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LONDON, UK / ACCESSWIRE / January 18, 2018 / Active-Investors.com has just released a free research report on AveXis, Inc. (NASDAQ: AVXS). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=AVXS as the Company's latest news hit the wire. On January 16, 2018, the clinical-stage gene therapy company that provides treatments for patients suffering from rare and life-threatening neurological genetic diseases, shared details about the expanded clinical development program for AVXS-101, which is its initial gene therapy candidate for the treatment of spinal muscular atrophy (SMA). Register today and get access to over 1000 Free Research Reports by joining our site below:
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AveXis already has two ongoing studies for SMA, i.e. pivotal trial in SMA Type-1 (STR1VE) and Phase-1 trial in SMA Type-2 (STRONG). Apart from that, the Company intends to expand the study of AVXS-101 into additional SMA populations such as pre-symptomatic, older pediatric Type-2 and Type-3 SMA patients. AveXis also declared that the first patient has been dosed in the Phase-1 trial of AVXS-101 in SMA Type-2.
SMA, the Leading Cause of Infant Mortality
Spinal muscular atrophy is a severe neuromuscular disease, in which the loss of motor neurons leads to progressive muscle weakness and paralysis. It is caused by a genetic defect in the SMN1 gene, which codes SMN, i.e. the protein necessary for survival of motor neurons. At present, SMA is the leading cause of infant mortality - its incidence is nearly one in 10,000 live births.
Type-1 SMA is the most serious form of SMA, characterized by motor neuron loss and related muscle deterioration, which often leads to mortality or the need for permanent ventilation support before the age of two for more than 90% patients. On the other hand, SMA Type-2 generally occurs between six to 18 months of age. Majority of the SMA Type-2 patients cannot walk without support and some cannot even stand without support. This form of SMA results in mortality in over 30% of patients by the age of 25.
How AveXis Treats SMA?
AVXS-101 is AveXis' proprietary gene therapy candidate for one-time treatment of SMA Type-1 and -2. AVXS-101 focuses on the monogenic root cause of SMA and prevents further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. Besides, AVXS-101 is also designed to target motor neurons, which causes rapid onset of effect and crosses the blood brain barrier to allow targeting of both central and systemic features.
Overview of the Ongoing Clinical Trials of AVXS-101
- STR1VE, the Pivotal Trial of AVXS-101 in SMA Type-1: STR1VE is an ongoing, open-label, single-arm, single dose, multi-center trial, which is designed to assess the efficacy and safety of a one-time IV infusion of AVXS-101 in patients with SMA Type-1. For this trial, AveXis aims to enroll at least 15 patients with SMA Type-1 who are less than six months of age at the time of gene therapy, and who have one or two copies of the SMN2 backup gene, as determined by genetic testing and bi-allelic SMN1 gene deletion or point mutations. So far, three patients have been dosed for the trial.
- STRONG, Phase-1 Trial of AVXS-101 in SMA Type-2: STRONG is an on-going, open-label, dose-comparison, multi-center Phase-1 trial, which has been specifically designed to gauge the safety, optimal dosing, and proof of concept for efficacy of AVXS-101 in two different age groups of patients with SMA Type-2. This Trial is based on the one-time intrathecal (IT) route of administration. For this trial, AveXis aims 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 are not able to stand or walk. In this trial, only one person has been dosed up till now.
Overview of the Three Proposed Trials in SMA
- STR1VE EU, Pivotal Trial of AVXS-101 in SMA Type 1 in Europe: STR1VE EU has been specifically designed to assess the safety and efficacy of a one-time IV dose of AVXS-101. It is meant to evaluate achievement of motor milestones, patients' ability to sit unassisted, and an efficacy measure defined by the time from birth to an event, wherein event means death or a situation requiring at least 16 hours per day of ventilation support for breathing for greater than two weeks in the absence of an acute reversible illness, or preoperatively. For this trial, AveXis aims to enroll approximately 30 patients with SMA Type-1 who are less than six months of age at the time of gene therapy.
This trial is based on single-arm design, using natural history of the disease as a comparator. Besides, the Company has also incorporated scientific advice from the European Medicines Agency into the protocol design and expects to start the trial in the first half of 2018.
- SPRINT, for Pre-Symptomatic SMA Types 1, 2, 3: The multi-national trial SPRINT is meant to assess appropriate clinical endpoints, such as developmental milestones, survival, bulbar function, and safety, of a one-time IV infusion of AVXS-101. For this trial, AveXis aims to enroll around 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. The Company intends to start this trial in the first half of 2018.
- REACH for Pediatric "All Comers" with SMA Types 1, 2, 3: The multi-national trial REACH is designed to evaluate a one-time IT dose of AVXS-101. For this trial, AveXis aims to enroll around 50 patients between six months and 18 years of age who do not qualify for other AVXS-101 trials at the time of gene therapy. The Company aims to commence this trial in end of 2018 or beginning of 2019.
Stock Performance Snapshot
January 17, 2018 - At Wednesday's closing bell, AveXis' stock slightly declined 0.13%, ending the trading session at $104.87.
Volume traded for the day: 2.91 million shares, which was above the 3-month average volume of 382.66 thousand shares.
Stock performance in the last month - up 5.99%; previous three-month period - up 2.80%; last six-month period - up 27.02%; past twelve-month period - up 99.71%.
After yesterday's close, AveXis' market cap was at $3.44 billion.
The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.9% at the end of the session.
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