AveXis Completes Enrollment of Phase 1 Clinical Trial

AveXis, Inc., announced patient enrollment has completed for the Phase 1 clinical trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1.
According to the company’s press release:

The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined by genetic testing. The trial includes two dosing cohorts:

• Cohort 1 includes three patients dosed at (6.7 X10¹³ vg/kg), aged six to seven months at time of dosing;
• Cohort 2 includes 12 patients dosed at (2.0 X10¹⁴ vg/kg), aged one to eight months at time of dosing.

Sean Nolan, President and Chief Executive Officer of AveXis commented:

We are pleased to have reached this important initial clinical milestone in our effort to bring AVXS -101 to patients who suffer from SMA Type 1, a devastating disease for which there are currently no FDA-approved therapies. The data from this initial trial will inform future clinical studies in Type 1 and potentially other types of SMA. We look forward to reviewing the data from this study over the coming year as we continue the development of AVXS-101.

Click here to view the full press release.