AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy

AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world’s first human gene therapy trial for the treatment of infants with spinal muscular atrophy (SMA). The trial (NCT02122952) opened for enrollment in April 2014.

“History in gene transfer therapy was made on May 13th, when an infant was successfully injected with about 400 trillion viral particles containing the SMN transgene without any complications to date. That historical moment was followed by the dosing of two additional infants in the low dose cohort,” commented John A. Carbona, Chief Executive Officer of AveXis.

AveXis also announced ongoing plans to modify the current trial design through the addition of a third cohort at a mid-range dose. This marks a departure from the originally-designed 2-cohort trial. “We are also on track to start our intrathecal trial in the first half of 2015 and very serious thought is being given to opening a trial to treat Type 2 patients in 2015. Right now, we are looking at patient recruitment as a gating issue,” said Allan Kaspar, Chief Scientific Officer of AveXis.

In addition, AveXis is announcing the start of its C.A.N. Program. This new initiative is designed to Contact All Neurologists in an effort to grow awareness of the ongoing and upcoming clinical trials in SMA, in addition to informing neurologists how they can help shape the future of patient recruitment in SMA trials. AveXis will also be reaching out to foundations and patient advocacy groups, both domestically and internationally, to increase the overall awareness of clinical trials.

For further details on the trial, enrollment criteria, eligibility and contact information please see the complete posting.

Cure SMA’s Support of Gene Therapy

In 2010, Cure SMA made an initial grant to Dr. Brian Kaspar to begin study of this approach, followed by a second grant in 2012 to allow the drug to treat older and bigger patients using a different delivery method directly into the central nervous system (CNS).

Using the data generated with our funding on the CNS delivery of the drug, Dr. Kaspar and his team were able to secure a $4 million grant from National Institute of Neurological Disorders and Stroke (NINDS) for additional development.

The technology for both systemic and CNS-delivered gene therapy was then licensed to AveXis in 2013 for clinical testing. The CNS-delivered drug is also advancing toward clinical trials, and we hope both routes of delivery will soon be tested in individuals with SMA.

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