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ROCKVILLE, Md., May 5, 2021 /PRNewswire/ --
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 of March 31, 2021
Conference call Wednesday, May 5th at 4:30 p.m. ET
REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ended March 31, 2021, and recent operational highlights.
"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 Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Additionally, we are pleased to announce the advancement of our Phase II AAVIATE® trial for the suprachoroidal delivery of RGX-314 for wet AMD where we have completed enrollment in the second cohort of patients and expanded the trial to evaluate suprachoroidal delivery of RGX-314 in patients who are positive for neutralizing antibodies. This cohort of patients may provide us additional information about the effects of RGX-314 in neutralizing antibody positive patients, potentially broadening the patient population that could be treated with this in-office delivery approach."
Mr. Mills added: "Beyond RGX-314, we have continued to advance our MPS II clinical program, enrolling additional patients in our ongoing Phase I/II trial of RGX-121 in younger patients, as well as dosing the first patient in our Phase I/II trial of RGX-121 in older patients. The ongoing Phase I/II trial of RGX-111 in patients with MPS I also continues to advance, having completed dosing in Cohort 1. We are also on track to file an IND for RGX-202 in mid-2021. We look forward to providing additional updates from our programs in 2021."
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)
Suprachoroidal Delivery of RGX-314 for the Treatment of Wet AMD
Suprachoroidal Delivery of RGX-314 for the Treatment of Diabetic Retinopathy (DR)
Research Program for the Treatment of Hereditary Angioedema (HAE)
Research Program for the Treatment of Neurodegenerative Diseases
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
RGX-202 for the Treatment of Duchenne Muscular Dystrophy (DMD)
RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
RGX-381 for the Treatment of Ocular Manifestations of CLN2 Disease
Current Good Manufacturing Practice (cGMP) Manufacturing Facility
NAV Technology Licensee Program Highlights
As of March 31, 2021, REGENXBIO's NAV Technology Platform was being applied in one marketed product and multiple clinical stage programs, with over 20 partnered programs in total. REGENXBIO's NAV Technology Licensees are advancing product candidates in a broad range of therapeutic areas and disease indications. Recent updates from NAV Technology Licensees include:
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, formerly Audentes 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
REGENXBIO's NAV Technology Platform is being applied in one marketed product, Zolgensma®. On April 27, 2021, Novartis AG reported first quarter 2021 global Zolgensma sales revenue of $319 million.
Cash Position: Cash, cash equivalents and marketable securities were $656.5 million as of March 31, 2021, compared to $522.5 million as of December 31, 2020. The increase was primarily attributable to $216.1 million of aggregate net proceeds received from the Company's follow-on public offering of common stock completed in January 2021, including the full exercise of the underwriters' option to purchase additional shares in connection with the offering. The increase was partially offset by net cash used in operating activities of $41.1 million, cash used to purchase property and equipment of $31.0 million, and Zolgensma royalties paid to Healthcare Royalty Management, LLC of $9.5 million during the three months ended March 31, 2021.
Revenues: Revenues were $18.9 million for the three months ended March 31, 2021, compared to $17.6 million for the three months ended March 31, 2020. The increase was primarily attributable to Zolgensma royalty revenues, which increased by $8.3 million, from $10.0 million for the first quarter of 2020 to $18.3 million for the first quarter of 2021. As reported by Novartis, sales of Zolgensma for the first quarter of 2021 increased by 88% as compared to the first quarter of 2020. The increase in revenues was partially offset by non-recurring revenue recognized during the three months ended March 31, 2020 related to new licenses to the NAV Technology Platform granted by REGENXBIO during the period.
Research and Development Expenses: Research and development expenses were $39.7 million for the three months ended March 31, 2021, compared to $37.0 million for the three months ended March 31, 2020. The increase was primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs, and clinical trial expenses for our lead product candidates.
General and Administrative Expenses: General and administrative expenses were $17.8 million for the three months ended March 31, 2021, compared to $14.8 million for the three months ended March 31, 2020. The increase was primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services.
Net Loss: Net loss was $50.1 million, or $1.20 basic and diluted net loss per share, for the three months March 31, 2021, compared to net loss of $40.0 million, or $1.08 basic and diluted net loss per share, for the three months ended March 31, 2020.
Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $656.5 million as of March 31, 2021, to fund its operations, including the completion of its internal manufacturing capabilities and clinical advancement of its product candidates, into the second half of 2023.
In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. To access the live call by phone, dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 7044809. To access a live or recorded webcast of the call, please visit the "Investors" section of the REGENXBIO website at www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
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, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2020, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
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Investor Relations and Corporate Communications
Brendan Burns, 212-600-1902
David Rosen, 212-600-1902
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SOURCE REGENXBIO Inc.