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REGENXBIO Reports Fourth Quarter and Full-Year 2019 Financial Results and Operational Highlights

ROCKVILLE, Md., Feb. 26, 2020 /PRNewswire/ --

  • Additional data from RGX-314 Phase I/IIa trial for wet AMD expected in 1H 2020
  • RGX-314 clinical trials for treatment of wet AMD and diabetic retinopathy expected to advance in 2020 using subretinal and suprachoroidal delivery approaches
  • Additional interim data from RGX-121 Phase I/II trial for MPS II expected in 2020
  • Plans to expand rare disease pipeline into neuromuscular disorders in 2H 2020
  • $400 million in cash, cash equivalents and marketable securities as of December 31, 2019
  • Conference call Wednesday, February 26th at 4:30 p.m. ET

REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced financial results for the fourth quarter and full year ended December 31, 2019, and recent operational highlights.

"In 2020, we are broadening our internal gene therapy pipeline using our proprietary NAV Technology Platform, and advancing key programs including RGX-314 for the treatment of retinal diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "As we drive the RGX-314 program forward with new trials in wet AMD and diabetic retinopathy planned for later this year, we are also advancing our RGX-121 program in MPS II based on encouraging data from the first cohort of patients. We are pleased with the advancement of the RGX-111 and RGX-501 programs, and look forward to submitting an IND for RGX-181 and providing updates for our research programs in HAE and neurodegenerative diseases later this year."

Mr. Mills added: "Based on our in-house research and development team's work, we also plan to expand our rare disease platform to include a potential treatment for a neuromuscular disorder using NAV AAV8, which we believe has the potential to treat patients who lack treatments or who are currently underserved by existing therapies. I look forward to providing further updates on this work in the second half of the year."

Recent Operational Highlights

Gene Therapy Using NAV Vectors for AAV-Mediated Antibody Delivery

  • RGX-314 for the Treatment of Wet AMD
  • 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-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-501 for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
  • Research Program for the Treatment of Neuromuscular Disorders

Operational Updates and Anticipated Milestones in 2020

  • Current Good Manufacturing Practice (cGMP) Manufacturing Facility

NAV Technology Licensee Program Highlights

As of December 31, 2019, REGENXBIO's NAV Technology Platform was being applied in one marketed product and more than 20 partnered product candidates in development. Fifteen of these partnered product candidates are in active clinical development. 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:

Marketed NAV Technology Products

  • On January 29, 2020, Novartis AG reported fourth quarter 2019 U.S. Zolgensma® sales revenue of $186 million, and REGENXBIO recognized royalty revenue of $10.7 million in the fourth quarter of 2019 as a result of these sales.
  • Novartis has reported that Zolgensma is currently under regulatory review in Europe with an anticipated regulatory decision in the first quarter of 2020 and in Japan with an anticipated regulatory decision in the first half of 2020. Zolgensma uses the NAV AAV9 vector.

Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $400.0 million as of December 31, 2019, compared to $470.6 million as of December 31, 2018. The decrease was primarily attributable to $107.7 million of net cash used in operating activities during 2019, partially offset by realized and unrealized gains of $37.8 million related to our marketable equity securities of Prevail Therapeutics Inc.

Revenues: Revenues were $11.8 million and $35.2 million for the three months and year ended December 31, 2019, respectively, compared to $40.8 million and $218.5 million for the three months and year ended December 31, 2018, respectively. The decreases were primarily attributable to $176.1 million of non-recurring revenue recognized in 2018 under REGENXBIO's March 2014 license agreement with AveXis, Inc., as amended, as well as $35.6 million of non-recurring revenue recognized in the fourth quarter of 2018 under REGENXBIO's license agreement with Abeona Therapeutics Inc. The decreases in revenue were partially offset by $10.7 million and $20.8 million of royalty revenue recognized during the three months and year ended December 31, 2019, respectively, related to net sales of Zolgensma. Commercial sales of Zolgensma commenced in the second quarter of 2019, and we are eligible to receive a milestone payment of $80.0 million from AveXis upon the achievement of $1.0 billion in cumulative net sales of Zolgensma.

Research and Development Expenses: Research and development expenses were $33.8 million and $124.2 million for the three months and year ended December 31, 2019, respectively, compared to $24.3 million and $83.9 million for the three months and year ended December 31, 2018, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs, expenses associated with conducting clinical trials for our lead product candidates, and externally sourced services for preclinical, regulatory and manufacturing-related activities.

