EXHIBIT 99.1
REGENXBIO Reports Second Quarter 2024 Financial Results and Recent Operational Highlights
ROCKVILLE, Md., August 1, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the second quarter ending June 30, 2024. Recent operational highlights, including acceleration of the late-stage pipeline, support meaningful value generation from the Company's leading portfolio of AAV Therapeutics.
“In the first half of 2024, REGENXBIO has made remarkable progress in accelerating and advancing the development of AAV Therapeutics that are well-positioned to make a profound impact for patients,” said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. “Looking ahead, we have multiple, exciting catalysts across our pipeline, including enrolling our first patient aged 1-3 in the AFFINITY DUCHENNE® trial and advancing RGX-202 into pivotal stage to address the significant ongoing unmet need in this community, initiating a rolling BLA for RGX-121 as the only one-time treatment for Hunter syndrome, and accelerating our End-of-Phase II meeting with the FDA for ABBV-RGX-314 in diabetic retinopathy. Each of these programs represent differentiated therapies that we expect will drive significant value.”
PROGRAM HIGHLIGHTS AND MILESTONES
Neuromuscular Disease: RGX-202 is an investigational one-time AAV Therapeutic designed to deliver a novel microdystrophin gene for improved function and outcomes in Duchenne.
Retinal Disease: ABBV-RGX‑314, being developed in collaboration with AbbVie, is a potential one-time treatment for chronic retinal conditions, including wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and diabetic macular edema (DME).
Neurodegenerative Disease: RGX-121 is a potential one-time AAV Therapeutic for the treatment of boys with MPS II.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $327.3 million as of June 30, 2024, compared to $314.1 million as of December 31, 2023. The increase was primarily attributable to $131.1 million of aggregate net proceeds received from the follow-on public offering of the Company’s common stock and pre-funded warrants completed in March 2024, and was partially offset by cash used to fund operating activities during the first half of 2024.
Revenues: Revenues were $22.3 million for the three months ended June 30, 2024, compared to $20.0 million for the three months ended June 30, 2023. The increase was primarily attributable to Zolgensma® royalty revenues, which increased from $19.0 million for the second quarter of 2023 to $21.8 million for the second quarter of 2024.
Research and Development Expenses: Research and development expenses were $48.9 million for the three months ended June 30, 2024, compared to $59.9 million for the three months ended June 30, 2023. The decrease was largely driven by manufacturing and clinical supply costs for ABBV-RGX-314 and RGX-202, and personnel-related costs as a result of reduced headcount. The decrease was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202.
General and Administrative Expenses: General and administrative expenses were $18.9 million for the three months ended June 30, 2024, compared to $23.7 million for the three months ended June 30, 2023. The decrease was primarily attributable to expenses for professional services and other corporate overhead costs.
Net Loss: Net loss was $53.0 million, or $1.05 basic and diluted net loss per share, for the three months ended June 30, 2024, compared to a net loss of $72.1 million, or $1.66 basic and diluted net loss per share, for the three months ended June 30, 2023.
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $327.3 million as of June 30, 2024 to fund its operations into 2026. 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 (including a potential, one-time $200.0 million milestone for achievement of first patient dosed in the first pivotal trial for suprachoroidal delivery for treatment of DR) and the potential monetization of a priority review voucher that may be received for RGX-121.
CONFERENCE CALL
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 (646) 307-1963 (domestic) or (800) 715-9871 (international) and enter the passcode 4849384. 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. 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. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO’s AAV Therapeutic platform, including Novartis’ ZOLGENSMA® for
children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com.
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 timing or likelihood of payments from AbbVie, the monetization of any priority review voucher, 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, 2023, 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.
REGENXBIO INC.
