On August 6, 2015 GTx, Inc. (Nasdaq: GTXI) reported financial results for the quarter and six months ended June 30, 2015, and highlighted recent accomplishments and developments (Press release, GTx, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076477 [SID:1234507058]). The Company has initiated its Phase 2 clinical trial of enobosarm in androgen receptor positive (AR+) triple negative breast cancer (TNBC) and anticipates initiating this quarter an additional Phase 2 clinical study of enobosarm to treat AR+ and estrogen receptor positive (ER+) breast cancer. The Company also is evaluating several selective androgen receptor modulator (SARM) compounds in preclinical models of Duchenne muscular dystrophy (DMD) where a SARM’s ability to increase muscle mass may prove beneficial to patients suffering from DMD.

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"I am pleased that we have initiated our Phase 2 clinical study of AR+ triple negative breast cancer, and I look forward to having our first patient enrolled this quarter," said Marc S. Hanover, CEO of GTx.

"Our primary focus continues to be developing enobosarm for the treatment of advanced breast cancer and the preclinical development of our newly licensed selective androgen receptor degrader program," said Dr. Robert J. Wills, Executive Chairman of GTx. "In addition, the extensive SARM data from our preclinical and clinical development efforts, together with input from leading experts in areas of muscle disorders, has encouraged us to undertake preclinical studies to determine whether SARMs offer a treatment option for DMD."

Corporate Highlights

Enobosarm, a SARM, is the Company’s lead product candidate and is being developed as a targeted treatment for two advanced breast cancer indications for (i) AR+ TNBC and (ii) ER+ and AR+ breast cancer. For both clinical trials, the primary efficacy objective will be clinical benefit, which is defined as a complete response, partial response or stable disease by Response Evaluation Criteria in Solid Tumors 1.1.

The Company initiated its open-label, proof-of-concept Phase 2 clinical trial of a daily dose of 18 mg of enobosarm in patients with advanced AR+ TNBC. The study will enroll up to 55 patients to obtain 41 evaluable patients for the primary efficacy objective defined as clinical benefit at 16 weeks. There will be two stages of evaluation in the clinical trial with the first stage assessment occurring following 16 weeks of treatment for the first 21 evaluable patients. If at least 2 of the 21 patients achieve clinical benefit, the trial will continue to enroll the second stage of the study.

In the third quarter of 2015, the Company plans to initiate an open-label Phase 2 clinical trial of enobosarm to assess clinical benefit in patients with ER+/AR+ advanced breast cancer. The study will enroll up to 118 patients to obtain 44 evaluable patients in each of two cohorts. One cohort will receive a daily dose of 9 mg of enobosarm and the other cohort a daily dose of 18 mg of enobosarm. There will be two stages of evaluation in the clinical trial with the first stage assessment occurring following 24 weeks of treatment for the first 18 evaluable patients in each of the two cohorts. If at least 3 of the 18 patients achieve clinical benefit in one or both cohorts, the trial will continue through the second stage for that cohort.

The Company is expecting data from the first stage of each Phase 2 clinical trial by the end of 2016.

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 variants, such as ARv-7.

The Company is conducting research in collaboration with the University of Tennessee Health Science Center to select and optimize appropriate drug development candidates to move into the preclinical studies required to support initial clinical trials.
SARM’s ability to increase muscle mass may prove beneficial as a treatment for DMD.

DMD is a rare genetic disorder affecting approximately one in 3,000 boys. DMD is characterized by progressive muscle degeneration and weakness and represents a serious unmet medical need.

The Company is undertaking preclinical studies and has initiated discussions with experts to better understand the potential of SARMs as a treatment for DMD.

GTx-758 (Capesaris) for the treatment of advanced prostate cancer.

All patients in the Company’s open-label Phase 2 clinical trial of GTx-758 in men with metastatic and high risk non-metastatic CRPC have reached the primary endpoint assessment. Both the 125 mg and 250 mg doses have demonstrated dose dependent increases from baseline in sex hormone binding globulin (SHBG), reductions in free testosterone and reductions in prostate specific antigen (PSA), confirming the mechanism of action of GTx-758. Efficacy and safety data from the clinical trial will be presented at an appropriate scientific meeting and submitted for publication.

The Company is discussing this data with potential strategic partners to determine their interest in partnering or acquiring this asset, as well as the library of ER alpha agonist compounds.

Second Quarter and Six Months 2015 Financial Results

As of June 30, 2015, cash and short-term investments were $39.4 million compared to $49.3 million at December 31, 2014.
Loss from operations for the quarter ended June 30, 2015 was $5.0 million compared to $10.9 million for the same period of 2014. Loss from operations for the six months ended June 30, 2015 was $10.0 million compared to $19.9 million for the same period of 2014.

Research and development expenses for the quarter ended June 30, 2015 were $3.0 million compared to $7.9 million for the same period of 2014.

General and administrative expenses for the quarter ended June 30, 2015 were $2.0 million compared to $3.1 million for the same period of 2014.

The Company recognized a non-cash loss of $43.0 million and $40.4 million for the quarter and six months ended June 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.

The net loss for the quarter ended June 30, 2015 was $48.0 million compared to a net loss of $10.9 million for the same period in 2014. The net loss for the quarter ended June 30, 2015 included the above mentioned non-cash loss of $43.0 million related to the change in the fair value of the Company’s warrant liability. The net loss for the six months ended June 30, 2015 was $50.3 million compared to $19.9 million for the same period of 2014. The net loss for the six months ended June 30, 2015 included a non-cash loss of $40.4 million related to the change in fair value of the Company’s warrant liability.

GTx had approximately 140.4 million shares outstanding as of June 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.