General and Administrative Expenses: General and administrative expenses were $14.5 million and $51.8 million for the three months and year ended December 31, 2019, respectively compared to $11.1 million and $36.9 million for the three months and year ended December 31, 2018, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services.

Net Loss: Net loss was $26.5 million, or $0.72 basic and diluted net loss per share, and $94.7 million, or $2.58 basic and diluted net loss per share, for the three months and year ended December 31, 2019, respectively, compared to net income of $4.3 million, or $0.12 basic and $0.11 diluted net income per share, and $99.9 million, or $2.99 basic and $2.73 diluted net income per share, for the three months and year ended December 31, 2018, respectively.

Financial Guidance

Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $400 million to fund the completion of its internal manufacturing capabilities and clinical advancement of its product candidates into 2022.

Conference Call

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 2439067. To access a live or recorded webcast of the call and accompanying slides, 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.

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'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, 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, 2019, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2020, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the 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. 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 AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.

 

REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands, except per share data)




December 31, 2019



December 31, 2018


Assets









Current assets









Cash and cash equivalents


$

69,514



$

75,561


Marketable securities



226,696




244,200


Accounts receivable



38,148




8,587


Prepaid expenses



6,475




5,734


Other current assets



4,199




3,831


Total current assets



345,032




337,913


Marketable securities



103,785




150,819


Accounts receivable



4,155




23,012


Property and equipment, net



28,973




28,702


Operating lease right-of-use assets



10,078





Restricted cash



1,330




1,053


Other assets



4,555




2,315


Total assets


$

497,908



$

543,814


Liabilities and Stockholders' Equity









Current liabilities









Accounts payable


$

6,409



$

4,412


Accrued expenses and other current liabilities



24,846




17,164


Deferred revenue






600


Operating lease liabilities



2,421





Total current liabilities



33,676




22,176


Deferred revenue



3,333




3,333


Operating lease liabilities



8,874





Deferred rent






1,098


Financing lease obligations






5,854


Other liabilities



1,828




2,505


Total liabilities



47,711




34,966


Stockholders' equity









Preferred stock; $0.0001 par value; 10,000 shares 
     authorized, and no shares issued and outstanding 
     
at December 31, 2019 and December 31, 2018







Common stock; $0.0001 par value; 100,000 shares 
     authorized at December 31, 2019 and December 31, 2018;
 
     36,992 and 36,120 shares issued and outstanding at 
     December 31, 2019 and December 31, 2018, respectively



4




4


Additional paid-in capital



627,810




592,580


Accumulated other comprehensive income (loss)



205




(720)


Accumulated deficit



(177,822)




(83,016)


Total stockholders' equity



450,197




508,848


Total liabilities and stockholders' equity


$

497,908



$

543,814


 

 

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

(unaudited)

(in thousands, except per share data)




Three Months Ended December 31,



Years Ended December 31,




2019



2018



2019



2018


Revenues

















License and royalty revenue


$

11,768



$

40,777



$

35,233



$

218,505


Total revenues



11,768




40,777




35,233




218,505


Operating Expenses

















Cost of revenues



3,791




2,843




8,241




9,640


Research and development



33,807




24,329




124,185




83,873


General and administrative



14,450




11,144




51,815




36,850


Other operating expenses (income)



44




11




(10)




42


Total operating expenses



52,092




38,327




184,231




130,405


Income (loss) from operations



(40,324)




2,450




(148,998)




88,100


Other Income

















Interest income from licensing



860




584




2,951




8,946


Investment income



10,609




2,893




48,559




7,070


Total other income



11,469




3,477




51,510




16,016


Income (loss) before income taxes



(28,855)




5,927




(97,488)




104,116


Income Tax Benefit (Expense)



2,391




(1,621)




2,755




(4,179)


Net income (loss)


$

(26,464)



$

4,306



$

(94,733)



$

99,937


Other Comprehensive Income (Loss)

















Unrealized gain (loss) on available-for-sale 
     securities, net



(158)




154




885




(5)


Total other comprehensive income (loss)



(158)




154




885




(5)


Comprehensive income (loss)


$

(26,622)



$

4,460



$

(93,848)



$

99,932


Net income (loss) per share:

















Basic


$

(0.72)



$

0.12



$

(2.58)



$

2.99


Diluted


$

(0.72)



$

0.11



$

(2.58)



$

2.73


Weighted-average common shares outstanding:

















Basic



36,905




35,951




36,690




33,427


Diluted



36,905




38,933




36,690




36,648


 

Contacts:

Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com

Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com

Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com

(PRNewsfoto/REGENXBIO Inc.)
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SOURCE REGENXBIO Inc.