AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
Company to expand study of AVXS-101 into additional SMA populations including pre-symptomatic, older pediatric Type 2 and Type 3 SMA patients
First patient dosed in Phase 1 trial of AVXS-101 in SMA Type 2
Chicago, Ill. (January 16, 2018) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the companys initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (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. Additionally, the company announced the first patient has been dosed in the Phase 1 trial of AVXS-101 in SMA Type 2.
focus has always been to serve the SMA community, and our expanded clinical development program is designed to evaluate the impact of AVXS-101 in a broader set of SMA patients, said Dr. Sukumar Nagendran, Chief Medical Officer of AveXis. We believe the year ahead has the potential to be one of significant clinical progress as we continue toward our ultimate goal of bringing AVXS-101 to the patients and families devastated by SMA.
Clinical Development Program Overview of AVXS-101 for the Treatment of SMA
Ongoing Clinical Trials