REGENXBIO Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Operational Highlights
- REGENXBIO prioritizes its pipeline for pivotal stages and expects the first BLA filing in 2024.
- The company reported $314 million in cash, cash equivalents, and marketable securities as of December 31, 2023, providing operational funding until the second half of 2025.
- Progress is seen in the Duchenne and retinal disease programs with new updates planned starting in March.
- REGENXBIO's strategic pipeline prioritization aims to reduce operating expenses and maximize value generation from its AAV Therapeutics.
- The company anticipates significant milestones and data readouts across all programs in 2024, marking a turning point in its journey.
- Program highlights include advancements in retinal disease treatments and neuromuscular disease therapies.
- Novartis AG, Rocket Pharmaceuticals, and Ultragenyx Pharmaceutical have investigational AAV Therapeutics in pivotal phases using REGENXBIO's NAV Technology.
- Financially, REGENXBIO's revenues, research and development expenses, and net loss for Q4 2023 reflect operational activities and revenue streams.
- The company's cash runway guidance of $314.1 million is expected to sustain operations until the second half of 2025.
- Revenues decreased for the three months and full year ended December 31, 2023, compared to the same periods in 2022, primarily due to lower Zolgensma royalty revenues.
- Net loss increased slightly for Q4 2023 compared to Q4 2022, and for the full year ended December 31, 2023, compared to 2022.
- Research and development expenses decreased slightly for Q4 2023 compared to Q4 2022, but increased for the full year ended December 31, 2023, compared to 2022.
- General and administrative expenses increased for the full year ended December 31, 2023, compared to 2022.
- The decrease in cash, cash equivalents, and marketable securities from 2022 to 2023 was driven by operational activities.
Insights
The financial results of REGENXBIO Inc. indicate a mixed performance, with a decrease in cash reserves from $565.2 million to $314.1 million year-over-year. This is a significant reduction and could be indicative of the high costs associated with advancing clinical trials, which is not uncommon in the biotech industry. However, the company's operational runway is projected to last into the second half of 2025, providing some assurance of financial stability in the near term.
Revenue decline, primarily from Zolgensma royalty revenues, could concern investors as it suggests either a saturation in market uptake or increased competition. Nonetheless, the company's reduced net loss from $280.3 million to $263.5 million year-over-year may be seen as a positive sign of cost management and operational efficiency. Stakeholders should closely monitor future revenue streams and the potential impact of the pipeline's progress on the company's financial health.
The clinical advancements in REGENXBIO's pipeline are noteworthy, particularly with ABBV-RGX-314, which is positioned to potentially redefine the standard-of-care in treating chronic retinal diseases. The reduction in treatment burden and sustained vision health could be a game-changer for patients with wet age-related macular degeneration and diabetic retinopathy. The success of the pivotal trials and subsequent regulatory submissions will be critical for the company's value proposition.
Similarly, RGX-202's potential impact on Duchenne muscular dystrophy, as indicated by the preliminary trial results, could have significant implications for treatment paradigms. The use of RGX-202 microdystrophin expression as a surrogate endpoint for accelerated approval is a strategic move that could expedite the drug's availability to patients, pending FDA endorsement of the approach. The neurodegenerative program, RGX-121 for MPS II, also holds promise with its primary endpoint achievement in the CAMPSIITE trial.
The strategic prioritization of REGENXBIO's pipeline aligns with current market trends, where precision medicine and gene therapies are gaining traction. The focus on AAV therapeutics for large commercial opportunities indicates a clear vision to capture significant market share in emerging treatment modalities. The collaboration with AbbVie and the potential for a new standard-of-care in retinal diseases could position REGENXBIO favorably within a competitive landscape.
It's important to consider the broader market dynamics, such as the increasing scrutiny on drug pricing and reimbursement challenges that could affect the adoption of new therapies. The long-term financial viability of REGENXBIO will hinge on the successful commercialization of its pipeline products and the ability to secure and grow revenue streams from partnerships and royalty agreements.
