Voyager reports 1b Parkinson's progress

Founded in 2013  lead clinical candidate is the VY-AADC, which is in open-label Phase 1b clinical trials for the treatment of advanced Parkinson's disease. Its preclinical programs comprise VY-SOD101 for a monogenic form of amyotrophic lateral sclerosis; VY-HTT01 for Huntington's disease; VY-FXN01 for Friedreich's ataxia; Tau program, for tauopathies, including Alzheimer's disease, PSP, and FTD; and VY-NAV01 for severe chronic pain.

Market cap about $400m, plenty of cash, no approved drugs and a burn rate of 70m on last report. This news seems to have had a negative SP effect.

[CAMBRIDGE, Mass., Nov. 07, 2018 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced positive longer-term results from the open-label, dose-escalating Phase 1b trial of VY-AADC demonstrating sustained improvements in patients’ motor function. Patients in the two highest dose cohorts (Cohorts 3 and 2) experienced mean improvements in diary on-time without troublesome dyskinesia (good ON time) of 1.7 hours per day at 18 months and 2.7 hours per day at two years, respectively.Having selected a dose for the Phase 2 trial between the two highest dose cohorts from the Phase 1b trial, Voyager performed a combined analysis of the outcomes from the ten patients in Cohorts 2 and 3. This combined analysis demonstrated an increase from baseline in good ON time of 2.4 hours per day at 12 months, the timepoint for the primary endpoint in the Phase 2 trial, and 2.6 hours per day at 18 months, the latest timepoint measured for both cohorts. Of the combined ten patients in Cohorts 2 and 3, seven patients would be eligible for the Phase 2 trial based on limits in severity of dyskinesia and minimum OFF time at baseline. For these seven patients, the Phase 2 trial relevant group, the improvements in good ON time were 2.8 hours at 12 months and 2.5 hours at 18 months. These results were achieved with clinically meaningful and sustained reductions in daily oral levodopa and related medications. ]

https://globenewswire.com/news-release/2018/11/07/1647433/0/en/Voyager-Therapeutics-Announces-Positive-Longer-Term-Data-for-VY-AADC-for-Parkinson-s-Disease.html