On Tuesday, MeiraGTx Holdings plc MGTX launched topline information from its scientific bridging examine of AAV-GAD for Parkinson’s illness, MGT-GAD-025.
MGT-GAD-025 is a 6-month, three-arm, randomized, double-blind, sham-controlled examine utilizing AAV-GAD drug product manufactured by MeiraGTx at its wholly-owned amenities with its business platform course of.
Individuals had idiopathic Parkinson’s illness, a historical past of levodopa responsiveness for a minimum of 12 months, and a UPDRS Half 3 rating of ≥25 factors within the “off” state.
Fourteen topics had been randomized to one in all three teams (excessive dose n=5, low dose n=5, and sham n=4)
The first goal of the examine was to judge the protection and tolerability of AAV-GAD, with exploratory efficacy endpoints together with the imply change from baseline to Week 26 in MDS-UPDRS Half 3 (motor examination) scores within the “off” state and the Parkinson’s Illness Questionnaire (PDQ-39) rating, a key patient-reported high quality of life measure in Parkinson’s illness.
AAV-GAD was protected and effectively tolerated, with no severe antagonistic occasions (SAEs) associated to AAV-GAD remedy.
At Week 26, the high-dose group demonstrated a statistically important 18-point common enchancment from baseline within the UPDRS Half 3 “off” remedy rating (p=0.03), with no important change within the sham or low dose teams.
Vital enhancements from baseline within the disease-specific, patient-reported high quality of life PDQ-39 rating had been demonstrated in each the excessive and low-dose teams with no important change within the sham group at Week 26:
- Within the high-dose AAV-GAD group, the PDQ-39 rating improved by 8 factors from baseline (p=0.02), the low-dose group improved by 6 factors from baseline (p=0.04), whereas the 0.2 level worsening within the sham surgical procedure group was not statistically important.
- A dose-response in PDQ-39 rating was noticed, with 100% of members within the high-dose group, 60% within the low-dose group, and 25% within the sham surgical procedure group reporting an enchancment.
- For the PDQ-39 rating, there was a pattern to significance between the excessive dose and sham surgical procedure teams at six months (n=4 evaluable per group).
Topics who accomplished this trial might enroll in a long-term follow-up examine, by which they are going to be monitored for 5 years after remedy.
Worth Motion: MGTX inventory is up 13.2% at $5.25 ultimately examine Tuesday.
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