- Focus on clinical stage AAV Therapeutic product candidates addressing large commercial opportunities and value generation
- Prioritized pipeline is expected to further progress to pivotal stage and first BLA filing in 2024
- New updates planned for Duchenne and in-office delivery retinal disease programs starting in March
$314 million - Conference call Tuesday, February 27, at 4:30 p.m. ET
"We started 2024 with amazing data from our AbbVie-partnered eye care programs and our treatments for Duchenne and Hunter syndrome. Our strategic pipeline prioritization at the end of 2023 created a sharpened focus for us and today we are rapidly advancing products through late-stage clinical trials. We believe this is the best way to support the creation of meaningful value," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We expect more important data readouts and milestones to be achieved across all programs and the initiation of new pivotal trials this year. 2024 will be a turning point in our journey to bring ground-breaking AAV Therapeutics to millions of patients."
PROGRAM HIGHLIGHTS AND MILESTONES
Retinal Disease: ABBV-RGX‑314, in collaboration with AbbVie, is a potential one-time treatment for wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal disease that is designed to continually produce an anti-VEGF protein in the eye. ABBV-RGX-314 is currently being evaluated in patients with wet AMD and DR in nine ongoing clinical trials, including two pivotal trials. A single ABBV-RGX-314 treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing retinal disease due to the treatment burden of chronic anti-VEGF injections.
- ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD
- Enrollment is on track in ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the
U.S. Food and Drug Administration and the European Medicines Agency in late 2025 through the first half of 2026.
- Enrollment is on track in ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
- REGENXBIO expects to share new program and data updates for the Phase II AAVIATE trial in mid-2024.
- In January 2024, REGENXBIO presented data from the AAVIATE® trial demonstrating that, at six months, patients treated with ABBV-RGX-314 continue to demonstrate stable vision and retinal anatomy while a meaningful reduction in anti-VEGF treatment burden was observed. The highest reduction was seen in dose level 3, demonstrating an
80% reduction in annualized injection rate with50% of patients remaining injection-free.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR
- REGENXBIO expects to share new program and data updates for the Phase II ALTITUDE® trial in Q2 2024.
- In November 2023, REGENXBIO presented data from the ALTITUDE trial showing that, at one year, dose level 2 in non-proliferative DR patients prevented disease progression as measured by the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale. Dose level 2 reduced the risk of patients developing vision-threatening events by
89% .
Neuromuscular Disease: RGX-202 is an investigational one-time AAV therapeutic designed to meaningfully impact disease by delivering a gene for a novel microdystrophin with important biology most similar to naturally occurring dystrophin that protects from the muscle degradation associated with Duchenne.
- REGENXBIO plans to share new updates from the Phase I/II AFFINITY DUCHENNE® trial at the Muscular Dystrophy Association Clinical and Scientific Meeting being held next week in
Orlando, FL and virtually. - REGENXBIO expects to make a pivotal dose determination in mid-2024. The Company also expects to share initial strength and functional assessment data for both dose levels and the initiation of a pivotal program in the second half of 2024.
- In February 2024, REGENXBIO reported interim data from the AFFINITY DUCHENNE trial, demonstrating that all three patients at dose level 1 indicate encouraging increases in expression of RGX-202 microdystrophin at three months and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement. In the third patient, RGX-202 microdystrophin expression was measured to be
83.4% . - REGENXBIO plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) filing using the accelerated approval pathway.
Neurodegenerative Disease: RGX-121 is an investigational one-time AAV therapeutic designed to change the course of disease by restoring the gene missing in boys with MPS II.
- On track to file a BLA in 2024 using the accelerated approval pathway. Approval of the planned BLA could result in receipt of a Priority Review Voucher in 2025.
- In February 2024, REGENXBIO reported the pivotal phase of the CAMPSIITE® trial achieved its primary endpoint, as treated patients achieved decreased cerebrospinal fluid (CSF) levels of D2S6 below maximum attenuated disease levels at 16 weeks (p value of 0.00016). Patients treated with RGX-121 have showed continued improvement in neurodevelopmental skill acquisition up to four years and discontinued intravenous enzyme therapy.
