Clinical Trials

Date: 2018-01-16

Type of information: update on patient enrollment

phase: 1

Announcement: update on patient enrollment

Company: Avexis (USA - IL)

Product: AVXS-101 (adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene)

Action mechanism:

• gene therapy. AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is the only clinical-stage gene therapy in development for SMA. AVXS-101 is designed to address the monogenetic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.
• AVXS-101 - ChariSMA™ is a recombinant AAV9 in which most of the AAV9’s naturally-occurring components have been removed and replaced with the SMN transgene and other elements such as the CB promoter and the Bovine Growth Hormone polyadenylation signal (BGH pA) added for hydrolytic enzymatic protection and mRNA stability. The viral vector will deliver the SMN transgene into the motor neurons. Protein synthesis from the SMN transgene will produce full-length, functional SMN protein.

Disease: spinal muscular atrophy (SMA) Type 2

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Country: USA

Trial details:

• The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of AVXS-101 in patients with Spinal Muscular Atrophy with 3 copies of SMN2. (NCT03381729)

Latest news: • On January 16, 2018, AveXis provided an overview of the expanded clinical development program for 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. Ongoing Clinical Trials: Phase 1 Trial of AVXS-101 in SMA Type 2 (STRONG): The on-going, open-label, dose-comparison, multi-center Phase 1 trial is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two distinct age groups of patients with SMA Type 2, utilizing a one-time intrathecal (IT) route of administration. The trial is expected to enroll 27 infants and children who are symptomatic with a genetic diagnosis consistent with SMA, including the bi-allelic deletion of SMN1 and three copies of SMN2 without the SMN2 genetic modifier, who are able to sit but have no historical or current ability to stand or walk. One patient has been dosed to date.    

Is general: Yes