Veru Announces FDA Grant of Fast Track Designation for Enobosarm for the Treatment of AR+ ER+ HER2- Metastatic Breast Cancer

Veru Inc. recently announced the US FDA has granted Fast Track designation to the Phase 3 registration program for the investigation of enobosarm, a selective androgen receptor targeting agonist, for the treatment of androgen receptor positive, estrogen receptor positive, human epidermal growth factor receptor 2 negative (AR+ER+HER2-) metastatic breast cancer patients who have shown previous disease progression on a nonsteroidal AI, fulvestrant, and CDK 4/6 inhibitor therapy, and who have AR% nuclei staining ≥40% in breast cancer tissue (third-line metastatic setting).

“We are very pleased that enobosarm has received Fast Track designation from the FDA, a distinction that underscores the urgent need for new, novel, targeted therapies for this important patient population suffering from this aggressive disease,” said Mitchell Steiner, MD, Chairman, President and Chief Executive Officer of Veru Inc. “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.

Enobosarm is an oral, first-in-class, new chemical entity, selective androgen receptor agonist that targets the androgen receptor in AR+ ER+ HER2- metastatic breast cancer without unwanted masculinizing side effects. Enobosarm is in clinical development for three indications: (i) a Phase 3 ARTEST clinical study evaluating enobosarm for the treatment of third line metastatic AR+ER+HER2- breast cancer patients whose disease has progressed after treatment with a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor with an androgen receptor nuclei staining of ≥40% which is enrolling; (ii) a planned Phase 3 ENABLAR-2 clinical study evaluating enobosarm + abemaciclib combination therapy as treatment of second line metastatic AR+ER+HER2- breast cancer patients whose breast cancer has progressed after treatment with palbociclib and either a nonsteroidal aromatase inhibitor or fulvestrant combination with an androgen receptor nuclei staining of ≥40%; and (iii) a planned Phase 2 clinical study of enobosarm + sabizabulin combination therapy in metastatic triple negative breast cancer after two systemic chemotherapies.

Breast cancer is the most commonly diagnosed cancer in women with an estimated 284,200 new cases and 44,130 deaths expected for 2021 in the US. It is expected that one in eight women will develop invasive breast cancer in their lifetime. Breast cancer is a heterogenous disease with diverse clinical and molecular characteristics. Estrogen is one of the main drivers of breast cancer proliferation, tumor progression, and metastasis. Consequently, treatments that target the estrogen receptor (ER) have been the mainstay of breast cancer therapy, but unfortunately almost all women will eventually develop resistance to endocrine therapies and alternative treatment approaches will be required including IV chemotherapy.

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)
• 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)
• Planned Q2 2022 Phase 2 study of sabizabulin + enobosarm combination therapy in metastatic triple negative breast cancer after two systemic chemotherapies.

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. We have 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 antiviral 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 our drug development programs. Its two main products are:

• ENTADFI (finasteride and tadalafil) capsules for oral use, a new treatment for benign prostatic hyperplasia
• 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.