AveXis has filed an S-1 form with the U.S. Securities and Exchange Commission (SEC) in regards to its initial public offering (IPO). No pricing details were given in the filing, but the offering is valued up to $115 million. The company intends to list on the Nasdaq Global Market under the symbol AVXS.
The underwriters for the offering are Goldman Sachs, Jefferies, BMO Capital Markets and Chardan.
This clinical-stage gene therapy company is dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Its initial product candidate is AVXS-101, a proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, the leading genetic cause of infant mortality.
SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients. The survival motor neuron (SMN) is a critical protein for normal motor neuron signaling and function. Patients with SMA Type 1 either carry a mutation in their SMN1 gene or their SMN1 genes have been deleted, which prevents them from producing adequate levels of functional SMN protein. AVXS-101 is designed to deliver a fully functional human SMN gene into the nuclei of motor neurons that then generates an increase in SMN protein levels. The company believes this will result in improved motor neuron function and patient outcomes.
AveXis has completed enrollment in its ongoing Phase 1 clinical trial, in which it has treated 15 SMA Type 1 patients as of December 31, 2015, and has observed a favorable safety profile that is generally well-tolerated and has also observed compelling preliminary evidence of efficacy, including improved motor function. The U.S. Food and Drug Administration (FDA) has granted AVXS-101 orphan drug designation for the treatment of all types of SMA and fast track designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1, the company plans to develop AVXS-101 to treat additional SMA types and develop other novel treatments for rare neurological genetic diseases.
The company intends to use the net proceeds from this offering to fund its clinical trials and further its research. The remainder will be used for working capital and general corporate purposes.
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