- Dr. Brian Kaspar, Senior Vice President and Chief Scientific Officer of AveXis, presented data from a dose-response study for the cerebrospinal fluid delivery of AVXS-101 that offers insight into vector distribution and its correlation with transgene expression, providing guidance for future AAV9-based clinical trials in SMA, as well as other neurodegenerative disorders.
- Dr. Douglas Sproule, Vice President of Clinical Development and Medical Affairs of AveXis, presented data suggesting pre-existing antibodies to AAV9 are quite uncommon in the pediatric population and should not impact use of gene therapy for the vast majority of SMA Type 1 patients.
- Dr. Linda Lowes, Director of Clinical Therapies Research and a member of the Center for Gene Therapy at the Research Institute of Nationwide Children's Hospital, presented research providing further evidence of the correlation between motor function and motor milestone achievement in patients with SMA Type 1, demonstrating that the degree of treatment outcome appears to be influenced by age at dosing and baseline motor function.
- In the meeting minutes, the FDA made a request that the company complete implementation of its potency assay qualification plan, presented in the meeting, prior to initiation of upcoming clinical studies. This assay utilizes the Delta 7 mouse model, which has been used historically to assess AVXS-101 potency but now incorporates additional elements to make it acceptable to global regulatory authorities. The company has already initiated the work necessary to address this request and expects to have the data ready to submit to the FDA in August.
- AveXis plans to initiate a pivotal study trial of AVXS-101 in SMA Type 1 in the U.S. and a Phase 1/2a trial of AVXS-101 in SMA Type 2 in the U.S. later in the third quarter of 2017, pending agreement from the FDA that these data are sufficient. AveXis expects to provide an update once the company has received FDA agreement that these data are sufficient to initiate the studies. The company also expects to provide design details for these studies at that time.
- Additionally, FDA is aligned with the company's proposed comparability protocol to assess the similarity of key characteristics of the Nationwide Children's Hospital product, used in the Phase 1 SMA Type 1 study, with the product derived from the new GMP manufacturing process. Data from this comparability work is ongoing and will include the above-mentioned potency qualification data, which will be incorporated into the data package along with the full Phase 1 clinical data that will be reviewed and discussed at the upcoming end-of-Phase 1 meeting. This meeting will help further inform the regulatory pathway options for AVXS-101. The company anticipates providing an update on the outcome of that meeting once the official minutes are available, which is anticipated to be in the fourth quarter of 2017.
- Cash Position: As of June 30, 2017, AveXis had $417.6 million in cash and cash equivalents.
- R&D Expenses: Research and development expenses were $45.2 million for the second quarter of 2017 (which included $3.7 million of non-cash stock-based compensation expense), compared to $10.4 million for the same period in 2016 (which included $1.3 million of non-cash stock-based compensation expense), resulting in an increase of $34.8 million. The increase was primarily attributable to product manufacturing expenses and associated accelerated spending, including increased headcount, in the company's product manufacturing facility, as well as expenses related to the conclusion of the Phase 1 clinical trial of AVXS-101 in SMA Type 1, licensing fees related to the company's planned new programs in Rett syndrome and genetic ALS and an increase in non-cash stock-based compensation expense.
- G&A Expenses: General and administrative expenses were $13.2 million for the second quarter of 2017 (which included $4.5 million of non-cash stock-based compensation expense), compared to $5.4 million for the same period in 2016 (which included $2.6 million of stock-based compensation expense), resulting in an increase of $7.8 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 $58.0 million, or $2.07 per share, for the second quarter of 2017, compared to a net loss of $15.7 million, or $0.68 per share, for the second quarter of 2016.
|Selected Financial Information (unaudited)|
|(In thousands, except share and per share data)|
|Three Months Ended June 30,||Six Months Ended June 30,|
|General and administrative||13,154||5,418||22,803||10,242|
|Research and development||45,206||10,380||65,521||26,445|
|Total Operating Expenses||58,360||15,798||88,324||36,687|
|Loss from operations||(58,360||)||(15,798||)||(88,324||)||(36,687||)|
|Weighted-average basic and diluted common shares outstanding||27,971,733||23,013,838||27,850,199||19,876,850|
|Basic and diluted net loss per common share||$||(2.07||)||$||(0.68||)||$||(3.15||)||$||(1.84||)|
|Balance Sheet Information:|
|(In thousands)||June 30,||December 31,|
|Cash and cash equivalents||$||417,620||$||240,430|
About AVXS-101AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is 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 crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.About Rett SyndromeRett syndrome (RTT) is a devastating, rare neurological disorder characterized by slowed growth, loss of normal movement and coordination and loss of communication skills. RTT is caused by an X-linked dominant mutation in the methyl CpG binding protein 2 ( MECP2) gene, which results in problems with the protein production critical for brain development. RTT occurs in approximately one of every 10,000 female births and usually begins to show signs and symptoms in infants between six and 18 months of age. Current treatments only offer symptomatic relief and do not target the genetic cause of the disease, leaving a significant unmet need. About Genetic Amyotrophic Lateral SclerosisAmyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Familial or inherited forms of ALS reflect five to 10 percent of ALS cases, or approximately one to two thousand people in the U.S., and can be caused by mutations in several genes known to be associated with ALS. Approximately 20 percent of these cases are caused by mutations in the gene that produces the copper zinc superoxide dismutase 1 (SOD1) enzyme, which leads to a progressive degeneration of motor neurons affecting movement and muscle control. ALS usually occurs in people between the ages of 40 and 70. Current treatments only offer modest benefits and do not target the genetic cause of the disease, leaving a significant unmet need. 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 an ongoing Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com. Forward-Looking StatementsThis 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, expectations regarding AveXis' research, development and regulatory plans for its programs for treatment of RTT and genetic ALS, its expectations regarding initiation of IND-enabling studies for these programs and timing of providing an update on these programs, the overall clinical development of AVXS-101, AveXis' research, development and regulatory plans for AVXS-101, including AveXis' commercial manufacturing process, AveXis' potency assay development and the expected timing of the submission of data regarding the potency assay to the FDA, the expected timing of the initiation of AveXis' planned future clinical trials, the expected timing of future meetings with the FDA, AveXis' ability to meet future commercial demand for AVXS-101 through its manufacturing facility and the possibility that AveXis may be able to utilize data from its Phase 1 clinical trial in the regulatory pathway of AVXS-101. 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.
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