REGENXBIO Reports First Quarter 2024 Financial Results and Recent Operational Highlights
REGENXBIO reported its first quarter 2024 financial results and recent operational highlights, showcasing progress towards BLA filing in 2024 and pivotal trial initiations for Duchenne and diabetic retinopathy. The company announced positive interim efficacy and safety data, cash reserves of $381 million, and planned meetings with the FDA. Program highlights include advancements in neuromuscular and retinal diseases. Financially, revenues decreased to $15.6 million, with increased cash position but higher R&D and G&A expenses leading to a net loss of $63.3 million.
Accelerating progress towards pivotal trials for Duchenne and diabetic retinopathy
Positive interim efficacy and safety data for Duchenne and diabetic retinopathy programs
Strong cash position of $381 million as of March 31, 2024, expected to fund operations into 2026
On track with planned BLA filing for RGX-121 and initiating pivotal trials for retinal diseases
Continued program advancements in neuromuscular and retinal diseases with positive data updates
Decrease in revenues to $15.6 million for the first quarter of 2024
Net loss of $63.3 million, attributed to higher R&D and G&A expenses
Reduction in Zolgensma royalty revenues impacting overall financials
Impact of reduced headcount on R&D and G&A expenses
Insights
- Company remains on track for its first BLA filing in 2024 and is accelerating progress toward pivotal trial initiation for Duchenne (H2 2024) and diabetic retinopathy (H1 2025)
- Dose level 2 selected as pivotal dose for RGX-202 treatment of Duchenne
- New positive interim efficacy and safety data announced for second boy at DL2
- AFFINITY DUCHENNE® trial expansion phase is underway with third and fourth boys dosed at DL2
- End-of-Phase II meeting with FDA planned for early Q3 and pivotal trial initiation anticipated in late Q3 to early Q4 2024
- Positive interim results from ALTITUDE® trial for diabetic retinopathy support plans for anticipated End-of-Phase II meeting with FDA in Q1 2025
$381 million - Conference call Wednesday, May 8, at 4:30 p.m. ET
"Exciting program and data updates continue in 2024 and demonstrate remarkable progress on how REGENXBIO is rapidly advancing products through late-stage development," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We expect to make significant progress across our prioritized pipeline, including advancing our Duchenne program into pivotal stage and filing a BLA for RGX-121 this year, as well as finalizing plans to initiate a pivotal program in diabetic retinopathy early next year. Regardless of today's landscape of Duchenne treatments, communication from the FDA continues to support the need for alternative gene therapies for rare diseases, including Duchenne. Our new, positive biomarker data from the AFFINITY DUCHENNE trial demonstrates the potential of RGX-202 as a meaningful and differentiated treatment option for the Duchenne community."
PROGRAM HIGHLIGHTS AND MILESTONES
Neuromuscular Disease: RGX-202 is a potential one-time AAV therapeutic for the treatment of Duchenne.
- As of May 3, 2024, RGX-202 continues to be well tolerated in all patients with no serious adverse events.
- In new data announced today from the second patient, aged 8.1 years, who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be
20.9% compared to control at three months. A reduction from baseline in serum creatinine kinase (CK) levels of90% was observed at 10 weeks. - Dose level 2 has been selected as the pivotal dose and the positive interim results enable rapid acceleration into pivotal development.
- REGENXBIO is now enrolling patients in an expedited dose level 2 expansion phase of the AFFINITY DUCHENNE trial accepted by the
U.S. Food and Drug Administration (FDA), and recently dosed two additional boys aged 5.8 and 8.5 years old. The Company expects to enroll up to a total of seven patients at the pivotal dose through early Q3 2024. - An end-of-Phase II (EOP2) meeting with FDA in early Q3 2024 is expected to support a final pivotal trial design. REGENXBIO plans to use RGX-202 microdystrophin expression as a surrogate endpoint likely to predict clinical benefit to support a Biologics License Application (BLA) filing using the accelerated approval pathway. Initiation of the pivotal trial is expected in late Q3 2024 to early Q4 2024.
- REGENXBIO also continues to expect to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024.
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.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR
- Based on positive interim results from the Phase II ALTITUDE trial to date, design and evaluation of two pivotal trials is on-going. These results also support discussion with the FDA at an EOP2 meeting anticipated in Q1 2025 that can enable rapid acceleration towards pivotal development. The Company expects to initiate the first pivotal trial in the first half of 2025.
