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GNMX: ASCEND Will Enroll To N=64, (consistent with initial trial design)

By Brian Marckx, CFA



Last week Aevi (GNMX) announced that they will go ahead and continue to enroll ASCEND to 64 patients, which was an option within the initial trial design. ASCEND was designed to enroll in sequential stages with the 9-gene cohort (subsequently modified to 8 genes) enrolling first (“part A”) followed by those with no genetic mutation (“part B”). Both parts are randomized 1:1 versus control. The study was designed with anticipated enrollment of up to n=64 in part A and n=42 in part B.

Indicating that after an interim look at the placebo arm, the decision was made to continue to enroll to n=64 in order to “ensure sufficient power to detect a robust treatment effect”. In other words, in order to further reduce the risk of placebo effect. Importantly, this decision was based solely on a statistically-derived estimate of placebo response based solely on the placebo arm (i.e. no inferences relative to effectiveness, or lack thereof, of AEVI-001 should be drawn by the decision to increase enrollment). As a reminder, greater than expected placebo effect in SAGA diluted treatment delta in that study. ASCEND was designed to reduce placebo noise – specifically, it includes only high-responders (i.e. those with mutations to nine specific genes in the mGluR network that showed a clinically and statistically significant response to AEVI-001 in SAGA). In addition, ~75% of ASCEND participants are expected to be ages 6 – 12 - this younger population should further enhance separation as compared to SAGA (ages 12 – 17). We continue to like the chances for success of ASCEND (see link to full report for more commentary on this topic), which is unchanged from our most recent update.

While GNMX’s press release notes that ASCEND has already enrolled more than 42 patients, their timelines have slipped a bit. Previously they had guided for Part A data in mid-2018, which is now updated to topline data in Q4 2018. While the delays have been unfortunate, given the potential opportunity of AEVI-001 in the treatment of a (significant) subset of the ADHD population and depending on the ultimate success of ASCEND, they could prove to be largely meaningless. And if the trial would have otherwise failed but will now succeed with the increase in enrollment, the additional ~4 - 6 month delay is completely immaterial.

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