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-- Initial public offering raised
-- Encouraging interim data for lead clinical program, AVXS-101, for Spinal Muscular Atrophy Type 1 –
-- Conference call
“The past six months has been a period of significant progress for
- Initial Public Offering: In the first quarter of 2016,
AveXiscompleted its initial public offering of 5,277,941 shares of common stock at an initial public offering price of $20.00per share, including a partial exercise of the over-allotment option by the underwriters of 527,941 shares, before underwriting discounts. The total estimated net proceeds from the initial public offering were approximately $98.2 million, after deducting underwriting discounts and estimated offering expenses.
- Enrollment completed in Phase 1 Trial of AVXS-101 for SMA Type 1: The trial enrolled a total of 15 patients who met enrollment criteria of clinical symptoms with diagnosis of SMA Type 1 before 6 months of age, with gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and two copies of the SMN2 backup gene, as determined by genetic testing. The trial includes two dosing cohorts: Cohort 1 of three patients dosed at the low dose of 6.7 X1013 vg/kg, aged six to seven months at time of dosing; Cohort 2 of 12 patients dosed at the proposed therapeutic dose of 2.0 X1014 vg/kg, aged one to eight months at time of dosing.
- Interim Phase 1 Data: In
January 2016, AveXisreported interim data through the December 31, 2015time period from the ongoing Phase 1 trial of AVXS-101 in SMA Type 1. The data reported in this release reflects that same dataset and time period. AveXisreported that AVXS-101 appears to have a favorable safety profile and appears to be generally well tolerated in patients studied as of December 31, 2015. As of that date, there had been a total of 10 serious adverse events (SAEs) reported; two of these were determined to be related to therapy and involved clinically asymptomatic, elevated liver function enzymes. Both cases have resolved.
December 31, 2015, no patient in either dosing cohort had experienced an “event.” An event is defined as death, or until a patient requires at least 16 hours per day of ventilation support for breathing for 14 consecutive days in the absence of an acute reversible illness, or perioperatively. The median event-free age of all 15 patients was 11.8 months, with the oldest patient at 25.8 months of age. In Cohort 1, all 3 patients were over 20 months of age and event-free. In Cohort 2, the first 6 patients to be treated were greater than 10.5 months of age and event-free.
December 31, 2015, mean increases of 6.0 points and 18.0 points in CHOP-INTEND scores were observed in Cohort 1 and Cohort 2, respectively. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) is a test developed to measure motor skills of patients with SMA Type 1, and other motor milestone development surveys and tests.
- Board of Directors Appointments: In
December 2015and January 2016, AveXisexpanded the board of directors with the appointments of Daniel G. Welch(Non-executive Chairman of the Board), currently an executive partner at Sofinnova Venturesand former Chairman, CEO and President of InterMune, Inc.; Terrence C. Kearney, former Chief Operating Officer for Hospira, Inc.where he previously held the position of Chief Financial Officer; and Frank Verwiel, M.D., formerly President, Chief Executive Officer and on the Board of Directors for Aptalis Pharma (formerly known as Axcan Pharma).
Upcoming Clinical Development Milestones
- Provide quarterly updates on the ongoing Phase 1 trial of AVXS-101 in SMA Type 1, starting in the first quarter of 2016.
- Initiate a Phase 1 safety and dosing study of AVXS-101 via intrathecal (IT) delivery in patients with SMA Type 2 in the second half of 2016.
- Report 13.6 months of data for all patients in the ongoing SMA Type 1 Phase 1 trial in the first quarter of 2017.
- Initiate pivotal trials of AVXS-101 in patients with SMA Type 1 in
the United Statesand Europein the first half of 2017.
Fourth Quarter 2015 Financial Results
- Cash Position: As of
December 31, 2015, AveXishad approximately $62.3 millionin cash, cash equivalents and marketable securities, which did not include total net proceeds of approximately $98.2 millionfrom the company's initial public offering of common stock in February 2016.
- R&D Expenses: Research and development expenses were
$8.7 millionfor the fourth quarter of 2015, compared to $3.4 millionfor the same period in 2014, an increase of $5.3 million. The increase in research and development expenses was primarily attributable to a $3.7 millionincrease in employee compensation and benefit expense and a $1.6 millionincrease in expenses necessary to support the advancement of our clinical and pre-clinical programs, primarily the Phase 1 trial of AVXS-101 in SMA Type 1.
- G&A Expenses: General and administrative expenses were
$4.4 millionfor the fourth quarter of 2015, compared to $0.3 millionfor the same period in 2014, an increase of $4.1 million. The increase in general and administrative expenses was primarily attributable to an increase in employee compensation and benefit expense to support our overall growth and expenses related to preparing for the initial public offering.
- Net Loss: Net loss was
$13.2 millionfor the fourth quarter of 2015, compared to net loss of $3.7 millionfor the fourth quarter of 2014. The net loss for the full year 2015 was $38.5 million, compared with a net loss of $15.7 millionin 2014.
Conference Call at
Analysts and investors can participate in the conference call by dialing (877) 508-0547 for domestic callers and (281) 973-6085 for international callers, using the conference ID 62607679. The webcast can be accessed live on the Events and Presentations page in the Investors and Media section of the
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 expected timing for initiating and reporting results from ongoing and planned clinical trials. 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 ability to obtain and maintain regulatory approval of AveXis’ product candidates; the scope, progress, expansion, and costs of developing and commercializing AveXis’ product candidates; AveXis’ ability to obtain and maintain intellectual property protection for our product candidates; AveXis’ ability to establish and maintain development partnerships; AveXis’ expectations regarding federal, state and foreign regulatory requirements; regulatory developments in
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein.
|Selected Financial Information|
|Consolidated Statement of Operations:|
|Three Months Ended||Twelve Months Ended|
|December 31,||December 31,|
|Cost of goods sold|
|General and administrative||4,428,279||268,598||11,079,512||1,869,899|
|Research and development||8,737,246||3,383,411||27,493,460||13,550,422|
|Total Operating Expenses||13,165,525||3,652,009||38,572,972||15,420,321|
|Loss from operations||(13,165,525||)||(3,652,009||)||(38,572,972||)||(15,420,321||)|
|Interest (income) expense||(4,660||)||(1,143||)||(14,570||)||131,527|
|Total Other (Income) Expense||(4,718||)||(1,143||)||(99,128||)||131,527|
|Loss from continuing operations, before tax||(13,160,807||)||(3,650,866||)||(38,473,844||)||(15,551,848||)|
|Income tax expense (benefit)||-||-||-||-|
|Loss from continuing operations||(13,160,807||)||(3,650,866||)||(38,473,844||)||(15,551,848||)|
|Loss from operations of discontinued cell banking business, net of tax||-||-||-||153,928|
|Condensed Consolidated Balance Sheets:|
|As of December 31,|
|Balance Sheet Data:||2015||2014|
|Cash and cash equivalents||62,251,860||3,119,713|
|Total stockholders' deficit||(58,550,520||)||(20,076,619||)|
Lauren Barbiero W2O Group646-564-2156 firstname.lastname@example.org Investor Inquiries: Jim Goff AveXis, Inc.650-862-4134 email@example.com