Full Press Release Details
REGENXBIO Reports Fourth Quarter and Full-Year 2018 Financial Results
and Recent Operational Highlights
ROCKVILLE, Md., Feb. 27, 2019 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced financial results for the fourth quarter and full year ended December 31, 2018, and recent operational highlights.
"2018 was a pivotal year for REGENXBIO. Significant clinical and regulatory progress was made in advancing our NAV Technology Platform across 18 diverse clinical stage programs, four of which are in our own expanding pipeline of gene therapy product candidates for the treatment of retinal, neurodegenerative and metabolic diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "In 2019, we are focused on advancing and expanding our ophthalmology franchise with RGX-314, our lead product candidate for subjects with wet AMD, by extending development of this asset as a one-time anti-VEGF treatment in another chronic retinal condition. In addition, we anticipate the launch of the first NAV Technology-based treatment, Novartis' ZOLGENSMA for the treatment of SMA Type I, in the United States, Japan and Europe in 2019. This would be a major commercial milestone for our NAV Technology Platform and represent a post-launch commercial revenue stream for REGENXBIO."
Mr. Mills added: "Our mission is to improve lives through the curative potential of gene therapy. We continue to execute on our strategic priorities including the expansion of RGX-314 development into additional retinal conditions, advancing our current clinical programs with RGX-314, RGX-121, RGX-111 and RGX-501 and enabling our NAV Technology licensee network to develop potentially life-changing treatments. We believe we are well-positioned for a transformative 2019 as we build on last year's pivotal achievements and broaden our NAV Technology footprint."
Recent Operational Highlights
NAV Technology Licensee Program Highlights
As of December 31, 2018, REGENXBIO's NAV Technology Platform was being applied in more than 20 partnered product candidates in development by NAV Technology Licensees. Fourteen of these partnered product candidates are in active clinical development, and one partnered product candidate has been submitted for Biologics License Application (BLA) approval with the FDA. Over 100 subjects have been treated in clinical trials sponsored by NAV Technology Licensees. REGENXBIO's NAV Technology Licensees are advancing product candidates in a broad range of therapeutic areas and disease indications, including two clinical trials started in 2018 in hemophilia A sponsored by Takeda Pharmaceutical Company Limited and Bayer AG. Recent updates from NAV Technology Licensees include:
Cash, cash equivalents and marketable securities were $470.6 million as of December 31, 2018, compared to $176.4 million as of December 31, 2017. Cash, cash equivalents and marketable securities as of December 31, 2018 include $180.0 million received in 2018 in connection with the amendment to the license agreement with AveXis, Inc. for the development and commercialization of treatments for SMA, as well as $189.1 million of aggregate net proceeds from a follow-on public offering of common stock completed in August 2018.
Revenues were $40.8 million and $218.5 million for the three months and year ended December 31, 2018, respectively, compared to $2.0 million and $10.4 million for the three months and year ended December 31, 2017, respectively. The increases in revenue were primarily attributable to $176.1 million of revenue recognized in 2018 under the amended license agreement with AveXis for the development and commercialization of treatments for SMA, as well as $35.6 million of revenue recognized in the fourth quarter of 2018 under the license agreement with Abeona for the development and commercialization of treatments for various diseases.
Research and development expenses were $24.3 million and $83.9 million for the three months and year ended December 31, 2018, respectively, compared to $14.2 million and $57.2 million for the three months and year ended December 31, 2017, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs and expenses associated with conducting clinical trials and externally sourced manufacturing-related services.
General and administrative expenses were $11.1 million and $36.9 million for the three months and year ended December 31, 2018, respectively, compared to $4.8 million and $27.2 million for the three months and year ended December 31, 2017, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services.
Net income was $4.3 million, or $0.12 basic and $0.11 diluted net income per share, and $99.9 million, or $2.99 basic and $2.73 diluted net income per share, for the three months and year ended December 31, 2018, respectively, compared to net losses of $16.0 million, or $0.51 basic and diluted net loss per share, and $73.2 million, or $2.45 basic and diluted net loss per share, for the three months and year ended December 31, 2017, respectively.
Based on its current operating plan, and excluding any commercial revenue from Novartis' ZOLGENSMA, subject to approval by regulatory authorities, REGENXBIO reiterates that it expects its balance in cash, cash equivalents and marketable securities to be between $330 million and $350 million as of December 31, 2019, which will be used to support the continued development of its lead product candidate programs. Importantly, REGENXBIO anticipates adding commercial revenue from ZOLGENSMA to its existing base of partner revenue this year, pending approval by regulatory authorities.
