On August 14, 2017 GTx, Inc. (Nasdaq: GTXI) reported financial results for the second quarter of 2017 and highlighted recent accomplishments and upcoming milestones (Press release, GTx, AUG 14, 2017, View Source [SID1234520208]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

During the quarter, GTx announced positive preliminary clinical data from an ongoing, open-label, Phase 2 proof-of-concept clinical trial of enobosarm in postmenopausal women with stress urinary incontinence (SUI). The Company also has continued to advance two additional R&D programs which are outlined below.

"We are impressed with the positive results in the first seven patients dosed in the SUI study. Stress urinary incontinence is a serious and often embarrassing condition which can have a significant negative impact on a patient’s quality of life. We are focused on potentially addressing this prevalent condition with a first-in-class, orally-administered therapy," said Robert J. Wills, Ph.D., Executive Chairman of GTx.

Corporate Highlights and Anticipated Milestones

Enobosarm in Stress Urinary Incontinence: The Company has an ongoing Phase 2 proof-of-concept clinical trial of enobosarm 3 mg in postmenopausal women with SUI. This open-label, non-placebo controlled proof-of-concept clinical trial is the first of its kind to evaluate an orally-administered selective androgen receptor modulator (SARM) for SUI.

In June 2017, the Company announced preliminary results from the first seven women in the ongoing trial, in which all of the women treated with enobosarm showed a clinically significant reduction (50 percent or greater) in incontinence episodes per day, as measured by mean stress leaks from baseline to week 12 of the trial.
Mean stress leaks decreased by 80.9 percent overall; stress leaks dropped from 5.7 to 1.1 leaks per day.
These results, as well as data from additional patients, will be presented at the International Continence Society (ICS) annual meeting in September 2017. The results from the first seven patients can be found here.
Encouraged by the results to date from its SUI proof-of-concept clinical trial, the Company is planning to initiate in the second half of 2017 a randomized, double-blinded Phase 2 clinical trial to assess the efficacy and safety of two doses of enobosarm (3 mg and 1 mg) per day against placebo with the expectation that top-line data from this trial will be available by the end of 2018.
Enobosarm in Breast Cancer:

The Company has an ongoing Phase 2 clinical trial of enobosarm in estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer.

The Company reported in November 2016 that the pre-specified threshold for success of the trial was already attained in the 9 mg cohort, with nine patients achieving a clinical benefit response (CBR) at 24 weeks among the first 22 evaluable patients in that cohort.
CBR is defined as a complete response, partial response or stable disease, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) at 24 weeks of treatment.
The Phase 2 clinical trial is fully enrolled with at least 44 evaluable patients enrolled in each of the 9 mg and 18 mg cohorts of the trial, and the Company expects to report top-line results in the third quarter of 2017.
The independent Safety Monitoring Committee established to monitor the safety of this clinical trial met on August 11, 2017, and recommended that the trial continue as planned.
The Company’s Phase 2 clinical trial of enobosarm in AR+ triple negative breast cancer completed the first stage of the two stage trial. Since there was insufficient CBR demonstrated among patients enrolled in the first stage of the trial, the Company has determined that the second and final stage of the trial will not continue.

One patient who achieved stable disease remains on study for approximately 10 months from the initiation of treatment.
Enobosarm in Duchenne muscular dystrophy (DMD): The Company has conducted preclinical studies evaluating SARMs in DMD. In preclinical models of DMD, GTx SARMs have increased body weight, lean mass and physical function.

The Company is pursuing a potential strategic collaboration with biopharma companies experienced in orphan drug development to continue the development of a SARM for the treatment of DMD.
SARDs in Prostate Cancer: The Company has a Selective Androgen Receptor Degrader (SARD) preclinical program to evaluate its novel SARD technology in castration-resistant prostate cancer (CRPC).

The Company has ongoing preclinical studies to select the most appropriate compound to advance into a first-in-human clinical trial.
Second Quarter 2017 Financial Results

As of June 30, 2017, cash and short-term investments were $11.4 million compared to $21.9 million at December 31, 2016. On August 10, 2017, the Company entered into a loan agreement (the "Loan Agreement") with J.R. Hyde, III, the largest shareholder of the Company and its Lead Director of its Board of Directors, and The Pyramid Peak Foundation, another large shareholder of the Company, (the "Lenders") for the Lenders to make available to the Company from time to time during the term of the Loan Agreement up to $15 million at an interest rate of 8% per annum (the "Loan"). The term of the Loan Agreement is for a period of nine (9) months and is unsecured. The Lenders have agreed that they will participate in any "qualified" financing (defined as the sale of equity by the Company during the term of the Loan Agreement which equals or exceeds $15 million) at least to the extent of any outstanding indebtedness under the Loan.
Research and development expenses for the quarter ended June 30, 2017 were $4.4 million compared to $4.1 million for the same period of 2016.
General and administrative expenses were $2.0 million for both the quarter ended June 30, 2017 and June 30, 2016.
Net loss for the quarter ended June 30, 2017 was $6.4 million compared to a net loss of $6.1 million for the same period in 2016.
Net loss for the six months ended June 30, 2017 was $12.7 million compared to a net loss of $3.9 million for the same period in 2016. The six months ended June 30, 2016 included a non-cash gain of $8.2 million due to the change in fair value of the Company’s warrant liability, recorded in the first quarter of 2016. During the first quarter of 2016, the Company modified its outstanding warrants with no further adjustment to the fair value of these warrants being required.
GTx had approximately 16 million shares of common stock outstanding as of June 30, 2017. Additionally, there remain warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share.