Veru Announces FDA Grant of Fast Track Designation for Sabizabulin for the Treatment of Hospitalized COVID-19 Patients at High Risk for Acute Respiratory Distress Syndrome

Veru Inc. recently announced the US FDA has granted Fast Track designation to the Phase 3 registration program for the investigation of sabizabulin, a novel, proprietary, oral cytoskeleton disruptor with both anti-viral and anti-inflammatory properties, to combat COVID-19 infection and the cytokine storm that is responsible for Acute Respiratory Distress Syndrome (ARDS) and death. FDA Fast Track Designation is intended to expedite the development and review of new drugs to treat serious medical conditions that fill unmet medical needs. The global pandemic disease caused by the novel coronavirus SARS-CoV-2 is entering its third year, and there remains an unmet medical need for new effective treatments for hospitalized patients with moderate to severe COVID-19 symptoms at high risk for ARDS and death.

“We are very pleased that the sabizabulin therapeutic for COVID-19 clinical program has received Fast Track designation from the FDA, a distinction that underscores the urgent need for new, novel, effective therapies to be used along with vaccinations to combat this COVID-19 pandemic,” said Mitchell Steiner, MD, Chairman, President and Chief Executive Officer of Veru Inc. “COVID-19 global cases, hospitalizations, and deaths are at the highest levels since the start of the pandemic. Some of the antibody drugs are not effective against the omicron variant. It is clear that an effective and safe oral therapeutic that prevents deaths in hospitalized patients with moderate to severe COVID-19 disease who are at high risk for ARDS is desperately needed. We strongly believe that sabizabulin with its anti-viral and anti-inflammatory properties and a favorable safety profile can be that greatly needed oral therapy for hospitalized COVID-19 patients with serious illness. We look forward to ongoing, productive regulatory interactions with the FDA, which are further enabled with this designation.”

Fast Track designation aims to expedite the development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to fill unmet medical needs. The purpose is to get important new drugs to patients faster. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. Drugs that are granted this designation are given the opportunity for more frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval: more frequent written communication with FDA about such things as the design of the proposed clinical trials and use of biomarker; Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met; and, Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA.

Microtubule trafficking is critical for viruses to be transported, replicated, assembled, and released from the cell. Microtubules also play a role in the inflammatory process including the cytokine release syndrome (cytokine storm). Sabizabulin is a cytoskeleton disruptor which blocks microtubule trafficking and has the potential to treat both the SARS-CoV-2 viral infection and the cytokine storm and septic shock that leads to ARDS and the high COVID-19 mortality rates.

The Phase 3 clinical trial is a double-blind, multicenter, multinational, randomized (2:1), placebo-controlled trial evaluating daily oral doses of 9 mg sabizabulin for up to 21 days versus placebo in 300 hospitalized patients (200 subjects treated with sabizabulin and 100 subjects receive placebo/standard of care) who have SARS-CoV-2 virus infection and who are at high risk for ARDS. Subjects in the sabizabulin and placebo arms will also be allowed to receive standard of care. The primary efficacy endpoint will be the proportion of patients that die on study up to Day 60. Secondary endpoints will include the proportion of patients without respiratory failure, days in ICU, WHO Ordinal Scale for Clinical Improvement change from baseline, days on mechanical ventilation, days in the hospital, and viral load. The study is being conducted in the United States, Brazil, Argentina, Mexico, Colombia and Bulgaria. Clinical results are expected in the first half of calendar year 2022.

Veru is an oncology biopharmaceutical company with a principal focus on developing novel medicines for the management of breast and prostate cancers. The company’s late-stage breast cancer development portfolio comprises enobosarm, a selective androgen receptor targeting agonist, and sabizabulin, a cytoskeleton disruptor.

Current studies on the two drugs include:

• Enrolling Phase 3 ARTEST study of enobosarm in androgen receptor positive, estrogen receptor positive, and human epidermal growth factor receptor two negative (AR+ ER+ HER2-) metastatic breast cancer with AR ≥ 40% (third-line metastatic setting), and which has been granted Fast Track designation by the FDA.
• Planned Q1 2022 Phase 3 ENABLAR-2 study of enobosarm + abemaciclib (a CDK 4/6 inhibitor) in AR+ ER+ HER2- metastatic breast cancer with AR ≥ 40% (second-line metastatic setting).
• Planned Q1 2022 Phase 2b study of sabizabulin in AR+ ER+ HER2- metastatic breast cancer with AR < 40% (third-line metastatic setting).

The company has determined that patients who have ≥ 40% androgen receptor nuclei staining by immunohistochemistry in their breast cancer tissue, a measure of AR expression, are most likely to respond to enobosarm. Consequently, Veru is developing a companion diagnostic to determine a patient’s androgen receptor expression status, and has partnered with Roche/Ventana Diagnostics, a world leader in oncology companion diagnostics, which will develop and, if it is approved, commercialize the companion AR diagnostic.

Veru’s late-stage prostate cancer portfolio comprises sabizabulin, VERU-100, a long-acting GnRH antagonist, and zuclomiphene citrate, an oral nonsteroidal estrogen receptor agonist.

Current studies on these drugs include:

• Enrolling Phase 3 VERACITY and ongoing Phase 2 studies of sabizabulin in metastatic castration and androgen receptor targeting agent resistant prostate cancer prior to IV chemotherapy.
• Enrolling Phase 2 dose-finding study of VERU-100 in advanced hormone-sensitive prostate cancer.
• Planned Phase 2b study of zuclomiphene citrate in men with advanced prostate cancer undergoing androgen deprivation therapy who suffer from hot flashes.

In addition, sabizabulin, which has dual anti-viral and anti-inflammatory effects, is currently enrolling in a Phase 3 study for the treatment of hospitalized COVID-19 patients at high risk for acute respiratory distress syndrome, also known as the cytokine storm.

Veru also has a commercial sexual health division, the proceeds of which help fund its drug development programs, comprised of:

• ENTADFI (finasteride and tadalafil) capsules for oral use, a new treatment for benign prostatic hyperplasia, for which commercialization launch plans are underway.
• FC2 Female Condom (internal condom), for the dual protection against unplanned pregnancy and the transmission of sexually transmitted infections which is sold in the US and globally.