ROCKVILLE, Md., May 3, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ended March 31, 2023, and recent operational highlights.
"REGENXBIO is well-positioned for a transformative year, with multiple clinical milestones and updates anticipated in 2023. Our goal in partnering our core expertise in gene therapy with AbbVie's leadership in eye care and their global infrastructure was to expand and advance the reach of our retinal programs for patients in need of a potential one-time gene therapy, and we are pleased to progress this program with a vision of benefiting patients worldwide. Based on the encouraging safety and clinical profiles, we believe ABBV-RGX-314 has the potential to be a highly differentiated product for millions of patients." said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We also continue to make tremendous strides in our rare disease pipeline, advancing clinical trials in Duchenne, Hunter and Hurler syndromes and Batten disease, and we anticipate more updates across each of these programs in 2023. Overall, with an active pipeline of clinical-stage products, we are making excellent progress advancing our '5x'25' strategy to bring leading, ground-breaking AAV Therapeutics to patients in need."
AbbVie Eye Care Collaboration Update
REGENXBIO today announced updates from its eye care collaboration with AbbVie to develop and commercialize a potential one-time gene therapy for the treatment of chronic retinal diseases. The updates include the transfer of the Investigational New Drug (IND) applications to AbbVie for all ongoing clinical trials and the expansion of the ATMOSPHERE® and ASCENT™ pivotal trials for the treatment of patients with wet age-related macular degeneration (wet AMD) using subretinal delivery to support new global registration plans. As part of the IND transfers, the investigational gene therapy RGX-314 has been renamed ABBV-RGX-314.
As outlined in the original collaboration agreement, beginning in 2023, AbbVie has been responsible for the majority of all development expenses for ABBV-RGX-314. The transfer of the INDs enables AbbVie to serve as lead regulatory party in the U.S. and globally. AbbVie will lead the clinical development and commercialization of ABBV-RGX-314 globally. AbbVie and REGENXBIO will continue to collaborate on additional trials of ABBV-RGX-314.
ABBV-RGX-314 is currently being evaluated in nine ongoing clinical trials in the U.S. and Canada, including two pivotal trials, a Phase II bridging study, a Long-term Follow-up study, and a Fellow Eye treatment study in patients with wet AMD, all utilizing subretinal delivery, as well as two Phase II clinical trials in patients with wet AMD and diabetic retinopathy (DR), and two corresponding Long-term Follow-up studies, all utilizing in-office suprachoroidal delivery.
The pivotal program utilizing subretinal delivery is now expected to add new sites in Europe, Japan, and Israel to advance global development plans. Additional clinical trial applications will be submitted to the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency in Japan. The ATMOSPHERE and ASCENT trials are expected to expand enrollment to approximately 540 and 660 patients, respectively. As prospectively allowed in the pivotal trial protocols, the enrollment expansion is also expected to support the increase in power of primary and secondary endpoints to enable additional global regulatory submissions and labelling options. The new global site plans and expanded enrollment targets are expected to support regulatory submissions with the U.S. Food and Drug Agency (FDA) and the EMA in late 2025 through the first half of 2026.
Also as previously announced, under the collaboration, REGENXBIO will continue to lead the manufacturing of ABBV-RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of ABBV-RGX-314 for commercial supply outside the U.S. Additionally, cGMP material produced by REGENXBIO's proprietary NAVXpress™ platform process has been incorporated into the ongoing clinical trials, including the ATMOSPHERE and ASCENT pivotal trials. REGENXBIO shall participate in U.S. commercialization efforts as provided under a mutually agreed upon commercialization plan. REGENXBIO and AbbVie will share equally in profits from net sales of ABBV-RGX-314 in the U.S. and AbbVie will pay REGENXBIO tiered royalties on net sales outside the U.S.
Program Highlights and Milestones
ABBV-RGX-314: ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for the treatment of wet AMD, DR and other additional chronic retinal conditions. ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).
RGX-202: RGX-202 is an investigational one-time AAV Therapeutic for the treatment of Duchenne Muscular Dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.
RGX-121: RGX-121 is an investigational one-time AAV Therapeutic for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme.
RGX-111: RGX-111 is an investigational one-time AAV Therapeutic for the treatment of severe Mucopolysaccharidosis Type I (MPS I), using the NAV AAV9 vector to deliver the α-l-iduronidase (IDUA) gene.
RGX-181: RGX-181 is an investigational one-time AAV Therapeutic for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, using the NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system.
RGX-381: RGX-381 is an investigational one-time AAV Therapeutic for the treatment of the ocular manifestations of CLN2 disease using the NAV AAV9 vector to deliver the TPP1 gene directly to the retina.
Operational Updates
NAV Technology Licensee Program Highlights
As of March 31, 2023, REGENXBIO's NAV Technology Platform was being applied in one marketed product and multiple clinical stage partnered programs, with the potential to impact a broad range of therapeutic areas and disease indications.
Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $473.5 million as of March 31, 2023, compared to $565.2 million as of December 31, 2022. The decrease was primarily driven by cash used to fund operating activities during the first quarter of 2023.
Revenues: Revenues were $19.1 million for the three months ended March 31, 2023, compared to $22.2 million for the three months ended March 31, 2022. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $21.5 million in the first quarter of 2022 to $16.1 million in the first quarter of 2023.
