REGENXBIO Reports Fourth Quarter and Full-Year 2020 Financial Results and Operational Highlights
- Pivotal program for subretinal delivery of RGX-314 for the treatment of wet AMD is active and enrolling patients; expected to support BLA filing in 2024
- Phase II trials of RGX-314 utilizing in-office suprachoroidal delivery for treatment of wet AMD and diabetic retinopathy are ongoing
- IND filing expected in mid-2021 for RGX-202, a novel, advanced microdystrophin gene therapy for the treatment of Duchenne Muscular Dystrophy
- Positive interim data from RGX-121 Phase I/II trial demonstrated consistent reductions in CNS biomarkers, continued neurocognitive development, and evidence of systemic effects
- Monetized portion of Zolgensma® royalties in
December 2020 for$200 million in gross proceeds $523 million in cash, cash equivalents and marketable securities as ofDecember 31, 2020 - Completed public offering of common stock in
January 2021 of approximately$230 million in gross proceeds - Conference call
Monday, March 1 st at4:30 p.m. ET
"Over the past year, we made great strides in driving our key internal clinical development programs forward while advancing the build-out of our manufacturing capabilities at our new headquarters, and strengthening our balance sheet," said
Recent Operational Highlights
Gene Therapy Using NAV® Vectors for AAV-Mediated Antibody Delivery
Pivotal Program for RGX-314 for the Treatment of Wet Age-related Macular Degeneration (wet AMD)REGENXBIO announced inJanuary 2021 that it completed an End of Phase 2 meeting with theU.S. Food and Drug Administration (FDA) to discuss the details of a pivotal program to support a Biologics License Application (BLA).REGENXBIO plans to conduct two randomized, well-controlled clinical trials to evaluate the efficacy and safety of RGX-314 in patients with wet AMD, enrolling approximately 700 patients total.REGENXBIO expects to submit a BLA based on these trials in 2024.- ATMOSPHERE™, the first of two planned pivotal trials, is active and enrolling patients. The trial will enroll approximately 300 patients across two RGX-314 dose arms versus ranibizumab. The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at one year.
- The second pivotal trial is expected to be similar in design to ATMOSPHERE and
REGENXBIO plans to initiate the trial in the second half of 2021. - The Company plans to incorporate its scalable suspension cell culture manufacturing process to support future commercialization upon completion of a bridging study and the pivotal trials. The bridging study is expected to initiate in the first half of 2021.
- In
February 2021 ,REGENXBIO presented additional positive data from the patients enrolled in the ongoing Phase I/II trial of RGX-314 for the treatment of wet AMD and its Long-Term Follow-Up study. - As of
January 22, 2021 , RGX-314 continued to be generally well-tolerated across all dose cohorts. - Durable treatment effect was observed in patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314, including stable visual acuity, decreased retinal thickness, and reductions in anti-VEGF injection burden.
- Long-term, durable treatment effect was demonstrated in Cohort 3 over three years, including mean improvement in vision and stable retinal thickness, and reductions in anti-VEGF treatment burden.
- Suprachoroidal Delivery of RGX-314 for the Treatment of Wet AMD
- In
January 2021 ,REGENXBIO announced that it had completed enrollment of patients in Cohort 1 of AAVIATE™, a Phase II trial for the treatment of wet AMD. The Company plans to report interim efficacy data from Cohort 1 in the third quarter of 2021. Enrollment of patients in Cohort 2 has begun and is expected to be complete in the second quarter of 2021. - Suprachoroidal Delivery of RGX-314 for the Treatment of Diabetic Retinopathy (DR)
REGENXBIO expects to report initial data from ALTITUDE, a Phase II trial for the treatment of DR, in 2021.- Research Program for the Treatment of Hereditary Angioedema (HAE)
- REGENXBIO expects to provide a program update in 2021.
- Research Program for the Treatment of Neurodegenerative Diseases
- REGENXBIO continues to collaborate with Neurimmune AG on research programs targeting both alpha synuclein and tau, and expects to provide a program update in 2021.
