REGENXBIO Reports Second Quarter 2019 Financial and Operating Results and Additional Positive Interim Phase I/IIa Trial Update for RGX-314 for the Treatment of Wet AMD
- Reports further positive interim update from RGX-314 Phase I/IIa trial for Wet AMD
- Well-tolerated at all doses
- Dose-dependent protein expression levels observed across all five dose cohorts
- 50% of subjects treated in Cohort 3 remain free of anti-VEGF injections at 18 months
- Company plans to present interim data update from all five dose cohorts in
October 2019 - Remains on-track to initiate Phase IIb trial in late 2019
- Continues dosing in the RGX-501 Phase I/II trial for HoFH and the RGX-121 Phase I/II trial for MPS II
- Expanded pipeline using NAV® Vectors to deliver therapeutic antibodies for the treatment of hereditary angioedema and neurodegenerative diseases
- Launch of first
FDA -approved gene therapy based onREGENXBIO's NAV Technology Platform,Novartis' Zolgensma® for the treatment of SMA $450 million in cash, cash equivalents and marketable securities as ofJune 30, 2019 - Webcast and conference call scheduled for today at
4:30 p.m. ET
"We are encouraged by the continued positive interim data from the Phase I/IIa RGX-314 trial and the potential of NAV gene therapy as a one-time treatment for wet AMD, and look forward to sharing six-month results from all five cohorts in the trial in
Mr. Mills added: "In addition to our excitement around RGX-314, the past quarter was highlighted by important milestones for
Lead Product Candidate Updates
Gene Therapy using NAV Vectors for AAV-Mediated Antibody Delivery:
- RGX-314 for the Treatment of Wet AMD
- As of
July 31, 2019 , 42 subjects across five dose cohorts have been treated in the Phase I/IIa trial of RGX-314. RGX-314 continues to be well-tolerated across all five dose cohorts, with no drug related serious adverse events (SAEs) reported. - Dose-dependent increases in RGX-314 protein expression levels, as measured from aqueous samples by electrochemiluminescence immunoassay (ECL) at approximately one month after administration of RGX-314, have been observed across all doses.
- 50% of subjects (3/6) in Cohort 3 continue to remain injection-free at 18 months.
REGENXBIO expects to present long-term clinical data from Cohort 3 out to 18 months and interim data from Cohort 4 and Cohort 5 inOctober 2019 .- Following the Type B meeting with the U.S. Food and Drug Administration (
FDA ) held in July 2019,REGENXBIO remains on track to initiate a Phase IIb trial for wet AMD in late 2019. - RGX-314 for the Treatment of Diabetic Retinopathy (DR)
REGENXBIO is on track to file a new Investigational New Drug (IND) application for a Phase II trial evaluating RGX-314 in subjects with DR in the second half of 2019.- Hereditary Angioedema (HAE)
REGENXBIO continues work on the HAE program and expects to provide an update in early 2020 on the preclinical studies, as well as plans for entering clinical trials.- Neurodegenerative Disease
REGENXBIO , in collaboration withNeurimmune AG , has initiated work to jointly develop novel therapies using AAV vectors to deliver human antibodies targeting abnormal tau and will provide program updates as the collaboration advances.
Gene Therapy using NAV Vectors for Rare Genetic Diseases:
- RGX-501 for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
- Dosing resumed and recruitment continues in the Phase I/II clinical trial evaluating RGX-501 for the treatment of HoFH.
REGENXBIO expects to report interim data from Cohort 2 with corticosteroid prophylaxis from the Phase I/II clinical trial in the second half of 2019.- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
- Recruitment, screening, and dosing continue in the first of two dose cohorts in the Phase I/II clinical trial evaluating RGX-121 for the treatment of MPS II.
