Full Press Release Details
REGENXBIO Reports Third Quarter 2021 Financial Results and Operational Highlights
ROCKVILLE, Md., November 2, 2021 (PR Newswire) -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the third quarter ended September 30, 2021, and recent operational highlights.
"We are encouraged by the clinical profile emerging from ongoing clinical trials evaluating the suprachoroidal delivery of RGX-314 for the treatment of wet AMD and diabetic retinopathy. Preliminary data highlight the potential of one-time, in-office delivery of RGX-314 which could provide sustainable, long-term anti-VEGF protein production in the eye for patients," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Moreover, our recently announced eye care collaboration with AbbVie, combining our AAV gene therapy expertise with AbbVie's global developmental and commercial infrastructure within eye care, will further support the broad potential of our RGX-314 program utilizing both subretinal and suprachoroidal delivery. We continue to expect this transaction to close by the end of this year."
Mr. Mills added: "For the remainder of 2021, we are focused on continuing to enroll patients in the ATMOSPHERETM trial and initiating our second pivotal trial for RGX-314 using subretinal delivery, sharing additional interim data from our AAVIATE trial and filing an IND for RGX-202 for the treatment of Duchenne muscular dystrophy. This year has been transformative for REGENXBIO, and I am grateful to our dedicated employees for their commitment to our mission to develop therapeutics for diseases that have significant unmet needs."
Eye Care Collaboration with AbbVie
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 September 30, 2021, REGENXBIO's NAV Technology Platform was being applied in one marketed product, and multiple clinical stage programs, 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 Products
REGENXBIO's NAV Technology Platform is being applied in one marketed product, Zolgensma . On October 26, 2021, Novartis AG reported third quarter 2021 global Zolgensma sales revenue of $375 million.
Cash Position: Cash, cash equivalents and marketable securities were $533.5 million as of September 30, 2021, compared to $522.5 million as of December 31, 2020. The increase was primarily attributable to $216.1 million of aggregate net proceeds received from the Company's follow-on public offering of common stock completed in January 2021, and was partially offset by net cash used in operating activities of $107.4 million, cash used to purchase property and equipment of $69.6 million, and
Zolgensma royalties paid to Healthcare Royalty Management, LLC of $33.3 million during the nine months ended September 30, 2021.
Revenues: Revenues were $30.8 million for the three months ended September 30, 2021, compared to $98.9 million for the three months ended September 30, 2020. The decrease was primarily attributable to an $80.0 million milestone fee recognized as revenue in the third quarter of 2020 upon the achievement of $1.0 billion of cumulative net sales of Zolgensma, and was partially offset by an increase in Zolgensma royalty revenues, which increased by $11.5 million, from $18.8 million for the third quarter of 2020 to $30.3 million for the third quarter of 2021. As reported by Novartis, sales of Zolgensma for the third quarter of 2021 increased by 29% as compared to the third quarter of 2020, driven by geographic expansion of product access.
Research and Development Expenses: Research and development expenses were $47.9 million for the three months ended September 30, 2021, compared to $44.0 million for the three months ended September 30, 2020. The increase was primarily attributable to personnel costs as a result of increased headcount, as well as laboratory and facilities costs.
General and Administrative Expenses: General and administrative expenses were $21.0 million for the three months ended September 30, 2021, compared to $15.9 million for the three months ended September 30, 2020. The increase was primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services
Net Loss: Net loss was $58.4 million, or $1.37 basic and diluted net loss per share, for the three months ended September 30, 2021, compared to net income of $8.8 million, or $0.24 basic and $0.23 diluted net income per share, for the three months ended September 30, 2020.
Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $533.5 million as of September 30, 2021 to fund its operations, including the completion of its internal manufacturing capabilities and clinical advancement of its product candidates, into the second half of 2023, excluding the effect of any potential payments that may be received under REGENXBIO's collaboration with AbbVie, which is expected to close by the end of 2021, subject to the satisfaction of customary closing conditions, including applicable regulatory approvals.
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 7878814. To access a live or recorded webcast of the call, 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," "assume," "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 proposed collaboration with AbbVie and REGENXBIO's future operations and clinical trials. 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 anticipated completion of REGENXBIO's proposed transaction with AbbVie, the outcome of REGENXBIO's proposed collaboration with AbbVie, 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, 2020 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. Except as required by law, 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.