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
- RGX-202 for the Treatment of Duchenne Muscular Dystrophy (DMD)
- In
January 2021 ,REGENXBIO announced the development of a potential one-time gene therapy for the treatment of DMD. RGX-202 is designed to deliver a novel microdystrophin transgene, which includes an extended coding region of the C-Terminal (CT) domain found in naturally occurring dystrophin, as well as other fundamental improvements. REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA in mid-2021.
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
- In
February 2021 ,REGENXBIO presented additional positive interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with MPS II. - As of
January 4, 2021 , RGX-121 is reported to be well-tolerated with no drug-related serious adverse events (SAEs) in eight patients dosed with RGX-121. - Biomarker data from patients in both cohorts indicate encouraging signals of I2S enzyme activity in the CNS following one-time administration of RGX-121, with consistent reductions of HS and D2S6, a component of HS. Patients in Cohorts 1 and 2 also demonstrated continued neurocognitive development and evidence of I2S enzyme activity in plasma and urine following administration of RGX-121.
REGENXBIO plans to evaluate a higher dose of RGX-121 in a third cohort of patients which is expected to begin enrolling in the first quarter of 2021.REGENXBIO expects to begin dosing patients in a Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with MPS II over the age of 5 years old in the first half of 2021.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
- In
December 2020 ,REGENXBIO announced dosing of the first patient in the Phase I/II trial of RGX-111 for the treatment of MPS I. Enrollment is ongoing.
- RGX-381 for the Treatment of Ocular Manifestations of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- REGENXBIO plans to submit an IND application, or foreign equivalent, for a Phase I/II study of RGX-381 in patients with CLN2 disease in the first half of 2021.
- RGX-181 for the Treatment of CLN2 Disease
- REGENXBIO expects to submit an IND for the intracisternal delivery of RGX-181 in the first quarter of 2021 and plans to initiate enrollment in a Phase I/II trial in the first half of 2021.
Operational Updates
- Current Good Manufacturing Practice (cGMP) Manufacturing Facility
- Construction of a new corporate, research and manufacturing headquarters in Rockville, Maryland continues, with plans to begin utilizing the new headquarters in the first half of 2021. The new cGMP production facility is expected to allow for production of NAV vectors at scales up to 2,000 liters using REGENXBIO's platform suspension cell culture process, which will complement REGENXBIO's current external manufacturing network and capabilities. The cGMP facility is expected to be operational starting in the first half of 2022.
NAV Technology Licensee Program Highlights
As of
- In
December 2020 , Eli Lilly and Company (Lilly) andPrevail Therapeutics Inc. (Prevail) announced a definitive agreement for Lilly to acquire Prevail, which closed inJanuary 2021 . Prevail licenses the NAV AAV9 vector for the development of gene therapy candidates for the treatment of Parkinson's disease and other neurodegenerative diseases.
- In
December 2020 , Rocket Pharmaceuticals, Inc. announced positive gene expression, clinical biomarker and preliminary functional data from their Phase I trial of RP-A501 for the treatment of Danon Disease. RP-A501 uses the NAV AAV9 vector.
Marketed NAV Technology Products
In
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
On
Financial Guidance
Based on its current operating plan,
Conference Call
In connection with this announcement,
About REGENXBIO Inc.
