September 9, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Dallas-based AveXis, Inc., announced yesterday that it had completed a $65 million Class D common stock financing. The company plans to use the funds to continue its ongoing clinical program in spinal muscular atrophy (SMA).
AveXis was founded in July 2013 and utilizes a synthetic biology platform using gene therapy techniques that include adeno-associated virus (AAV). It is focused on developing a treatment for spinal muscular atrophy, a rare orphan disease, which is the most common cause of infant mortality in the world, and the second most common inherited autosomal recessive disorder. There is currently no treatment for SMA.
The company was founded by the leadership of BioLife Cell Bank and was financed by White Rock Capital Texas, Inc. and others. The most recent round of financing was led by funds and accounts managed by T. Rowe Price Associates, Inc., a new investor. Existing investors participated, including Deerfield Management, Roche Venture Fund and Venrock. New participants included Janus Capital Management LLC, Adage Capital Management, LP, RA Capital Management, QVT Financial LP, Rock Springs Capital Management LP, Foresite Capital Management, LLC, RTW Investments, LLC and Boxter Capital of Tavistock Life Sciences.
“We are pleased to have such a robust syndicate of well-respected investors, led by T. Rowe Price funds and accounts, join with our existing investors in this financing,” said Sean Nolan, president and chief executive officer of AveXis in a statement. “This points to the broad enthusiasm surrounding our SMA clinical program and the progress we have made to date. We are now well-positioned to continue development of our novel gene therapy for patients suffering from SMA Type 1, a rare genetic disease and the most common genetic cause of infant mortality.”
Nolan was appointed to head the company in June. Before joining AveXis, he was the chief business officer at InterMune, Inc. . InterMune is a company focused on orphan diseases, specifically fibrotic disorders, and was acquired by Roche in 2014.
Also in June, the company announced the European Medicines Agency (EMA) had granted its gene transfer therapeutic Orphan Drug designation. The U.S. Food and Drug Administration (FDA) gave it Orphan Drug designation in October 2014.
The company’s gene therapy platform is ChariSMA, is a self-complementary adeno-associated virus, serotype 9 (scAAV9) that carries the cDNA of the human survival motor neuron (SMN) gene under control of the cytomegalovirus (CMV) enhanced chicken beta-actin hybrid promoter (CB), which is also known as scAAV9.CB.SMN. Less technically, it is a way of introducing fully functioning copies of a SMN gene into cells in order to produce more of the healthy SMN protein.
AveXis is currently running a Phase I dose-ranging clinical trial of scAAV9.CB.SMN in patients with SMA Type 1.
“SMA is a devastating genetic disease with no approved treatments,” said Taymour Tamaddon, portfolio manager with the T. Rowe Price Health Sciences Fund in a statement. “We believe the AveXis program holds great promise for patients suffering from SMA and we are pleased to support the company.”
