Chicago-based AveXis (AVXS 85.05, +12.27) trades to all-time highs today, up about 16.9% in reaction to both last night’s Q4 report and topline results from the company’s Phase 1 trial of AVXS-101 in SMA Type 1.
For those who may not be familiar, AVXS is a clinical-stage gene therapy company that develops treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company’s initial proprietary gene therapy candidate, AVXS-101, recently completed a Phase 1 clinical trial for the treatment of SMA Type 1.
Specifically, as of January 20, 2017, AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new safety or tolerability concerns identified. The company also noted that as of January 20, 2017, 12 of 12 patients in the cohort of patients who received the proposed one-time therapeutic dose of AVXS-101 (Cohort 2) had reached 13.6 months of age event-free, where the expected event-free survival rate based on natural history of the disease is 25%. The median age at last follow-up for Cohort 2 was 20.2 months, with the oldest patient at 31.1 months of age.
Further, as of January 20, 2017, mean increases from baseline in CHOP INTEND scores of 7.7 points in Cohort 1 and 24.7 points in Cohort 2 were observed, showing improvement in motor function. In Cohort 2 there were mean increases in CHOP INTEND of 9.8 points one month after gene therapy and 15.4 points three months following gene therapy.
Also, as of January 20, 2017, 11 of 12 patients in Cohort 2 achieved head control, nine of 12 patients could roll a minimum of 180 degrees from back to both left and right, and 11 of 12 patients could sit with assistance.
Lastly, for the end-of-study assessment, AVXS evaluated three validated and well-established measures of sitting unassisted for periods of increasing duration. Nine of 12 patients could sit unassisted for at least five seconds, seven of 12 patients could sit unassisted for at least 10 seconds and five of 12 patients could sit unassisted for 30 seconds or more. Two patients could walk independently, and each had achieved earlier and important developmental milestones such as standing with support, standing alone and walking with support.
In terms of the Q4 print, AVXS reported a worse than expected loss per share of $0.92 and did not record any revenues in the period. Looking ahead, AVXS gave some upcoming clinical development milestones:
- The company plans to conduct a Type B meeting with the U.S. Food and Drug Administration (FDA) to discuss chemistry manufacturing and controls (CMC); provide an update based on receipt of meeting minutes in the second quarter of 2017.
- Will initiate a pivotal trial in U.S. of AVXS-101 via intravenous (IV) delivery in patients with SMA Type 1 in the second quarter of 2017, pending a successful outcome of the Type B CMC meeting.
- Will also initiate a Phase 1 safety and dose escalation study of AVXS-101 via intrathecal (IT) delivery in patients with SMA Type 2 in the second quarter of 2017, pending a successful outcome of the Type B CMC meeting.
- Conduct an end-of-Phase 1 meeting with FDA in the second or third quarter of 2017.
- Conduct a comprehensive clinical program review with the EMA, to be scheduled in the second or third quarter of 2017.
- Initiate pivotal trial in the European Union of AVXS-101 using IV delivery in patients with SMA Type 1 in the second half of 2017.
- Five preclinical and clinical abstracts will be presented at the Annual Meeting of the American Academy of Neurology in Boston, April 22-28, 2017, including results from the Phase 1 trial of AVXS-101 in SMA Type 1, including developmental milestones videos.