Full Press Release Details
REGENXBIO Reports First Quarter 2016 Financial Results and Recent Operational Highlights
ROCKVILLE, Md., May 5, 2016 REGENXBIO Inc. (REGENXBIO or the Company) (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 and recent operational highlights for the quarter ended March 31, 2016.
first quarter of 2016, we made significant progress toward this year s goals of initiating two clinical studies and filing an Investigational New Drug application to support the initiation of a third study, said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. Currently, REGENXBIO and our partners at the University of Pennsylvania are recruiting participants in the Phase I/II clinical trial of RGX-501 for the treatment of homozygous familial
hypercholesterolemia. In addition, we are completing steps that will enable us to file an Investigational New Drug application for RGX-111 for the treatment of Mucopolysaccharidosis Type I. REGENXBIO also continues to build our team and capabilities
to support the activities that will best position us to advance our mission of developing and commercializing gene therapy products that transform the lives of people suffering from severe diseases.
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 March 31, 2016, REGENXBIO s NAV Technology Platform was 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 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 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 Annual Report on Form 10-K for the year
ended December 31, 2015, available on the SEC s website at www.sec.gov. Additional factors may be described in those sections of REGENXBIO s Quarterly Report on Form 10-Q for the quarter ended March 31,
2016, to be filed with the SEC in the second 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.
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
thousands, except per share data)
| March 31, | December 31, | |||||||
| 2016 | 2015 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 35,511 | $ | 54,116 | ||||
| Marketable securities | 65,544 | 60,025 | ||||||
| Accounts receivable | 2,036 | 2,136 | ||||||
| Prepaid expenses | 1,189 | 1,020 | ||||||
| Other current assets | 1,300 | 851 | ||||||
| Total current assets | 105,580 | 118,148 | ||||||
| Marketable securities | 107,553 | 102,226 | ||||||
| Property and equipment, net | 1,328 | 538 | ||||||
| Cost method investments | 300 | 300 | ||||||
| Other assets | 223 | 168 | ||||||
| Total assets | $ | 214,984 | $ | 221,380 | ||||
| Liabilities and Stockholders Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 1,113 | $ | 1,014 | ||||
| Accrued expenses and other current liabilities | 4,633 | 3,198 | ||||||
| Advance payments | 35 | 127 | ||||||
| Total current liabilities | 5,781 | 4,339 | ||||||
| Deferred rent, net of current portion | 602 | 233 | ||||||
| Total liabilities | 6,383 | 4,572 | ||||||
| Stockholders equity | 208,601 | 216,808 | ||||||
| Total liablities and stockholders equity | $ | 214,984 | $ | 221,380 |
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
thousands, except per share data)
| Three Months Ended March 31, | ||||||||
| 2016 | 2015 | |||||||
| Revenues | ||||||||
| License revenue | $ | 328 | $ | 100 | ||||
| Reagent sales | 59 | 104 | ||||||
| Grant revenue | 6 | 440 | ||||||
| Total revenues | 393 | 644 | ||||||
| Expenses | ||||||||
| Costs of revenues | ||||||||
| Licensing costs (including amounts to related parties) | 66 | 20 | ||||||
| Costs of reagent sales (including amounts to related parties) | 30 | 33 | ||||||
| Research and development (including amounts to related parties) | 6,183 | 2,791 | ||||||
| General and administrative (including amounts to related parties) | 5,479 | 1,716 | ||||||
| Other operating expenses (income) | (114 | ) | 77 | |||||
| Total operating expenses | 11,644 | 4,637 | ||||||
| Loss from operations | (11,251 | ) | (3,993 | ) | ||||
| Other Income (Expense) | ||||||||
| Investment income | 483 | 2 | ||||||
| Interest expense | (20 | ) | ||||||
| Total other income (expense) | 483 | (18 | ) | |||||
| Net loss | $ | (10,768 | ) | $ | (4,011 | ) | ||
| Other Comprehensive Income | ||||||||
| Unrealized gain on available-for-sale securities | 994 | |||||||
| Total other comprehensive income | 994 | |||||||
| Comprehensive loss | $ | (9,774 | ) | $ | (4,011 | ) | ||
| Reconciliation of net loss to net loss applicable to common stockholders | ||||||||
| Net loss | $ | (10,768 | ) | $ | (4,011 | ) | ||
| Net decretion and dividends on convertible preferred stock | 755 | |||||||
| Net gain on extinguishment of convertible preferred stock | 759 | |||||||
| Net loss applicable to common stockholders | $ | (10,768 | ) | $ | (2,497 | ) | ||
| Basic and diluted net loss per common share | $ | (0.41 | ) | $ | (0.94 | ) | ||
| Weighted-average basic and diluted common shares | 26,327 | 2,645 |
Laura Bagby, 312-448-8098