Full Press Release Details
REGENXBIO Reports Third Quarter 2019 Financial and Operating Results and Recent Operational Highlights
ROCKVILLE, Md., November 5, 2019 (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 September 30, 2019, and recent operational highlights.
"We continue to make significant advances in our pipeline and NAV Technology Platform, expanding the pipeline with programs to deliver therapeutic antibodies, beginning an exciting collaboration with Neurimmune, and initiating construction of a new manufacturing facility," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Our focus on patients drives us to build upon the clinical progress of gene therapy options for both large indications and rare diseases, and we are excited to lead the field in innovative approaches. We are also very encouraged by the early adoption of the first approved NAV Technology-based therapy, Novartis' Zolgensma , for the treatment of SMA."
"We anticipate a short delay in the initiation of our Phase IIb trial for RGX-314 in wet AMD and our investigative new drug, or IND, application filing for diabetic retinopathy, following notification from the FDA regarding certain third-party commercially-available surgical devices that were used to deliver RGX-314 in our Phase I/IIa trial," added Mr. Mills. "The notification is not related to the RGX-314 gene therapy itself, and we have not received reports of any device-related concerns or complications in the subjects already dosed in the Phase I/IIa trial. Assessments and monitoring of all enrolled subjects in the trial continue to be performed as usual. We are working with the FDA to address this matter, and as a result, we now plan to initiate our Phase IIb trial for RGX-314 in wet AMD and file our IND for diabetic retinopathy in Q1 2020,"
"We are encouraged by the data from the Phase I/IIa clinical trial of RGX-314 in patients with wet AMD," said Dr. Stephen Pakola, Senior Vice President and Chief Medical Officer of REGENXBIO. "These data demonstrate significant long-term reduction of anti-VEGF treatment burden while maintaining or
improving vision and retinal thickness after a single administration of our gene therapy. We look forward to initiating our Phase IIb trial of RGX-314 in wet AMD, and preparing to file an IND to evaluate RGX-314 in diabetic retinopathy, while continuing our collaboration with Clearside to evaluate the in-office suprachoroidal administration of RGX-314. We have also achieved important clinical development milestones in our other gene therapy programs, including completing dosing of Cohort 2 in our RGX-501 trial for HoFH and Cohort 1 in our RGX-121 trial for MPS II. Our internal pipeline of novel gene therapies positions us to develop meaningful potential treatments for a broad spectrum of patients with significant unmet needs."
Product Candidate Updates
Gene Therapy using NAV Vectors for AAV-Mediated Antibody Delivery:
Gene Therapy using NAV Vectors for Rare Genetic Diseases:
Other Recent Operational Highlights
NAV Technology Licensee Program Highlights
As of October 31, 2019, REGENXBIO's NAV Technology Platform was being applied in one marketed product, Novartis AG's Zolgensma, and more than 20 partnered product candidates in development by NAV Technology Licensees. Fifteen of these partnered product candidates are in active clinical development. 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:
On October 22, 2019, Novartis AG reported Q3 2019 U.S. Zolgensma sales revenue of $160 million, and REGENXBIO recognized royalty revenue of $9.2 million in Q3 2019 as a result of these sales.
Partnered Product Candidates
Cash, cash equivalents and marketable securities were $417.1 million as of September 30, 2019, compared to $470.6 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities during the nine months ended September 30, 2019, was primarily attributable to $82.2 million of net cash used in operating activities during the period, partially offset by an unrealized gain of $29.4 million related to our marketable equity securities of Prevail Therapeutics Inc.
Revenues were $14.7 million for the three months ended September 30, 2019, compared to $5.3 million for the three months ended September 30, 2018. The increase was primarily attributable $9.2 million of royalty revenue recognized during the third quarter of 2019 related to net sales of Zolgensma. Commercial sales of Zolgensma commenced in the second quarter of 2019, and we are also eligible to receive a milestone payment of $80.0 million from AveXis, Inc. upon the achievement of $1 billion in cumulative net sales of Zolgensma.
Research and development expenses were $35.7 million for the three months ended September 30, 2019, compared to $18.5 million for the three months ended September 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs and external expenses associated with conducting clinical trials and manufacturing-related services.
General and administrative expenses were $12.4 million for the three months ended September 30, 2019, compared to $9.0 million for the three months ended September 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount.
Net loss was $34.6 million, or $0.94 basic and diluted net loss per share, for the three months ended September 30, 2019, compared to net loss of $19.2 million, or $0.56 basic and diluted net loss per share, for the three months ended September 30, 2018.
Based on its current operating plan, REGENXBIO now expects its balance in cash, cash equivalents and marketable securities to be at least $365 million as of December 31, 2019.
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 1159968. 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," "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, research and development activities, preclinical studies, 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, 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, 2018, 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.
