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, Inna Demender 2, Shai Rubnov2, Uri Danon2, Ehud Marom2, Ron Milo3,4, Ronit Gilad5, Boaz Weller6, Adi Vaknin-Dembinsky7, Mark Hellmann8, Arnon Karni9,10
1Shamir (Assaf Harofeh) Medical Center, 2Mapi Pharma Ltd., 3Barzilai Medical Center, 4Ben-Gurion University of the Negev, 5Kaplan Medical Center, 6Bnei Zion Medical Center, 7Hadassah Medical Center, 8Rabin Medical Center, Belinson Hospital, 9Ichilov Medical Center, 10Tel Aviv University
Objective:
To assess the safety and efficacy of GA Depot treatment (for up to 19 months) in seven primary progressive MS (PPMS) subjects.
Background:
PPMS is characterized by worsening neurologic function (accumulation of disability) from the onset of symptoms, without early relapses or remissions. GA long-acting injection (GA Depot) consists of extended-release microspheres containing GA, administered intramuscularly (IM) once every 28 days. Results of GA Depot phase IIa for three years 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 years, subjects diagnosed with PPMS with signs of disease progression (rate of ≥ 1 point increase / year in EDSS score) in the year prior to screening, EDSS score at baseline ≥2.0 and ≤ 6.5. Patients are receiving GA Depot IM at dose of 40 mg every 28 days. 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:

AEs mainly included mild injection site reactions. No unexpected AEs were reported.  EDSS score remained stable for all patients and no 12 weeks confirmed disability progression (CDP) was detected. Mean 9HPT and T25FW scores remain stable or slightly improved. MRI analysis (n=5) revealed one new, one active T2 and one new and active T1 lesions for the same patient. Gd-enhancing lesions were noted in two patients.

Conclusions:
These interim results suggest a possible safe and effective therapy with GA Depot for patients with PPMS which encourage us to continue this on-going investigation.