NAV® TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS
Novartis AG reported fourth quarter and full year 2023 global sales of Zolgensma, for the treatment of spinal muscular atrophy, of
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were
Revenues: Revenues were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Net Loss: Net loss was
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of
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. 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 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, which will be filed with the SEC in the first quarter of 2024 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the
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) | ||||||||
December 31, 2023 | December 31, 2022 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 34,522 | $ | 96,952 | ||||
Marketable securities | 240,736 | 267,690 | ||||||
Accounts receivable, net | 24,790 | 28,082 | ||||||
Prepaid expenses | 14,520 | 13,900 | ||||||
Other current assets | 20,403 | 9,352 | ||||||
Total current assets | 334,971 | 415,976 | ||||||
Marketable securities | 38,871 | 200,560 | ||||||
Accounts receivable, net | 701 | 1,504 | ||||||
Property and equipment, net | 132,103 | 141,685 | ||||||
Operating lease right-of-use assets | 60,487 | 65,116 | ||||||
Restricted cash | 2,030 | 2,030 | ||||||
Other assets | 4,807 | 6,397 | ||||||
Total assets | $ | 573,970 | $ | 833,268 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 22,786 | $ | 27,213 | ||||
Accrued expenses and other current liabilities | 49,703 | 46,794 | ||||||
Deferred revenue | 148 | 1,829 | ||||||
Operating lease liabilities | 7,068 | 5,997 | ||||||
Liability related to sale of future royalties | 50,567 | 48,601 | ||||||
Total current liabilities | 130,272 | 130,434 | ||||||
Operating lease liabilities | 82,222 | 88,802 | ||||||
Liability related to sale of future royalties | 43,485 | 89,005 | ||||||
Other liabilities | 6,249 | 8,832 | ||||||
Total liabilities | 262,228 | 317,073 | ||||||
Stockholders' equity | ||||||||
Preferred stock; no shares issued and outstanding | — | — | ||||||
Common stock; 44,046 and 43,299 shares issued | 4 | 4 | ||||||
Additional paid-in capital | 1,021,214 | 973,145 | ||||||
Accumulated other comprehensive loss | (4,429) | (15,401) | ||||||
Accumulated deficit | (705,047) | (441,553) | ||||||
Total stockholders' equity | 311,742 | 516,195 | ||||||
Total liabilities and stockholders' equity | $ | 573,970 | $ | 833,268 | ||||
REGENXBIO INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) | |||||||||||||||
Three Months | Years | ||||||||||||||
Ended December 31, | Ended December 31, | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Revenues | |||||||||||||||
License and royalty revenue | $ | 22,213 | $ | 31,345 | $ | 90,242 | $ | 112,724 | |||||||
Total revenues | 22,213 | 31,345 | 90,242 | 112,724 | |||||||||||
Operating Expenses | |||||||||||||||
Cost of revenues | 11,238 | 12,783 | 37,213 | 54,545 | |||||||||||
Research and development | 55,681 | 62,505 | 232,266 | 242,453 | |||||||||||
General and administrative | 19,079 | 21,210 | 88,494 | 85,281 | |||||||||||
Other operating expenses (income) | 118 | (7,382) | 397 | (6,679) | |||||||||||
Total operating expenses | 86,116 | 89,116 | 358,370 | 375,600 | |||||||||||
Loss from operations | (63,903) | (57,771) | (268,128) | (262,876) | |||||||||||
Other Income (Expense) | |||||||||||||||
Interest income from licensing | (141) | 77 | 25 | 342 | |||||||||||
Investment income | 2,366 | 2,026 | 11,319 | 5,383 | |||||||||||
Interest expense | (1,363) | (4,310) | (6,862) | (23,254) | |||||||||||
Total other income (expense) | 862 | (2,207) | 4,482 | (17,529) | |||||||||||
Loss before income taxes | (63,041) | (59,978) | (263,646) | (280,405) | |||||||||||
Income Tax Benefit | 152 | 43 | 152 | 84 | |||||||||||
Net loss | $ | (62,889) | $ | (59,935) | $ | (263,494) | $ | (280,321) | |||||||
Other Comprehensive Loss | |||||||||||||||
Unrealized gain (loss) on available-for-sale securities, net | 2,984 | 2,855 | 10,972 | (12,832) | |||||||||||
Total other comprehensive income (loss) | 2,984 | 2,855 | 10,972 | (12,832) | |||||||||||
Comprehensive loss | $ | (59,905) | $ | (57,080) | $ | (252,522) | $ | (293,153) | |||||||
Net loss per share, basic and diluted | $ | (1.43) | $ | (1.38) | $ | (6.02) | $ | (6.50) | |||||||
Weighted-average common shares outstanding, basic and diluted | 44,001 | 43,296 | 43,734 | 43,152 | |||||||||||
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com
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SOURCE REGENXBIO Inc.
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