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will get a mid-range dose in the trial. In total, Carbona says the company hopes to have dosed 18 total children both intravenously and intrathecally in both trials.
Good results will help AveXis edge into a competitive space with some large, well-financed players. The company, which was founded in 2013, is a relative newcomer to the SMA field.
In 2012, Isis announced it was partnering with Biogen Idec (NASDAQ: BIIB) on an experimental antisense drug for SMA. The drug candidate is delivered through an injection into the spine, and earlier this month, Isis reported that it had received a $7 million payment from Biogen to advance an ongoing open-label extension study, which is evaluating its drug candidate in a phase III trial with 110 children.
Roche and PTC Therapeutics (NASDAQ: PTCT) are also developing a treatment via a partnership began in 2011. They’re trying to use a process called “alternative splicing” to generate drugs that would prompt a backup SMN gene to produce more of the vital protein.
In November, the two companies announced that they started a Phase 1b/2a study in both adult and pediatric patients with the help of the SMA Foundation. They aim to enroll 48 patients in the trial.
Founded two years ago,AveXis was formed out of another Dallas venture, the BioLife Cell Bank, which stores stem and fat cells. Carbona, who had been CEO of the cell bank, says he saw the possibility of gene therapy for SMA treatment and left to form AveXis. The company has licensed its technology from Ohio State University and Brian Kaspar, a principal investigator at The Research Institute at Nationwide Children’s Hospital and an associate professor at the university’s medical school.
Last year, AveXis entered into an agreement with Washington, D.C.-based Regenx to use one of the company’s viral vectors to deliver ChariSMA (several other gene therapy upstarts, like AAVLife, Audentes Therapeutics, and Dimension Therapeutics, are similarly using RegenX vectors). The company has obtained orphan status for its drug candidate.
I spoke to Carbona right after his red-eye flight to Dallas from San Francisco, where he had attended the J.P. Morgan Healthcare Conference, the biotech industry’s biggest annual gathering. In addition to securing the new funds, he says the company is working on doing pre-clinical and then clinical trials in Europe. AveXis is aims to have drugs on the market by 2018, he added.
There’s a long road ahead, but AveXis’s progress is sure to be closely watched by its rivals.