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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): March 1, 2022

 

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.)

 

9804 Medical Center Drive 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	Trading symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.0001 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 March 1, 2022, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter and year ended December 31, 2021. 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 March 1, 2022 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: March 1, 2022		By:		/s/ Patrick J. Christmas II
				Patrick J. Christmas II
				Executive Vice President, Chief Legal Officer

 

 

EXHIBIT 99.1

                                                        

                

 

 

REGENXBIO Reports Fourth Quarter and Full-Year 2021 Financial Results and Recent Operational Highlights

 

 

 

 

ROCKVILLE, Md., March 1, 2022 (PRNewswire) -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the fourth quarter ended December 31, 2021, and recent operational highlights.

 

“I am pleased with our performance as an organization over this past quarter, which was highlighted by the successful closing of our eye care collaboration agreement with AbbVie,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We believe this collaboration, along with the continued positive interim data we presented at recent medical meetings, provides strong validation supporting the development of RGX-314 as a potential one-time gene therapy for the treatment of some of the largest ophthalmic markets. We look forward to working with AbbVie and providing additional data from our Phase II trials in diabetic retinopathy and wet AMD.”

 

Mr. Mills continued: “Our 2021 momentum has continued into the new year with a number of recent events that we believe raise the profile of our exciting neurodegenerative and neuromuscular pipeline. Most recently, we presented positive interim data at the 18^th Annual WORLDSymposium that demonstrated that RGX-121 and RGX-111 were well tolerated with encouraging evidence of CNS biomarker activity and improvements in neurodevelopmental function. We are looking forward to advancing RGX-202 for the treatment of Duchenne this year, following the FDA’s clearance of our Investigational New Drug application and granting of Rare Pediatric Disease Designation and Orphan Drug Designation, which mark important milestones and acknowledgement of the need for new treatment options for patients with Duchenne. RGX-202 is our first home-grown program leveraging our proprietary NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain. We believe that RGX-202 has the potential to address the limitations of

 

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other treatment approaches for this severe, degenerative disease, and we look forward to initiating this clinical trial.”  

 

 

Program Highlights and Milestones

 

RGX-314: RGX-314 is an investigational one-time gene therapy being developed for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other additional chronic retinal conditions. RGX-314 uses the NAV AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).

 

 

 

 

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RGX-202: RGX-202 is an investigational one-time gene therapy for the treatment of Duchenne Muscular Dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.

 

RGX-121: RGX-121 is an investigational one-time gene therapy for the treatment of Mucopolysaccharidosis (MPS II), also known as Hunter Syndrome, using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme.

 

 

RGX-111: RGX-111 is an investigational one-time gene therapy for the treatment of severe Mucopolysaccharidosis Type I (MPS I), using the NAV AAV9 vector to deliver the α-l-iduronidase (IDUA) gene.

 

 

Operational Updates

 

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NAV Technology Licensee Program Highlights

As of December 31, 2021, REGENXBIO's NAV® Technology Platform was being applied in one marketed product and multiple clinical stage partnered programs, with the potential to impact a broad range of therapeutic areas and disease indications.

 

Financial Results

 

Cash Position: Cash, cash equivalents and marketable securities were $849.3 million as of December 31, 2021, compared to $522.5 million as of December 31, 2020. The increase was primarily attributable to a $370.0 million upfront payment received upon the closing of the AbbVie eye care collaboration agreement in November 2021 and $216.1 million of aggregate net proceeds received from a follow-on public offering of common stock completed in January 2021, and was partially offset by cash used to fund operating activities and capital expenditures during the year ended December 31, 2021.

 

Revenues: Revenues were $398.7 million and $470.3 million for the three months and year ended December 31, 2021, respectively, compared to $21.4 million and $154.6 million for the three months and year ended December 31, 2020, respectively. The increases were primarily attributable to revenue of $370.0 million recognized in the fourth quarter of 2021 under our eye care collaboration agreement with AbbVie, and increases in Zolgensma royalty revenues, which increased by $7.1 million and $33.3 million for the three months and year ended December 31, 2021, respectively, as compared to the same periods in 2020. The increase in revenues for the year ended December 31, 2021 was partially offset by an $80.0 million milestone payment recognized as revenue in the third quarter of 2020 upon the achievement of $1.0 billion in cumulative net sales of Zolgensma. As reported by Novartis, sales of Zolgensma for the

 

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fourth quarter of 2021 increased by 35% as compared to the fourth quarter of 2020, driven by geographic expansion of product access.

 

Research and Development Expenses: Research and development expenses were $48.0 million and $181.4 million for the three months and year ended December 31, 2021, respectively, compared to $47.2 million and $166.3 million for the three months and year ended December 31, 2020, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs, and costs associated with clinical trial and regulatory activities for our lead product candidates.

 

General and Administrative Expenses: General and administrative expenses were $22.0 million and $79.3 million for the three months and year ended December 31, 2021, respectively, compared to $17.6 million and $63.8 million for the three months and year ended December 31, 2020. The increases were primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services.

 

Net Income (Loss): Net income was $294.0 million, or $6.87 basic and $6.67 diluted net income per share, for the three months ended December 31, 2021, compared to a net loss of $46.2 million, or $1.24 basic and diluted net loss per share, for the three months ended December 31, 2020. Net income was $127.8 million, or $3.01 basic and $2.91 diluted net income per share, for the year ended December 31, 2021, compared to a net loss of $111.3 million, or $2.98 basic and diluted net loss per share, for the year ended December 31, 2020.

 

Financial Guidance

 

Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $849.3 million as of December 31, 2021 to fund its operations, including the completion of its internal manufacturing capabilities and clinical advancement of its product candidates, into 2025.

 

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 7635958. 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," “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 and

 

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clinical trials. 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, 2021, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2022, 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. 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.

 

 

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REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)

 

 

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REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

(unaudited)

(in thousands, except per share data)

 

 

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Contacts:

Dana Cormack

Corporate Communications

dcormack@regenxbio.com

 

Investors:

Chris Brinzey, ICR Westwicke

339-970-2843

Chris.brinzey@westwicke.com