- 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 FDA and the European Medicines Agency in the first half of 2026.
- 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 Q3 2024.
Neurodegenerative Disease: RGX-121 is a potential one-time AAV therapeutic for the treatment of 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.
NAV® TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS
Novartis AG reported first quarter 2024 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 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 Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
REGENXBIO INC. | ||||||||
CONSOLIDATED BALANCE SHEETS | ||||||||
(unaudited) | ||||||||
(in thousands) | ||||||||
March 31, 2024 | December 31, 2023 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 112,975 | $ | 34,522 | ||||
Marketable securities | 225,728 | 240,736 | ||||||
Accounts receivable, net | 15,828 | 24,790 | ||||||
Prepaid expenses | 13,590 | 14,520 | ||||||
Other current assets | 27,297 | 20,403 | ||||||
Total current assets | 395,418 | 334,971 | ||||||
Marketable securities | 41,807 | 38,871 | ||||||
Accounts receivable | 523 | 701 | ||||||
Property and equipment, net | 127,662 | 132,103 | ||||||
Operating lease right-of-use assets | 57,558 | 60,487 | ||||||
Restricted cash | 2,030 | 2,030 | ||||||
Other assets | 4,217 | 4,807 | ||||||
Total assets | $ | 629,215 | $ | 573,970 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 31,356 | $ | 22,786 | ||||
Accrued expenses and other current liabilities | 33,129 | 49,703 | ||||||
Deferred revenue | 13 | 148 | ||||||
Operating lease liabilities | 7,066 | 7,068 | ||||||
Liability related to sale of future royalties | 38,615 | 50,567 | ||||||
Total current liabilities | 110,179 | 130,272 | ||||||
Operating lease liabilities | 80,183 | 82,222 | ||||||
Liability related to sale of future royalties | 44,702 | 43,485 | ||||||
Other liabilities | 3,485 | 6,249 | ||||||
Total liabilities | 238,549 | 262,228 | ||||||
Stockholders' equity | ||||||||
Preferred stock; no shares issued and outstanding | — | — | ||||||
Common stock; 49,043 and 44,046 shares issued | 5 | 4 | ||||||
Additional paid-in capital | 1,162,267 | 1,021,214 | ||||||
Accumulated other comprehensive loss | (3,229) | (4,429) | ||||||
Accumulated deficit | (768,377) | (705,047) | ||||||
Total stockholders' equity | 390,666 | 311,742 | ||||||
Total liabilities and stockholders' equity | $ | 629,215 | $ | 573,970 | ||||
REGENXBIO INC. | ||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||
(unaudited) | ||||||||
(in thousands, except per share data) | ||||||||
Three Months Ended March 31, | ||||||||
2024 | 2023 | |||||||
Revenues | ||||||||
License and royalty revenue | $ | 15,622 | $ | 19,138 | ||||
Total revenues | 15,622 | 19,138 | ||||||
Operating Expenses | ||||||||
Cost of revenues | 4,283 | 4,112 | ||||||
Research and development | 54,844 | 58,516 | ||||||
General and administrative | 18,291 | 22,634 | ||||||
Impairment of long-lived assets | 2,101 | — | ||||||
Other operating expenses (income) | (34) | 33 | ||||||
Total operating expenses | 79,485 | 85,295 | ||||||
Loss from operations | (63,863) | (66,157) | ||||||
Other Income (Expense) | ||||||||
Interest income from licensing | 37 | 70 | ||||||
Investment income | 2,469 | 2,166 | ||||||
Interest expense | (1,973) | (2,755) | ||||||
Total other income (expense) | 533 | (519) | ||||||
Net loss | $ | (63,330) | $ | (66,676) | ||||
Other Comprehensive Income | ||||||||
Unrealized gain on available-for-sale securities, net | 1,200 | 3,779 | ||||||
Total other comprehensive income | 1,200 | 3,779 | ||||||
Comprehensive loss | $ | (62,130) | $ | (62,897) | ||||
Net loss per share, basic and diluted | $ | (1.38) | $ | (1.53) | ||||
Weighted-average common shares outstanding, basic and diluted | 45,733 | 43,451 | ||||||
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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