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, except per share data) |
||||||||
|
|
|||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
$ |
338,426 |
$ |
69,514 |
||||
Marketable securities |
137,314 |
226,696 |
||||||
Accounts receivable, net |
42,999 |
38,148 |
||||||
Prepaid expenses |
10,505 |
6,475 |
||||||
Other current assets |
1,953 |
4,199 |
||||||
Total current assets |
531,197 |
345,032 |
||||||
Marketable securities |
46,809 |
103,785 |
||||||
Accounts receivable |
3,267 |
4,155 |
||||||
Property and equipment, net |
56,467 |
28,973 |
||||||
Operating lease right-of-use assets |
63,815 |
10,078 |
||||||
Restricted cash |
1,330 |
1,330 |
||||||
Other assets |
5,279 |
4,555 |
||||||
Total assets |
$ |
708,164 |
$ |
497,908 |
||||
Liabilities and Stockholders' Equity |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ |
10,622 |
$ |
6,409 |
||||
Accrued expenses and other current liabilities |
49,082 |
24,846 |
||||||
Deferred revenue |
449 |
— |
||||||
Operating lease liabilities |
2,500 |
2,421 |
||||||
Liability related to sale of future royalties |
18,794 |
— |
||||||
Total current liabilities |
81,447 |
33,676 |
||||||
Deferred revenue |
3,783 |
3,333 |
||||||
Operating lease liabilities |
70,153 |
8,874 |
||||||
Liability related to sale of future royalties |
174,504 |
— |
||||||
Other liabilities |
524 |
1,828 |
||||||
Total liabilities |
330,411 |
47,711 |
||||||
Stockholders' equity |
||||||||
Preferred stock; no shares issued and outstanding at |
— |
— |
||||||
Common stock; 37,476 and 36,992 shares issued and outstanding at |
4 |
4 |
||||||
Additional paid-in capital |
667,181 |
627,810 |
||||||
Accumulated other comprehensive income (loss) |
(360) |
205 |
||||||
Accumulated deficit |
(289,072) |
(177,822) |
||||||
Total stockholders' equity |
377,753 |
450,197 |
||||||
Total liabilities and stockholders' equity |
$ |
708,164 |
$ |
497,908 |
|
||||||||||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS |
||||||||||||||||
(unaudited) |
||||||||||||||||
(in thousands, except per share data) |
||||||||||||||||
Three Months |
Years |
|||||||||||||||
Ended |
Ended |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||
Revenues |
||||||||||||||||
License and royalty revenue |
$ |
21,445 |
$ |
11,768 |
$ |
154,567 |
$ |
35,233 |
||||||||
Total revenues |
21,445 |
11,768 |
154,567 |
35,233 |
||||||||||||
Operating Expenses |
||||||||||||||||
Cost of revenues |
10,257 |
3,791 |
35,714 |
8,241 |
||||||||||||
Research and development |
47,180 |
33,807 |
166,294 |
124,185 |
||||||||||||
General and administrative |
17,571 |
14,450 |
63,817 |
51,815 |
||||||||||||
Provision for credit losses and other |
88 |
44 |
7,975 |
(10) |
||||||||||||
Total operating expenses |
75,096 |
52,092 |
273,800 |
184,231 |
||||||||||||
Loss from operations |
(53,651) |
(40,324) |
(119,233) |
(148,998) |
||||||||||||
Other Income |
||||||||||||||||
Interest income from licensing |
130 |
860 |
4,271 |
2,951 |
||||||||||||
Investment income |
13,794 |
10,609 |
9,723 |
48,559 |
||||||||||||
Interest expense |
(771) |
— |
(771) |
— |
||||||||||||
Total other income |
13,153 |
11,469 |
13,223 |
51,510 |
||||||||||||
Loss before income taxes |
(40,498) |
(28,855) |
(106,010) |
(97,488) |
||||||||||||
Income Tax Benefit (Expense) |
(5,743) |
2,391 |
(5,240) |
2,755 |
||||||||||||
Net loss |
$ |
(46,241) |
$ |
(26,464) |
$ |
(111,250) |
$ |
(94,733) |
||||||||
Other Comprehensive Income (Loss) |
||||||||||||||||
Unrealized gain (loss) on available-for-sale securities, net |
(623) |
(158) |
(565) |
885 |
||||||||||||
Total other comprehensive income (loss) |
(623) |
(158) |
(565) |
885 |
||||||||||||
Comprehensive loss |
$ |
(46,864) |
$ |
(26,622) |
$ |
(111,815) |
$ |
(93,848) |
||||||||
Net loss per share: |
||||||||||||||||
Basic |
$ |
(1.24) |
$ |
(0.72) |
$ |
(2.98) |
$ |
(2.58) |
||||||||
Diluted |
$ |
(1.24) |
$ |
(0.72) |
$ |
(2.98) |
$ |
(2.58) |
||||||||
Weighted-average common shares outstanding: |
||||||||||||||||
Basic |
37,418 |
36,905 |
37,281 |
36,690 |
||||||||||||
Diluted |
37,418 |
36,905 |
37,281 |
36,690 |
Contacts:
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
brendan@argotpartners.com
Media:
david.rosen@argotpartners.com
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