Marinus Pharmaceuticals (NASDAQ:MRNS) announces clinical and regulatory updates for its orphan seizure programs in tuberous sclerosis complex (TSC), CDKL5 deficiency disorder (CDD) and PCDH19-related epilepsy (PCDH19-RE).
The company intends to initiate a Phase 2 trial evaluating the safety and tolerability of ganaxolone in TSC in H1 2020. The study will enroll approx. 20-40 patients ages 2 to 65. The primary endpoint is percent change in 28-day primary seizure frequency through the end of the 12-week treatment period relative to the 4-week baseline period.
The European Medicines Agency has granted orphan drug designation for ganaxolone for the treatment of CDD.
Topline data from Phase 3 Marigold study evaluating the use of oral ganaxolone in children and young adults with CDD is expected in Q3 2020.
International site initiation and enrollment is continuing in Phase 3 Violet Study, evaluating oral ganaxolone in children with PCDH19-RE. The study will enroll up to 70 patients between the ages of 1 and 17. The Company remains on-track to report top-line data in 2021.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.