REGENXBIO expects to present an interim data update from the Phase I/II clinical trial in the second half of 2019.- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
REGENXBIO expects to file an IND or foreign equivalent for the first-in-human clinical trial evaluating RGX-181 for the treatment of CLN2 in the second half of 2019.- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
- Recruitment, screening and additional site activations are ongoing in the Phase I clinical trial evaluating RGX-111 for the treatment of MPS I.
Other Recent Operational Highlights
- In
May 2019 ,REGENXBIO announced its plans to construct a current good manufacturing practice (cGMP) production facility inRockville, Maryland . The new cGMP production facility will be integrated intoREGENXBIO's previously announced new 139,000 square foot headquarters, for which construction is currently underway, and will allow for production of NAV Technology-based vectors at scales up to 2,000 liters usingREGENXBIO's platform suspension cell culture process. The facility will be designed to meet global regulatory requirements and is expected to be operational in 2021.
NAV Technology Licensee Program Highlights
As of
Marketed Products
- In
May 2019 ,Novartis announced theFDA approval of Zolgensma as a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.Novartis has stated that it anticipates regulatory approvals of Zolgensma in Japan and Europe in the second half of 2019.REGENXBIO earned a$3.5 million milestone payment upon Zolgensma's approval inthe United States and is eligible to receive an additional$80 million milestone payment upon the achievement of$1 billion in cumulative net sales of Zolgensma.REGENXBIO will receive tiered royalties on sales of Zolgensma up to a low double-digit percentage. Zolgensma uses the NAV AAV9 vector.
Partnered Product Candidates
- In
July 2019 ,REGENXBIO announced that it granted a non-exclusive worldwide license for its NAV AAV9 vector toPfizer Inc. for the development and commercialization of gene therapy for the treatment of Friedreich's ataxia, the most common hereditary ataxia. In return, Pfizer will provideREGENXBIO with an upfront payment, andREGENXBIO has the potential to receive ongoing fees, development and commercial milestone payments and royalties on net sales of products incorporating the NAV AAV9 vector. - In
July 2019 ,Abeona Therapeutics, Inc. announced positive data from its ongoing Phase I/II clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA). ABO-102 uses the NAV AAV9 vector. - In
June 2019 ,Rocket Pharmaceuticals, Inc. announced it dosed the first subject in its Phase I clinical trial of RP-A501 for the treatment of Danon disease. RP-A501 uses the NAV AAV9 vector.
Financial Results
Cash, cash equivalents and marketable securities were
Revenues were
Research and development expenses were
General and administrative expenses were
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," "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 INC. |
||||||||
CONSOLIDATED BALANCE SHEETS |
||||||||
(unaudited) |
||||||||
(in thousands, except per share data) |
||||||||
June 30, 2019 |
December 31, 2018 |
|||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
$ |
55,142 |
$ |
75,561 |
||||
Marketable securities |
286,354 |
244,200 |
||||||
Accounts receivable |
9,679 |
8,587 |
||||||
Prepaid expenses |
6,036 |
5,734 |
||||||
Other current assets |
2,281 |
3,831 |
||||||
Total current assets |
359,492 |
337,913 |
||||||
Marketable securities |
108,194 |
150,819 |
||||||
Accounts receivable |
23,955 |
23,012 |
||||||
Property and equipment, net |
27,330 |
28,702 |
||||||