CONSOLIDATED BALANCE SHEETS
(unaudited)
(in thousands)
|
| June 30, 2024 |
|
| December 31, 2023 |
| ||
Assets |
|
|
|
|
|
| ||
Current assets |
|
|
|
|
|
| ||
Cash and cash equivalents |
| $ | 57,765 |
|
| $ | 34,522 |
|
Marketable securities |
|
| 232,592 |
|
|
| 240,736 |
|
Accounts receivable, net |
|
| 22,809 |
|
|
| 24,790 |
|
Prepaid expenses |
|
| 10,021 |
|
|
| 14,520 |
|
Other current assets |
|
| 23,496 |
|
|
| 20,403 |
|
Total current assets |
|
| 346,683 |
|
|
| 334,971 |
|
Marketable securities |
|
| 36,943 |
|
|
| 38,871 |
|
Accounts receivable |
|
| 464 |
|
|
| 701 |
|
Property and equipment, net |
|
| 123,969 |
|
|
| 132,103 |
|
Operating lease right-of-use assets |
|
| 56,344 |
|
|
| 60,487 |
|
Restricted cash |
|
| 2,030 |
|
|
| 2,030 |
|
Other assets |
|
| 2,946 |
|
|
| 4,807 |
|
Total assets |
| $ | 569,379 |
|
| $ | 573,970 |
|
Liabilities and Stockholders’ Equity |
|
|
|
|
|
| ||
Current liabilities |
|
|
|
|
|
| ||
Accounts payable |
| $ | 16,362 |
|
| $ | 22,786 |
|
Accrued expenses and other current liabilities |
|
| 42,488 |
|
|
| 49,703 |
|
Deferred revenue |
|
| 21 |
|
|
| 148 |
|
Operating lease liabilities |
|
| 7,302 |
|
|
| 7,068 |
|
Liability related to sale of future royalties |
|
| 32,100 |
|
|
| 50,567 |
|
Total current liabilities |
|
| 98,273 |
|
|
| 130,272 |
|
Operating lease liabilities |
|
| 78,234 |
|
|
| 82,222 |
|
Liability related to sale of future royalties |
|
| 41,079 |
|
|
| 43,485 |
|
Other liabilities |
|
| 3,526 |
|
|
| 6,249 |
|
Total liabilities |
|
| 221,112 |
|
|
| 262,228 |
|
Stockholders’ equity |
|
|
|
|
|
| ||
Preferred stock; no shares issued and outstanding |
|
| — |
|
|
| — |
|
Common stock; 49,317 and 44,046 shares issued |
|
| 5 |
|
|
| 4 |
|
Additional paid-in capital |
|
| 1,171,894 |
|
|
| 1,021,214 |
|
Accumulated other comprehensive loss |
|
| (2,266 | ) |
|
| (4,429 | ) |
Accumulated deficit |
|
| (821,366 | ) |
|
| (705,047 | ) |
Total stockholders’ equity |
|
| 348,267 |
|
|
| 311,742 |
|
Total liabilities and stockholders’ equity |
| $ | 569,379 |
|
| $ | 573,970 |
|
REGENXBIO INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands, except per share data)
| Three Months |
|
| Six Months |
| ||||||||||
| Ended June 30, |
|
| Ended June 30, |
| ||||||||||
| 2024 |
|
| 2023 |
|
| 2024 |
|
| 2023 |
| ||||
Revenues |
|
|
|
|
|
|
|
|
|
|
| ||||
License and royalty revenue | $ | 22,295 |
|
| $ | 19,977 |
|
| $ | 37,917 |
|
| $ | 39,115 |
|
Total revenues |
| 22,295 |
|
|
| 19,977 |
|
|
| 37,917 |
|
|
| 39,115 |
|
Operating Expenses |
|
|
|
|
|
|
|
|
|
|
| ||||
Cost of revenues |
| 10,579 |
|
|
| 9,475 |
|
|
| 14,862 |
|
|
| 13,587 |
|
Research and development |
| 48,869 |
|
|
| 59,886 |
|
|
| 103,713 |
|
|
| 118,402 |
|
General and administrative |
| 18,855 |
|
|
| 23,698 |
|
|
| 37,146 |
|
|
| 46,332 |
|
Impairment of long-lived assets |
| — |
|
|
| — |
|
|
| 2,101 |
|
|
| — |
|
Other operating expenses (income) |
| 29 |
|
|
| 26 |
|
|
| (5 | ) |
|
| 59 |
|
Total operating expenses |
| 78,332 |
|
|
| 93,085 |
|
|
| 157,817 |
|
|
| 178,380 |
|
Loss from operations |
| (56,037 | ) |
|
| (73,108 | ) |
|
| (119,900 | ) |
|
| (139,265 | ) |
Other Income (Expense) |
|
|
|
|
|
|
|
|
|
|
| ||||
Interest income from licensing |
| 29 |
|
|
| 40 |
|
|
| 66 |
|
|
| 110 |
|
Investment income |
| 3,468 |
|
|
| 2,127 |
|
|
| 5,937 |
|
|
| 4,293 |
|
Interest expense |
| (449 | ) |
|
| (1,120 | ) |
|
| (2,422 | ) |
|
| (3,875 | ) |
Total other income |
| 3,048 |
|
|
| 1,047 |
|
|
| 3,581 |
|
|
| 528 |
|
Net loss | $ | (52,989 | ) |
| $ | (72,061 | ) |
| $ | (116,319 | ) |
| $ | (138,737 | ) |
Other Comprehensive Income |
|
|
|
|
|
|
|
|
|
|
| ||||
Unrealized gain on available-for-sale securities, net |
| 963 |
|
|
| 1,524 |
|
|
| 2,163 |
|
|
| 5,303 |
|
Total other comprehensive income |
| 963 |
|
|
| 1,524 |
|
|
| 2,163 |
|
|
| 5,303 |
|
Comprehensive loss | $ | (52,026 | ) |
| $ | (70,537 | ) |
| $ | (114,156 | ) |
| $ | (133,434 | ) |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Net loss per share, basic and diluted | $ | (1.05 | ) |
| $ | (1.66 | ) |
| $ | (2.41 | ) |
| $ | (3.19 | ) |
Weighted-average common shares outstanding, basic and diluted |
| 50,601 |
|
|
| 43,531 |
|
|
| 48,167 |
|
|
| 43,491 |
|
###
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com