– On track to request pre-BLA meeting with
– Intends to initiate pivotal trial in SMA Type 1 in
– Anticipates IND submissions for Rett syndrome and genetic ALS in late 2018/early 2019 –
– Conference call and webcast
"2017 was marked by significant progress for
Recent Corporate Highlights
- Expanded Product Development Pipeline: On
June 7, 2017 ,AveXis obtained exclusive worldwide rights to AAV9 fromREGENXBIO for Rett syndrome ( MECP2 gene) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 ( SOD1 ) gene.AveXis had previously licensed the preclinical data developed by Dr.Brian Kaspar from Nationwide Children's Hospital. These are rare, life-threatening, neurological monogenic diseases that have significant unmet need with either minimally effective or no available treatment options.AveXis expects to leverage its Good Manufacturing Practice manufacturing platform for these programs, whichAveXis believes will reduce overall development timelines. - Announced Alignment with the
FDA on Next Steps in Regulatory Pathway for AVXS-101 in SMA Type 1: OnJanuary 4, 2018 , following the receipt of minutes from the end-of-Phase 1 meeting with theFDA ,AveXis announced plans to address detailed information requests from the agency by submitting the requested information to the investigational new drug (IND) application on an ongoing basis, and plans to submit the request for a pre-BLA meeting in the second quarter of 2018. - Acquired Exclusive Worldwide Rights from
REGENXBIO to Entire NAV Technology Platform to Develop Treatments for SMA : OnJanuary 8, 2018 ,AveXis announced an amended licensing agreement, which expands the exclusive, worldwide license agreement to REGENXBIO's entire NAV platform for SMA, and permits license assignment byAveXis upon a change of control without consent fromREGENXBIO . - Initiated Phase 1 Trial of AVXS-101 in SMA Type 2 (STRONG) Using Intrathecal Delivery and Product from the Company's Good Manufacturing Practice Commercial Process: On
January 16, 2018 ,AveXis announced the first patient was dosed in STRONG. Two patients have been dosed to date, with a four-week interval between the dosing of the first three patients for each dose cohort per the trial protocol. - Completed Public Offering with Net Proceeds of
$431.9 Million : OnJanuary 22, 2018 ,AveXis closed an underwritten public offering with net proceeds of$431.9 million .AveXis intends to use the net proceeds to fund its research, manufacturing and clinical activities to support its programs in SMA, Rett syndrome and genetic ALS; to fund pre-commercial activities, including medical affairs, development of commercial initiatives for the potential launch of AVXS-101; licensing activities; and for general corporate purposes and working capital. - Expanded SMA Clinical Development Program: On
January 26, 2018 ,AveXis announced plans to initiate three studies to further evaluate AVXS-101, including in new SMA patient populations. - Initiated Screening for Remaining Patients in U.S. Pivotal Trial of AVXS-101 for SMA Type 1 (STR1VE): On
January 30, 2018 , following review of safety data and early signals of efficacy from the first three patients dosed, and with agreement from theFDA ,AveXis initiated screening for the remaining patients in the trial. To date, five patients have been dosed.
Upcoming Milestones
- Pre-BLA Meeting Request:
AveXis expects to submit the request for a pre-BLA meeting related to AVXS-101 in SMA Type 1 in the second quarter of 2018. - Pivotal Trial of AVXS-101 in SMA Type 1 in
Europe (STR1VE EU): The single-arm trial is expected to initiate in the first half of 2018 and enroll approximately 30 patients who are less than six months of age and naïve to SMA treatment. STR1VE EU is designed to evaluate a one-time intravenous (IV) infusion of AVXS-101, including safety, event-free survival and achievement of the developmental milestone of sitting without support for 10 seconds, and will study children up to 18 months of age. - Pre-Symptomatic SMA Types 1, 2, 3 (
SPRINT ): The multi-cohort, multi-national trial is expected to initiate in the first half of 2018 and enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic.SPRINT is designed to evaluate appropriate clinical endpoints, including developmental milestones, survival, bulbar function, and safety of a one-time IV infusion of AVXS-101. The primary efficacy endpoint by cohort is as follows:
- Cohort with two copies of SMN2 : proportion of patients who achieve functional independent sitting for at least 30 seconds, up to 18 months of age;
- Cohort with three copies of SMN2 : proportion of patients who achieve the ability to stand without support for at least three seconds, up to 24 months of age;
- Cohort with four copies of SMN2 : proportion of patients who do not manifest symptoms consistent with SMA Type 3 based on a scaled score on Bayley V.3 Gross and Fine Motor Subtests within 1.5 standard deviations of chronological development reference standard, as assessed at 36 months of age. - Pediatric SMA Types 1, 2, 3 (REACH) : Expected to initiate late in the fourth quarter of 2018 or early 2019.