Operating lease right-of-use assets |
5,904 |
— |
||||||
Restricted cash |
1,053 |
1,053 |
||||||
Other assets |
3,211 |
2,315 |
||||||
Total assets |
$ |
529,139 |
$ |
543,814 |
||||
Liabilities and Stockholders' Equity |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ |
5,851 |
$ |
4,412 |
||||
Accrued expenses and other current liabilities |
16,534 |
17,164 |
||||||
Deferred revenue |
— |
600 |
||||||
Operating lease liabilities |
2,276 |
— |
||||||
Total current liabilities |
24,661 |
22,176 |
||||||
Deferred revenue |
3,333 |
3,333 |
||||||
Operating lease liabilities |
4,654 |
— |
||||||
Deferred rent |
— |
1,098 |
||||||
Financing lease obligations |
— |
5,854 |
||||||
Other liabilities |
1,899 |
2,505 |
||||||
Total liabilities |
34,547 |
34,966 |
||||||
Stockholders' equity |
||||||||
Preferred stock; $0.0001 par value; 10,000 shares authorized, |
— |
— |
||||||
Common stock; $0.0001 par value; 100,000 shares authorized |
4 |
4 |
||||||
Additional paid-in capital |
610,891 |
592,580 |
||||||
Accumulated other comprehensive income (loss) |
471 |
(720) |
||||||
Accumulated deficit |
(116,774) |
(83,016) |
||||||
Total stockholders' equity |
494,592 |
508,848 |
||||||
Total liabilities and stockholders' equity |
$ |
529,139 |
$ |
543,814 |
REGENXBIO INC. |
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CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) |
||||||||||||||||
(unaudited) |
||||||||||||||||
(in thousands, except per share data) |
||||||||||||||||
Three Months Ended June 30, |
Six Months Ended June 30, |
|||||||||||||||
2019 |
2018 |
2019 |
2018 |
|||||||||||||
Revenues |
||||||||||||||||
License and royalty revenue |
$ |
7,881 |
$ |
40,031 |
$ |
8,765 |
$ |
172,422 |
||||||||
Total revenues |
7,881 |
40,031 |
8,765 |
172,422 |
||||||||||||
Operating Expenses |
||||||||||||||||
Cost of revenues |
1,927 |
3,872 |
1,956 |
6,280 |
||||||||||||
Research and development |
29,483 |
21,486 |
54,686 |
41,036 |
||||||||||||
General and administrative |
13,405 |
8,318 |
24,963 |
16,698 |
||||||||||||
Other operating expenses (income) |
(62) |
5 |
(62) |
33 |
||||||||||||
Total operating expenses |
44,753 |
33,681 |
81,543 |
64,047 |
||||||||||||
Income (loss) from operations |
(36,872) |
6,350 |
(72,778) |
108,375 |
||||||||||||
Other Income |
||||||||||||||||
Interest income from licensing |
762 |
6,898 |
1,375 |
8,253 |
||||||||||||
Investment income |
34,524 |
1,196 |
37,519 |
2,055 |
||||||||||||
Total other income |
35,286 |
8,094 |
38,894 |
10,308 |
||||||||||||
Income (loss) before income taxes |
(1,586) |
14,444 |
(33,884) |
118,683 |
||||||||||||
Income Tax Benefit (Expense) |
129 |
(3,850) |
199 |
(3,850) |
||||||||||||
Net income (loss) |
$ |
(1,457) |
$ |
10,594 |
$ |
(33,685) |
$ |
114,833 |
||||||||
Other Comprehensive Income (Loss) |
||||||||||||||||
Unrealized gain (loss) on available-for-sale |
530 |
132 |
1,151 |
(56) |
||||||||||||
Total other comprehensive income (loss) |
530 |
132 |
1,151 |
(56) |
||||||||||||
Comprehensive income (loss) |
$ |
(927) |
$ |
10,726 |
$ |
(32,534) |
$ |
114,777 |
||||||||
Net income (loss) per share: |
||||||||||||||||
Basic |
$ |
(0.04) |
$ |
0.33 |
$ |
(0.92) |
$ |
3.60 |
||||||||
Diluted |
$ |
(0.04) |
$ |
0.30 |
$ |
(0.92) |
$ |
3.29 |
||||||||
Weighted-average common shares outstanding: |
||||||||||||||||
Basic |
36,669 |
32,082 |
36,518 |
31,858 |
||||||||||||
Diluted |
36,669 |
35,272 |
36,518 |
34,884 |
Zolgensma® is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.
Contacts:
Investors:
heather@argotpartners.com
Media:
david.rosen@argotpartners.com
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