Drug Development

AveXis shares soar after it outlines FDA’s support for a looming pivotal study on SMA

AveXis CEO Sean Nolan

AveXis CEO Sean Nolan

Shares of AveXis $AVXS ripped higher after the market close on Tuesday after the gene therapy biotech outlined the FDA’s acceptance of a small, single arm pivotal study for rare cases of spinal muscular atrophy Type 1 (SMA).

According to the Chicago-based biotech, regulators signed off on a 20-patient study that would use a historical comparison for evaluating efficacy. And that lays a straight path to a pivotal study now slated to begin in the first half of a looming 2017.

AveXis shares shot up 25% on the trial plans.

Here’s the summary:

At the Type B meeting and in the meeting minutes, the FDA acknowledged the company’s rationale for a single-arm pivotal study and provided a number of constructive suggestions to help optimize such a trial design. The FDA also indicated its preference for a design with co-primary endpoints consisting of a measure of developmental milestone achievement (such as sitting unassisted) along with a clinically meaningful measure of survival (such as time to an “event” as described above). Based on FDA’s suggestions as well as other expert input, AveXis continues to evaluate a number of the details of the trial design. More specific information will be made available at the time the study is initiated, which is expected in the first half of 2017.

“We believe the Type B meeting had a positive tone, with FDA offering a number of constructive suggestions which we believe will better enable implementation of a pivotal study design that is most appropriate for the patients suffering from this devastating disease,” said AveXis CEO Sean Nolan in a statement. “With the feedback needed from the FDA to move forward with our pivotal trial, we plan to proceed as expeditiously as possible to begin the study in the first half of 2017.”

Not only that, the FDA — which appears to be quite accommodating in AveXis’s summary — is also willing to consider an approval on the Phase I study. At least one analyst says these developments could pose a problem for Biogen and Ionis, which are partnered on nusinersen for SMA. Notes Jefferies’ Brian Abrahams:

While early data for AVXS’s AVXS-101 has looked promising in SMA, it is hard to compare between treatments and BIIB retains a first mover advantage that could potentially limit uptake of ‘101. Additionally, nusinersen is also being explored in studies of Type 2/3 SMA, which represent additional significant market opportunities, and the possibility of broad initial approval across all subtypes remains; in contrast, AVXS is just beginning to explore such additional SMA populations. Systemic administration of AVXS-101 could, however, offer greater convenience, at least in Type 1 pts. We would not rule out the possibility the therapies could be used in the same pts, though pricing could be an issue. We currently model a 2017 nusinersen launch with probability-adjusted revenues potentially reaching $1.5B by 2025.


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RAPS Regulatory Convergence 2017