Research and Development Expenses: Research and development expenses were $58.5 million for the three months ended March 31, 2023, compared to $55.6 million for the three months ended March 31, 2022. The increase was primarily attributable to personnel costs as a result of increased headcount and laboratory and facilities costs, driven primarily by the activation of the REGENXBIO Manufacturing Innovation Center in mid-2022.
General and Administrative Expenses: General and administrative expenses were $22.6 million for the three months ended March 31, 2023, compared to $22.3 million for the three months ended March 31, 2022.
Net Loss: Net loss was $66.7 million, or $1.53 basic and diluted net loss per share, for the three months ended March 31, 2023, compared to a net loss of $76.7 million, or $1.79 basic and diluted net loss per share, for the three months ended March 31, 2022.
Financial Guidance
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $473.5 million as of March 31, 2023, to fund its operations into 2025. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under our ABBV-RGX-314 collaboration.
Conference Call
In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.
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 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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 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 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, 2022, 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.
REGENXBIO INC. | ||||||||
CONSOLIDATED BALANCE SHEETS | ||||||||
(unaudited) | ||||||||
(in thousands) | ||||||||
March 31, 2023 | December 31, 2022 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 70,091 | $ | 96,952 | ||||
Marketable securities | 261,726 | 267,690 | ||||||
Accounts receivable | 18,861 | 28,082 | ||||||
Prepaid expenses | 15,521 | 13,900 | ||||||
Other current assets | 23,003 | 9,352 | ||||||
Total current assets | 389,202 | 415,976 | ||||||
Marketable securities | 141,709 | 200,560 | ||||||
Accounts receivable, net | 1,300 | 1,504 | ||||||
Property and equipment, net | 141,573 | 141,685 | ||||||
Operating lease right-of-use assets | 63,726 | 65,116 | ||||||
Restricted cash | 2,030 | 2,030 | ||||||
Other assets | 8,290 | 6,397 | ||||||
Total assets | $ | 747,830 | $ | 833,268 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 23,283 | $ | 27,213 | ||||
Accrued expenses and other current liabilities | 31,709 | 46,794 | ||||||
Deferred revenue | 1,311 | 1,829 | ||||||
Operating lease liabilities | 6,303 | 5,997 | ||||||
Liability related to sale of future royalties | 49,728 | 48,601 | ||||||
Total current liabilities | 112,334 | 130,434 | ||||||
Operating lease liabilities | 86,992 | 88,802 | ||||||
Liability related to sale of future royalties | 77,382 | 89,005 | ||||||
Other liabilities | 5,983 | 8,832 | ||||||
Total liabilities | 282,691 | 317,073 | ||||||
Stockholders' equity | ||||||||
Preferred stock; no shares issued and outstanding | — | — | ||||||
Common stock; 43,465 and 43,299 shares issued | 4 | 4 | ||||||
Additional paid-in capital | 984,986 | 973,145 | ||||||
Accumulated other comprehensive loss | (11,622) | (15,401) | ||||||
Accumulated deficit | (508,229) | (441,553) | ||||||
Total stockholders' equity | 465,139 | 516,195 | ||||||
Total liabilities and stockholders' equity | $ | 747,830 | $ | 833,268 | ||||
REGENXBIO INC. | ||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||
(unaudited) | ||||||||
(in thousands, except per share data) | ||||||||
Three Months Ended March 31, | ||||||||
2023 | 2022 | |||||||
Revenues | ||||||||
License and royalty revenue | $ | 19,138 | $ | 22,218 | ||||
Total revenues | 19,138 | 22,218 | ||||||
Operating Expenses | ||||||||
Cost of revenues | 4,112 | 15,717 | ||||||
Research and development | 58,516 | 55,627 | ||||||
General and administrative | 22,634 | 22,318 | ||||||
Other operating expenses | 33 | 83 | ||||||
Total operating expenses | 85,295 | 93,745 | ||||||
Loss from operations | (66,157) | (71,527) | ||||||
Other Income (Expense) | ||||||||
Interest income from licensing | 70 | 94 | ||||||
Investment income | 2,166 | 799 | ||||||
Interest expense | (2,755) | (6,130) | ||||||
Total other income (expense) | (519) | (5,237) | ||||||
Loss before income taxes | (66,676) | (76,764) | ||||||
Income Tax Benefit | — | 41 | ||||||
Net loss | $ | (66,676) | $ | (76,723) | ||||
Other Comprehensive Income (Loss) | ||||||||
Unrealized gain (loss) on available-for-sale securities, net | 3,779 | (9,381) | ||||||
Total other comprehensive income (loss) | 3,779 | (9,381) | ||||||
Comprehensive loss | $ | (62,897) | $ | (86,104) | ||||
Net loss per share, basic and diluted | $ | (1.53) | $ | (1.79) | ||||
Weighted-average common shares outstanding, basic and diluted | 43,451 | 42,944 | ||||||
Contacts:
Dana Cormack
Corporate Communications
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Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
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| Last Trade: | US$7.51 |
| Daily Change: | -0.78 -9.41 |
| Daily Volume: | 706,435 |
| Market Cap: | US$372.120M |
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