REGENXBIO Reports Second Quarter 2020 Financial Results and Operational Highlights
"Now halfway through 2020 and with the backdrop of the challenging COVID-19 pandemic, I am pleased that we have made significant progress in our gene therapy programs and continue to make important decisions with respect to our pipeline and plans. We expect to initiate the RGX-
"Finally, we have made the difficult decision to discontinue the development of RGX-501 for the treatment of HoFH. This program was one of our first clinical-stage gene therapy programs, and we are grateful for the support of the HoFH patient community over the years. We will look for opportunities to support the continued advancement of this program through business development."
Recent Operational Highlights
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody Delivery
- RGX-314 for the Treatment of Wet Age-related Macular Degeneration (wet AMD)
- On
August 4, 2020 ,REGENXBIO reported data from the RGX-314 Phase I/IIa trial using subretinal delivery. RGX-314 was generally well-tolerated as ofJuly 13, 2020 . For Cohorts 4 and 5: - Durable treatment effect was observed with stable to improved visual acuity and retinal thickness at one year
- Meaningful reductions in anti-VEGF treatment burden were demonstrated over one year
- Dose-dependent intraocular RGX-314 protein expression levels were observed over one year
REGENXBIO expects to initiate a pivotal program for the subretinal delivery of RGX-314 for the treatment of wet AMD by the end of 2020.REGENXBIO plans to dose patients in AAVIATE, the Phase II trial for the suprachoroidal delivery of RGX-314 using the SCS Microinjector™ for the treatment of wet AMD, in the third quarter of 2020.- Interim data is expected to be reported from the first cohort by the end of 2020.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
REGENXBIO expects to initiate a Phase II trial in the second half of 2020 and interim data is expected to be reported in 2021.- Research Program for the Treatment of Hereditary Angioedema (HAE)
- Preclinical data from the HAE program was presented at the
American Society of Gene andCell Therapy (ASGCT) 23rd Annual Meeting inMay 2020 , demonstrating biological activity in a model of inflammatory edema and inhibition of kallikrein comparable to other anti-kallikrein treatments. REGENXBIO expects to provide a program update in the second half of 2020.- Research Program for the Treatment of Neurodegenerative Diseases
REGENXBIO continues to collaborate withNeurimmune AG to design and develop vectorized antibody therapies targeting both alpha synuclein and tau.REGENXBIO expects to provide a program update in the second half of 2020.
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
- In July,
REGENXBIO announced the completed dosing of three patients in Cohort 2 of its Phase I/II trial. As ofJune 24, 2020 , RGX-121 was reported to be well-tolerated in patients across two dose levels, with no drug-related serious adverse events (SAEs). - The Company expects to provide a data and program update in the second half of 2020.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
- In July,
REGENXBIO reported data under a single-patient investigator-initiated Investigational New Drug application (IND) conducted at CHOC Children's, which demonstrated encouraging biomarker activity and continued progression of neurocognitive development. As ofJune 9, 2020 , RGX-111 was reported to be well-tolerated in this patient, with no drug-related SAEs. - Recruitment and patient screening are ongoing in the Company's Phase I/II clinical trial evaluating RGX-111 for the treatment of MPS I.
REGENXBIO expects to provide a program update by the end of 2020.- RGX-381 for the Treatment of Ocular Manifestations of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- RGX-381 is a new program targeting the ocular manifestations of CLN2 disease in patients and is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the retina.
REGENXBIO believes that one-time administration of RGX-381 could provide a durable source of TPP1 activity in the retina, thereby potentially preventing visual decline. There is currently no available treatment for ocular manifestations of CLN2 disease. REGENXBIO expects to submit an IND for a Phase I/II study of RGX-381 in patients with CLN2 disease in the second half of 2020 and plans to initiate enrollment in the first half of 2021.- The company has initiated two non-interventional natural history studies to assess ocular manifestations in CLN2 disease.
- The Orphan Drug Designation and Rare Pediatric Disease Designation granted to the RGX-181 program also apply to the RGX-381 program.
- RGX-181 for the Treatment of CLN2 Disease
REGENXBIO expects to submit an IND for the intracisternal delivery of RGX-181 by the end of 2020, and plans to initiate enrollment in a Phase I/II trial in the first half of 2021.- RGX-501 for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO has discontinued internal clinical development of RGX-501 for the treatment of HoFH in order to focus on furthering other gene therapy programs in its pipeline. The Company plans to evaluate strategic alternatives to support the continued advancement of this program, including through partnering.- Research Program for the Treatment of Neuromuscular Disorders
REGENXBIO expects to announce plans for clinical development of a potential treatment for a neuromuscular disorder using NAV AAV8 in the second half of 2020.