Zolgensma is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
CONSOLIDATED BALANCE SHEETS
| September 30, 2021 | December 31, 2020 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 228,387 | $ | 338,426 | ||||
| Marketable securities | 111,473 | 137,314 | ||||||
| Accounts receivable, net | 46,017 | 42,999 | ||||||
| Prepaid expenses | 18,401 | 10,505 | ||||||
| Other current assets | 5,886 | 1,953 | ||||||
| Total current assets | 410,164 | 531,197 | ||||||
| Marketable securities | 193,640 | 46,809 | ||||||
| Accounts receivable, net | 2,730 | 3,267 | ||||||
| Property and equipment, net | 122,231 | 56,467 | ||||||
| Operating lease right-of-use assets | 61,742 | 63,815 | ||||||
| Restricted cash | 1,330 | 1,330 | ||||||
| Other assets | 8,558 | 5,279 | ||||||
| Total assets | $ | 800,395 | $ | 708,164 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 19,845 | $ | 10,622 | ||||
| Accrued expenses and other current liabilities | 49,694 | 49,082 | ||||||
| Deferred revenue | 395 | 449 | ||||||
| Operating lease liabilities | 1,329 | 2,500 | ||||||
| Liability related to sale of future royalties | 35,508 | 18,794 | ||||||
| Total current liabilities | 106,771 | 81,447 | ||||||
| Deferred revenue | 3,531 | 3,783 | ||||||
| Operating lease liabilities | 83,880 | 70,153 | ||||||
| Liability related to sale of future royalties | 144,315 | 174,504 | ||||||
| Other liabilities | 484 | 524 | ||||||
| Total liabilities | 338,981 | 330,411 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock; no shares issued and outstanding at September 30, 2021 and December 31, 2020 | - | - | ||||||
| Common stock; 42,752 and 37,476 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | 4 | 4 | ||||||
| Additional paid-in capital | 917,950 | 667,181 | ||||||
| Accumulated other comprehensive loss | (1,285 | ) | (360 | ) | ||||
| Accumulated deficit | (455,255 | ) | (289,072 | ) | ||||
| Total stockholders' equity | 461,414 | 377,753 | ||||||
| Total liabilities and stockholders' equity | $ | 800,395 | $ | 708,164 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(in thousands, except per share data)
| Three Months | Nine Months | |||||||||||||||
| Ended September 30, | Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenues | ||||||||||||||||
| License and royalty revenue | $ | 30,773 | $ | 98,912 | $ | 71,692 | $ | 133,122 | ||||||||
| Total revenues | 30,773 | 98,912 | 71,692 | 133,122 | ||||||||||||
| Operating Expenses | ||||||||||||||||
| Cost of revenues | 14,105 | 17,364 | 28,775 | 25,457 | ||||||||||||
| Research and development | 47,855 | 43,968 | 133,459 | 119,114 | ||||||||||||
| General and administrative | 21,030 | 15,859 | 57,293 | 46,246 | ||||||||||||
| Provision for credit losses and other | 5,131 | 7,770 | 5,781 | 7,887 | ||||||||||||
| Total operating expenses | 88,121 | 84,961 | 225,308 | 198,704 | ||||||||||||
| Income (loss) from operations | (57,348 | ) | 13,951 | (153,616 | ) | (65,582 | ) | |||||||||
| Other Income (Expense) | ||||||||||||||||
| Interest income from licensing | 117 | 1,444 | 700 | 4,141 | ||||||||||||
| Investment income (loss) | 5,535 | (6,607 | ) | 6,514 | (4,071 | ) | ||||||||||
| Interest expense | (6,709 | ) | - | (19,777 | ) | - | ||||||||||
| Total other income (expense) | (1,057 | ) | (5,163 | ) | (12,563 | ) | 70 | |||||||||
| Income (loss) before income taxes | (58,405 | ) | 8,788 | (166,179 | ) | (65,512 | ) | |||||||||
| Income Tax Benefit (Expense) | - | 3 | (4 | ) | 503 | |||||||||||
| Net income (loss) | $ | (58,405 | ) | $ | 8,791 | $ | (166,183 | ) | $ | (65,009 | ) | |||||
| Other Comprehensive Income (Loss) | ||||||||||||||||
| Unrealized gain (loss) on available-for-sale securities, net | (30 | ) | (487 | ) | (925 | ) | 58 | |||||||||
| Total other comprehensive income (loss) | (30 | ) | (487 | ) | (925 | ) | 58 | |||||||||
| Comprehensive income (loss) | $ | (58,435 | ) | $ | 8,304 | $ | (167,108 | ) | $ | (64,951 | ) | |||||
| Net income (loss) per share: | ||||||||||||||||
| Basic | $ | (1.37 | ) | $ | 0.24 | $ | (3.93 | ) | $ | (1.75 | ) | |||||
| Diluted | $ | (1.37 | ) | $ | 0.23 | $ | (3.93 | ) | $ | (1.75 | ) | |||||
| Weighted-average common shares outstanding: | ||||||||||||||||
| Basic | 42,629 | 37,342 | 42,324 | 37,234 | ||||||||||||
| Diluted | 42,629 | 38,877 | 42,324 | 37,234 |
Investor Relations and Corporate Communications
Brendan Burns, 212-600-1902
David Rosen, 212-600-1902