REGENXBIO Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Operational Highlights
- 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 in cash, cash equivalents and marketable securities as ofDecember 31, 2023 , expected to fund operational runway into the second half of 2025- Conference call
Tuesday, February 27 , at4:30 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
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 theEuropean 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 with 50% 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 inOrlando, 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
CONFERENCE CALL
In connection with this announcement,
ABOUT
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,
Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of
CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands) |
||||||||
|
|
|||||||
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 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) |
|||||||||||||||
Three Months |
Years |
||||||||||||||
Ended |
Ended |
||||||||||||||
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:
Corporate Communications
Dcormack@regenxbio.com
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com
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