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– Product used in the pivotal trial will be from the AveXis GMP manufacturing process; pivotal trial to commence immediately –
– Conference call and webcast today at 8:00 a.m. EDT –
“We are pleased to reach this outcome following a thorough review by the
With the pivotal trial now starting,
U.S. Pivotal Trial in SMA Type 1 (STR1VE)
The open-label, single-arm, single-dose, multi-center trial – known as STR1VE – is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 of 1.1 x 1014 vg/kg, which is equivalent to the proposed therapeutic dose received by the second dosing cohort in the Phase 1 trial, in patients with SMA Type 1. Based on the data derived from the company’s new analytical methods that were submitted and reviewed by
The trial will enroll 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. There will be at least a four-week dosing interval between dosing of the first three patients to allow review of the safety analysis from six time points (days one, two, seven, 14, 21 and 30), as well as early signals of efficacy, prior to dosing of the next patient.
The intent-to-treat population is defined as patients who are less than six months of age and symptomatic at the time of gene therapy, with two copies of the SMN2 gene as determined by genetic testing, bi-allelic SMN1 gene deletion and no c.859G>C mutation in SMN2.
The co-primary efficacy outcome measures of the trial will include:
- The achievement of the developmental milestone of independent sitting for at least 30 seconds at 18 months of age; and,
- Event-free survival at 14 months of age, with an event defined as 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 perioperatively.
Co-secondary outcome measures will include:
- The ability to thrive, defined as the ability to: remain independent from feeding support, tolerate thin liquids and maintain weight; and,
- The ability to remain independent of ventilatory support at 18 months of age.
The trial is projected to be conducted at 16 sites in
“We are appreciative of the detailed reviews and timely feedback we have received from the FDA,” said James L’Italien, PhD, Chief Regulatory and Quality Officer for
Today’s Conference Call Information
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births and is the leading genetic cause of infant mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients.
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Types 1 and 2, designed to address the monogenic 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 crossing the blood brain barrier to allow targeting of both central and systemic features.
For additional information, please visit www.avexis.com.
This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis’ research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in patients with SMA Type 1 and SMA Type 2, the expected timing of the initiation of AveXis’ planned clinical trials in SMA Type 1 and SMA Type 2 and the results of these trials the overall clinical development of AVXS-101, if approved, AveXis’ research, development and regulatory plans for AVXS-101, including AveXis’ commercial manufacturing process, AveXis’ ability to meet future commercial demand for AVXS-101 through its manufacturing facility and expectations regarding AveXis’ research, development and regulatory plans for its programs for treatment of RTT and genetic ALS. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis’ product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the "Risk Factors" and the "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of AveXis’ Annual Report on Form 10-K for the year ended
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein.