Watch Out, Ionis Pharma: Could This Little Biotech’s Experimental SMA Treatment Be a Threat?

October 14, 2016
By Alex Keown, BioSpace.com Breaking News Staff

CHICAGO – AveXis Inc. is riding high off of the Breakthrough Therapy Designation the company received from the U.S. Food and Drug Administration in July for AVX-101, its lead development candidate for the treatment of spinal muscular atrophy in infants.

Since the designation was awarded, shares of the Illinois-based company have been on an upward trend, jumping past rival Ionis Pharmaceuticals , which is developing a similar treatment. Shares of AveXis are trading at $46.98 this morning, while Ionis is trading at $30.19 per share. However, writing in the Motley Fool, Cory Renauer suggests by stepping back, AveXis isn’t as big a threat to Ionis to Ionis as one might think, simply based off of stock prices.

For Ionis, its SMA drug Nusinersen, co-developed with Biogen, is the first antisense drug from Ionis’ neurological disease franchise to advance to regulatory review, the company said. In August, the company presented positive Phase III data of their joint investigational treatment, which resulted in a nice payment of $75 million for Carlsbad, Calif.-based Ionis. Since the Phase III trial, Biogen has applied for a priority review of the drug, which would shorten the regulatory review period to about six months. The company is anticipating a review soon.

In contrast, AVX-101 has not even completed its Phase I trial, Renauer said. The trial is expected to end by the end of the year, with a second trial, designed to support a New Drug Application, hoped for next year.

Renauer said if all goes well for Biogen and Ionis, they could find out if the FDA will approve nusinersen for commercialization by April, before AveXis could begin its own trial for the same patient population. That could mean bad news for AveXis, which would likely send its stock tumbling.

“If nusinersen is already available when AveXis is ready to submit an AVXS-101 application, there's little chance the FDA would stretch its limited resources to speed along a second SMA treatment,” Renauer said.

Spinal muscular atrophy a genetic condition which affects the motor neurons that control muscle movement. According to the Muscular Dystrophy Association, “babies born with the most severe type of SMA of SMA -- Type 1 -- are not able to sit up unsupported and the majority don’t survive beyond the age of two years.” SMA appears in about one out of every 60,000 babies, the MDA said on its website.

Another key as to why AVXS-101 is not likely to be a threat to Ionis, at least any time soon, is that the drug has not yet been tested head to head with nusinersen, Renauer said.

Phase III data for nusinersen showed the patients receiving the drug experienced a statistically significant improvement in the achievement of motor milestones (kicking feet, crawling and standing) compared to those who did not receive treatment.

Renauer said AveXis’ drug has also shown positive results in a very small patient test. In a 15-patient test, eight of 12 babies treated with a higher dosage of AVXS-101, were able to achieve similar results to the Ionis drug. However, four of the patients experienced treatment-related side effects, Renauer said.

Those reasons means AveXis is not likely a threat to Ionis. In fact, Renauer said investors in AveXis should be nervous given the bar that nusinersen has set.