REGENXBIO Reports First Quarter 2021 Financial Results and Operational Highlights
- Enrollment ongoing in ATMOSPHERE™, the first of two planned pivotal trials for the subretinal delivery of RGX-314 for the treatment of wet AMD
- Continued progress and expansion of Phase II AAVIATE® trial of RGX-314 utilizing in-office suprachoroidal delivery for the treatment of wet AMD
- Began dosing in Phase I/II trial of RGX-121, a one-time gene therapy for MPS II, in pediatric patients over 5 years old
- Completed dosing in Cohort 1 of ongoing trial of RGX-111, a one-time gene therapy for MPS I
- IND filing expected in mid-2021 for RGX-202, a novel, advanced microdystrophin gene therapy for treatment of Duchenne Muscular Dystrophy
$657 million in cash, cash equivalents and marketable securities as ofMarch 31, 2021 - Conference call
Wednesday, May 5 th at4:30 p.m. ET
"In the first quarter of 2021, we were able to build upon the important clinical advancements we made in 2020, including continued enrollment of patients in the Company's first pivotal trial for the treatment of wet AMD," 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)- Enrollment is ongoing in ATMOSPHERE™, the first of two planned pivotal trials to evaluate the efficacy and safety of RGX-314 in patients with wet AMD using the subretinal delivery approach.
- The randomized, well-controlled 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 manufacturing bridging study is now active to align with plans to incorporate
REGENXBIO's scalable suspension cell culture manufacturing process to support potential future commercialization of RGX-314. - The open-label study will enroll approximately 60 patients to evaluate two manufacturing process formulations of RGX-314. The primary endpoint of the trial is RGX-314 protein concentration in the aqueous humor at six months.
- Suprachoroidal Delivery of RGX-314 for the Treatment of Wet AMD
REGENXBIO plans to report interim data from Cohort 1 of AAVIATE, a Phase II trial of RGX-314 for the treatment of wet AMD, in the third quarter of 2021.REGENXBIO has completed dosing of patients in Cohort 2 of AAVIATE and expects to report interim data from Cohort 2 in the second half of 2021.- In addition,
REGENXBIO has expanded AAVIATE, and began dosing in a third cohort of patients. - Cohort 3 will evaluate the efficacy, safety and tolerability of RGX-314 in up to 20 patients who are neutralizing antibody (NAb) positive.
- The same dose evaluated in Cohort 2, 5.0x1011 genomic copies per eye (GC/eye) of RGX-314, will be delivered to patients in Cohort 3 via a single injection. As with Cohorts 1 and 2, patients in Cohort 3 will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.
- Suprachoroidal Delivery of RGX-314 for the Treatment of Diabetic Retinopathy (DR)
REGENXBIO continues to enroll patients in Cohort 1 in ALTITUDE™, a Phase II trial for the treatment of DR, and expects to report initial data 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)
REGENXBIO expects to submit an Investigational New Drug application (IND) to the FDA for RGX-202 for the treatment of DMD in mid-2021.
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
- In
April 2021 ,REGENXBIO announced that the first patient had been dosed in Cohort 3 of the ongoing Phase I/II trial of RGX-121 for the treatment of MPS II in patients up to 5 years old. Patients in the third cohort will receive a dose of 2.0x1011 GC/g of brain mass of RGX-121, delivered directly to the cerebrospinal fluid (CSF). - In addition, the first patient has been dosed in a second Phase I/II trial of RGX-121 for the treatment of pediatric patients with MPS II over the age of 5 years old.
- Up to six patients are expected to be enrolled in the multicenter, open-label trial, and RGX-121 will be administered at a dose level of 6.5x1010 GC/g of brain mass, delivered directly to the CSF.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
REGENXBIO has completed dosing of patients in Cohort 1 of the Phase I/II trial of RGX-111 for the treatment of MPS I.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- An IND was submitted to the
U.S. Food and Drug Administration (FDA), after which the FDA notifiedREGENXBIO in a letter that its proposed trial had been placed on clinical hold and the agency requested more information to support the initial dose selection and certain study drug administration procedures.REGENXBIO is evaluating theFDA's requests and plans to provide an update on the program in the second half of 2021.
- RGX-381 for the Treatment of Ocular Manifestations of CLN2 Disease
- Based on communication with the FDA and the update from the RGX-181 program,
REGENXBIO now expects to provide a program update for RGX-381 in the second half of 2021.
Operational Updates
- Current Good Manufacturing Practice (cGMP) Manufacturing Facility
REGENXBIO expects to begin utilizing its new headquarters inRockville, Maryland in the first half of 2021, and the cGMP facility which is expected to allow for production of NAV vectors at scales up to 2,000 liters usingREGENXBIO's platform suspension cell culture process is on track to be operational starting in the first half of 2022.