- Rett Syndrome and Genetic ALS: Expected to submit IND applications for AVXS-201 for Rett syndrome ( MECP2 ) and AVXS-301 for genetic ALS ( SOD1 ) in late 2018/early 2019.
Fourth Quarter and Full Year 2017 Financial Results
- Cash Position: As of
December 31, 2017 ,AveXis had$324.1 million in cash and cash equivalents. This amount does not include the net proceeds from the recently completed public offering described above. - R&D Expenses: Research and development expenses were
$51.4 million for the fourth quarter of 2017 (which included$4.9 million of non-cash stock-based compensation expense), compared to$18.3 million for the same period in 2016 (which included$1.6 million of non-cash stock-based compensation expense), an increase of$33.1 million . The increase in research and development expenses was primarily attributable to increases in third-party clinical and manufacturing research and development spending related to AveXis' clinical trials and clinical trial product manufacturing, salaries and personnel-related expenses driven by increased headcount across all research, development and manufacturing functions, license fees and other research and development expenses. - G&A Expenses: General and administrative expenses were
$31.1 million for the fourth quarter of 2017 (which included$3.0 million of non-cash stock-based compensation expense), compared to$7.2 million for the same period in 2016 (which included$2.4 million of stock-based compensation expense), an increase of$23.9 million . The increase in general and administrative expenses was primarily attributable to a loss incurred on a non-cash common stock settlement and increases in pre-commercial marketing expenses, salaries and personnel-related costs driven by increased headcount across all general and administrative functions to support our overall growth, and legal, professional and consulting fees and other administrative costs. - Net Loss: Net loss was
$81.7 million , or$2.55 per share, for the fourth quarter of 2017, compared to a net loss of$25.4 million , or$0.92 per share, for the fourth quarter of 2016.
Selected Financial Information
| Operating Results (In thousands, except per share data): | |||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2017 | 2016 | 2017 | 2016 | ||||||||||||
| Revenue | $ | - | $ | - | $ | - | $ | - | |||||||
| Operating Expenses: | |||||||||||||||
| General and administrative | 31,078 | 7,198 | 69,976 | 24,523 | |||||||||||
| Research and development | 51,445 | 18,350 | 150,391 | 58,892 | |||||||||||
| Total Operating Expenses | 82,523 | 25,548 | 220,367 | 83,415 | |||||||||||
| Loss from operations | (82,523 | ) | (25,548 | ) | (220,367 | ) | (83,415 | ) | |||||||
| Interest income | 858 | 173 | 2,316 | 403 | |||||||||||
| Net Loss | $ | (81,665 | ) | $ | (25,375 | ) | $ | (218,051 | ) | $ | (83,012 | ) | |||
| Weighted-average basic and diluted common shares outstanding | 32,030 | 27,678 | 29,935 | 22,648 | |||||||||||
| Basic and diluted net loss per common share | $ | (2.55 | ) | $ | (0.92 | ) | $ | (7.28 | ) | $ | (3.67 | ) | |||
| Balance Sheet Information (In thousands): | |||||||||||||||
| December 31, | December 31, | ||||||||||||||
| 2017 | 2016 | ||||||||||||||
| Cash and cash equivalents | $ | 324,117 | $ | 240,430 | |||||||||||
| Total assets | 391,578 | 270,575 | |||||||||||||
| Total liabilities | 58,838 | 24,443 | |||||||||||||
| Accumulated deficit | $ | (359,613 | ) | $ | (141,562 | ) | |||||||||
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 1599399. The webcast can be accessed live on the Events and Presentations page in the Investors and Media section of the
About SMA
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 and 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. SMA Type 2 typically presents between six and 18 months of age, and those affected will never walk without support and most will never stand without support. SMA Type 2 results in mortality in more than 30 percent of patients by the age of 25.
About Rett Syndrome
Rett syndrome is a devastating, rare neurological disorder characterized by slowed growth, loss of normal movement and coordination and loss of communication skills. Rett syndrome is caused by an X-linked dominant mutation in the methyl CpG binding protein 2 (MECP2) gene, which results in problems with MECP2 protein production critical for brain development. Rett syndrome occurs in approximately one of every 10,000 female births in the U.S., and affected infants usually begin to show signs and symptoms 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 Sclerosis
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects motor neurons in the brain and the spinal cord. Inherited forms of ALS comprise five to 10 percent of ALS cases, or approximately 1,000 to 2,000 people in the U.S., and can be caused by mutations in several genes known to be associated with ALS. Approximately 15 to 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 AVXS-101
AveXis' initial product candidate, AVXS-101, is its proprietary gene therapy currently in development for the one-time treatment of SMA Types 1 and 2, 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
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' clinical 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 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.
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