A phase I, placebo controlled, dose escalation safety and pharmacokinetic study of (Z)-Endoxifen in healthy female volunteers.

This double-blinded study consisted of two parts:

1) A three-arm placebo controlled study with our proprietary formula of Topical (Z)-Endoxifen amongst 24 volunteers; and

2) A three-arm placebo controlled study with our proprietary Oral (Z)-Endoxifen amongst 24 volunteers, in both single and multiple dose administrations. One woman in the Topical Part (receiving placebo) was dropped due to lack of compliance and replaced.


Parameter Topical (Z)-Endoxifen Oral (Z)-Endoxifen
Safety There were no reported significant adverse safety events There were no clinically significant safety signals and no clinically significant adverse events in participants receiving oral (Z)-Endoxifen
Tolerability Topical (Z)-Endoxifen at the dose levels and for the dosing duration utilized in the study was well tolerated by the study subjects Oral (Z)-Endoxifen was well tolerated at each dose level and for the dosing duration utilized in the study
Pharmacokinetics Higher levels of (Z)-Endoxifen was achieved in the blood of subject who received higher doses of the topical drug Oral (Z)-Endoxifen demonstrated blood levels that have been associated with a therapeutic effect in the adjuvant setting in women with breast cancer


This study is designed to determine if Oral (Z)-Endoxifen “turns’ down” or reduces tumor cell activity in patients with newly diagnosed estrogen receptor positive breast cancer. Participating patients will receive Oral (Z)-Endoxifen for at least 21 days prior to surgery, or during the “Window of Opportunity.” Tissue samples obtained from the initial biopsy and again at surgery will be analyzed and results compared to determine if cancer cell activity is lower following Oral (Z)-Endoxifen administration. If a reduction in tumor cell activity is reduced in at least two of the first eight patients, then the study will be expanded to enroll an additional 17 patients.

This study is being conducted by Dr. Vinod Ganju, Peninsula & South Eastern Haematology & Oncology Group, Franksten, Victoria, Australia.


Interim Results: The open-label study was designed to permit an interim analysis of the Ki-67 change. The requirement was to achieve a meaningful Ki-67 change in at least two of eight patients. Interim results are as follows: All patients (N=6) experienced a significant reduction in Ki-67. A summary of these results includes:

  • Ki-67 was reduced by more than 50% in every patient in the window of opportunity between initial biopsy and surgery, with an overall reduction of 74%.
  • All six patients had a Ki-67 below 25% after treatment. In a paper entitled, “Prognostic value of different cut-off levels of Ki-67 in breast cancer: a systematic review and meta-analysis of 64,196 patients,” Ki-67 was an independent prognostic value for predicting overall survival in ER+ breast cancer patients. Ki-67 levels below 25% were associated with the lowest risk of death in this systematic review and meta-analysis.  
  • Treatment ranged from 16-40 days with an average of 22 days.
  • There were no safety or tolerability issues, including vasomotor symptoms such as hot flashes and night sweats, which are often a tolerability challenge for patients on tamoxifen.


This study was designed to determine the safety and tolerability of AT-301 nasal spray which is being developed for at home use for patients recently diagnosed with COVID-19.

The study enrolled a total of 32 healthy adult subjects into either a single ascending dose group or a multiple ascending dose group each with two different doses.

This study was conducted in Australia.

Blinded Preliminary Results:

  • AT-301 appears safe and well tolerated at single or multiple doses for 14 days.
  • There were no serious adverse events, no discontinuations, and only one subject of the 32 subjects experienced adverse events that were considered moderate in severity. All other adverse events were considered mild.

Interest in the Expanded Access program must be provided to Atossa Therapeutics by a medically qualified physician, by filling out the Expanded Access Request form below:

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    Expanded Access Policy: Purpose & Philosophy

    Atossa Therapeutics is a clinical-stage biopharmaceutical company developing novel, proprietary therapeutics and delivery methods for breast cancer and other breast conditions.

    Atossa Therapeutics’ development resources are focused on conducting clinical studies to fully answer important scientific questions about the potential risks and benefits of the investigational products, and to obtain marketing approval by the FDA and other regulatory health authorities.

    Atossa Therapeutics is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Atossa Therapeutics’ investigational products by contacting the Company.

    The purpose of this policy is to describe the requirements for Expanded Access to Atossa Therapeutics investigational products to patients outside of a clinical study. To read our full expanded access policy please click the button below:

    Expanded Access Policy