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AveXis Announce Phase 1 Trial of AVXS-101 for SMA Type 2

15 December 2017

The Food and Drug Administration (FDA) has granted permission to AveXis to begin a second Phase 1 trial of their gene therapy drug, AVXS-101, with SMA Type 2 patients in the United States.

AVXS-101, which has “Breakthrough Therapy” status to facilitate the drug approval process (for more information click here), uses harmless, genetically engineered viruses to increase SMN protein levels. A Phase I trial of AVXS-101 with SMA Type 1 patients has recently been completed and showed that the therapy is safe, well tolerated, and able to improve and help sustain muscle function in young infants with the condition (for more information click here).

This new Phase 1 trial, which has been named STRONG, is designed to assess the safety and optimal drug dose of intrathecal injections of AVXS-101 (i.e. the drug will be directly administered into the fluid bathing the spinal cord and brain). The open-label trial will also provide preliminary, proof-of-concept information on the effectiveness of the therapy, and is to be initiated immediately.

Two different doses of AVXS-101 will be tested, and the impact of the drug will be assessed against historical data of SMA Type 2 progression, rather than a placebo control.

The STRONG trial will involve approximately 27 infants and children with SMA Type 2, and will broaden our knowledge of the impact of AVXS-101 across the SMA disease spectrum. To be enrolled, patients are required to have complete SMN1 gene deletion and three copies of the SMN2 gene. Consistent with an SMA Type 2 diagnosis, only those patients able to sit, but not stand, will be eligible.

AVXS-101 will be produced according to the company’s Good Manufacturing Practice (GMP) commercial manufacturing process, which helps to ensure that patients receive a safe and effective product (for more information click here).

This new trial will complement two recently announced Pivotal trials of AveXis that are being conducted in the US and Europe (click here for more information).


Further Information

AveXis Press Release