NAV Technology Licensee Program Highlights
As of
- In
January 2021 , Ultragenyx Pharmaceutical Inc. announced FDA Clearance of an IND for UX701, a gene therapy for the treatment of Wilson Disease. Ultragenyx is expected to begin enrolling patients in a single-protocol Phase 1/2/3 study in the second half of 2021. UX701 uses the NAV AAV9 vector.
- In
April 2021 , Astellas Gene Therapies, formerlyAudentes Therapeutics, Inc. , announced that the first patient has been dosed in its Phase I/II FORTIS trial evaluating AT845 in patients with late-onset Pompe disease. AT845 uses the NAV AAV8 vector.
Marketed NAV Technology Products
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
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 |
$ |
291,482 |
$ |
338,426 |
||||
Marketable securities |
149,398 |
137,314 |
||||||
Accounts receivable, net |
41,039 |
42,999 |
||||||
Prepaid expenses |
13,839 |
10,505 |
||||||
Other current assets |
2,880 |
1,953 |
||||||
Total current assets |
498,638 |
531,197 |
||||||
Marketable securities |
215,598 |
46,809 |
||||||
Accounts receivable, net |
2,859 |
3,267 |
||||||
Property and equipment, net |
89,342 |
56,467 |
||||||
Operating lease right-of-use assets |
62,607 |
63,815 |
||||||
Restricted cash |
1,330 |
1,330 |
||||||
Other assets |
9,068 |
5,279 |
||||||
Total assets |
$ |
879,442 |
$ |
708,164 |
||||
Liabilities and Stockholders' Equity |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ |
11,311 |
$ |
10,622 |
||||
Accrued expenses and other current liabilities |
41,605 |
49,082 |
||||||
Deferred revenue |
395 |
449 |
||||||
Operating lease liabilities |
1,843 |
2,500 |
||||||
Liability related to sale of future royalties |
28,807 |
18,794 |
||||||
Total current liabilities |
83,961 |
81,447 |
||||||
Deferred revenue |
3,729 |
3,783 |
||||||
Operating lease liabilities |
75,078 |
70,153 |
||||||
Liability related to sale of future royalties |
161,722 |
174,504 |
||||||
Other liabilities |
448 |
524 |
||||||
Total liabilities |
324,938 |
330,411 |
||||||
Stockholders' equity |
||||||||
Preferred stock; no shares issued and outstanding |
— |
— |
||||||
Common stock; 42,505 and 37,476 shares issued |
4 |
4 |
||||||
Additional paid-in capital |
895,079 |
667,181 |
||||||
Accumulated other comprehensive loss |
(1,368) |
(360) |
||||||
Accumulated deficit |
(339,211) |
(289,072) |
||||||
Total stockholders' equity |
554,504 |
377,753 |
||||||
Total liabilities and stockholders' equity |
$ |
879,442 |
$ |
708,164 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) |
||||||||
Three Months Ended |
||||||||
2021 |
2020 |
|||||||
Revenues |
||||||||
License and royalty revenue |
$ |
18,884 |
$ |
17,644 |
||||
Total revenues |
18,884 |
17,644 |
||||||
Operating Expenses |
||||||||
Cost of revenues |
4,851 |
3,409 |
||||||
Research and development |
39,722 |
37,035 |
||||||
General and administrative |
17,838 |
14,833 |
||||||
Provision for credit losses and other |
515 |
67 |
||||||
Total operating expenses |
62,926 |
55,344 |
||||||
Loss from operations |
(44,042) |
(37,700) |
||||||
Other Income (Expense) |
||||||||
Interest income from licensing |
29 |
848 |
||||||
Investment income (loss) |
580 |
(3,186) |
||||||
Interest expense |
(6,702) |
— |
||||||
Total other income (expense) |
(6,093) |
(2,338) |
||||||
Loss before income taxes |
(50,135) |
(40,038) |
||||||
Income Tax Expense |
(4) |
— |
||||||
Net loss |
$ |
(50,139) |
$ |
(40,038) |
||||
Other Comprehensive Loss |
||||||||
Unrealized loss on available-for-sale securities, net |
(1,008) |
(785) |
||||||
Total other comprehensive loss |
(1,008) |
(785) |
||||||
Comprehensive loss |
$ |
(51,147) |
$ |
(40,823) |
||||
Net loss per share, basic and diluted |
$ |
(1.20) |
$ |
(1.08) |
||||
Weighted-average common shares outstanding, basic and diluted |
41,819 |
37,104 |
Contacts:
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
brendan@argotpartners.com
Media:
david.rosen@argotpartners.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/regenxbio-reports-first-quarter-2021-financial-results-and-operational-highlights-301284916.html
SOURCE