On October 29, 2015 GTx, Inc. (Nasdaq: GTXI) reported financial results for the quarter and nine months ended September 30, 2015, and highlighted recent accomplishments and developments (Press release, GTx, OCT 29, 2015, View Source;p=RssLanding&cat=news&id=2103981 [SID:1234507837]). The Company’s lead program has patients enrolling in two Phase 2 clinical trials of enobosarm: one in women with estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and another in women with AR+ triple negative breast cancer (TNBC).

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In October, the Company received FDA clearance to initiate a Phase 2 proof-of-concept clinical trial of enobosarm to treat postmenopausal women with stress urinary incontinence (SUI) and plans to initiate the trial in the first quarter of 2016.

"While our primary focus continues to be the development of enobosarm to treat advanced breast cancer, we are expanding the potential utility of our SARM portfolio with the initiation of a proof of concept trial in SUI," said Dr. Robert J. Wills, Executive Chairman of GTx. "Together with our preclinical development in Duchenne muscular dystrophy, we hope to leverage other SARM compounds in our portfolio to create near term value for indications outside of oncology."

Corporate Highlights

Enobosarm, a selective androgen receptor modulator (SARM), is the Company’s lead product candidate and is being developed as a targeted treatment for two advanced breast cancer indications for (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical studies, the primary efficacy objective will be clinical benefit, which is defined as a complete response, partial response or stable disease. Preliminary data from the first stage of both Phase 2 clinical trials are expected by the end of 2016.

Initiated enrollment of an open-label, Phase 2 clinical trial of enobosarm to assess clinical benefit in women with metastatic or locally advanced, ER+/AR+ breast cancer. The study will enroll up to 118 patients to obtain data from 88 evaluable patients to assess the primary efficacy objective of clinical benefit following 24 weeks of treatment.

Initiated enrollment of an open-label, proof-of-concept Phase 2 clinical trial of enobosarm in women with advanced AR+ TNBC. The study will enroll up to 55 patients to obtain data from 41 evaluable patients to assess the primary efficacy objective of clinical benefit following 16 weeks of treatment.

SARMs ability to increase muscle mass may prove beneficial in other non-oncology indications. The Company is exploring SARMs as potential treatments for stress urinary incontinence (SUI) in postmenopausal women where pelvic floor muscle weakness leads to incontinence, as well as Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration and weakness.

The FDA has accepted the investigational new drug (IND) application for a Phase 2 proof-of-concept clinical trial of enobosarm to treat postmenopausal women with SUI. The Company plans to initiate the trial in the first quarter of 2016 and anticipates top-line data by the end of 2016.
The Company’s preclinical studies have continued to confirm beneficial effects from SARMs in mice genetically altered to simulate DMD, compared to control groups. DMD mice were treated with three different SARM compounds, including enobosarm, and each cohort demonstrated increases in body weight, muscle mass, muscle performance (grip strength) and cardiac function compared to control groups. The Company has initiated discussions with potential strategic partners who have proven experience in developing and commercializing therapies for DMD.
Selective Androgen Receptor Degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer, including those who do not respond or are resistant to currently approved therapies. The Company believes that its SARD compounds will degrade multiple forms of the androgen receptor, including AR variants, such as AR-V7.

The Company continues to make progress towards identifying potential clinical SARD candidates with the goal of initiating preclinical studies in the first quarter of 2016.

Third Quarter and Nine Months 2015 Financial Results

As of September 30, 2015, cash and short-term investments were $34.5 million compared to $49.3 million at December 31, 2014.
Research and development expenses for the quarter ended September 30, 2015 were $3.8 million compared to $3.4 million for the same period of 2014.

General and administrative expenses for the quarter ended September 30, 2015 were $2.0 million compared to $1.6 million for the same period of 2014.

The Company recognized a non-cash gain of $40.7 million and $352,000 for the quarter and nine months ended September 30, 2015, respectively, due to the change in fair value of the Company’s warrant liability. The Company classified the warrants issued in its November 2014 private placement as a liability due to certain provisions of the warrants that may require the Company, or its successor, to pay cash to warrant holders under certain circumstances through December 31, 2016. The Company anticipates recognizing non-cash gains or losses resulting from the revaluation of these warrants to fair value each reporting period through the earlier of December 31, 2016 or the exercise in full of these warrants.

Net income for the quarter ended September 30, 2015 was $34.9 million compared to a net loss of $4.9 million for the same period in 2014. Net income for the quarter ended September 30, 2015 included the above mentioned non-cash gain of $40.7 million related to the change in the fair value of the Company’s warrant liability. The net loss for the nine months ended September 30, 2015 was $15.5 million compared to $24.9 million for the same period of 2014. The net loss for the nine months ended September 30, 2015 included a non-cash gain of $352,000 related to the change in fair value of the Company’s warrant liability.

GTx had approximately 140.4 million shares outstanding as of September 30, 2015. Additionally, there remain warrants outstanding to purchase approximately 64.3 million shares of GTx common stock at an exercise price of $0.85 per share.