On September 25, 2017 Atossa Genetics Inc. (NASDAQ:ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions reported that it has contracted with a leading European hospital to conduct a Phase 2 study of its proprietary topical Endoxifen for the treatment of women with mammographic breast density, or MBD (Press release, Atossa Genetics, SEP 25, 2017, View Source [SID1234520621]). Studies by others have shown that a reduction in MBD reduces the risk of developing breast cancer. The Phase 2 study will be conducted at Stockholm South General Hospital in Sweden and will be led by principal investigator Dr. Per Hall, MD, Ph.D., Head of the Department of Medical Epidemiology and Biostatistics at Karolinska Institutet.
“Dr. Hall is widely regarded as a leading researcher in the field of breast cancer and prevention. He is heading the unique KARMA (Karolinska Mammography Project for Risk Prediction of Breast Cancer) Cohort, including over 70,000 women, which is regarded as the best characterized breast cancer cohort in the world and serves as a resource for studies about breast cancer risk assessment and prevention, and the Karisma Intervention Study, which is studying the change in MBD in women taking various doses of oral tamoxifen. We are honored to be working with Dr. Hall and his colleagues on our Phase 2 study of MBD as their unique experience and qualifications are simply unmatched anywhere in the world,” said Dr. Steven C. Quay, CEO and President of Atossa Genetics.
Atossa plans to apply for approval from the Institutional Review Board and Swedish regulatory authority (Medical Products Agency) within the next 30 days. The placebo-controlled, double-blinded study is expected to enroll up to 480 subjects. The primary endpoint is MBD reduction, which will be measured after six and twelve months of dosing, as well as safety and tolerability.
Atossa’s Proprietary Endoxifen
Endoxifen is an active metabolite of tamoxifen. Tamoxifen is an FDA-approved drug to prevent new breast cancer as well as recurrent breast cancer in breast cancer patients. Tamoxifen itself must be broken down by the liver into active compounds (metabolites), of which Endoxifen is the most active. Atossa has completed a comprehensive Phase 1 clinical study using both a topical and an oral formulation of Endoxifen. Preliminary results from the topical arm of the study indicated that the topical formulation was safe, well tolerated and that topical Endoxifen crossed the skin barrier in a dose-dependent fashion.
Topical Endoxifen Opportunity: MBD. Atossa is developing its proprietary topical Endoxifen to reduce MBD, which has been shown in studies conducted by others to be an independent risk factor for developing breast cancer. To date, 30 U.S. states require that findings of MBD be directly communicated to the patient. Although oral tamoxifen has been shown to reduce MBD, the benefit-risk ratio is generally not acceptable to most physicians and their patients. For example, it is estimated that only ~ 2% of women at high-risk of developing breast cancer, including those with MBD, take oral tamoxifen to prevent breast cancer because of the risk of, or actual side-effects of, oral tamoxifen.
Oral Endoxifen Opportunity. Approximately one million breast cancer survivors take oral tamoxifen annually; however, up to half of them do not properly metabolize tamoxifen and do not have desired levels of Endoxifen (meaning they are “refractory”). Low Endoxifen levels in breast cancer patients taking oral tamoxifen are associated with an increased risk of recurrence or the development of new breast tumors. Providing oral Endoxifen directly to the patient without having to be metabolized by the liver may help to address this problem.
Based on the number of women at high-risk of developing breast cancer and the number of patients who have survived breast cancer but are refractory to tamoxifen, Atossa estimates that the potential markets for its proprietary oral and topical formulations of Endoxifen could each potentially exceed $1 billion in annual sales.