REGENXBIO Reports Second Quarter 2024 Financial Results and Recent Operational Highlights
Rhea-AI Summary
REGENXBIO (Nasdaq: RGNX) reported its Q2 2024 financial results and recent operational highlights. The company is progressing its late-stage pipeline with the first Biologics License Application (BLA) expected in 2024. Key highlights include:
- Positive data from Phase II AFFINITY DUCHENNE trial of RGX-202 demonstrating high microdystrophin expression.
- End-of-Phase II meeting with FDA for RGX-202, supporting an accelerated approval pathway.
- End-of-Phase II meeting for ABBV-RGX-314 for diabetic retinopathy moved to Q4 2024.
- $327 million in cash and equivalents as of June 30, 2024, funding operations into 2026.
- Q2 revenues increased to $22.3 million, primarily due to Zolgensma royalties.
- R&D expenses decreased to $48.9 million, driven by reduced headcount and other costs.
- Net loss narrowed to $53 million, from $72.1 million in the same period last year.
Positive
- Positive Phase II data for RGX-202 with high microdystrophin expression.
- End-of-Phase II meeting with FDA supports accelerated approval for RGX-202.
- ABBV-RGX-314 End-of-Phase II meeting for diabetic retinopathy advanced to Q4 2024.
- $327 million in cash, funding operations into 2026.
- Q2 revenues increased to $22.3 million due to Zolgensma royalties.
- R&D expenses decreased to $48.9 million.
Negative
- Net loss of $53 million in Q2 2024.
- General and administrative expenses were $18.9 million.
News Market Reaction 1 Alert
On the day this news was published, RGNX declined 2.03%, reflecting a moderate negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
- 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)
- New, positive data from Phase II AFFINITY DUCHENNE® trial of RGX-202 demonstrates consistent high expression of microdystrophin across treated patients in all age groups
- Successful End-of-Phase II meeting with FDA for RGX-202 supports plans for using the accelerated approval pathway and pivotal initiation in Q4 2024
- End-of-Phase II meeting for ABBV-RGX-314 for diabetic retinopathy accelerated to Q4 2024 to support global pivotal program initiation in H1 2025
$327 million - Conference call Thursday, August 1, at 4:30 p.m. ET
"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.
- REGENXBIO recently announced new, positive data from two patients treated with dose level 2 (DL2), demonstrating consistent, high microdystrophin expression across treated patients in the Phase II AFFINITY DUCHENNE® trial of RGX-202. In patients aged 5.8 and 8.5 at dosing, RGX-202 microdystrophin expression was measured to be
77.2% , and46.5% , respectively, compared to control at three months. As of July 8, 2024, RGX-202 has been well tolerated with no serious adverse events. All seven patients who completed three-month trial assessments indicate meaningful increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement. - REGENXBIO expects to complete enrollment in the DL2 expansion cohort in early Q3 2024 and has initiated enrollment in the cohort for boys aged 1-3. The Company remains on track to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024.
- REGENXBIO recently held a successful End-of-Phase II (EOP2) meeting with the FDA and is moving forward with plans to initiate a pivotal trial in Q4 2024. Discussions with the FDA continue to support use of microdystrophin as a surrogate endpoint reasonably likely to predict clinical benefit for accelerated approval. The Company expects to share the pivotal trial design in late Q3 to early Q4 2024.
- RGX-202 is manufactured using REGENXBIO's proprietary, high-yielding NAVXpress™ platform process. This suspension-based manufacturing process has demonstrated scalability up to 2,000L with consistent yield and product purity. The REGENXBIO Manufacturing Innovation Center has the capacity and yields to produce up to 2,500 doses of RGX-202 per year to support future commercialization.
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).
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR
- Based on positive interim results from the Phase II ALTITUDE® trial to date, the design and evaluation of two pivotal trials is on-going. With AbbVie, REGENXBIO has accelerated a planned EOP2 meeting with the FDA, now expected in Q4 2024. The Company expects to initiate the first global pivotal trial in the first half of 2025.
- The ALTITUDE trial is now enrolling a new cohort of patients with center-involved diabetic macular edema (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. DME is a vision-threatening complication of diabetic retinopathy; an estimated 34 million people globally have DME.
- ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD
- 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 the first half of 2026.
- The open label fellow eye study evaluating ABBV-RGX-314 in patients treated in the subretinal Phase I/IIa study is now fully enrolled. This study is designed to monitor safety, immune responses, and efficacy of ABBV-RGX-314 treatment in the fellow eye and these study data are intended to support the inclusion of bilateral use in the product label. Bilateral disease impacts a significant number of patients with wet AMD.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
- As of July 29, 2024, ABBV-RGX-314 at dose level 3 with short course prophylactic steroid eye drops continues to be well tolerated with no drug-related SAEs and no cases of intraocular inflammation, endophthalmitis, vasculitis, retinal artery occlusion, choroidal effusion, or hypotony. Mild episcleritis occurred in 3 patients, all resolved and completed treatment with topical steroids. There were no cases of elevated intraocular pressure.
- Based on this favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase II AAVIATE® trial is initiating enrollment in 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.
Neurodegenerative Disease: RGX-121 is a potential one-time AAV Therapeutic for the treatment of boys with MPS II.
- REGENXBIO completed a successful pre-BLA meeting with the FDA and will initiate a rolling BLA submission using the accelerated approval pathway in Q3 2024, potentially making RGX-121 the first approved gene therapy and one-time treatment for MPS II.
- REGENXBIO expects to share additional safety and efficacy data from the Phase I/II/III CAMPSIITE® trial in the second half of 2024.
- Approval of the planned BLA 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 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
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
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SOURCE REGENXBIO Inc.
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