On September 14, 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 preliminary results from its Phase 1 dose escalation study of its proprietary topical Endoxifen (Press release, Atossa Genetics, SEP 14, 2017, View Source [SID1234520512]). All objectives were successfully met:

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Safety: There were no clinically significant safety signals and no clinically significant adverse events in participants receiving topical Endoxifen.

Tolerability: Topical Endoxifen was well tolerated at each dose level and for the dosing duration utilized in the study.

Pharmacokinetics: Topical Endoxifen crossed the skin barrier when applied daily to the breast, as demonstrated by low but measurable Endoxifen blood levels detected in a dose-dependent fashion.
These data demonstrate the suitability of topical endoxifen for further clinical development.

Atossa expects to announce results from the oral arm of the Phase 1 study in the next 30-60 days.

The Phase 1 Study

The Phase 1 study was a double-blind, placebo-controlled, repeat dose study of 48 healthy female subjects. Atossa assessed safety, tolerability and the pharmacokinetics of proprietary formulations of both topical and oral Endoxifen dosage forms in varying dose levels over 28 days. The study was conducted in two parts based on route of administration. Results from the oral arm of the study are expected in the next 30 to 60 days.

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.

Topical Endoxifen. A condition called breast density (or, MBD), typically diagnosed by a mammogram, has been shown to be an independent breast cancer risk factor. To date, 30 states require that findings of MBD be directly communicated to the patient. We believe a topical form of Endoxifen could potentially reduce MBD. Although oral tamoxifen has been shown to reduce MBD, the benefit-cost ratio is 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. Therefore we expect our next study to focus on the potential for Atossa’s topical Endoxifen to reduce MBD.

Oral Endoxifen. Although approximately one million breast cancer survivors take tamoxifen annually, up to half of them do not benefit from tamoxifen, meaning they are "refractory," for a number of reasons including that they do not properly metabolize tamoxifen. 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. Thus providing oral Endoxifen directly to the patient without having to be metabolized 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 not benefiting from 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.

Next Steps

"Based on these positive preliminary results, we are advancing our topical Endoxifen into Phase 2 studies," commented Dr. Steven C. Quay, CEO and President. "We look forward to announcing the results from the oral arm of our Phase 1 study in the coming 30 to 60 days," continued Dr. Quay.

Breast Cancer Statistics

The American Cancer Society (ACS) estimates that approximately 250,000 women will be diagnosed with breast cancer in the United States this year and that approximately 40,000 will die from the disease. It is the second leading cause of cancer death in American women. Although about 100 times less common than women, breast cancer also affects men. The ACS estimates that the lifetime risk of men getting breast cancer is about 1 in 1,000; 2,470 new cases of invasive breast cancer will be diagnosed; and 460 men will die from breast cancer in 2017.