Glatiramer Acetate Depot (Extended-Release) Phase IIa Study in Patients with Primary Progressive Multiple Sclerosis: Safety and Efficacy Snapshot
Shlomo Flechter1, Laura Popper2, Nadav Bleich Kimelman2, Shai Rubnov2, Uri Danon2, Ehud Marom2, Ron Milo3,4, Ronit Gilad5, Boaz Weller6, Adi Vaknin-Dembinsky7, Mark Hellman8, Alla Shifrin9, Arnon Karni10,11
1Sheba Medical Center, 2Mapi Pharma Ltd., 3Barzilai Medical Center, 4Ben-Gurion University of the Negev, 5Kaplan Medical Center, 6Bnai Zion Medical Center, 7Hadassah Medical Center, 8Rabin Medical Center, Belinson Hospital, 9Rambam Medical Center, 10Tel Aviv Sourasky Medical Center, 11Sackler faculty of Medicine, Tel Aviv University
Objective:
 To assess the safety and efficacy of GA Depot treatment (for up to 144 weeks) in 16 primary progressive MS (PPMS) subjects treated with GA Depot.
Background:
PPMS is characterized by worsening neurologic function from the onset of symptoms, without early relapses or remissions. GA Depot consists of extended-release microspheres containing GA, administered intramuscularly (IM) once every 28 days. Results of a 5 year GA Depot phase IIa trial in relapsing remitting MS suggest that GA Depot is safe, tolerable and efficacious. The IM administration route together with the slow release formulation may result in a noted effect on PPMS patients as well.
Design/Methods:
Eligibility criteria included: age 18-65, s PPMS diagnosis with rapid disease progression (rate of ≥ 1 point increase/year on EDSS score) in the year prior to screening, EDSS score of 2.0 - 6.5 at baseline. Safety is assessed by analysis of adverse events, CBC and blood chemistry. Efficacy is assessed by EDSS, 9HPT, T25FW tests, as well as by MRI analysis.
Results:
88.2% of the AEs recorded in the study were mild. Most common AEs included injection site reactions and general weakness. No unexpected AEs were reported.  Two SAEs were reported (one related and one not related to study drug). EDSS score remained stable for all patients and no 12 weeks confirmed disability progression (CDP) was detected. Mean 9HPT score and T25FW remained stable. MRI analysis (compared to baseline) revealed activity which included T2/FLAIR lesions (new or enlarging) in four patients. Three of these patients had also Gd+T1 lesions.
Conclusions:

These interim snapshot data suggest that GA Depot is a safe and effective treatment for patients with PPMS, as demonstrated by stable mean EDSS, mean 9HPT and mean T25FW data, which encourage us to continue this on-going investigation.