REGENXBIO Reports Third Quarter 2019 Financial and Operating Results and Recent Operational Highlights
- Initiation of RGX-314 Phase IIb trial for wet AMD and IND filing for Phase II trial in diabetic retinopathy now expected in Q1 2020 as a result of partial clinical hold following
FDA notification related to certain commercial, third-party surgical devices; matter is not related to RGX-314 gene therapy candidate - Positive interim update from RGX-314 Phase I/IIa trial for wet AMD presented in October at AAO
- Well-tolerated at all doses
- Anti-VEGF treatment burden was decreased by over 80% in Cohort 5, with 75% of subjects anti-VEGF injection-free up to 6 months after RGX-314 administration
- 50% of subjects in Cohort 3 were anti-VEGF injection-free at 1.5 years after RGX-314 administration
- Completed dosing of Cohort 2 in the RGX-501 Phase I/II trial for HoFH and Cohort 1 in the RGX-121 Phase I/II trial for MPS II; interim updates expected at year end
- Revenue includes
$9.2 million in royalties from the Q3 2019 sales ofNovartis' Zolgensma® $417 million in cash, cash equivalents and marketable securities as ofSeptember 30, 2019 ; expect to end 2019 with cash, cash equivalents and marketable securities of at least$365 million - Webcast and conference call scheduled for today at
4:30 p.m. ET
"We continue to make significant advances in our pipeline and NAV Technology Platform, expanding the pipeline with programs to deliver therapeutic antibodies, beginning an exciting collaboration with Neurimmune, and initiating construction of a new manufacturing facility," said
"We anticipate a short delay in the initiation of our Phase IIb trial for RGX-314 in wet AMD and our investigative new drug, or IND, application filing for diabetic retinopathy, following notification from the
"We are encouraged by the data from the Phase I/IIa clinical trial of RGX-314 in patients with wet AMD," said Dr.
Product Candidate Updates
Gene Therapy using NAV Vectors for AAV-Mediated Antibody Delivery:
- RGX-314 for the Treatment of Wet Age-related Macular Degeneration (wet AMD) and Diabetic Retinopathy (DR)
REGENXBIO has been informed by theU.S. Food and Drug Administration (FDA ) that they are assessing aspects of the surgical delivery system, specifically the use of certain third-party commercially-available devices that are used to deliver RGX-314 in our Phase I/IIa trial for the treatment of wet AMD. As previously announced, this trial has completed dosing of all 42 patients with RGX-314. While discussions with theFDA are ongoing, however, the IND for the trial has been placed on a partial clinical hold, which is not related to the gene therapy product candidate itself. The Company is not aware of any delivery system concerns or complications from the Phase I/IIa trial, nor is it aware of any concerns or actions related to these third-party device suppliers or other companies using similar devices in AAV gene therapy surgical delivery systems. Regular assessments and safety monitoring of all subjects enrolled in the Phase I/IIa trial continue to be performed as normal.- The Company is conducting a review of the third-party device components and believes that there are readily available and suitable alternatives for these third-party devices, if needed.
REGENXBIO will continue to work closely with theFDA regarding the potential replacement, testing or modification of third-party devices. As a result of these activities,REGENXBIO now expects to initiate the RGX-314 Phase IIb trial for wet AMD and file an IND for the Phase II trial in DR in the first quarter of 2020. - In
October 2019 ,REGENXBIO presented interim results from all five dose cohorts of the RGX-314 Phase I/IIa dose escalation clinical trial in subjects with wet AMD at theAmerican Academy of Ophthalmology Annual Meeting (AAO). As ofOctober 9, 2019 , RGX-314 continued to be well-tolerated across all cohorts, with no drug-related serious adverse events (SAEs) reported. Up to six months after administration of RGX-314, subjects in Cohort 5 demonstrated a reduction of over 80% from the mean annualized injection rate during the 12 months prior to administration of RGX-314, and 75% of subjects had not received anti-VEGF injections, with mean improvement in vision and retinal thickness. Durable effects on vision and retinal thickness had been demonstrated over 1.5 years in Cohort 3, and 50% of subjects remained free of anti-VEGF injections more than 1.5 years after RGX-314 administration. REGENXBIO continues to evaluate in-office delivery of RGX-314 to the suprachoroidal space. Clinical plans for this route of administration are expected to be announced in 2020.- Hereditary Angioedema (HAE)
REGENXBIO expects to provide an update in early 2020 on the preclinical studies of a new gene therapy product candidate for the treatment of HAE, as well as plans for clinical development.- Neurodegenerative Diseases
REGENXBIO initiated design and development of vectorized antibody therapies in collaboration withNeurimmune AG , using Neurimmune's Reverse Translational Medicine™ platform along with the Company's NAV Technology Platform. Initially, the companies are focusing on diseases associated with the accumulation and deposition of the microtubule-associated protein tau (tauopathies).
