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AveXis Reports on Rett Gene Therapy Program: AVXS-201

February 28, 2018

AveXis Reports on the Rett Syndrome Gene Therapy Program – Clinical Trial Likely to Start Within a Year

In June of 2017 AveXis announced that based on encouraging data generated by RSRT’s Gene Therapy Consortium it would advance the lead MECP2 gene therapy candidate toward clinical trials.

Yesterday the company provided an update on its Rett Syndrome program during a conference call to report their 2017 financial results, recent corporate accomplishments and upcoming milestones.

We encourage you to listen to the conference call, which is interesting and encouraging. Please note that the Q&A at the end of the call addresses a number of relevant questions raised by analysts about the Rett program. Brian Kaspar, Chief Scientific Officer of AveXis and past member of the Gene Therapy Consortium presented preclinical data with these slides. A press release is also available.

Below are key highlights from the call.

  • AveXis is on-track to meet with the FDA next quarter to present data from the Spinal Muscular Atrophy Type 1 (SMA 1) gene therapy product, AVXS-101. This data package submission (called Biologics License Application) is the last step before the FDA can approve AVXS-101 and make it commercially available.
  • Initiated a Phase 1 trial of AVXS-101 in SMA Type 2 using an intrathecal route of delivery (injection into the spinal fluid). This is the same delivery route that will be used in the Rett Syndrome trial. The first two patients that have been treated with this delivery method have reported no adverse effects.
  • Will be initiating a pivotal trial for AVXS-101 in Europe. Pivotal means that the trial is intended to provide sufficient data for approval by the European FDA equivalent, the EMA. It’s important for all of our European families to know that AveXis has the infrastructure and desire to market their products in Europe as well as the US.
  • Obtained exclusive rights from REGENXBIO to the AAV9 gene therapy delivery system and are expanding their gene therapy program to pursue Rett Syndrome (AVXS-201) and amyotrophic lateral sclerosis (AVXS-301).
  • Preclinical mouse studies using AVXS-201 led to the longest living Rett mouse to date with sustained symptom reduction.
  • Primate studies using AVXS-201 show no signs of adverse effects or over-expression of MECP2.
  • Production of clinical trial grade AVXS-201 using AveXis’ own scalable manufacturing platform is underway.
  • Plan to submit Investigational New Drug (IND) applications for both Rett and ALS, in late 2018/early 2019. An IND is the proposal to the FDA to begin human trials. If the FDA has no substantial issues with the application, a clinical trial may proceed 30 days after the IND submission.

We are delighted with the progress that AveXis has made in advancing the AVXS-201 program. Pending FDA approval it’s likely that a gene therapy clinical trial will start within a year. We will inform the community when details on the trial become available.

We profusely thank all of our Rett families and donors, in the US and beyond, who have played such a critical role in getting us to this point.