AveXis Offers Easily The Best Treatment Option For SMA Type-1 Patients

AveXis Inc AVXS announced top-line data from the phase I study for AVXS-101, an AAV [adeno-associated virus] -based gene therapy [GT], for spinal muscular atrophy [SMA] type 1.

AVXS-101 could generate sales of $1.8 billion by 2030, an estimate that is 70 percent risk-adjusted, Chardan Capital Markets’ Gbola Amusa said in a report.

Amusa has a Buy rating and a price target of $100 for AveXis. Given the breakthrough, event-free results of AVXS-101, AveXis continues to be a Top Pick for 2017 at Chardan Capital Markets.

AVXS-101 Prospects

The analyst mentioned that the phase I top-line results showed:

1. No new treatment-related safety or tolerability findings
2. No new events reported “in a disease characterized by the rapid (1-2 year) onset of events.”

Related Link: Citron Research On AveXis: This Will Be One Of 2017's Biotech Blowups

“Unheard of milestones are being achieved in all patients in Cohort 2, with some children being able to walk independently,” Amusa added. He commented that with this quality of data, AveXis had a good change of filing on phase I data to the FDA [Food and Drug Administration] and EMA [European Medicines Agency].

The company is scheduled for a CMC [chemistry, manufacturing, and controls] meeting with the FDA on May 1, 2017, and could have the end of phase I meeting during Q2 or Q3 of 2017.