On March 12, 2018 GTx, Inc. (Nasdaq:GTXI) reported financial results for the fourth quarter and year ended December 31, 2017 and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAR 12, 2018, View Source;p=RssLanding&cat=news&id=2337621 [SID1234524675]).

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"2017 was a transformational year for GTx, marked by positive results from our Phase 2 proof-of-concept clinical trial in post-menopausal women with stress urinary incontinence and the initiation of the ASTRID trial, a Phase 2 placebo-controlled clinical trial of enobosarm for the treatment of SUI," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "We believe that there is a significant opportunity for an effective oral therapy to treat SUI and look forward to results from the ASTRID trial in the second half of 2018."

Clinical Highlights and Anticipated Milestones

Stress Urinary Incontinence (SUI) Phase 2 Proof-of-Concept Trial:

Last week at the Society of Urodynamics, Female Pelvic Medicine, & Urogenital Reconstruction (SUFU) Meeting and at the International Continence Society (ICS) annual meeting in 2017, positive results were presented from the Company’s Phase 2 proof-of-concept (POC) clinical trial of enobosarm 3 mg administered orally in post-menopausal women with SUI. The results, including additional positive results presented at SUFU in a subset of women with both urge and stress incontinence, are summarized as follows:

At the end of the 12-week treatment period, all of the 18 enobosarm-treated women showed a clinically meaningful reduction in stress urinary incontinence episodes per day (the primary endpoint of the trial).

Mean stress leaks decreased by 81 percent from baseline;
Stress leaks decreased from a mean of 5.17 leaks/day at baseline to 1.00 leak/day;
All 18 patients demonstrated clinically meaningful reductions in stress urinary incontinence episodes per day, compared to baseline, of at least 50 percent; and
Median Medical, Epidemiologic and Social Aspects of Aging (MESA) scores for SUI decreased from 79.5 percent to 44.5 percent.
The reduction in incontinence episodes was sustained, or durable, well beyond the 12-week treatment period.

Patients are being followed for up to seven months post-treatment to assess enobosarm’s duration of effect, and to date no patient, including nine patients who have reached seven months, has returned to her baseline level of SUI episodes.
Additional positive results in subset of postmenopausal women suggest dual treatment effect on both urge incontinence and stress urinary incontinence.

While all of the women in the trial had predominant SUI, some also experienced urge incontinence (UI). Eleven of the 18 women completing 12 weeks of treatment were determined to have both SUI and UI at baseline, also known as mixed incontinence.

Mean urge leaks decreased by 68 percent from baseline;
Urge leaks decreased from a mean of 1.41 leaks/day at baseline, to 0.45 leaks/day;
9 of 11 women demonstrated a reduction in their number of UI leaks, compared to baseline, with 8 of 11 demonstrating a clinically meaningful reduction in their UI episodes per day of at least 50 percent; and
Median Medical, Epidemiologic and Social Aspects of Aging (MESA) scores for UI decreased from 56 percent to 22 percent.
Magnetic resonance imaging (MRI) was used to quantitatively measure muscle in the pelvic floor of 17 women at 12 weeks compared to their baseline. The results showed a statistically significant increase in several important measurements and support the mechanism of action of enobosarm on the pelvic floor.

At week 12, mean levator ani muscle thickness increased 1.15 mm (p=0.006) from a baseline measurement of 4.79 mm;
At week 12, mean inner urethral muscle diameter increased 0.7 mm (p=0.002) from a baseline measurement of 10.7 mm; and
At week 12, mean outer urethral muscle diameter increased 0.7 mm (p=0.0003) from a baseline measurement of 15.4 mm.
There were no serious adverse events reported during the trial and reported adverse events were minimal and included headaches, nausea, fatigue, hot flashes, insomnia, muscle weakness and acne. Mild transient elevations in liver enzymes that were within normal limits were observed, except for one patient with levels greater than 1.5 times the upper limit of normal which returned to normal following her 12-week treatment period. Reductions in total cholesterol, LDL-C, HDL-C and triglycerides were also observed.

SUI Phase 2 Placebo-Controlled ASTRID Clinical Trial:

Based on the positive results from the Phase 2 POC trial, the Company initiated a randomized, double-blinded, placebo-controlled, Phase 2 trial to assess the efficacy and safety of enobosarm administered orally in approximately 400 post-menopausal women with SUI compared to placebo. More information about the ASTRID (Assessing Enobosarm for Stress Urinary Incontinence Disorder) trial, which is ongoing, can be found here. Top-line results from the Phase 2 placebo-controlled clinical trial are expected in the second half of 2018.

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 mechanistic preclinical studies designed to select the most appropriate compound to potentially advance into a first-in-human clinical trial.

Fourth Quarter and Year-End 2017 Financial Results

As of December 31, 2017, cash and short-term investments were $43.9 million compared to $21.9 million at December 31, 2016.
Research and development expenses for the quarter ended December 31, 2017 were $6.9 million compared to $4.6 million for the same period of 2016. Research and development expenses for the year ended December 31, 2017 were $21.5 million compared to $17.2 million for the year ended December 31, 2016.
General and administrative expenses for the quarter ended December 31, 2017 were $2.5 million compared to $2.3 million for the same period of 2016. General and administrative expenses for the year ended December 31, 2017 were $9.2 million compared to $8.7 million for the year ended December 31, 2016.
The net loss for the quarter ended December 31, 2017 was $9.3 million compared to a net loss of $6.9 million for the same period in 2016.
The net loss for the year ended December 31, 2017 was $30.4 million compared to a net loss of $17.7 million for the year ended December 31, 2016. The net loss for the year ended December 31, 2016 included a non-cash gain of $8.2 million related to the change in the fair value of the Company’s warrant liability. 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 21.5 million shares of common stock outstanding as of December 31, 2017. Additionally, there are warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share and approximately 3.3 million shares of GTx common stock at an exercise price of $9.02.