Featured Company News - AveXis Intends To Initiate Pivotal Trial Of AVXS-101 In SMA Type 1 On An Immediate Basis
LONDON, UK / ACCESSWIRE / October 3, 2017 / Pro-Trader Daily takes a look at the latest corporate events and news making the headlines for AveXis, Inc. (NASDAQ: AVXS), following which we have published a free report that can be viewed by signing up at http://protraderdaily.com/optin/?symbol=AVXS. The Company announced on September 29, 2017, that the US Food and Drug Administration (FDA) has notified AveXis that it may initiate a pivotal trial of AVXS-101. The FDA's approval is based on the review of data following the Chemistry, Manufacturing, and Controls (CMC) Type B meeting, including the potency assay. The pivotal trial of AVXS-101 would be conducted for patients suffering from spinal muscular atrophy (SMA) Type 1 using the intravenous (IV) formulation produced by the Company's Good Manufacturing Practice (GMP) commercial manufacturing process. AveXis intends to initiate this trial immediately. For immediate access to our complimentary reports, including today's coverage, register for free now at:
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SMA, A Life-Threatening Disease
SMA is a severe neuromuscular disease, which is characterized by the loss of motor neurons. This could lead to progressive muscle weakness as well as paralysis.
SMA is primarily caused by a genetic defect in the SMN1 gene that codes SMN, a protein essential for the survival of motor neurons. It is the leading cause of infant mortality – nearly one in 10,000 live births get SMA.
Type 1 SMA is the most severe form. It is characterized by motor neuron loss and associated muscle deterioration, which leads to mortality, or the need for permanent ventilation support before the age of two for more than 90% patients.
About AVXS-101
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Types 1 and 2. It has been designed to tackle the monogenic root cause of SMA and also avert further muscle degeneration by treating the defective and/or loss of the primary SMN gene. AVXS-101 targets motor neurons, providing rapid onset of effect and crossing the blood-brain barrier to allow targeting of both central and systemic features.
About the US Pivotal Trial in SMA Type 1 - STR1VE
STR1VE is an open-label, single-arm, single-dose, and multi-center trial. It has been designed to assess the efficacy and safety of a one-time IV infusion of AVXS-101 of 1.1x 1014 vg/kg, which is equal to the proposed therapeutic dose received by the second dosing cohort in the Phase-1 trial, in patients with SMA Type 1.
AveXis had submitted data about its new analytical methods, which was subsequently reviewed by the FDA. On the basis of this data and a direct test with the improved PCR method, it has been determined that the dose used in AveXis' Phase-1 trial of AVXS-101 in SMA Type 1 was 1.1x 1014 vg/kg. Moreover, extensive testing in the SMN delta 7-mouse potency assay has also verified the equivalence of dose-response between the products produced by the Phase-1 and Phase-3 manufacturing processes.
The trial encompasses a minimum of 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. The trial would provide for a dosing interval of at least four weeks between dosing of the first three patients to allow review of the safety analysis from six time points (i.e. days 1, 2, 7, 14, 21, and 30), as well as early signals of efficacy, prior to dosing of the next patient.
AveXis plans to conduct the trial at 16 sites in the United States, namely: (i) Ann and Robert H. Lurie Children's Hospital of Chicago, (ii) Boston Children's Hospital, (iii) Children's Hospital Colorado, (iv) Children's Hospital of Philadelphia, (v) Columbia University, (vi) David Geffen School of Medicine at UCLA, (vii) Duke University, (viii) Johns Hopkins Pediatric Neurology, (ix) Nationwide Children's Hospital, (x) Oregon Health and Science University, (xi) Stanford University Medical Center, (xii) University of Central Florida College of Medicine, (xiii) University of Texas Southwestern Medical Center, (xiv) University of Utah, (xv) University of Wisconsin, and (xvi) Washington University School of Medicine.
Co-Primary and Co-Secondary Outcome Measures
The co-primary efficacy outcome measures of the trial include the following:
The achievement of the developmental milestone of independent sitting for at least 30 seconds at 18 months of age;
Event-free survival at 14 months of age, where an event means either death or at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or preoperatively.
The co-secondary outcome measures include the following:
The ability to thrive, which is the ability to remain independent from feeding support, tolerate thin liquids, and maintain weight;
The ability to remain independent of ventilator support at 18 months of age.
An Important Milestone for AveXis
James L'Italien, PhD, Chief Regulatory and Quality Officer at AveXis, welcomed the detailed reviews and timely feedback from the FDA. He looks forward to the end-of-Phase-1 meeting with the FDA, to discuss further steps in the regulatory process for AVXS-101. The meeting is scheduled for end of the fourth quarter.
Sean Nolan, President and Chief Executive Officer (CEO) at AveXis, also expressed his pleasure on reaching this positive outcome. He said this approval is the result of a thorough review by the FDA of the comprehensive information supplied by the Company to address the commitments made during the CMC Type B meeting in May 2017. In fact, AveXis is eager to initiate its pivotal trial of AVXS-101 in SMA Type 1 in the US, utilizing its product from the GMP process. He mentioned that moving AVXS-101 back into the clinic is an important milestone, not just for AveXis but also for the patients that the Company strives to serve.
AveXis facility – the Main Production Site
AveXis plans to start the pivotal trial on an immediate basis. The AveXis facility would be the production site for supplying the pivotal as well as future trials.
Moreover, if AVXS-101 gets approved for marketing, the same site would be leveraged to meet projected commercial demand.
Ongoing Discussions with FDA
Now that the pivotal trial is in its starting phase, AveXis and the FDA are continuing their discussions on important topics such as dosing, for intrathecal administration of AVXS-101 for the planned clinical trial in patients with SMA Type 2.
AveXis can expect a follow-up or update on this program in the fourth quarter of 2017.
Last Close Stock Review
At the closing bell, on Monday, October 02, 2017, AveXis' stock slipped 1.00%, ending the trading session at $95.76. A total volume of 401.68 thousand shares have exchanged hands. The Company's stock price skyrocketed 16.60% in the last three months, 26.05% in the past six months, and 132.37% in the previous twelve months. Moreover, the stock soared 100.63% since the start of the year. The stock currently has a market cap of $2.91 billion.
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