- 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 10 14 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.
- 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.
- 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, 2017 at the American Academy of Neurology.
- Cash Position: As of September 30, 2017, AveXis had $374.2 million in cash and cash equivalents.
- R&D Expenses: Research and development expenses were $33.4 million for the third quarter of 2017 (which included $5.0 million of non-cash stock-based compensation expense), compared to $14.1 million for the same period in 2016 (which included $2.7 million of 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 million for the third quarter of 2017 (which included $5.3 million of non-cash stock-based compensation expense), compared to $7.1 million for the same period in 2016 (which included $2.7 million of 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 $1.52 per share, for the third quarter of 2017, compared to a net loss of $21.1 million, or $0.87 per share, for the third quarter of 2016.
|Selected Financial Information (unaudited)|
|(In thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|General and administrative||16,095||7,083||38,898||17,325|
|Research and development||33,425||14,098||98,946||40,542|
|Total Operating Expenses||49,520||21,181||137,844||57,867|
|Loss from operations||(49,520||)||(21,181||)||(137,844||)||(57,867||)|
|Weighted-average basic and diluted common shares outstanding||31,941,185||24,166,113||29,228,847||20,958,421|
|Basic and diluted net loss per common share||$||(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|
About AVXS-101AVXS-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.About AveXis, Inc.AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company's initial proprietary gene therapy candidate, AVXS-101, is in the pivotal phase of study for the treatment of SMA Type 1. The company also intends to expand the study of gene therapy into SMA Type 2 and two additional rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 ( SOD1) gene. For additional information, please visit www.avexis.com. Forward-Looking Statements 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 FDA, the overall clinical development of AVXS-101 and expectations regarding AveXis' research, development and regulatory plans for its programs for treatment of RTT and genetic ALS. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis' product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the "Risk Factors" and the "Management's Discussion and Analysis of Financial Condition and Results of Operations" section of AveXis' Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect AveXis' results. There can be no assurance that the actual results or developments anticipated by AveXis will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, AveXis. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. AveXis cautions investors not to rely too heavily on the forward-looking statements AveXis makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). AveXis undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Media Inquiries:Lauren BarbieroW2O Group646-564-2156 email@example.com Investor Inquiries:Jim GoffAveXis, Inc.650-862-4134 firstname.lastname@example.org