On March 3, 2016 GTx, Inc. (Nasdaq: GTXI) reported financial results for the fourth quarter and year ended December 31, 2015, and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAR 3, 2016, View Source;p=RssLanding&cat=news&id=2145524 [SID:1234509359]). The Company is currently enrolling patients in three clinical trials: two trials evaluating enobosarm as a potential treatment for women with advanced breast cancer, and another assessing enobosarm as a potential treatment for stress urinary incontinence in postmenopausal women.

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"In 2015, we continued to make important progress across our pipeline assets, and while our primary focus continues to be the development of enobosarm to potentially treat advanced breast cancer and our SARD technology, we have diversified our therapeutic opportunities to include SARMs for stress urinary incontinence and Duchenne muscular dystrophy," said Dr. Robert J. Wills, Executive Chairman of GTx.

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product candidate, a selective androgen receptor modulator (SARM), is being developed as a targeted treatment for two advanced breast cancer indications: (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical trials, the primary efficacy endpoint will be clinical benefit, which is defined as a complete response, partial response or stable disease.

ER+/AR+ breast cancer: currently expect to enroll the first stage of the open-label, Phase 2 clinical trial of enobosarm in women with metastatic or locally advanced, ER+/AR+ breast cancer by mid-year in order to report preliminary results by the end of 2016. While the first stage of the trial will evaluate 18 patients for each of the two dosing arms, 9 mg and 18 mg of enobosarm, the trial is designed to enroll up to 118 patients to obtain data from 44 evaluable patients in each study arm (a total of 88 evaluable patients) to assess the primary efficacy objective of clinical benefit response following 24 weeks of treatment.

AR+ TNBC: currently expect to enroll the first stage of the open-label, proof-of-concept Phase 2 clinical trial of 18 mg of enobosarm in women with advanced AR+ TNBC by mid-year in order to report preliminary results by the end of 2016. While the first stage will include 21 evaluable patients, the trial is designed to enroll up to 55 patients in total in order to obtain data from 41 evaluable patients to assess the primary efficacy objective of clinical benefit response following 16 weeks of treatment.

SARMs in Non-Oncologic Indications: The Company is exploring SARMs as potential treatments for both stress urinary incontinence (SUI) and Duchenne muscular dystrophy (DMD), a rare disease characterized by progressive muscle degeneration and weakness.

SUI: enrolled first patient in a Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm to treat up to 35 postmenopausal women with SUI, the first clinical trial to evaluate a SARM for SUI. Top-line data from the Phase 2 clinical trial is anticipated by the end of 2016.

DMD: 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 one of three different SARM compounds, including enobosarm, and each SARM cohort demonstrated increases in body weight, muscle mass, muscle performance (grip strength) and cardiac function, compared to control groups;
Histologically, in SARM-treated mice, skeletal muscles demonstrated a reduction in necrosis, fibrosis, and centrally nucleated cells, which are accepted markers of skeletal muscle atrophy; and

A SARM also markedly reduced the fibrosis of cardiac muscle, which is relevant since most DMD patients will develop cardiomyopathy as the disease progresses.

SARDs in Prostate Cancer: Selective Androgen Receptor Degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), 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 splice variants, such as AR-V7.

CRPC: lead SARD compounds are currently being evaluated in additional preclinical studies to select the best SARD compounds for continued development, as well as to develop data necessary to initiate first in human clinical trials in 2017.

Fourth Quarter and Year-End 2015 Financial Results

As of December 31, 2015, cash and short-term investments were $29.3 million compared to $49.3 million at December 31, 2014.
Research and development expenses for the quarter ended December 31, 2015 were $3.9 million compared to $3.3 million for the same period of 2014. Research and development expenses for the year ended December 31, 2015 were $13.6 million compared to $20.9 million for the year ended December 31, 2014.

General and administrative expenses for the quarter ended December 31, 2015 were $2.1 million compared to $2.2 million for the same period of 2014. General and administrative expenses for the year ended December 31, 2015 were $8.2 million compared to $9.5 million for the year ended December 31, 2014.

The net loss for the quarter ended December 31, 2015 was $3.2 million compared to a net loss of $14.5 million for the same period in 2014. The net loss for the quarters ended December 31, 2015 and December 31, 2014 included a non-cash gain of $2.7 million and a non-cash loss of $8.8 million, respectively, related to the change in the fair value of the Company’s warrant liability. The net loss for the year ended December 31, 2015 was $18.7 million compared to a net loss of $39.4 million for the year ended December 31, 2014. The net loss for the years ended December 31, 2015 and December 31, 2014 included a non-cash gain of $3.1 million and a non-cash loss of $8.8 million, respectively, related to the change in the fair value of the Company’s warrant liability.

GTx had approximately 140.4 million shares of common stock outstanding as of December 31, 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.