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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

FORM 8-K

 

Current Report

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): November 5, 2019

 

REGENXBIO Inc.

(Exact Name of Registrant as Specified in its Charter)

 

 

Delaware		001-37553		47-1851754
(State or other jurisdiction of incorporation)		(Commission File Number)		(I.R.S. Employer Identification No.)

 

9600 Blackwell Road, Suite 210 Rockville, Maryland	20850
(Address of principal executive offices)	(Zip Code)

(240) 552-8181

(Registrant’s telephone number, including area code)

N/A

(Former name or former address, if changed since last report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

☐	Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐	Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐	Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐	Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class	Ticker symbol(s)	Name of each exchange on which registered
Common Stock, $0.0001 par value per share	RGNX	The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 under the Securities Act of 1933 (17 CFR 230.405) or Rule 12b-2 under the Securities Exchange Act of 1934 (17 CFR 240.12b-2).

Emerging growth company  ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

 

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Item 2.02.

Results of Operations and Financial Condition.

On November 5, 2019, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter ended September 30, 2019. The press release is furnished as Exhibit 99.1 to this Current Report on Form 8‑K and is incorporated by reference herein.

The information in Item 2.02 of this Current Report on Form 8‑K and Exhibit 99.1 attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to liability under that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 9.01.

Financial Statements and Exhibits.

(d)Exhibits

 

Exhibit No.		Description
99.1		Press release dated November 5, 2019 relating to REGENXBIO Inc.’s financial results.
104		The cover page from this Current Report on Form 8-K, formatted in Inline XBRL.

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SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

		REGENXBIO INC.
Date: November 5, 2019		By:		/s/ Patrick J. Christmas II
				Patrick J. Christmas II
				Senior Vice President, General Counsel

 

 

EXHIBIT 99.1

 

 

                                                        

 

 

FOR IMMEDIATE RELEASE

 

REGENXBIO Reports Third Quarter 2019 Financial and Operating Results and Recent Operational Highlights

 

 

ROCKVILLE, Md., November 5, 2019 (PRNewswire) -- 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 quarter ended September 30, 2019, and recent operational highlights.

 

“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 Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “Our focus on patients drives us to build upon the clinical progress of gene therapy options for both large indications and rare diseases, and we are excited to lead the field in innovative approaches. We are also very encouraged by the early adoption of the first approved NAV Technology-based therapy, Novartis’ Zolgensma^®, for the treatment of SMA.”

 

“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 FDA regarding certain third-party commercially-available surgical devices that were used to deliver RGX-314 in our Phase I/IIa trial,” added Mr. Mills. “The notification is not related to the RGX-314 gene therapy itself, and we have not received reports of any device-related concerns or complications in the subjects already dosed in the Phase I/IIa trial. Assessments and monitoring of all enrolled subjects in the trial continue to be performed as usual. We are working with the FDA to address this matter, and as a result, we now plan to initiate our Phase IIb trial for RGX-314 in wet AMD and file our IND for diabetic retinopathy in Q1 2020,”

 

“We are encouraged by the data from the Phase I/IIa clinical trial of RGX-314 in patients with wet AMD,” said Dr. Stephen Pakola, Senior Vice President and Chief Medical Officer of REGENXBIO. “These data demonstrate significant long-term reduction of anti-VEGF treatment burden while maintaining or

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improving vision and retinal thickness after a single administration of our gene therapy. We look forward to initiating our Phase IIb trial of RGX-314 in wet AMD, and preparing to file an IND to evaluate RGX-314 in diabetic retinopathy, while continuing our collaboration with Clearside to evaluate the in-office suprachoroidal administration of RGX-314. We have also achieved important clinical development milestones in our other gene therapy programs, including completing dosing of Cohort 2 in our RGX-501 trial for HoFH and Cohort 1 in our RGX-121 trial for MPS II. Our internal pipeline of novel gene therapies positions us to develop meaningful potential treatments for a broad spectrum of patients with significant unmet needs.”

 

Product Candidate Updates

 

Gene Therapy using NAV Vectors for AAV-Mediated Antibody Delivery:

 

 

 

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Gene Therapy using NAV Vectors for Rare Genetic Diseases:

 

 

 

 

 

 

Other Recent Operational Highlights

 

 

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NAV Technology Licensee Program Highlights

 

As of October 31, 2019, REGENXBIO's NAV Technology Platform was being applied in one marketed product, Novartis AG’s Zolgensma, and more than 20 partnered product candidates in development by NAV Technology Licensees. 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 Products

 

On October 22, 2019, Novartis AG reported Q3 2019 U.S. Zolgensma sales revenue of $160 million, and REGENXBIO recognized royalty revenue of $9.2 million in Q3 2019 as a result of these sales.

 

Partnered Product Candidates

 

 

 

 

Financial Results

 

Cash, cash equivalents and marketable securities were $417.1 million as of September 30, 2019, compared to $470.6 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities during the nine months ended September 30, 2019, was primarily attributable to $82.2 million of net cash used in operating activities during the period, partially offset by an unrealized gain of $29.4 million related to our marketable equity securities of Prevail Therapeutics Inc.

 

Revenues were $14.7 million for the three months ended September 30, 2019, compared to $5.3 million for the three months ended September 30, 2018. The increase was primarily attributable $9.2 million of royalty revenue recognized during the third quarter of 2019 related to net sales of Zolgensma. Commercial sales of Zolgensma commenced in the second quarter of 2019, and we are also eligible to receive a milestone payment of $80.0 million from AveXis, Inc. upon the achievement of $1 billion in cumulative net sales of Zolgensma.

 

Research and development expenses were $35.7 million for the three months ended September 30, 2019, compared to $18.5 million for the three months ended September 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs and external expenses associated with conducting clinical trials and manufacturing-related services.

 

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General and administrative expenses were $12.4 million for the three months ended September 30, 2019, compared to $9.0 million for the three months ended September 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount.

 

Net loss was $34.6 million, or $0.94 basic and diluted net loss per share, for the three months ended September 30, 2019, compared to net loss of $19.2 million, or $0.56 basic and diluted net loss per share, for the three months ended September 30, 2018.

 

Financial Guidance

 

Based on its current operating plan, REGENXBIO now expects its balance in cash, cash equivalents and marketable securities to be at least $365 million as of December 31, 2019.

 

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 1159968. 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.

 

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, research and development activities, preclinical studies, 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, 2018, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and

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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. 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.

 

 

 

 

 

 

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REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands, except per share data)

 

 

 

 

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REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

(unaudited)

(in thousands, except per share data)

 

 

Zolgensma^® is a registered trademark of AveXis. All other trademarks referenced herein are registered trademarks of REGENXBIO.

 

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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