REGENXBIO Reports Third Quarter 2024 Financial Results and Recent Operational Updates
Rhea-AI Summary
REGENXBIO reported Q3 2024 financial results with revenues of $24.2 million, down from $28.9 million in Q3 2023. The company posted a net loss of $59.6 million ($1.17 per share). Cash position stood at $278.6 million, expected to fund operations into 2026.
Key operational updates include advancement in Phase I/II AFFINITY DUCHENNE trial of RGX-202, BLA submission initiation for RGX-121 (expected completion in Q1 2025), and positive Phase II data for ABBV-RGX-314. The company accelerated End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy to Q4 2024, with global pivotal program initiation planned for H1 2025.
Positive
- Cash runway extended into 2026 with $278.6 million in cash and equivalents
- BLA submission for RGX-121 initiated, potential Priority Review Voucher in 2025
- Positive Phase II data for ABBV-RGX-314 showing 97% reduction in anti-VEGF treatment burden
Negative
- Revenue decreased to $24.2M from $28.9M YoY in Q3
- Net loss of $59.6M in Q3 2024
- Zolgensma royalty revenues declined to $23.9M from $28.4M YoY
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- Advancement in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne Muscular Dystrophy; pivotal trial initiation and first functional data expected this month
- BLA submission for RGX-121 initiated and expected to complete in Q1 2025
- Positive Phase II data support bilateral administration of subretinal ABBV-RGX-314; data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile
- End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy accelerated to Q4 2024 to support global pivotal program initiation in H1 2025
- Conference call today at 4:30 p.m. ET
"It has been a turning point year for REGENXBIO, as we are on the cusp of advancing AFFINITY DUCHENNE® to pivotal phase, completing our first BLA for MPS II and entering pivotal phase in a second indication within our global eyecare collaboration with AbbVie," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "The rapid progress we are making in RGX-202, which continues to demonstrate its potential to be a best-in-class gene therapy for Duchenne, is highly encouraging, and the near-term filing of our BLA for RGX-121 represents a major milestone for the patient community in need of a treatment to address both the neurocognitive and systemic effects of MPS II. The recent fellow eye data presented at the American Academy of Ophthalmology meeting demonstrate the potential of ABBV-RGX-314 to preserve vision long-term for patients with wet AMD as a one-time treatment for both eyes. Each of these programs represent one-time treatments with the potential to transform the trajectory and management of disease for patients in need of new and better options, and we look forward to continued momentum and important milestones in the last quarter of the year."
PROGRAM HIGHLIGHTS AND MILESTONES
Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy designed to deliver a differentiated, novel microdystrophin gene for improved function and outcomes for patients living with Duchenne.
- In the Phase I/II AFFINITY DUCHENNE trial of RGX-202, the last patient has been dosed in the dose level 2 (pivotal dose) expansion cohort for ages 4-11 and the first patient has been dosed in the cohort for ages 1-3. Patients with Duchenne under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the
U.S. - A clinical trials application (CTA) for RGX-202 has been authorized by Health Canada. REGENXBIO expects to initiate sites in
Canada in H1 2025. - REGENXBIO plans to share a full program update this month, including pivotal trial design and plans for accelerated approval, as well as initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial.
Retinal Disease: ABBV-RGX‑314, being developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
ABBV-RGX-314 for Treatment of DR (Suprachoroidal Delivery)
- Based on positive interim results to date from the Phase II ALTITUDE® trial, AbbVie and REGENXBIO have accelerated a planned End-of-Phase II meeting with the
U.S. Food and Drug Administration (FDA) expected this quarter. The Company expects to initiate the first global pivotal trial in H1 2025. - The ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema (DME). DME is a vision-threatening complication of DR; an estimated 34 million people globally have DME. Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.
ABBV-RGX-314 for the Treatment of Wet AMD (Subretinal Delivery)
- Enrollment is on track in the ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the FDA and European Medicines Agency in H1 2026.
- Positive data from the Phase II fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314 in patients with bilateral wet AMD were recently presented at the American Academy of Ophthalmology annual meeting. As of September 11, 2024, ABBV-RGX-314 was well tolerated in the treated fellow eye, with no drug-related serious adverse events and no cases of intraocular inflammation observed. At nine months post-administration, patients saw a
97% reduction in anti-VEGF treatment burden and sustained vision and anatomy. These results support the potential of ABBV-RGX-314 to treat bilateral disease at an expected commercial launch.
ABBV-RGX-314 for the Treatment of Wet AMD (Suprachoroidal Delivery)
- Based on a favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase II AAVIATE® trial is enrolling a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short course prophylactic steroid eye drops. At dose level 3, patients receiving ABBV-RGX-314 demonstrated an
80% reduction in annualized injection rate, with50% of patients remaining injection-free at six months. Patients also demonstrated stable Best Corrected Visual Acuity and central retinal thickness.
