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Chicago, Ill. – January 4, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced patient enrollment has completed for the Phase 1 clinical trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1.
The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined by genetic testing. The trial includes two dosing cohorts:
“We are pleased to have reached this important initial clinical milestone in our effort to bring AVXS -101 to patients who suffer from SMA Type 1, a devastating disease for which there are currently no FDA-approved therapies,” said Sean Nolan, President and Chief Executive Officer of AveXis. “The data from this initial trial will inform future clinical studies in Type 1 and potentially other types of SMA. We look forward to reviewing the data from this study over the coming year as we continue the development of AVXS-101.”
Phase 1 Trial Design
The clinical protocol requires that each patient receive a one-time dosage of AVXS-101, by intravenous injection over a one hour period. The patient remains at the clinical trial site for 48 hours after dosing for monitoring prior to discharge, and weekly follow-up evaluations are conducted for one month after dosing. After the first month, additional evaluations are conducted monthly for 23 months.
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.
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. SMA Type 1 is the leading genetic cause of infant mortality.
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