Full Press Release Details
REGENXBIO Reports Fourth Quarter and Full-Year 2015 Financial Results and Recent Operational Progress
ROCKVILLE, Md., March 3, 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 reported financial results
for the fourth quarter and full year ended December 31, 2015. REGENXBIO also provided updates on lead program progress and reiterated its full-year 2016 cash burn guidance.
REGENXBIO has made significant progress over the past year toward our goal of changing the lives of people with severe diseases through gene therapy.
During 2015, we strengthened our leadership team, built out our manufacturing capabilities and raised more than $240 million in capital to advance our pipeline. At the end of 2015, in collaboration with our development partners at the University of
Pennsylvania, an IND for our lead program, RGX-501, became active. We believe all of our lead product candidate programs remain on track based on our previous guidance and I am also excited to announce that we intend to file an IND application for
our fourth lead program, RGX-121 for the treatment of mucopolysaccharidosis Type II, in the first half of 2017, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. In 2016, we plan to focus on advancing our lead
gene therapy product candidates into the clinic, expanding our pipeline and continuing to grow the REGENXBIO team with a goal of delivering important gene therapy treatments in areas of unmet patient need.
Fourth Quarter and Full-Year 2015 Financial Results
2016 Financial Guidance
REGENXBIO s balance sheet
was strengthened during 2015, with aggregate gross proceeds of $100.5 million raised from two private financings as well as $145.2 million raised from its initial public offering, net of underwriting discounts and commissions and offering expenses.
REGENXBIO ended 2015 with $216.4 million in cash, cash equivalents and
marketable securities. In 2016, REGENXBIO expects its cash burn to be between $60 million and $70 million, which will support the continuing development of its lead product candidate programs and
the anticipated growth of its internal pipeline.
Additional Corporate Updates
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 December 31,
2015, REGENXBIO s NAV Technology Platform is being applied in the development of 28 product candidates for a variety of diseases, including five internally developed candidates and 23 partnered candidates developed by REGENXBIO s
Forward Looking Statements
release contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIO s expected cash burn for 2016, 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 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 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 its product candidates and technology; REGENXBIO s ability to establish and
maintain development partnerships, including those with NAV Technology Licensees; REGENXBIO s expectations regarding REGENXBIO s expenses and revenue, the sufficiency of REGENXBIO s cash resources and needs for additional
financing, REGENXBIO s expectations regarding federal, state and foreign regulatory requirements; 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2015, available on
the SEC s website at www.sec.gov. Additional factors may be described in those sections of REGENXBIO s Annual Report on Form 10-K for the year ended December 31, 2015, to be filed with the SEC in
the first quarter of 2016. In addition to the risks described above and in Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other 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.
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.
| As of December 31, | ||||||||
| 2015 | 2014 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 54,116 | $ | 1,121 | ||||
| Marketable securities | 60,025 | |||||||
| Accounts receivable | ||||||||
| Trade receivables | 2,136 | 805 | ||||||
| Related party receivables | 750 | |||||||
| Unbilled receivables | 327 | |||||||
