Drug Development

Voyager shares spike on an early glimpse of promise for Parkinson’s gene therapy

Bernard Ravina, Voyager Therapeutics

Bernard Ravina, VP of Clinical Development, Voyager Therapeutics

Shares of Voyager Therapeutics $VYGR took off in after-market trading on Wednesday after the biotech spotlighted signs of success from a small number of patients treated with their gene therapy for Parkinson’s.

This once-off gene treatment is designed to amp up the effectiveness of levodopa, giving patients back better motor control that is lost as the disease progresses, while cutting back on the dosage they have to take to manage the disease.

Voyager’s stock shot up 35% on the enthusiasm for these data.

The key here is that in two doses tested in a single arm study among only 10 patients, the higher dose in particular appeared to have the greatest positive impact.

“At 12 months in Cohort 2, treatment with VY-AADC01 resulted in a 14-point, or 44%, improvement in UPDRS-III off medication, a 9-point, or 55% improvement in UPDRS-III on medication, and a 2.2 hour, or 48%, decrease in diary off-time from baseline,” said Bernard Ravina, vice president of clinical development at Voyager Therapeutics. “Very importantly, these improvements in motor function occurred with a substantial 34% reduction in daily doses of oral levodopa and related medications at six months in Cohort 2 that was maintained at 12 months, a magnitude that we find compelling and consistent with the mechanism of action of VY-AADC01. The dose-escalating portion of this trial continues and we plan to complete Cohort 3 enrollment in early 2017, report 6-month data from this Cohort as well as longer-term data from Cohorts 1 and 2 in mid-2017, and present results from this trial at a medical conference in the first half of next year.”

Voyager CEO Steven Paul

Voyager CEO Steven Paul

All 10 patients had passed the 6-month mark after therapy and 5 of the low-dose group and 3 of the high-dose arm hit a full 12 months.

This is a long way from pivotal data, which will require a larger group of patients covering a longer period of time to test durability. But it does give Voyager CEO Steven Paul the kind of proof-of-concept data needed to demonstrate that the biotech is on the right track, with a better idea of the dose needed to make a positive difference for these patients as he and his team ponder mounting a late-stage study in 2017.

So far, so good.

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