Neurodegenerative Disease: RGX-121 is a potential first-in-class treatment for MPS II.
- REGENXBIO initiated a rolling BLA submission for RGX-121 using the accelerated approval pathway in Q3 2024. The BLA submission is expected to be complete in Q1 2025.
- RGX-121 is on track to be the potential first gene therapy and one-time treatment approved for MPS II. Approval of RGX-121 could result in receipt of a Priority Review Voucher in 2025.
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 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 rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, 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
Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO.
REGENXBIO INC. CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands) | ||||||||
September 30, 2024 | December 31, 2023 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 56,617 | $ | 34,522 | ||||
Marketable securities | 198,843 | 240,736 | ||||||
Accounts receivable, net | 23,604 | 24,790 | ||||||
Prepaid expenses | 11,002 | 14,520 | ||||||
Other current assets | 23,330 | 20,403 | ||||||
Total current assets | 313,396 | 334,971 | ||||||
Marketable securities | 23,108 | 38,871 | ||||||
Accounts receivable | 404 | 701 | ||||||
Property and equipment, net | 120,551 | 132,103 | ||||||
Operating lease right-of-use assets | 55,293 | 60,487 | ||||||
Restricted cash | 2,030 | 2,030 | ||||||
Other assets | 4,332 | 4,807 | ||||||
Total assets | $ | 519,114 | $ | 573,970 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 19,522 | $ | 22,786 | ||||
Accrued expenses and other current liabilities | 48,642 | 49,703 | ||||||
Deferred revenue | 144 | 148 | ||||||
Operating lease liabilities | 7,720 | 7,068 | ||||||
Liability related to sale of future royalties | 26,697 | 50,567 | ||||||
Total current liabilities | 102,725 | 130,272 | ||||||
Operating lease liabilities | 76,342 | 82,222 | ||||||
Liability related to sale of future royalties | 35,052 | 43,485 | ||||||
Other liabilities | 3,579 | 6,249 | ||||||
Total liabilities | 217,698 | 262,228 | ||||||
Stockholders' equity | ||||||||
Preferred stock; no shares issued and outstanding | — | — | ||||||
Common stock; 49,534 and 44,046 shares issued | 5 | 4 | ||||||
Additional paid-in capital | 1,182,956 | 1,021,214 | ||||||
Accumulated other comprehensive loss | (582) | (4,429) | ||||||
Accumulated deficit | (880,963) | (705,047) | ||||||
Total stockholders' equity | 301,416 | 311,742 | ||||||
Total liabilities and stockholders' equity | $ | 519,114 | $ | 573,970 | ||||
REGENXBIO INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) | |||||||||||||||
Three Months | Nine Months | ||||||||||||||
Ended September 30, | Ended September 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Revenues | |||||||||||||||
License and royalty revenue | $ | 24,197 | $ | 28,914 | $ | 62,114 | $ | 68,029 | |||||||
Total revenues | 24,197 | 28,914 | 62,114 | 68,029 | |||||||||||
Operating Expenses | |||||||||||||||
Cost of revenues | 12,387 | 12,388 | 27,249 | 25,975 | |||||||||||
Research and development | 54,429 | 58,183 | 158,142 | 176,585 | |||||||||||
General and administrative | 19,422 | 23,083 | 56,568 | 69,415 | |||||||||||
Impairment of long-lived assets | — | — | 2,101 | — | |||||||||||
Other operating expenses | 37 | 220 | 32 | 279 | |||||||||||
Total operating expenses | 86,275 | 93,874 | 244,092 | 272,254 | |||||||||||
Loss from operations | (62,078) | (64,960) | (181,978) | (204,225) | |||||||||||
Other Income (Expense) | |||||||||||||||
Interest income from licensing | 25 | 56 | 91 | 166 | |||||||||||
Investment income | 3,276 | 4,660 | 9,213 | 8,953 | |||||||||||
Interest expense | (820) | (1,624) | (3,242) | (5,499) | |||||||||||
Total other income | 2,481 | 3,092 | 6,062 | 3,620 | |||||||||||
Net loss | $ | (59,597) | $ | (61,868) | $ | (175,916) | $ | (200,605) | |||||||
Other Comprehensive Income | |||||||||||||||
Unrealized gain on available-for-sale securities, net | 1,684 | 2,685 | 3,847 | 7,988 | |||||||||||
Total other comprehensive income | 1,684 | 2,685 | 3,847 | 7,988 | |||||||||||
Comprehensive loss | $ | (57,913) | $ | (59,183) | $ | (172,069) | $ | (192,617) | |||||||
Net loss per share, basic and diluted | $ | (1.17) | $ | (1.41) | $ | (3.59) | $ | (4.60) | |||||||
Weighted-average common shares outstanding, basic and diluted | 50,800 | 43,945 | 49,051 | 43,644 | |||||||||||
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
George E. MacDougall
Investor Relations
IR@regenxbio.com
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SOURCE REGENXBIO Inc.