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– First patient has been dosed in pivotal trial of AVXS-101 for SMA Type 1 –
– Conference call and webcast
“We had several significant accomplishments this quarter, and the recent dosing of our first patient in the pivotal study for SMA Type 1 using AVXS-101 product derived from our GMP process is a very exciting milestone for the company and the patients we serve,” said
Initiated Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New Good Manufacturing Practice (GMP) Commercial Process: On
- The open-label, single-arm, single-dose, multi-center trial – known as STR1VE – is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 of 1.1 x 1014 vg/kg, which is equivalent to the proposed therapeutic dose (Cohort 2) in the Phase 1 trial, in patients with SMA Type 1.
- The trial will enroll a minimum of 15 patients with SMA Type 1 who are less than six months of age at the time of gene therapy, and who have one or two copies of the SMN2 backup gene as determined by genetic testing and bi-allelic SMN1 gene deletion or point mutations. The intent to treat (ITT) population defined in the protocol includes symptomatic infants less than six months of age with the bi-allelic deletion of the SMN1 gene, only two copies of the SMN2 backup gene and no exon 7 modifier. There will be at least a four-week dosing interval between dosing of the first three patients to allow review of the safety analysis as well as early signals of efficacy, prior to dosing of the next patient.
First Patient Dosed in Pivotal Trial of AVXS-101 in SMA Type 1: Today,
Data from Phase 1 Trial of AVXS-101 in SMA Type 1 Presented at the International Annual Congress of the
Analysis Presented at WMS of Seven Patients in Cohort 2 of the Phase 1 Trial of AVXS-101 in SMA Type 1 Demonstrated Cognitive, Fine Motor and Language Abilities Within the
- All patients evaluated demonstrated fine motor, cognition and language ability within the normal range. Natural history indicates patients will rarely achieve the ability to speak or retain any significant motor ability.
- In normal children, average composite scores for language and cognition abilities are 90-109, and fine motor skills are reported as a scaled score in which 10 is the mean.
• Seven of 7 (100%) patients demonstrated age-appropriate language (94-106), cognition (90-105) and fine motor skills (9-12) when compared to healthy age-matched peers.
Data from Phase 1 Trial of AVXS-101 in SMA Type 1 Published in
- All 15 patients (100%) were alive and event-free at 20 months of age, in contrast to the eight percent event-free rate demonstrated in an independent, peer-reviewed natural history study for patients with SMA Type 1. The median age at last follow-up was 25.7 months and 30.8 months for 12 patients in Cohort 2 and the three patients in the low-dose cohort (Cohort 1), respectively. Patients in Cohort 2 continued to demonstrate improvements in motor milestones: the majority of patients (92%) achieved head control, rolling over (75%), sitting with assistance (92%) and sitting without assistance for at least 5 seconds (92%). Two patients in the cohort could crawl, pull to a stand, as well as stand and walk independently.
- All patients who were free of respiratory or feeding support on
January 20, 2017, continued without the need for these elements of supportive care.
- Eleven of 12 (92%) patients were able to speak; three more patients than previously reported on
April 25, 2017at the American Academy of Neurology.
AveXis Initiated Additional Formal Clinical and Quality Scientific Advice Proceedings with the
Phillip B. Donenberg Appointed as Chief Financial Officer: On
SMA Type 2 Trial:
SMA Type 1 End-of-Phase 1 Meeting with
SMA Type 1 EU Trial:
Third Quarter 2017 Financial Results
- Cash Position: As of
September 30, 2017, AveXishad $374.2 millionin cash and cash equivalents.
- R&D Expenses: Research and development expenses were
$33.4 millionfor the third quarter of 2017 (which included $5.0 millionof non-cash stock-based compensation expense), compared to $14.1 millionfor the same period in 2016 (which included $2.7 millionof non-cash stock-based compensation expense), resulting in an increase of $19.3 million. The increase was primarily attributable to product manufacturing expenses and associated accelerated spending, including increased headcount in AveXis’ product manufacturing facility, as well as expenses related to the conclusion of the Phase 1 clinical trial of AVXS-101 in SMA Type 1, commencement of AveXis’ pivotal trial of AVXS-101 and an increase in non-cash stock-based compensation expense.
- G&A Expenses: General and administrative expenses were
$16.1 millionfor the third quarter of 2017 (which included $5.3 millionof non-cash stock-based compensation expense), compared to $7.1 millionfor the same period in 2016 (which included $2.7 millionof stock-based compensation expense), resulting in an increase of $9.0 million. The increase was primarily attributable to increases in salaries and personnel-related costs; legal, professional and consulting fees; other administrative costs driven by increased headcount across all general and administrative functions to support the company’s overall growth, and non-cash stock-based compensation expense.
- Net Loss: Net loss was
$48.6 million, or $1.52per share, for the third quarter of 2017, compared to a net loss of $21.1 million, or $0.87per share, for the third quarter of 2016.
|Selected Financial Information (unaudited)|
|(In thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|General and administrative||16,095||7,083||38,898||17,325|
|Research and development||33,425||14,098||98,946||40,542|
|Total Operating Expenses||49,520||21,181||137,844||57,867|
|Loss from operations||(49,520||)||(21,181||)||(137,844||)||(57,867||)|
|Weighted-average basic and diluted common shares outstanding||31,941,185||24,166,113||29,228,847||20,958,421|
|Basic and diluted net loss per common share||$||(1.52||)||$||(0.87||)||$||(4.67||)||$||(2.75||)|
|Balance Sheet Information:|
|(In thousands)||September 30,||December 31,|
|Cash and cash equivalents||$||374,211||$||240,430|
Conference Call Information
Analysts and investors can participate in the conference call by dialing (844) 889-6863 for domestic callers and (661) 378-9762 for international callers, using the conference ID 3899689. The webcast can be accessed live on the Events and Presentations page in the Investors and Media section of the
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births. SMA is the leading genetic cause of infant mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients.
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1, designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons, providing rapid onset of effect and crossing the blood brain barrier to allow effective targeting of both central and systemic features.
For additional information, please visit www.avexis.com.
This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis’ research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in children with SMA Type 1 and Type 2, the expected timing of dosing patients in the pivotal trial for AVXS-101, the initiation of AveXis’ planned future clinical trials in SMA Type 1 and Type 2, the expected timing of future meetings with the
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein.