We currently have three programs underway: two using our proprietary endoxifen (oral and topical formulations) and the other using our patented microcatheter technology.
We are developing both oral and a topical formulation of endoxifen, which are now in a Phase 1 dose-finding clinical study being conducted by a leading Clinical Research Organization in Australia. The study is a placebo-controlled, repeat dose study of 48 healthy female volunteers. The primary end point is to assess the pharmacokinetics of both formulations of endoxifen over 28 days. The secondary endpoint is to assess safety and tolerability. Subject to positive results from this study, we plan to advance to one or more Phase 2 clinical studies in the second half of 2017.
Oral Endoxifen. Endoxifen is the most active metabolite (ingredient) in the FDA-approved drug tamoxifen, which is recommended for breast cancer survivors to prevent recurrence as well as the development of new cancer. Research indicates that low endoxifen levels in breast cancer patients taking oral tamoxifen correlate with a higher risk of recurrence and new cancer as compared to breast cancer patients with adequate endoxifen levels. We believe that up to 50% of the approximately one million patients taking tamoxifen in the United States each year are refractory, meaning that they have inadequate endoxifen levels (for any number of reasons including low levels of a liver enzyme) and they have an increased risk for breast cancer recurrence. Subject to favorable results from our Phase 1 study, we are planning to begin a Phase 2 study of oral endoxifen in the second half of 2017 for these patients who are refractory to tamoxifen.
We are also evaluating endoxifen in the neo-adjuvant setting, meaning it would be use to treat breast cancer before surgery to remove the cancerous tumor.
Topical Endoxifen. The topical formulation is being developed for women with a condition call high breast density, which has been shown in third-party studies to result in a higher risk of developing breast cancer. Subject to favorable results from our Phase 1 study, we are planning to begin a Phase 2 study of topical endoxifen for women with high breast density. The goal of this program is to reduce high breast density, which should result in the lowering of the risk of breast cancer.
Topical endoxifen is also being evaluated for its potential in other breast conditions.
Our key objectives are to advance our programs through Phase 2 trials and then evaluate further development independently or with partners.
Our third program uses our patented microcatheter technology to deliver drugs through the nipple directly to the site of the cancer. The goals of this delivery method are to increase the amount of the drug getting to the targeted area while reducing the side effects caused by delivering the drug through the blood stream.
We believe our patented intraductal microcatheter technology may be useful in delivering a number of drugs directly to the breast tissue. The initial drug we are studying using our microcatheters is fulvestrant. Fulvestrant is FDA-approved for metastatic breast cancer. It is administered as a monthly intramuscular injection of two injections, typically into the buttocks. We own one issued patent and several pending applications directed to the treatment of breast conditions, including cancer, by the intraductal administration of therapeutics, including fulvestrant.
We are currently conducting a Phase 2 study using our microcatheter technology to deliver fulvestrant at Montefiore Medical Center. This trial is a Phase 2 study in women with ductal carcinoma in situ (DCIS) or Stage 1 or 2 breast cancer (invasive ductal carcinoma) scheduled for mastectomy or lumpectomy within 30 to 45 days. This study is assessing the safety, tolerability, cellular activity and distribution of fulvestrant when delivered directly into breast milk ducts of these patients compared to those who receive the same drug by injection. Of the 30 patients required for full enrollment, six will receive the standard intramuscular injection of fulvestrant and 24 will receive fulvestrant with our microcatheter device.
The primary endpoint of the clinical trial is to compare the safety, tolerability and distribution of fulvestrant between the two routes of administration (intramuscular injection or through our microcatheters). The secondary endpoint of the study is to determine if there are changes in the expression of Ki67 as well as estrogen and progesterone receptors between a pre-fulvestrant biopsy and post-fulvestrant surgical specimens. Digital breast imaging before and after drug administration in both groups will also be performed to determine the effect of fulvestrant on any lesions as well as breast density of the participant.