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 usingREGENXBIO's platform suspension cell culture process, which will complementREGENXBIO's current external manufacturing network and capabilities. The cGMP facility is expected to be operational starting in late 2021.
Marketed NAV Technology Product Highlights
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,
Q2 Earnings 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," "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,
SCS Microinjector™ is a trademark of Clearside Biomedical, Inc. Zolgensma® is a registered trademark of
CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands, except per share data) |
|||||
|
|
||||
Assets |
|||||
Current assets |
|||||
Cash and cash equivalents |
$ |
94,222 |
$ |
69,514 |
|
Marketable securities |
174,964 |
226,696 |
|||
Accounts receivable |
42,876 |
38,148 |
|||
Prepaid expenses |
9,749 |
6,475 |
|||
Other current assets |
7,271 |
4,199 |
|||
Total current assets |
329,082 |
345,032 |
|||
Marketable securities |
70,054 |
103,785 |
|||
Accounts receivable |
3,618 |
4,155 |
|||
Property and equipment, net |
32,696 |
28,973 |
|||
Operating lease right-of-use assets |
8,635 |
10,078 |
|||
Restricted cash |
1,330 |
1,330 |
|||
Other assets |
4,323 |
4,555 |
|||
Total assets |
$ |
449,738 |
$ |
497,908 |
|
Liabilities and Stockholders' Equity |
|||||
Current liabilities |
|||||
Accounts payable |
$ |
10,901 |
$ |
6,409 |
|
Accrued expenses and other current liabilities |
25,839 |
24,846 |
|||
Deferred revenue |
450 |
— |
|||
Operating lease liabilities |
3,013 |
2,421 |
|||
Total current liabilities |
40,203 |
33,676 |
|||
Deferred revenue |
4,007 |
3,333 |
|||
Operating lease liabilities |
7,085 |
8,874 |
|||
Other liabilities |
582 |
1,828 |
|||
Total liabilities |
51,877 |
47,711 |
|||
Stockholders' equity |
|||||
Preferred stock; |
— |
— |
|||
Common stock; |
4 |
4 |
|||
Additional paid-in capital |
648,729 |
627,810 |
|||
Accumulated other comprehensive income |
750 |
205 |
|||
Accumulated deficit |
(251,622) |
(177,822) |
|||
Total stockholders' equity |
397,861 |
450,197 |
|||
Total liabilities and stockholders' equity |
$ |
449,738 |
$ |
497,908 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) |
|||||||||||||
Three Months Ended |
Six Months Ended |
||||||||||||
2020 |
2019 |
2020 |
2019 |
||||||||||
Revenues |
|||||||||||||
License and royalty revenue |
$ |
16,566 |
$ |
7,881 |
$ |
34,210 |
$ |
8,765 |
|||||
Total revenues |
16,566 |
7,881 |
34,210 |
8,765 |
|||||||||
Operating Expenses |
|||||||||||||
Cost of revenues |
4,684 |
1,927 |
8,093 |
1,956 |
|||||||||
Research and development |
38,111 |
29,483 |
75,146 |
54,686 |
|||||||||
General and administrative |
15,554 |
13,405 |
30,387 |
24,963 |
|||||||||
Other operating expenses (income) |
50 |
(62) |
117 |
(62) |
|||||||||
Total operating expenses |
58,399 |
44,753 |
113,743 |
81,543 |
|||||||||
Loss from operations |
(41,833) |
(36,872) |
(79,533) |
(72,778) |
|||||||||
Other Income |
|||||||||||||
Interest income from licensing |
1,849 |
762 |
2,697 |
1,375 |
|||||||||
Investment income |
5,722 |
34,524 |
2,536 |
37,519 |
|||||||||
Total other income |
7,571 |
35,286 |
5,233 |
38,894 |
|||||||||
Loss before income taxes |
(34,262) |
(1,586) |
(74,300) |
(33,884) |
|||||||||
Income Tax Benefit |
500 |
129 |
500 |
199 |
|||||||||
Net loss |
$ |
(33,762) |
$ |
(1,457) |
$ |
(73,800) |
$ |
(33,685) |
|||||
Other Comprehensive Income |
|||||||||||||
Unrealized gain on available-for-sale securities, net |
1,330 |
530 |
545 |
1,151 |
|||||||||
Total other comprehensive income |
1,330 |
530 |
545 |
1,151 |
|||||||||
Comprehensive loss |
$ |
(32,432) |
$ |
(927) |
$ |
(73,255) |
$ |
(32,534) |
|||||
Basic and diluted net loss per share |
$ |
(0.91) |
$ |
(0.04) |
$ |
(1.98) |
$ |
(0.92) |
|||||
Weighted-average basic and diluted common shares outstanding |
37,257 |
36,669 |
37,180 |
36,518 |
Contacts:
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com
Media:
david.rosen@argotpartners.com
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