Clinical Trials

Date: 2018-01-16

Type of information: Initiation of the trial

phase:

Announcement: initiation of the trial

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 1

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Country:

Trial details:

Latest news:

• On January 16, 2018, AveXis provided an overview of the expanded clinical development program for the company’s 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. Planned Trials in SMA:
Pediatric “All Comers” with SMA Types 1, 2, 3 (REACH): 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.