Gene Therapy using NAV Vectors for Rare Genetic Diseases:
- RGX-501 for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO completed dosing of subjects in Cohort 2 in the Phase I/II clinical trial evaluating RGX-501 for the treatment of HoFH with corticosteroid prophylaxis and expects to report interim data at the end of 2019.- The primary endpoint of this trial is safety and tolerability, and secondary endpoints include changes in low-density lipoprotein (LDL-C) and other lipid outcome measures.
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
REGENXBIO completed dosing of subjects in Cohort 1 in the Phase I/II clinical trial evaluating RGX-121 for the treatment of MPS II. The Company expects to report an interim data update from the Phase I/II clinical trial at the end of 2019.- The primary endpoint of this trial includes safety and tolerability, and secondary endpoints include change in biomarkers as measured in cerebrospinal fluid, serum and urine, and change in neurodevelopmental parameters.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
- RGX-111 was recently administered to a subject with MPS I through an investigator-initiated study at
CHOC Children's Hospital , following review and agreement by theFDA . As ofNovember 4, 2019 , RGX-111 has been well-tolerated. - Recruitment, screening and additional site activations are ongoing in the Phase I clinical trial evaluating RGX-111 for the treatment of MPS I.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) "Batten" Disease
REGENXBIO intends to submit an IND (or foreign equivalent) for the first-in-human clinical trial evaluating RGX-181 in the second half of 2020, following additional preclinical development and analyses to support clinical development.- Preclinical data presented at the
European Society of Gene & Cell Therapy (ESGCT) 27th Annual Congress highlighted outcomes from a murine mouse model for CLN2 Batten disease.
Other Recent Operational Highlights
- In
October 2019 ,REGENXBIO exercised the full option withClearside Biomedical, Inc. for exclusive worldwide rights to Clearside's proprietary, in-office SCS Microinjector™ for the delivery of AAV vectors to the suprachoroidal space to treat a number of indications.REGENXBIO continues to evaluate RGX-314 using Clearside's SCS Microinjector for in-office, non-surgical delivery into the suprachoroidal space to treat wet AMD, DR and other conditions for which anti-VEGF treatment is currently the standard of care, while continuing to advance its RGX-314 subretinal delivery program currently in development. - Construction of
REGENXBIO's current good manufacturing practice (cGMP) production facility continues as planned. The new cGMP production facility is designed to allow for production of NAV Technology-based vectors at scales up to 2,000 liters usingREGENXBIO's platform suspension cell culture process. The facility is expected to be operational in 2021.
NAV Technology Licensee Program Highlights
As of
Marketed Products
On
Partnered Product Candidates
- In
August 2019 ,Audentes Therapeutics, Inc. stated that enrollment in the pivotal expansion cohort data of ASPIRO, the clinical trial evaluating AT132 in subjects with X-Linked Myotubular Myopathy (XLMTM), is expected to be complete in the fall of 2019. The Biologics License Application (BLA) submission for AT132 is planned for mid-2020, and the Marketing Authorization Application (MAA) submission is planned for the second half of 2020. InOctober 2019 ,Audentes Therapeutics, Inc. announced new data from ASPIRO at the 24th International Annual Congress of theWorld Muscle Society . AT132 uses the NAV AAV8 vector. - In
September 2019 ,Ultragenyx Pharmaceutical, Inc. presented positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401 for the treatment of glycogen storage disease type Ia (GSDIa) at theSociety for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium. DTX401 uses the NAV AAV8 vector. - In
July 2019 ,REGENXBIO granted a non-exclusive worldwide license to the NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of Friedreich's ataxia toPfizer, Inc.