| Prepaid expenses | 1,020 | 28 | ||||||
| Other current assets | 851 | |||||||
| Total current assets | 118,148 | 3,031 | ||||||
| Marketable securities | 102,226 | |||||||
| Property and equipment, net | 538 | |||||||
| Cost method investments | 300 | 303 | ||||||
| Deferred issuance costs | 157 | |||||||
| Other assets | 168 | |||||||
| Total assets | $ | 221,380 | $ | 3,491 | ||||
| Liabilities, Convertible Preferred Stock and Stockholders Equity (Deficit) | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 1,014 | $ | 334 | ||||
| Accrued expenses and other current liabilities | 3,198 | 1,115 | ||||||
| Due to related party under services agreement | 1,423 | |||||||
| Related party promissory notes | 2,403 | |||||||
| Other related party payables | 3,761 | |||||||
| Advance payments | 127 | 153 | ||||||
| Total current liabilities | 4,339 | 9,189 | ||||||
| Deferred rent, net of current portion | 233 | |||||||
| Total liabilities | 4,572 | 9,189 | ||||||
| Convertible preferred stock | 12,593 | |||||||
| Stockholders equity (deficit) | 216,808 | (18,291 | ) | |||||
| Total liabilities, convertible preferred stock and stockholders equity (deficit) | $ | 221,380 | $ | 3,491 |
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
thousands, except per share data)
| Three Months Ended December 31, | Years Ended December 31, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| Revenues | ||||||||||||||||
| License revenue | $ | 4,390 | $ | 650 | $ | 5,025 | $ | 4,355 | ||||||||
| License revenue from related party | $ | 70 | 2,000 | 220 | ||||||||||||
| Reagent sales | 48 | $ | 12 | 257 | 326 | |||||||||||
| Grant revenue | 3 | $ | 419 | 306 | 1,219 | |||||||||||
| Total revenues | 4,441 | $ | 1,151 | 7,588 | 6,120 | |||||||||||
| Expenses | ||||||||||||||||
| Costs of revenues | ||||||||||||||||
| Licensing costs (including amounts to related parties) | 878 | $ | 144 | 1,405 | 885 | |||||||||||
| Costs of reagent sales (including amounts to related parties) | 5 | $ | 8 | 98 | 122 | |||||||||||
| Research and development (including amounts to related parties) | 4,812 | $ | 2,089 | 17,279 | 4,961 | |||||||||||
| General and administrative (including amounts to related parties) | 4,232 | $ | 1,121 | 11,912 | 3,851 | |||||||||||
| Foreign currency transaction losses | 15 | $ | 19 | 57 | 30 | |||||||||||
| Other operating income | $ | (15 | ) | (26 | ) | (47 | ) | |||||||||
| Total operating expenses | 9,942 | $ | 3,366 | 30,725 | 9,802 | |||||||||||
| Loss from operations | (5,501 | ) | $ | (2,215 | ) | (23,137 | ) | (3,682 | ) | |||||||
| Other Income (Expense) | ||||||||||||||||
| Investment income | 323 | $ | 346 | |||||||||||||
| Interest expense | $ | (118 | ) | (20 | ) | (321 | ) | |||||||||
| Total other income (expense) | 323 | $ | (118 | ) | 326 | (321 | ) | |||||||||
| Net loss | $ | (5,178 | ) | $ | (2,333 | ) | $ | (22,811 | ) | $ | (4,003 | ) | ||||
| Other Comprehensive Loss | ||||||||||||||||
| Unrealized loss on available-for-sale securities | (693 | ) | $ | (719 | ) | |||||||||||
| Total other comprehensive loss | (693 | ) | $ | (719 | ) | |||||||||||
| Comprehensive loss | $ | (5,871 | ) | $ | (2,333 | ) | $ | (23,530 | ) | $ | (4,003 | ) | ||||
| Reconciliation of net loss to net loss applicable to common stockholders | ||||||||||||||||
| Net loss | $ | (5,178 | ) | $ | (2,333 | ) | $ | (22,811 | ) | $ | (4,003 | ) | ||||
| Accretion and dividends on convertible preferred stock | $ | (223 | ) | (1,747 | ) | (815 | ) | |||||||||
| Net gain on extinguishment of convertible preferred stock | $ | 759 | ||||||||||||||
| Net loss applicable to common stockholders | $ | (5,178 | ) | $ | (2,556 | ) | $ | (23,799 | ) | $ | (4,818 | ) | ||||
| Basic and diluted net loss per common share | $ | (0.20 | ) | $ | (0.97 | ) | $ | (2.59 | ) | $ | (1.82 | ) | ||||
| Weighted-average basic and diluted common shares | 26,312 | 2,644 | 9,173 | 2,643 |
Elizabeth Broder, 646-378-2945
Laura Bagby, 312-448-8098