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-- Significant progress across operating functions including licensing, clinical trials and regulatory interactions --
-- AVXS-101 pre-BLA meeting with
“Throughout the first quarter and in recent weeks, we have made significant progress in executing against our strategic plan and are quite pleased that
Recent Corporate Highlights
Entered Agreement to be Acquired by
Presented Data at the 2018 Annual Meeting of the
- STR1VE Data as of
April 11, 2018
- Eleven patients were enrolled in the trial, and six patients were symptomatic and at least one-month post gene therapy treatment.
- All symptomatic patients (6 of 6) were alive and event-free. AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated. At the time of gene transfer, the mean age was 3.2 months, with the oldest patient being 5.0 months of age.
- In the six patients who were at least one-month post gene transfer, a cumulative total of 25 adverse events (AEs) were reported. Two patients experienced transient elevations in transaminases greater than 3x ULN that were not clinically significant and all resolved with prednisolone treatment without any clinical manifestations or sequelae. There were no serious adverse events (SAEs) reported.
- Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) scores increased by an average of 7.8 at one month after gene transfer (in six patients) and 17.3 at three months after gene transfer (in three patients), reflecting improvement in motor function. These data correlate to CHOP-INTEND achievement by the proposed therapeutic dose cohort (Cohort 2) in the Phase 1 trial, which experienced mean increases of 9.8 points at one month and 15.4 points at three months. Early CHOP-INTEND increases have been observed to be associated with eventual milestone achievement.
- 24-Month Follow-Up Data from Phase 1 Trial
- Twenty-four months following gene transfer, 15 of 15 (100%) patients were alive and without need for permanent ventilation.
- The median age at last follow-up was 27.8 months and 30.7 months for patients in Cohort 2 and low-dose cohort (Cohort 1), respectively. Natural history indicates only eight percent of untreated patients with SMA Type 1 survive event-free at 20 months of age. After the 24-month follow-up, to date, 11 patients have enrolled in the Long-Term Follow-Up (LTFU) trial for ongoing evaluation.
- Patients in Cohort 2 continued to achieve new milestones during the LTFU trial.
- Two additional patients achieved the ability to sit unassisted for 30 seconds or more. Eleven of 12 (92%) patients could sit unassisted.
- Two additional patients achieved the ability to stand with assistance. Four of 12 (33%) patients could stand with assistance.
- Three of the four patients achieving these new milestones were on AVXS-101 alone (one sitting and two standing with assistance).
- The oldest child from Cohort 2 at the time of last visit in the LTFU study was 46.2 months old and 40.6 months post gene therapy.
- AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new treatment-related safety or tolerability concerns identified at the 24-month follow-up.
Dosed Fourth Patient in Phase 1 Trial of AVXS-101 in SMA Type 2 (STRONG): Following review of safety data from the first three patients dosed in Cohort 1 using the lower dose (6.0 x 1013 vg), the first patient has been dosed in Cohort 2, the higher dose cohort (1.2 X 1014 vg). Four total patients with SMA Type 2 have now been treated with AVXS-101 in the STRONG study. Three patients less than 60 months of age will be enrolled in Cohort 2 and, if safety is established according to the Data Safety Monitoring Board, an additional 21 patients will be enrolled in Cohort 2 until there are a total of 12 patients less than 24 months, and 12 patients at least 24 months but less than 60 months of age.
Initiated Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3 Trial (
Awarded SAKIGAKE Designation for SMA Type 1: On
Entered into Licensing Agreement with Généthon: On
Expanded Relationship with
Underwritten Public Offering
- Pre-BLA Meeting with
FDA: Meeting is scheduled for June 2018. AveXisexpects to provide an update when the final meeting minutes are received, currently anticipated to be late July or early August 2018.
- Pivotal Trial of AVXS-101 in SMA Type 1 in
Europe(STR1VE EU): Expected to initiate in the first half of 2018.
- Phase 3 Trial of AVXS-101 in 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.
First Quarter 2018 Financial Results
- Cash Position: As of
March 31, 2017, AveXishad $586.8 millionin cash and cash equivalents.
- R&D Expenses: Research and development expenses were
$199.7 millionfor the first quarter of 2018 (which included $7.4 millionof non-cash stock-based compensation expense), compared to $20.3 millionfor the same period in 2017 (which included $2.3 millionof non-cash stock-based compensation expense), an increase of $179.4 million. The increase in research and development expenses was primarily attributable to fees associated with our licensing agreements, specifically the agreement with REGENXBIO(including the amendment thereto), for which $130.7 millionof expense was recognized in the first quarter of 2018. Increased spending was also attributable to increased headcount, further development of our manufacturing facility and our STR1VE and STRONG clinical trial starts.
- G&A Expenses: General and administrative expenses were
$22.7 millionfor the first quarter of 2018 (which included $5.6 millionof non-cash stock-based compensation expense), compared to $9.6 millionfor the same period in 2017 (which included $2.8 millionof stock-based compensation expense), an increase of $13.1 million. The increase in general and administrative expenses was primarily attributable to increases in headcount and personnel related costs as well as market research and professional fees.
- Net Loss: Net loss was
$222.1 million, or $6.20per share, for the first quarter of 2018, compared to a net loss of $29.7 million, or $1.07per share, for the first quarter of 2017.
|Selected Financial Information|
|Operating Results (In thousands, except per share data):|
|Three Months Ended March 31,|
General and administrative
Research and development
|Total operating expenses||222,456||29,965|
|Loss from operations||(222,456||)||(29,965||)|
|Interest income, net||354||246|
|Weighted-average basic and diluted common shares outstanding||35,842||27,734|
|Basic and diluted net loss per common share||$||(6.20||)||$||(1.07||)|
|Balance Sheet Information (in thousands):|
|March 31,||December 31,|
|Cash and cash equivalents||$||586,808||$||324,117|
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. SMA Type 3 typically presents in early childhood to early adulthood, and those affected may lose the ability to walk over time. The incidence per live births among subtypes is approximately 60, 27 and 13 percent for SMA Type 1, Type 2 and Type 3, respectively.
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.
AveXis’ initial product candidate, AVXS-101, is its proprietary gene therapy currently in development for the one-time treatment of SMA Types 1, 2 and 3, 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 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 is for informational purposes only and does not constitute an offer to buy or a solicitation of an offer to sell any securities of
This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, the proposed acquisition of