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New trials planned for gene therapy product AVXS-101

New trials planned for gene therapy product AVXS-101AveXis announced its plans to expand the clinical development program for the company’s gene therapy candidate, AVXS-101, for the treatment of SMA. In addition to the ongoing pivotal trial in SMA Type 1 (STR1VE) and the ongoing Phase 1 trial in SMA Type 2 (STRONG), the company plans to initiate three studies to further evaluate AVXS-101, including in new SMA patient populations:

STR1VE EU – in SMA Type 1 in Europe

The planned trial is expected to enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy. The trial is designed to evaluate safety and efficacy of a one-time IV dose of AVXS-101. AveXis incorporated scientific advice from the European Medicines Agency into the protocol design, and expects to initiate the trial in the first half of 2018.

SPRINT – In pre-Symptomatic SMA Types 1, 2, 3

The planned multi-national trial is expected to enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy. AveXis expects to initiate the trial in the first half of 2018, and will provide more design details at the time of initiation.

REACH – In paediatric “All Comers” with SMA Types 1, 2, 3

The planned multi-national trial is expected to enroll approximately 50 patients between approximately six months and 18 years of age who do not qualify for other AVXS-101 trials at the time of gene therapy. The trial is designed to evaluate a one-time IT dose of AVXS-101. AveXis expects to initiate the trial in late Q4 2018 or early 2019 and will provide more trial design details at the time of initiation.

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