Atossa Therapeutics Provides Five-Year (Z)-Endoxifen Treatment Update on FDA-Approved "Expanded Access" Program for a U.S. Breast Cancer Patient - Seite 2

The FDA authorized a single-patient study under its Expanded Access or compassionate use program for this woman to receive 4 mg/day oral (Z)-endoxifen for 20 days before surgery.

Surgery. The patient had a unilateral mastectomy. After (Z)-endoxifen neoadjuvant treatment, the Ki-67 expression in the tumor was reduced 50%, from 20% to 10%. Both the relative response, a 50% reduction, and the absolute value at the time of surgery have been found in large trials to be an indicator of favorable long-term outcomes.

Adjuvant treatment. Following surgery, her physician recommended adjuvant therapy but, like many women, she was not a candidate for tamoxifen due to her low liver enzyme (CYP2D6) activity. She also did not want to take drugs to suppress her ovarian function and go into early menopause, which meant aromatase inhibitors were not an option. Given the strong and rapid response to (Z)-endoxifen, it was recommended that she continue taking (Z)-endoxifen. This led to an additional compassionate use authorization by the FDA to allow this patient to continue taking 4 mg/day (Z)-endoxifen in the adjuvant setting.

She has now completed five years of daily (Z)-endoxifen. As of the date of this press release, she has not had a recurrence of breast cancer or a new cancer in the contralateral breast, as assessed by clinical examination and mammography. Additionally, the treatment has been well tolerated, and there were no vasomotor symptoms commonly associated with tamoxifen (for example, night sweats and hot flashes).

Under the FDA Expanded Access IND program, the use of Atossa's proprietary (Z)-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: the 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 does not interfere with investigational trials that could support a medical product's development or marketing approval for the treatment indication; alternative 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; and alternative 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.