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. |
||||||||
September 30, 2019 |
December 31, 2018 |
|||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
$ |
79,594 |
$ |
75,561 |
||||
Marketable securities |
252,125 |
244,200 |
||||||
Accounts receivable |
19,175 |
8,587 |
||||||
Prepaid expenses |
6,920 |
5,734 |
||||||
Other current assets |
2,414 |
3,831 |
||||||
Total current assets |
360,228 |
337,913 |
||||||
Marketable securities |
85,408 |
150,819 |
||||||
Accounts receivable |
24,345 |
23,012 |
||||||
Property and equipment, net |
28,287 |
28,702 |
||||||
Operating lease right-of-use assets |
5,905 |
— |
||||||
Restricted cash |
1,053 |
1,053 |
||||||
Other assets |
4,011 |
2,315 |
||||||
Total assets |
$ |
509,237 |
$ |
543,814 |
||||
Liabilities and Stockholders' Equity |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ |
9,608 |
$ |
4,412 |
||||
Accrued expenses and other current liabilities |
19,231 |
17,164 |
||||||
Deferred revenue |
— |
600 |
||||||
Operating lease liabilities |
2,506 |
— |
||||||
Total current liabilities |
31,345 |
22,176 |
||||||
Deferred revenue |
3,333 |
3,333 |
||||||
Operating lease liabilities |
4,320 |
— |
||||||
Deferred rent |
— |
1,098 |
||||||
Financing lease obligations |
— |
5,854 |
||||||
Other liabilities |
1,844 |
2,505 |
||||||
Total liabilities |
40,842 |
34,966 |
||||||
Stockholders' equity |
||||||||
Preferred stock; $0.0001 par value; 10,000 shares |
— |
— |
||||||
Common stock; $0.0001 par value; 100,000 shares |
4 |
4 |
||||||
Additional paid-in capital |
619,386 |
592,580 |
||||||
Accumulated other comprehensive income (loss) |
363 |
(720) |
||||||
Accumulated deficit |
(151,358) |
(83,016) |
||||||
Total stockholders' equity |
468,395 |
508,848 |
||||||
Total liabilities and stockholders' equity |
$ |
509,237 |
$ |
543,814 |
REGENXBIO INC. |
||||||||||||||||
Three Months Ended |
Nine Months Ended |
|||||||||||||||
2019 |
2018 |
2019 |
2018 |
|||||||||||||
Revenues |
||||||||||||||||
License and royalty revenue |
$ |
14,700 |
$ |
5,306 |
$ |
23,465 |
$ |
177,728 |
||||||||
Total revenues |
14,700 |
5,306 |
23,465 |
177,728 |
||||||||||||
Operating Expenses |
||||||||||||||||
Cost of revenues |
2,494 |
517 |
4,450 |
6,797 |
||||||||||||
Research and development |
35,692 |
18,508 |
90,378 |
59,544 |
||||||||||||
General and administrative |
12,402 |
9,008 |
37,365 |
25,706 |
||||||||||||
Other operating expenses (income) |
8 |
(2) |
(54) |
31 |
||||||||||||
Total operating expenses |
50,596 |
28,031 |
132,139 |
92,078 |
||||||||||||
Income (loss) from operations |
(35,896) |
(22,725) |
(108,674) |
85,650 |
||||||||||||
Other Income |
||||||||||||||||
Interest income from licensing |
716 |
109 |
2,091 |
8,362 |
||||||||||||
Investment income |
431 |
2,122 |
37,950 |
4,177 |
||||||||||||
Total other income |
1,147 |
2,231 |
40,041 |
12,539 |
||||||||||||
Income (loss) before income taxes |
(34,749) |
(20,494) |
(68,633) |
98,189 |
||||||||||||
Income Tax Benefit (Expense) |
165 |
1,292 |
364 |
(2,558) |
||||||||||||
Net income (loss) |
$ |
(34,584) |
$ |
(19,202) |
$ |
(68,269) |
$ |
95,631 |
||||||||
Other Comprehensive Income (Loss) |
||||||||||||||||
Unrealized gain (loss) on available-for-sale |
(108) |
(103) |
1,043 |
(159) |
||||||||||||
Total other comprehensive income (loss) |
(108) |
(103) |
1,043 |
(159) |
||||||||||||
Comprehensive income (loss) |
$ |
(34,692) |
$ |
(19,305) |
$ |
(67,226) |
$ |
95,472 |
||||||||
Net income (loss) per share: |
||||||||||||||||
Basic |
$ |
(0.94) |
$ |
(0.56) |
$ |
(1.86) |
$ |
2.94 |
||||||||
Diluted |
$ |
(0.94) |
$ |
(0.56) |
$ |
(1.86) |
$ |
2.67 |
||||||||
Weighted-average common shares outstanding: |
||||||||||||||||
Basic |
36,813 |
33,988 |
36,618 |
32,576 |
||||||||||||
Diluted |
36,813 |
33,988 |
36,618 |
35,875 |
Zolgensma® is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of
Contacts:
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
heather@argotpartners.com
Media:
david.rosen@argotpartners.com
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