Full Press Release Details
REGENXBIO Reports First Quarter 2020 Financial Results and Operational Highlights
ROCKVILLE, Md., May 7, 2020 (PRNewswire) -- 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 quarter ended March 31, 2020, and recent operational highlights.
"Our overall focus at REGENXBIO during the COVID-19 pandemic is on the health and safety of our people and our communities, as well as the important work that we need to continue in order to help patients," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We recently reported additional positive data from our RGX-314 Phase I/IIa wet AMD trial which demonstrated long-term, sustained clinical outcomes in patients after a one-time administration of RGX-314 gene therapy. We look forward to initiating a pivotal program for the subretinal delivery of RGX-314 for wet AMD as well as additional studies using the suprachoroidal delivery approach in both wet AMD and diabetic retinopathy in 2020. We also anticipate providing updates throughout 2020 from our other programs, including the Phase I/II trials of RGX-121 for the treatment of MPS II and RGX-501 for the treatment of HoFH."
Mr. Mills added: "We continue to track the positive progress of Novartis' Zolgensma which uses the NAV AAV9 vector for the treatment of pediatric patients with spinal muscular atrophy (SMA). We are encouraged by the recent regulatory updates from Japan and Europe, and we look forward to continued sales growth for Zolgensma. After more than a decade of steadfast effort and focus, we remain dedicated and committed to improving lives through the curative potential of gene therapy."
Recent Operational Highlights
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody Delivery
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
NAV Technology Licensee Program Highlights
As of March 31, 2020, REGENXBIO's NAV Technology Platform was being applied in one marketed product, and the clinical development of 15 partnered product candidates, with over 20 partnered programs in total. REGENXBIO's NAV Technology Licensees are advancing product candidates in a broad range of therapeutic areas and disease indications. Recent updates from NAV Technology Licensees include:
Marketed NAV Technology Product Highlights
Cash Position: Cash, cash equivalents and marketable securities were $356.6 million as of March 31, 2020, compared to $400.0 million as of December 31, 2019. The decrease was primarily attributable to net cash used in operating activities of $35.6 million and cash used to purchase property and equipment of $4.6 million.
Revenues: Revenues were $17.6 million for the three months ended March 31, 2020, compared to $0.9 million for the three months ended March 31, 2019. The increase was primarily attributable to $10.0 million of royalty revenue recognized on net sales of Zolgensma in the first quarter of 2020, as well as
$7.2 million of license revenue recognized from a new license granted to Ultragenyx Pharmaceutical Inc. during the period. Commercial sales of Zolgensma commenced in the second quarter of 2019, and REGENXBIO is eligible to receive a milestone payment of $80.0 million from AveXis, Inc. upon the achievement of $1.0 billion in cumulative net sales of Zolgensma.
Research and Development Expenses: Research and development expenses were $37.0 million for the three months ended March 31, 2020, compared to $25.2 million for the three months ended March 31, 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount, laboratory and facilities costs, expenses associated with conducting clinical trials for our lead product candidates and externally sourced services for preclinical, regulatory and manufacturing-related activities.
General and Administrative Expenses: General and administrative expenses were $14.8 million for the three months ended March 31, 2020, compared to $11.6 million for the three months ended March 31, 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount and professional fees for advisory and other services.
Net Loss: Net loss was $40.0 million, or $1.08 basic and diluted net loss per share, for the three months March 31, 2020, compared to net loss of $32.2 million, or $0.89 basic and diluted net loss per share, for the three months ended March 31, 2019.
Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $356.6 million to fund the completion of its internal manufacturing capabilities and clinical advancement of its product candidates into 2022.
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 3893881. 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, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, 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, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (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.
SCS Microinjector is a trademark of Clearside Biomedical, Inc. Zolgensma is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.
CONSOLIDATED BALANCE SHEETS
(in thousands, except per share data)
| March 31, 2020 | December 31, 2019 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 69,411 | $ | 69,514 | ||||
| Marketable securities | 209,846 | 226,696 | ||||||
| Accounts receivable | 44,522 | 38,148 | ||||||
| Prepaid expenses | 7,422 | 6,475 | ||||||
| Other current assets | 7,032 | 4,199 | ||||||
| Total current assets | 338,233 | 345,032 | ||||||
| Marketable securities | 77,361 | 103,785 | ||||||
| Accounts receivable | 4,373 | 4,155 | ||||||
| Property and equipment, net | 30,414 | 28,973 | ||||||
| Operating lease right-of-use assets | 9,375 | 10,078 | ||||||
| Restricted cash | 1,330 | 1,330 | ||||||
| Other assets | 2,850 | 4,555 | ||||||
| Total assets | $ | 463,936 | $ | 497,908 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 9,008 | $ | 6,409 | ||||
| Accrued expenses and other current liabilities | 20,327 | 24,846 | ||||||
| Operating lease liabilities | 2,454 | 2,421 | ||||||
| Total current liabilities | 31,789 | 33,676 | ||||||
| Deferred revenue | 3,333 | 3,333 | ||||||
| Operating lease liabilities | 7,990 | 8,874 | ||||||
| Other liabilities | 672 | 1,828 | ||||||
| Total liabilities | 43,784 | 47,711 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at March 31, 2020 and December 31, 2019 | - | - | ||||||
| Common stock; $0.0001 par value; 100,000 shares authorized at March 31, 2020 and December 31, 2019; 37,190 and 36,992 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively | 4 | 4 | ||||||
| Additional paid-in capital | 638,588 | 627,810 | ||||||
| Accumulated other comprehensive income (loss) | (580 | ) | 205 | |||||
| Accumulated deficit | (217,860 | ) | (177,822 | ) | ||||
| Total stockholders' equity | 420,152 | 450,197 | ||||||
| Total liabilities and stockholders' equity | $ | 463,936 | $ | 497,908 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(in thousands, except per share data)
| Three Months Ended March 31, | ||||||||
| 2020 | 2019 | |||||||
| Revenues | ||||||||
| License and royalty revenue | $ | 17,644 | $ | 884 | ||||
| Total revenues | 17,644 | 884 | ||||||
| Operating Expenses | ||||||||
| Cost of revenues | 3,409 | 29 | ||||||
| Research and development | 37,035 | 25,203 | ||||||
| General and administrative | 14,833 | 11,558 | ||||||
| Other operating expenses | 67 | - | ||||||
| Total operating expenses | 55,344 | 36,790 | ||||||
| Loss from operations | (37,700 | ) | (35,906 | ) | ||||
| Other Income (Loss) | ||||||||
| Interest income from licensing | 848 | 613 | ||||||
| Investment income (loss) | (3,186 | ) | 2,995 | |||||
| Total other income (loss) | (2,338 | ) | 3,608 | |||||
| Loss before income taxes | (40,038 | ) | (32,298 | ) | ||||
| Income Tax Benefit | - | 70 | ||||||
| Net loss | $ | (40,038 | ) | $ | (32,228 | ) | ||
| Other Comprehensive Income (Loss) | ||||||||
| Unrealized gain (loss) on available-for-sale securities, net | (785 | ) | 621 | |||||
| Total other comprehensive income (loss) | (785 | ) | 621 | |||||
| Comprehensive loss | $ | (40,823 | ) | $ | (31,607 | ) | ||
| Basic and diluted net loss per share | $ | (1.08 | ) | $ | (0.89 | ) | ||
| Weighted-average basic and diluted common shares outstanding | 37,104 | 36,366 |
Investor Relations and Corporate Communications
Heather Savelle, 212-600-1902
David Rosen, 212-600-1902