CONSOLIDATED BALANCE SHEETS
(in thousands, except per share data)
| September 30, 2019 | December 31, 2018 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 79,594 | $ | 75,561 | ||||
| Marketable securities | 252,125 | 244,200 | ||||||
| Accounts receivable | 19,175 | 8,587 | ||||||
| Prepaid expenses | 6,920 | 5,734 | ||||||
| Other current assets | 2,414 | 3,831 | ||||||
| Total current assets | 360,228 | 337,913 | ||||||
| Marketable securities | 85,408 | 150,819 | ||||||
| Accounts receivable | 24,345 | 23,012 | ||||||
| Property and equipment, net | 28,287 | 28,702 | ||||||
| Operating lease right-of-use assets | 5,905 | - | ||||||
| Restricted cash | 1,053 | 1,053 | ||||||
| Other assets | 4,011 | 2,315 | ||||||
| Total assets | $ | 509,237 | $ | 543,814 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 9,608 | $ | 4,412 | ||||
| Accrued expenses and other current liabilities | 19,231 | 17,164 | ||||||
| Deferred revenue | - | 600 | ||||||
| Operating lease liabilities | 2,506 | - | ||||||
| Total current liabilities | 31,345 | 22,176 | ||||||
| Deferred revenue | 3,333 | 3,333 | ||||||
| Operating lease liabilities | 4,320 | - | ||||||
| Deferred rent | - | 1,098 | ||||||
| Financing lease obligations | - | 5,854 | ||||||
| Other liabilities | 1,844 | 2,505 | ||||||
| Total liabilities | 40,842 | 34,966 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at September 30, 2019 and December 31, 2018 | - | - | ||||||
| Common stock; $0.0001 par value; 100,000 shares authorized at September 30, 2019 and December 31, 2018; 36,840 and 36,120 shares issued and outstanding at September 30, 2019 and December 31, 2018, respectively | 4 | 4 | ||||||
| Additional paid-in capital | 619,386 | 592,580 | ||||||
| Accumulated other comprehensive income (loss) | 363 | (720 | ) | |||||
| Accumulated deficit | (151,358 | ) | (83,016 | ) | ||||
| Total stockholders' equity | 468,395 | 508,848 | ||||||
| Total liabilities and stockholders' equity | $ | 509,237 | $ | 543,814 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(in thousands, except per share data)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2019 | 2018 | 2019 | 2018 | |||||||||||||
| Revenues | ||||||||||||||||
| License and royalty revenue | $ | 14,700 | $ | 5,306 | $ | 23,465 | $ | 177,728 | ||||||||
| Total revenues | 14,700 | 5,306 | 23,465 | 177,728 | ||||||||||||
| Operating Expenses | ||||||||||||||||
| Cost of revenues | 2,494 | 517 | 4,450 | 6,797 | ||||||||||||
| Research and development | 35,692 | 18,508 | 90,378 | 59,544 | ||||||||||||
| General and administrative | 12,402 | 9,008 | 37,365 | 25,706 | ||||||||||||
| Other operating expenses (income) | 8 | (2 | ) | (54 | ) | 31 | ||||||||||
| Total operating expenses | 50,596 | 28,031 | 132,139 | 92,078 | ||||||||||||
| Income (loss) from operations | (35,896 | ) | (22,725 | ) | (108,674 | ) | 85,650 | |||||||||
| Other Income | ||||||||||||||||
| Interest income from licensing | 716 | 109 | 2,091 | 8,362 | ||||||||||||
| Investment income | 431 | 2,122 | 37,950 | 4,177 | ||||||||||||
| Total other income | 1,147 | 2,231 | 40,041 | 12,539 | ||||||||||||
| Income (loss) before income taxes | (34,749 | ) | (20,494 | ) | (68,633 | ) | 98,189 | |||||||||
| Income Tax Benefit (Expense) | 165 | 1,292 | 364 | (2,558 | ) | |||||||||||
| Net income (loss) | $ | (34,584 | ) | $ | (19,202 | ) | $ | (68,269 | ) | $ | 95,631 | |||||
| Other Comprehensive Income (Loss) | ||||||||||||||||
| Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | (108 | ) | (103 | ) | 1,043 | (159 | ) | |||||||||
| Total other comprehensive income (loss) | (108 | ) | (103 | ) | 1,043 | (159 | ) | |||||||||
| Comprehensive income (loss) | $ | (34,692 | ) | $ | (19,305 | ) | $ | (67,226 | ) | $ | 95,472 | |||||
| Net income (loss) per share: | ||||||||||||||||
| Basic | $ | (0.94 | ) | $ | (0.56 | ) | $ | (1.86 | ) | $ | 2.94 | |||||
| Diluted | $ | (0.94 | ) | $ | (0.56 | ) | $ | (1.86 | ) | $ | 2.67 | |||||
| Weighted-average common shares outstanding: | ||||||||||||||||
| Basic | 36,813 | 33,988 | 36,618 | 32,576 | ||||||||||||
| Diluted | 36,813 | 33,988 | 36,618 | 35,875 |
Zolgensma is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.
Investor Relations and Corporate Communications
Heather Savelle, 212-600-1902
David Rosen, 212-600-1902