The FDA appears to have lost its appetite for an accelerated marketing application that could pave the way to an early OK for Voyager’s lead gene therapy. The company announced after the market closed Wednesday that the agency’s view has shifted dramatically.
After tantalizing investors in July with its claim that FDA officials said in a Type C exchange that they were open to a filing based on the safety and efficacy seen in a Phase II study for VY-AADC in Parkinson’s disease, the new word is that regulators are now viewing the mid-stage study as a necessary preliminary step to a pivotal trial. In an addendum that arrived in late October, the biotech said, the FDA now says that “although the data from the Phase II trial may support the safety and efficacy of VY-AADC and could be considered in the BLA review, the FDA currently considers the Phase II trial as an early phase exploratory study.”
Voyager’s stock $VYGR took a 12% dive in after-market trading.
But it gets worse when you plumb the company’s SEC filing on its latest 10K. The biotech says it decided to skip a formal sit-down with regulators in a Type C meeting after they got the initial written responses on the timeline from the FDA. But along with the note on their current view regarding Phase II’s exploratory status, the company adds:
Consistent with this statement, the FDA further informed us that it has significant concerns regarding the ability of the Phase 2 trial by itself to provide substantial evidence of safety and effectiveness and that the FDA generally requires at least two adequate and well-controlled clinical trials to provide substantial evidence of effectiveness for submission of a marketing application
Their gene therapy is designed to dispatch the AADC gene directly into neurons of the putamen where dopamine receptors are located, enabling the neurons to express the AADC enzyme and convert levodopa into dopamine to better control symptoms of the disease.
In an update on their Phase Ib trial, the biotech separately noted that they picked their Phase II dose after seeing results for the two highest dose cohorts in their study, which included a small group of 10 patients.
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.
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