AveXis Expands Clinical Testing of Gene Therapy, AVXS-101, to Include Milder SMA Types

AveXis plans to launch clinical trials of its gene therapy AVXS-101 in a wider range of spinal muscular atrophy (SMA) patients — mirroring the company’s belief that the treatment may also benefit those with milder forms of the disease.

The planned studies include one enrolling presymptomatic patients with SMA type 1, 2, and 3. Another will recruit patients with these same disease subtypes, but who do not qualify for other trials.

“Our focus has always been to serve the SMA community, and our expanded clinical development program is designed to evaluate the impact of AVXS-101 in a broader set of SMA patients,” Sukumar Nagendran, AveXis’ chief medical officer, said in a press release.

AveXis is currently running a Phase 3 trial, called STR1VE (NCT03306277) in infants with SMA type 1. Researchers also recently dosed the first patient in a second study, the Phase 1 STRONG trial (NCT03381729) in children with SMA type 2.

Both studies, taking place at sites across the U.S., are still recruiting participants.

In addition to studies that will include type 3 patients, the company is also launching a European pivotal trial in type 1 infants. That study will be called STR1VE EU.

The trial in presymptomatic patients with type 1, 2, or 3 SMA, called SPRINT, will launch in the coming months, the company said. Participants will receive one intravenous injection of AVXS-101.

A so-called pediatric “all comers” trial, including patients not eligible for other studies, is not due to start until year’s end or in early 2019. This trial will be called REACH, and will include children ages six months to 18 years. As in the STRONG study, patients enrolled in REACH will receive a one-time injection of the gene therapy into the spinal canal.

AveXis said it will provide more information about these trials as they get underway.

“We believe the year ahead has the potential to be one of significant clinical progress as we continue toward our ultimate goal of bringing AVXS-101 to the patients and families devastated by SMA,” said Nagendran.

The Phase 3 STR1VE study in SMA type 1, and the Phase 1 STRONG study in SMA type 2, continue to enroll patients. For more information about these trials — including locations and contact details — see the trials’ registration pages: here (Phase 3) and here (Phase 1).

AVXS-101 is composed of a harmless virus that delivers a functional SMN gene — which is faulty in SMA — to nerve cells that control movement. The goal is to prevent the cells from degenerating.