Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced results from the open-label portion of the six month Phase II Daisy PETAL study (901 study) using its proprietary, orally-active nonpeptide Gonadotropin-Releasing Hormone (GnRH) receptor antagonist, elagolix, in patients with endometriosis.
"The previously announced improvements across all endometriosis-associated pain domains, during the eight week placebo controlled portion of this Daisy PETAL study, continued to show sustained effect through the open-label, single-arm portion of the 901 Study up to twenty-four weeks of treatment," said Chris O'Brien, M.D., Chief Medical Officer at Neurocrine. "We are pleased with the persistence of effect and the safety profile that elagolix continues to display. It is encouraging to see further improvement in dysmenorrhea, non-menstrual pelvic pain and dyspareunia with continued treatment."
Non-Menstrual Pelvic Pain
The previously reported eight week top-line data demonstrated that elagolix is associated with a statistically significant reduction in Non-Menstrual Pelvic Pain daily scores when compared to placebo (ITT population, ANCOVA). This latest week twenty-four data show a further decrease in non-menstrual pelvic pain scores for those subjects who were initially randomized to the 150mg elagolix arm. Additionally, for those subjects who received elagolix 150mg after spending the initial eight weeks in the placebo group, the non-menstrual pelvic pain scores were reduced at Week 24.
Dysmenorrhea
The previously reported eight week top-line data demonstrated that elagolix is associated with a statistically significant reduction in dysmenorrhea daily pain scores when compared to placebo (ITT population, ANCOVA). The new week twenty-four data show a further decrease in dysmenorrhea pain scores for those subjects who were initially randomized to the 150mg elagolix arm. Additionally, for those subjects who received elagolix 150mg after spending the initial eight weeks in the placebo group, the dysmenorrhea pain scores were reduced at Week 24.
Dyspareunia
The previously reported eight week top-line data demonstrated that elagolix is associated with a statistically significant reduction in dyspareunia daily pain scores when compared to placebo (ITT population, ANCOVA). This week twenty-four data show a further decrease in dyspareunia pain scores for those subjects who were initially randomized to the 150mg elagolix arm. Additionally, for subjects who received elagolix 150mg after spending the initial eight weeks in the placebo group, the dyspareunia pain scores were reduced at Week 24.
Additional Endpoints
Utilizing the Patient Global Impression of Change (PGIC); a 1-7 scale where a score of 4 is "no change," 3 is "minimally improved," 2 is "much improved," and 1 is "very much improved;" elagolix showed improvement. At Week 24 the PGIC percentage of subjects scoring "much improved" or "very much improved" was 86% for those subjects on 150mg elagolix for all 24 weeks, and 74% for those subjects who received elagolix 150mg after spending eight weeks in the placebo group.
The Endometriosis Health Profile 5 (EHP-5) assesses the impact of endometriosis symptoms on five domains utilizing a 0-100 scale. The baseline mean score, for the EHP-5 core pain domain, across all subjects was 54. The EHP-5 core pain domain score showed improvement for subjects randomized to elagolix. At Week 24, the EHP-5 pain score decreased by 36 for those subjects on 150mg of elagolix for the entire 24 weeks of treatment, and by 30 for those subjects who received elagolix 150mg after spending eight weeks in the placebo group.
The Composite Pelvic Signs and Symptoms Scale (CPSSS), a 0-15 scale, was assessed at screening, Week 8, and Week 24 (Baseline score of 9.5). At Week 24 the reduction in the overall CPSSS score was a mean reduction of -5.5. During the placebo controlled portion of the study, the reduction from the Baseline score in the CPSSS showed a statistically significant improvement with 150mg of elagolix, -4.5; vs. placebo, -2.2; (p <0.0001, ITT population, ANCOVA), as previously reported.
Safety Profile
In this clinical trial, the elagolix safety profile was consistent with previous trials. The discontinuation rate from the clinical trial due to adverse events was 5.1%. The most common adverse events during the 6 months of treatment were generally mild and transient: headache, nausea or hot flushes were reported by 9.9% of subjects. There were no elagolix treatment-related Serious Adverse Events.
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