Repros Therapeutics
Inc.^® RPRX today reported top line results from its Phase 2 study of
vaginally administered Proellex in the treatment of symptomatic fibroids. The
results from the study suggest the 12 mg dose may provide clinical benefit to
women suffering from symptomatic uterine fibroids. The Company plans to
request an end of Phase 2 meeting with the FDA as soon as practicable.
The primary endpoint in the study was change in Pictorial Blood Loss
Assessment (PBAC). Secondary endpoints included change in Uterine Fibroid
Symptom Quality of Life (UFSQOL) and change in fibroid volume by MRI
assessment.
A total of 40 women were enrolled into the study into one of four groups, 3 mg
(n=9), 6 mg (n=9), 12 mg (n=12) and 24 mg (n=10). The following table reports
the data from those subjects that completed the study. Unexpectedly, the 24 mg
dose exhibited lower exposure than the 12 mg dose which, the Company believes,
may result from the hydrophobic nature of the active ingredient and the low
volume of excipient used in order to prevent vaginal leakage. Based on these
findings, the Company plans to only pursue the 12 mg dose in Phase 3. The MRI
report for the 24 mg end of dosing visit is not available at this time but is
not expected to be an improvement on the 12 mg dose. In the table, the larger
the reduction (negative number), the better the result.
Dose (n=subjects Mean % Change in Mean % Change in Mean % Change in Fibroid
completing study) PBAC (StDev) UFSQOL (StDev) Volume (StDev)
3 mg (n=8) -58.3 (40.4) -59.3 (41.35) 1 (17.78)
6 mg (n=7) 56.8 (139.3) -6.47 (72.5) -3.1 (20.04)
12 mg (n=11) -84.6 (24.9) -79.1 (31.72) -28.0 (13.3)
24 mg (n=5) -85.5 (20.4) -65 (78.3) Not available
The 12 mg dose exhibited a statistically significant difference (p<0.05)
compared to the pooled data from the 3 and 6 mg doses for all three measures.
The 6 mg dose group data was affected by a single subject (given the small
number of subjects) that exhibited continuous uterine bleeding. This subject
also recorded the lone SAE probably associated with administration of the drug
following completion of the dosing period. The Company believes this
observation was associated with the low level of exposure of the 6 mg dose as
well as sporadic (poor compliance) administration of the drug in this
individual as exhibited by trough level analysis of the active ingredient at
each clinical visit (every two weeks).
The reduction in fibroid volume was significant for the 12 mg dose as compared
to either the 3 or 6 mg dose group (p=0.002 and p=0.01, respectively). The
magnitude of the change and statistical significance are remarkable given the
small number of subjects enrolled in the study.
58% of the women on the 12 mg dose ceased menstruation whereas 18% of the
combined 3 and 6 mg doses stopped. The Company believes the incomplete
cessation of menses at the 12 mg dose was a result of sporadic (poor
compliance) administration of the drug in some of the subjects which was
confirmed by evidence, on occasion, of no trough levels of active drug in
those individuals that did not cease menstruation.
The Company has put in place an extension study to begin to build the 1 year
exposure safety data base that will be required for an eventual NDA
submission. To date, 15 of the 40 subjects have elected to enroll in the
extension study.
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