“Obviously, we had hoped for a different outcome" says Peter Nordkild, CEO of Asarina Pharma. “To all women living with PMDD I can only express my shared disappointment about the study outcome. Maybe more than most we understand the enormity of the need for a treatment for PMDD, but also the incredibly complex symptomatology. We hope that our research around allopregnanolone will play an important part in the collective understanding of PMDD, and that this study has been valuable in raising awareness of and attention to this devastating condition.”
Asarina Pharma Chief Medical Officer Märta Segerdahl: “It is comforting to see that Sepranolone remains safe and well tolerated. We know that levels of allopregnanolone are elevated in the brain in both menstrual migraine and Tourette patients, and that Sepranolone is the body’s endogenous compound that inhibits the effect of allopregnanolone. We remain optimistic that Sepranolone may be developed as an effective treatment for Tourette syndrome and menstrual migraine.”
ABOUT THE STUDY
The phase IIb study was a randomized, double-blind, placebo-controlled study taking place in 14 study centers in Sweden, the UK, Poland and Germany. 206 patients, average age 33 years, were randomized after completing the screening. 547 menstrual cycles were evaluated, the average menstrual cycle length in baseline cycles was 28.1 days with 90 percent of cycles having less than ±2 days cycle length difference. Patients administered their own 0.4 mL injections using a prefilled single-use syringe. Two doses were administered, 10 and 16 mg per dose. Treatment began 14 days prior to estimated start of next menstruation, running every second day during the luteal phase up until the beginning of menstruation, with a maximum 7 doses per cycle. Patients filled in a daily rating of severity of problems, DRSP, of 11 PMDD symptoms.
The primary endpoint of the study was calculated from the late luteal phase total symptom score (LmaxSum21), as the difference between the average scores of two baseline cycles and the average score in the treatment cycles. The reduction in these scores was compared between actively treated and placebo treated patients. The average baseline score was 85 points, corresponding to patients having moderate to severe PMDD symptoms. Study treatment reduced the effect score by 27.9 points in the placebo group, compared to 30.3 in the active Sepranolone group, resulting in minimal to mild PMDD symptoms during treatment. The high placebo response may reflect the effect for some study patients of receiving positive attention of their disease having been unrecognized for many years.
In all, more than 2,000 doses of Sepranolone were taken during the study. One out of five patients reported injection site discomfort, no one reported severe discomfort. Overall, study data demonstrate that Sepranolone was well-tolerated with no safety signals.
In summary, Asarina Pharma’s Phase IIb PMDD study did not meet its primary or secondary endpoints. However, Sepranolone treatment did show numerical improvement in all symptoms compared with placebo though these differences did not reach statistical significance.
ASARINA PHARMA GOING FORWARD
Asarina Pharma will continue to develop Sepranolone for menstrual migraine and Tourette syndrome in their respective fully funded Phase II trials. The menstrual migraine study is presently 80% recruited, and despite Coronavirus slowdown restrictions the company remains optimistic about delivering topline results in Spring 2021.
A webcast with the possibility to ask questions on the results is scheduled at 09.30 CET on April 22: