Full Press Release Details
REGENXBIO Reports Second Quarter 2016 Financial Results and Recent Operational Highlights
ROCKVILLE, Md., August 9, 2016 -- REGENXBIO Inc. (Nasdaq: RGNX), a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy based on its proprietary NAV Technology Platform, today announced financial results for the quarter ended June 30, 2016 and recent operational highlights.
"This quarter has seen great progress in the advancement and expansion of the pipeline of gene therapies based on our NAV Technology Platform through our internal development efforts and the activities of our NAV Technology Licensees," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We look forward to enrolling the first patients in the RGX-501 Phase I/II clinical trial in the coming months, and expect to file INDs for our other three lead programs in 2017. Our NAV Technology Licensees continued their work to advance NAV-based gene therapies, with new progress reported by AveXis during the second quarter of 2016 in the treatment of spinal muscular atrophy using the NAV AAV9 vector, and the addition of our ninth licensee, Biogen, in a partnership that is focused on treatments for inherited blindness using NAV AAV8 and AAV9 vectors."
Recent Operational Highlights
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. REGENXBIO's NAV Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO's mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO's NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third-party NAV Technology Platform licensees. As of June 30, 2016, REGENXBIO's NAV Technology Platform was being applied in the development of 29 product candidates for a variety of diseases, including five internally developed candidates and 24 partnered candidates developed by REGENXBIO's licensees.
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, REGENXBIO's research, development and regulatory plans for RGX-111, RGX-121, RGX-314, RGX-501 and other gene therapies. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of REGENXBIO's development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Meaningful factors which could cause actual results to differ include, but are not limited to, the timing of enrollment, commencement and completion of REGENXBIO's clinical trials; the timing and success of preclinical studies and clinical trials conducted by REGENXBIO, its development partners and its NAV Technology Licensees; the ability to obtain and maintain regulatory approval to conduct clinical trials and to commercialize REGENXBIO's product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIO's product candidates; REGENXBIO's ability to obtain and maintain intellectual property protection for our product candidates and technology; trends and challenges in REGENXBIO's business and the markets in which REGENXBIO operates; REGENXBIO's ability to attract or retain key personnel; the size and growth of the potential markets for REGENXBIO's product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of REGENXBIO's product candidates; REGENXBIO's ability to establish and maintain development partnerships, including those with NAV Technology Licensees; REGENXBIO's expenses and revenue, the sufficiency of REGENXBIO's cash resources and needs for additional financing, regulatory developments in
the United States and foreign countries, as well as other factors discussed in 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, 2015 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2016, which are available on the SEC's website at www.sec.gov. Additional factors may be set forth in those sections of REGENXBIO's Form 10-Q for the quarter ended June 30, 2016, to be filed with the SEC in the third quarter of 2016. In addition to the risks described above and in REGENXBIO's filings with the SEC, other unknown or unpredictable factors also could affect REGENXBIO's results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. 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. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO 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). REGENXBIO 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.
| June 30, 2016 | December 31, 2015 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 30,083 | $ | 54,116 | ||||
| Marketable securities | 64,124 | 60,025 | ||||||
| Accounts receivable | 783 | 2,136 | ||||||
| Prepaid expenses | 1,480 | 1,020 | ||||||
| Other current assets | 1,437 | 851 | ||||||
| Total current assets | 97,907 | 118,148 | ||||||
| Marketable securities | 104,510 | 102,226 | ||||||
| Property and equipment, net | 2,864 | 538 | ||||||
| Cost method investments | 300 | 300 | ||||||
| Restricted cash | 225 | - | ||||||
| Other assets | 266 | 168 | ||||||
| Total assets | $ | 206,072 | $ | 221,380 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 2,076 | $ | 1,014 | ||||
| Accrued expenses and other current liabilities | 7,073 | 3,198 | ||||||
| Advance payments | - | 127 | ||||||
| Total current liabilities | 9,149 | 4,339 | ||||||
| Deferred rent, net of current portion | 748 | 233 | ||||||
| Total liabilities | 9,897 | 4,572 | ||||||
| Stockholders' equity | 196,175 | 216,808 | ||||||
| Total liabilities and stockholders' equity | $ | 206,072 | $ | 221,380 |
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(in thousands, except per share data)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2016 | 2015 | 2016 | 2015 | |||||||||||||
| Revenues | ||||||||||||||||
| License revenue | $ | 2,245 | $ | 470 | $ | 2,573 | $ | 570 | ||||||||
| License revenue from related party | - | 1,000 | - | 1,000 | ||||||||||||
| Reagent sales | 107 | 44 | 166 | 148 | ||||||||||||
| Grant revenue | 23 | (151 | ) | 29 | 289 | |||||||||||
| Total revenues | 2,375 | 1,363 | 2,768 | 2,007 | ||||||||||||
| Expenses | ||||||||||||||||
| Costs of revenues | ||||||||||||||||
| Licensing costs (including amounts to related parties) | 449 | 294 | 515 | 314 | ||||||||||||
| Costs of reagent sales (including amounts to related parties) | 49 | 16 | 79 | 49 | ||||||||||||
| Research and development (including amounts to related parties) | 10,680 | 4,012 | 16,863 | 6,803 | ||||||||||||
| General and administrative (including amounts to related parties) | 6,169 | 3,397 | 11,648 | 5,113 | ||||||||||||
| Other operating expenses (income) | (20 | ) | (60 | ) | (134 | ) | 17 | |||||||||
| Total operating expenses | 17,327 | 7,659 | 28,971 | 12,296 | ||||||||||||
| Loss from operations | (14,952 | ) | (6,296 | ) | (26,203 | ) | (10,289 | ) | ||||||||
| Other Income (Expense) | ||||||||||||||||
| Investment income | 515 | 6 | 998 | 8 | ||||||||||||
| Interest expense | - | - | - | (20 | ) | |||||||||||
| Total other income (expense) | 515 | 6 | 998 | (12 | ) | |||||||||||
| Net loss | $ | (14,437 | ) | $ | (6,290 | ) | $ | (25,205 | ) | $ | (10,301 | ) | ||||
| Other Comprehensive Income | ||||||||||||||||
| Unrealized gain on available-for-sale securities | 246 | - | 1,240 | - | ||||||||||||
| Total other comprehensive income | 246 | - | 1,240 | - | ||||||||||||
| Comprehensive loss | $ | (14,191 | ) | $ | (6,290 | ) | $ | (23,965 | ) | $ | (10,301 | ) | ||||
| Reconciliation of net loss to net loss applicable to common stockholders | ||||||||||||||||
| Net loss | $ | (14,437 | ) | $ | (6,290 | ) | $ | (25,205 | ) | $ | (10,301 | ) | ||||
| Net accretion and dividends on convertible preferred stock | - | (2,502 | ) | - | (1,747 | ) | ||||||||||
| Net gain on extinguishment of convertible preferred stock | - | - | - | 759 | ||||||||||||
| Net loss applicable to common stockholders | $ | (14,437 | ) | $ | (8,792 | ) | $ | (25,205 | ) | $ | (11,289 | ) | ||||
| Basic and diluted net loss per common share | $ | (0.55 | ) | $ | (3.24 | ) | $ | (0.96 | ) | $ | (4.21 | ) | ||||
| Weighted-average basic and diluted common shares | 26,362 | 2,712 | 26,344 | 2,679 |
Elizabeth Broder, 646-378-2945
Laura Bagby, 312-448-8098