Neurocrine Biosciences (NBIX -3.8%) and Voyager Therapeutics (VYGR -1.5%) announced data from Phase Ib (PD-1101) three-year efficacy and safety study, demonstrating that a one-time treatment with NBIb-1817 (VY-AADC) showed sustained improvement in motor function including greater “On” time and reduction in the amount of medications in patients with Parkinson’s disease.
NBIb-1817 reduced average “Off” time (times when the medication stops working and symptoms recur) by up to -1.91 hours and improved average “On” time without troublesome dyskinesia by up to +2.23 hours after three years across three cohorts.
In addition, 14 out of 15 patients treated with NBIb-1817 continued to show an improvement in disease staging after three years, as assessed by the modified Hoehn & Yahr scale.
These new data, along with two-year data from another open-label Phase Ib trial, PD-1102, were presented at the MDS Virtual Congress.
On the safety front, NBIb-1817 was well-tolerated in both studies, with no study drug-related serious adverse events reported. The most common adverse events reported were headache, hypoesthesia, and musculoskeletal pain (in PD-1101), and upper respiratory tract infection, headache, nausea, and depression (in PD-1102).
The company plans to re-initiate enrollment in registrational RESTORE-1 Trial of NBIb-1817 later this year, after the enrollment was paused amid COVID-19.
NBIb-1817 is an investigational gene therapy product designed to deliver the AADC gene directly in brain cells where it can convert levodopa to dopamine.
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