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Atossa Genetics Announces FDA Approval of Endoxifen for "Expanded Access" as Preoperative Systemic Endocrine Therapy for a U.S. Breast Cancer Patient

SEATTLE, Dec. 03, 2018 (GLOBE NEWSWIRE) -- Atossa Genetics Inc. (NASDAQ: ATOS), a clinical-stage biopharmaceutical company developing novel therapeutics and delivery methods to treat breast cancer and other breast conditions, today announced that the FDA has approved an "expanded access" of Atossa's proprietary oral Endoxifen in the preoperative setting in a U.S. patient awaiting surgery for breast cancer.   

Steven C. Quay, Ph.D., MD, President and CEO commented, "A physician recently contacted Atossa and requested our proprietary oral Endoxifen for a pre-menopausal, estrogen-receptor positive (ER+) breast cancer patient awaiting surgery. In this setting, the recommended preoperative endocrine systemic therapy is typically an aromatase inhibitor and a drug for ovarian suppression. The patient's physician was reluctant to use this preoperative therapy as it typically induces menopause and can have other potentially serious side effects. We worked with the physician to apply to the FDA to provide "expanded access" (formerly known as compassionate use) to this patient under an Investigational New Drug (IND) application. We are very pleased to report that the FDA approved this single-patient study, and the patient is currently receiving our proprietary oral Endoxifen preoperatively."

Quay added, "This is the same clinical setting as our Phase 2 'window of opportunity' study of preoperative systemic oral Endoxifen in breast cancer patients which is open for enrollment in Australia."

Under the FDA Expanded Access IND program, the use of Atossa's proprietary Endoxifen is restricted to this patient only.  

About FDA Expanded Access

Sometimes called "compassionate use," expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Expanded access may be appropriate when all the following apply: patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition; there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; patient enrollment in a clinical trial is not possible; potential patient benefit justifies the potential risks of treatment; providing the investigational medical product will not interfere with investigational trials that could support a medical product's development or marketing approval for the treatment indication.

Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

For more information, see the FDA website: FDA Expanded Use Website

About Atossa's Proprietary Endoxifen in Breast Cancer

Endoxifen is a cytochrome P450 mediated, active metabolite of tamoxifen, an FDA-approved drug to prevent new as well as recurrent disease in breast cancer patients. Tamoxifen itself must be broken down by the liver into active compounds (metabolites), of which Endoxifen is the most active. Studies by others have shown that breast cancer patients with endoxifen levels of 30 nM and above have a lower risk for developing future breast cancer. Up to half of the patients taking tamoxifen do not produce therapeutic levels of endoxifen, frequently because of limited liver metabolism capability.

Studies by others indicate that endoxifen's molecular mechanisms of action are concentration dependent and different than that of other anti-estrogens, including fulvestrant. Pathway analysis of differentially regulated genes revealed substantial differences related to endoxifen concentrations including significant induction of cell cycle arrest and markers of apoptosis following treatment with high, but not low, concentrations of endoxifen.

Many patients taking tamoxifen eventually stop responding, becoming tamoxifen refractory. In a small study by others of oral endoxifen use by tamoxifen refractory patients, endoxifen provided an acceptable safety profile and promising antitumor activity. Another class of drugs called aromatase inhibitors are also frequently used to treat breast cancer; however, they are not FDA-approved for use in pre-menopausal women and can only be given to pre-menopausal women in conjunction with drugs for ovarian suppression/oblation, which can cause cardiovascular and other toxicities. Additionally, 20-30% of those taking aromatase inhibitors may experience factures, bone pain or osteoporosis.

Because of these potential advantages, Atossa is developing oral and topical forms of Endoxifen.

Atossa's Phase 2 Study of Preoperative Systemic Oral Endoxifen

Atossa is currently conducting a Phase 2 study of its oral Endoxifen in Australia in the "window of opportunity" between diagnosis of breast cancer and surgery. "Once a patient is diagnosed with breast cancer, there is a window of time, typically a few weeks, before surgery is performed," commented Dr. Quay. "Our goal with this study is to show that our proprietary oral Endoxifen can modify the cancer activity in ER+ breast cancer during this 'window of opportunity.' Because our Phase 1 study indicated that our oral Endoxifen reaches therapeutic levels within 8 hours and therapeutic steady-state levels in only seven days, we are optimistic we can achieve a valuable treatment effect," added Dr. Quay. 

The Pilot Phase of the study will initially enroll up to eight newly-diagnosed patients with ER+ and HER2 negative (HER2-) stage 1 or 2 invasive breast cancer, requiring mastectomy or lumpectomy. Patients will receive Atossa's proprietary oral Endoxifen for at least 21 days from the time of diagnosis up to the day of surgery. Provided tumor activity reduction is demonstrated in at least two patients, an additional 17 patients will be enrolled for a total of 25. The primary endpoint is to determine if the administration of oral Endoxifen reduces the tumor activity as measured by Ki-67, which is a marker of cellular proliferation. The secondary endpoints are safety and tolerability and assessment of the study drug on expression levels of both estrogen and progesterone receptors. The impact on additional markers of cellular activity will also be explored. The Phase 2 study is being conducted on behalf of Atossa by CPR Pharma Services Pty Ltd., Thebarton, SA, Australia. CPR Pharma recently completed the successful Phase 1 study of Atossa's oral and topical Endoxifen in women.

About Atossa Genetics

Atossa Genetics Inc., is a clinical-stage biopharmaceutical company developing novel therapeutics and delivery methods to treat breast cancer and other breast conditions. For more information, please visit

Forward-Looking Statements

Forward-looking statements in this press release, which Atossa undertakes no obligation to update, are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with any variation between preliminary and final clinical results, actions and inactions by the FDA, the outcome or timing of regulatory approvals needed by Atossa including those needed to commence studies, lower than anticipated rate of patient enrollment, estimated market size of drugs under development, the safety and efficacy of Atossa's products and services, performance of clinical research organizations and investigators, obstacles resulting from proprietary rights held by others with respect to fulvestrant, such as patent rights, potential market sizes for Atossa's drugs under development and other risks detailed from time to time in Atossa's filings with the Securities and Exchange Commission, including without limitation its periodic reports on Form 10-K and 10-Q, each as amended and supplemented from time to time.

Atossa Genetics Company Contact:

Atossa Genetics Inc.
Kyle Guse
CFO and General Counsel
Office: 866 893-4927

Investor Relations Contact:

Scott Gordon
CorProminence LLC
377 Oak Street
Concourse 2
Garden City, NY 11530
Office: (516) 222-2560

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Dec 03, 2018