GTx Updates Phase 2 Enobosarm Clinical Trial Results in Stress Urinary Incontinence at 2018 AUA Annual Meeting
Updated patient durability results from proof-of-concept trial presented; reductions in incontinence episodes continue to be sustained for up to 7 months following completion of enobosarm treatment
Top-line results from ASTRID, an ongoing Phase 2 placebo-controlled clinical trial, are expected early in the fourth quarter of 2018
“The overall treatment effect of enobosarm in this proof-of-concept trial demonstrates the potential of enobosarm to effectively treat SUI, the most common type of incontinence suffered by women,” said Kenneth M. Peters, M.D., Chairman of Urology, Oakland University William Beaumont School of Medicine, and the principal investigator in the trial. “We now look forward to completing the ASTRID trial, which was designed with the same primary endpoint as the proof-of-concept trial, to determine whether clinically meaningful improvements in SUI are similarly achieved in this almost 500-person double-blinded, placebo-controlled clinical trial.”
Stress Urinary Incontinence Summary of Results
Consistent with previous findings, 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;
Median Medical, Epidemiologic and Social Aspects of Aging (
MESA) scores for SUI decreased from 79.5 percent to 44.5 percent; and
- There were no serious adverse events reported 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.
Dr. Peters provided an update on the reduction in incontinence episodes (a secondary endpoint of the trial), which 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 the 17 patients who have reached seven months, has returned to her baseline level of SUI episodes.
The Company has previously reported positive results from the proof-of-concept clinical trial that demonstrates a reduction in urinary incontinence episodes in a subset of women who had both SUI and urge incontinence (UI) at baseline. In addition, a previous presentation of the results also highlighted statistically significant magnetic resonance imaging results showing increases in several important measurements that support the mechanism of action of enobosarm on the pelvic floor. A more detailed summary of these results can be found here.
About the Phase 2 Proof-of-Concept Clinical Trial
The single-arm, open-label Phase 2 clinical trial is evaluating enobosarm in postmenopausal women with SUI, and is the first clinical trial to evaluate an orally-administered selective androgen receptor modulator (SARM) for SUI. This clinical trial is closed to enrollment; more information about the clinical trial can be found here.
About the Phase 2 ASTRID Clinical Trial
In addition to the Phase 2 proof-of-concept clinical trial being presented at AUA, GTx also has a larger, ongoing, placebo-controlled Phase 2 clinical trial evaluating enobosarm in postmenopausal women with SUI. The study, called ASTRID (Assessing Enobosarm for Stress Urinary Incontinence Disorder), completed enrollment (n=493) recently and is being conducted at over 60 clinical trial centers across the United States. Top-line results are expected early in the fourth quarter of this year. To learn more about the ASTRID clinical trial, click here.
About Enobosarm and SUI
Enobosarm (GTx-024), a selective androgen receptor modulator (SARM), has been evaluated in 25 completed or ongoing clinical trials enrolling over 2,100 subjects, in which approximately 1,500 subjects were treated with enobosarm at doses ranging from 0.1 mg to 100 mg. At all evaluated dose levels, enobosarm was observed to be generally safe and well tolerated. The rationale for evaluating enobosarm as a treatment for SUI is supported by preclinical in vivo data demonstrating increases in pelvic floor muscle mass following treatment with GTx’s SARM compounds, including enobosarm, and the proof-of-concept Phase 2 clinical trial of enobosarm 3 mg for the treatment of postmenopausal women with SUI.
About Stress Urinary Incontinence
Stress urinary incontinence (SUI) refers to the unintentional leakage of urine during activities that increase abdominal pressure such as coughing, sneezing or physical exercise. SUI, the most common type of incontinence suffered by women, affects up to 35 percent of adult women. There are a variety of treatments that are used to treat SUI in women, such as behavioral modification and pelvic floor physical therapy, especially as initial treatment options. As the condition worsens however, bulking agents and surgical procedures are often the most widely used treatments.
Forward-Looking Information is Subject to Risk and Uncertainty
This press release contains forward-looking statements based upon
GTx’s current expectations. Forward-looking statements involve risks and
uncertainties, and include, but are not limited to, statements relating
to GTx’s ongoing clinical development of its selective androgen receptor
modulator (SARM) compounds. GTx’s actual results and the timing of
events could differ materially from those anticipated in such
forward-looking statements as a result of these risks and uncertainties,
which include, without limitation, the risks (i) that the Phase 2
placebo-controlled clinical study being conducted by GTx for the
treatment of stress urinary incontinence (SUI) may not be completed on
schedule; (ii) that additional clinical development of GTx’s SARM
compound for the treatment of SUI will be required beyond the ongoing
study; and (iii) any future development of SARMs in SUI is contingent on
obtaining sufficient additional capital to permit such development,
which it may be unable to do. In addition, GTx will continue to need
additional funding and may be unable to raise capital when needed, which
would force GTx to delay, reduce or eliminate its product candidate
development programs and potentially cease operations. GTx’s actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of these
risks and uncertainties. You should not place undue reliance on these
forward-looking statements, which apply only as of the date of this
press release. GTx’s quarterly report on Form 10-Q for the period ended
Kimberly Minarovich or Sam Martin, 212-600-1902
Red House Consulting
Denise Powell, 510-703-9491