In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. To access the live call by phone, dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 1564206. To access a live or recorded webcast of the call and
accompanying slides, please visit the "Investors" section of the REGENXBIO website at www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
Forward-Looking Statements
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2018, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
CONSOLIDATED BALANCE SHEETS
(in thousands, except per share data)
| December 31, 2018 | December 31, 2017 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 75,561 | $ | 46,656 | ||||
| Marketable securities | 244,200 | 114,122 | ||||||
| Accounts receivable | 8,587 | 473 | ||||||
| Prepaid expenses | 5,734 | 5,334 | ||||||
| Other current assets | 3,831 | 1,412 | ||||||
| Total current assets | 337,913 | 167,997 | ||||||
| Marketable securities | 150,819 | 15,616 | ||||||
| Accounts receivable | 23,012 | - | ||||||
| Property and equipment, net | 28,702 | 13,977 | ||||||
| Restricted cash | 1,053 | 225 | ||||||
| Other assets | 2,315 | 862 | ||||||
| Total assets | $ | 543,814 | $ | 198,677 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 4,412 | $ | 4,832 | ||||
| Accrued expenses and other current liabilities | 17,164 | 9,605 | ||||||
| Deferred revenue | 600 | - | ||||||
| Total current liabilities | 22,176 | 14,437 | ||||||
| Deferred revenue | 3,333 | - | ||||||
| Deferred rent, net of current portion | 1,098 | 1,211 | ||||||
| Financing lease obligation | 5,854 | - | ||||||
| Other liabilities | 2,505 | - | ||||||
| Total liabilities | 34,966 | 15,648 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at December 31, 2018 and December 31, 2017 | - | - | ||||||
| Common stock; $0.0001 par value; 100,000 shares authorized at December 31, 2018 and December 31, 2017; 36,120 and 31,295 shares issued and outstanding at December 31, 2018 and December 31, 2017, respectively | 4 | 3 | ||||||
| Additional paid-in capital | 592,580 | 371,497 | ||||||
| Accumulated other comprehensive loss | (720 | ) | (715 | ) | ||||
| Accumulated deficit | (83,016 | ) | (187,756 | ) | ||||
| Total stockholders' equity | 508,848 | 183,029 | ||||||
| Total liabilities and stockholders' equity | $ | 543,814 | $ | 198,677 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(in thousands, except per share data)
| Three Months Ended December 31, | Years Ended December 31, | |||||||||||||||
| 2018 | 2017 | 2018 | 2017 | |||||||||||||
| Revenues | ||||||||||||||||
| License revenue | $ | 40,777 | $ | 2,040 | $ | 218,505 | $ | 10,385 | ||||||||
| Other revenues | - | - | - | 8 | ||||||||||||
| Total revenues | 40,777 | 2,040 | 218,505 | 10,393 | ||||||||||||
| Operating Expenses | ||||||||||||||||
| Costs of revenues | ||||||||||||||||
| Licensing costs | 2,843 | (382 | ) | 9,640 | 1,703 | |||||||||||
| Other | - | - | - | 6 | ||||||||||||
| Research and development | 24,329 | 14,170 | 83,873 | 57,224 | ||||||||||||
| General and administrative | 11,144 | 4,808 | 36,850 | 27,229 | ||||||||||||
| Other operating expenses | 11 | 42 | 42 | 116 | ||||||||||||
| Total operating expenses | 38,327 | 18,638 | 130,405 | 86,278 | ||||||||||||
| Income (loss) from operations | 2,450 | (16,598 | ) | 88,100 | (75,885 | ) | ||||||||||
| Other Income | ||||||||||||||||
| Interest income from licensing | 584 | - | 8,946 | - | ||||||||||||
| Investment income | 2,893 | 601 | 7,070 | 2,716 | ||||||||||||
| Total other income | 3,477 | 601 | 16,016 | 2,716 | ||||||||||||
| Income (loss) before income taxes | 5,927 | (15,997 | ) | 104,116 | (73,169 | ) | ||||||||||
| Income Tax Expense | (1,621 | ) | - | (4,179 | ) | - | ||||||||||
| Net income (loss) | $ | 4,306 | $ | (15,997 | ) | $ | 99,937 | $ | (73,169 | ) | ||||||
| Other Comprehensive Income (Loss) | ||||||||||||||||
| Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 154 | (161 | ) | (5 | ) | (682 | ) | |||||||||
| Total other comprehensive income (loss) | 154 | (161 | ) | (5 | ) | (682 | ) | |||||||||
| Comprehensive income (loss) | $ | 4,460 | $ | (16,158 | ) | $ | 99,932 | $ | (73,851 | ) | ||||||
| Net income (loss) per share: | ||||||||||||||||
| Basic | $ | 0.12 | $ | (0.51 | ) | $ | 2.99 | $ | (2.45 | ) | ||||||
| Diluted | $ | 0.11 | $ | (0.51 | ) | $ | 2.73 | $ | (2.45 | ) | ||||||
| Weighted-average common shares outstanding: | ||||||||||||||||
| Basic | 35,951 | 31,178 | 33,427 | 29,878 | ||||||||||||
| Diluted | 38,933 | 31,178 | 36,648 | 29,878 |
Heather Savelle, 212-600-1902
David Rosen, 212-600-1902