk for Acute Respiratory Distress Syndrome (ARDS). The Independent Data Safety Monitoring Committee unanimously recommended that the Phase 3 study be halted early due to efficacy, and they further remarked that no safety concerns were identified.

Sabizabulin is an oral cytoskeleton disruptor that blocks microtubule trafficking. The investigational treatment is expected to provide both antiviral and anti-inflammatory effects, thereby treating both the SARS-CoV-2 infection and the cytokine storm and septic shock that lead to ARDS.

Phase 3 COVID-19 Trial Design

The Phase 3 COVID-19 study is a double-blind, randomized, placebo-controlled Phase 3 clinical trial evaluating oral, once-a-day dosing of sabizabulin 9 mg versus placebo in approximately 210 hospitalized moderate to severe COVID-19 patients (≥WHO 4) who were at high risk for ARDS and death. Patients were randomized in a 2:1 ratio to the sabizabulin treatment group versus placebo. Patients in both treatment groups were allowed to receive standard of care including remdesivir, dexamethasone, anti-IL6 receptor antibodies, and JAK inhibitors. The trial was conducted in the United States, Brazil, Colombia, Argentina, Mexico, and Bulgaria. COVID-19 infections treated in the study included the Delta and Omicron variants. A planned interim analysis was conducted in the first 150 patients randomized into the study. The primary efficacy endpoint was the proportion of patients that died by Day 60.

Clinical Efficacy and Safety Results

The prespecified primary endpoint was death at or before day 60. Sabizabulin treatment resulted in a clinically and statistically meaningful 55% relative reduction in deaths (p=0.0029) in the intent to treat population. Placebo group (n=52) had a 45% mortality rate compared to the sabizabulin-treated group (n=98) which had a 20% mortality rate. The secondary efficacy endpoints are still being analyzed at the time of this release.

Sabizabulin treatment was well tolerated in this patient population with no clinically relevant safety observations in the sabizabulin treated group compared to placebo.

Regulatory Discussions and Planning

The Company plans to meet with FDA to discuss next steps including the submission of an emergency use authorization application. As previously disclosed, the FDA granted Fast Track designation to the sabizabulin COVID-19 clinical program in January 2022, which the Company hopes will help streamline the emergency use authorization process.

The Company has scaled up manufacturing processes to produce commercial drug supply to address anticipated drug needs following potential FDA authorization.

The Company has been in discussions with BARDA and other US government agencies in an effort to secure an advance purchasing agreement of drug product for the U.S.

"This study represents a significant milestone in the global fight against COVID-19 as sabizabulin is the first drug to demonstrate a clinically and statistically meaningful reduction in deaths in hospitalized patients with moderate to severe COVID-19," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru. "We strongly believe that sabizabulin, with its dual anti-viral and anti-inflammatory properties which demonstrated positive efficacy and safety results in the Phase 3 COVID-19 study, can be that greatly needed oral therapy for hospitalized moderate to severe COVID-19 patients," Dr. Steiner continued.

"What makes these findings more relevant is that the pharmacological activity of sabizabulin is independent of COVID-19 variant type. Pending upcoming discussion with FDA, this treatment option may be made available soon so we can be ready for when the next clinically important wave of COVID infections comes," said Gary Barnette, PhD, Chief Scientific Officer of Veru.

"We expect new COVID-19 variant infections and new challenges in the treatment of hospitalized patients, particularly as the country heads into the fall and winter seasonal cycle. With the results of this Phase 3 COVID-19 study, we now have a treatment option for the sickest hospitalized COVID patients," said Alan Skolnick, M.D., Principal Investigator with HD Research, who conducted this Phase 3 COVID-19 study at Memorial Hermann Memorial City Medical Center in Houston TX. "We have battled this pandemic for almost two and a half years now. A 55% reduction in deaths in hospitalized patients is tremendously meaningful to patients, their families, doctors, nurses, hospital staff and the communities they serve," added Dr. Skolnick.