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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): February 28, 2023

 

 

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)	(IRS Employer Identification No.)
9804 Medical Center Drive
Rockville, Maryland				20850
(Address of Principal Executive Offices)				(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: (240) 552-8181

 

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:

☐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 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

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 February 28, 2023, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter and year ended December 31, 2022. 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 February 28, 2023 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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SIGNATURES

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:	February 28, 2023	By:	/s/ Patrick J. Christmas II
			Patrick J. Christmas II Executive Vice President, Chief Legal Officer

 

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EXHIBIT 99.1

 

 

 

 

REGENXBIO Reports Fourth Quarter 2022 and Full-Year 2022 Financial Results and Recent Operational Highlights

 

 

 

ROCKVILLE, Md., February 28, 2023 (PRNewswire) -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the fourth quarter and full-year ended December 31, 2022, and recent operational highlights.

 

“REGENXBIO enters the year with a strong pipeline of AAV Therapeutics. One year ago, we introduced our ‘5x’25’ strategy and I remain confident that we will achieve this goal,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “Our lead candidate, RGX-314, is being developed in partnership with AbbVie for the treatment of wet AMD and diabetic retinopathy, which present opportunities to make an impact in eye care for millions of patients. In 2022, we made meaningful progress with our RGX-314 trial enrollment objectives and geographic expansion planning. We also established in-house manufacturing to meet global clinical and commercial regulatory standards. We remain committed to developing treatments for rare diseases and made significant advancements with our pipeline of treatments for diseases such as Batten, Duchenne, Hurler and Hunter Syndrome, the latter of which we expect will support a second BLA filing using the accelerated approval pathway. Our history, science, resources, people and values combine to make us an industry leader in gene therapy and in the development of potentially ground-breaking therapies. We look forward to providing additional updates about our progress in 2023.”

 

 

Program Highlights and Milestones

 

RGX-314: RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie 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 AAV Therapeutic 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.

 

 

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RGX-121: RGX-121 is an investigational one-time AAV Therapeutic for the treatment of Mucopolysaccharidosis Type II (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 AAV Therapeutic for the treatment of severe Mucopolysaccharidosis Type I (MPS I), using the NAV AAV9 vector to deliver the α-l-iduronidase (IDUA) gene.

 

 

RGX-181: RGX-181 is an investigational one-time AAV Therapeutic for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, using the NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system.

 

 

RGX-381: RGX-381 is an investigational one-time AAV Therapeutic for the treatment of the ocular manifestations of CLN2 disease using the NAV AAV9 vector to deliver the TPP1 gene directly to the retina.

 

 

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Operational Updates

 

NAV Technology Licensee Program Highlights

As of December 31, 2022, 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 $565.2 million as of December 31, 2022, compared to $849.3 million as of December 31, 2021. The decrease was primarily driven by cash used to fund operating activities and capital expenditures and temporary unrealized losses on marketable debt securities during the year ended December 31, 2022.

 

Revenues: Revenues were $31.3 million and $112.7 million for the three months and year ended December 31, 2022, respectively, compared to $398.7 million and $470.3 million for the three months and year ended December 31, 2021, respectively. The decreases were primarily attributable to non-recurring revenue of $370.0 million recognized in the fourth quarter of 2021 upon the effectiveness of our eye care collaboration with AbbVie. The decrease in revenues for the year ended December 31, 2022 was partially offset by Zolgensma royalty revenues, which increased from $95.0 million in 2021 to $101.9 million in 2022.

 

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Research and Development Expenses: Research and development expenses were $62.5 million and $242.5 million for the three months and year ended December 31, 2022, respectively, compared to $48.0 million and $181.4 million for the three months and year ended December 31, 2021, respectively. The increases were primarily attributable to personnel costs as a result of increased headcount, and costs associated with clinical trials and manufacturing-related activities for our lead product candidates.

 

General and Administrative Expenses: General and administrative expenses were $21.2 million and $85.3 million for the three months and year ended December 31, 2022, respectively, compared to $22.0 million and $79.3 million for the three months and year ended December 31, 2021, respectively. The increase in general and administrative expenses for the year ended December 31, 2022 was primarily attributable to corporate overhead expenses.

 

Net Income (Loss): Net loss was $59.9 million, or $1.38 basic and diluted net loss per share, for the three months ended December 31, 2022, compared to net income of $294.0 million, or $6.87 basic and $6.67 diluted net income per share, for the three months ended December 31, 2021. Net loss was $280.3 million, or $6.50 basic and diluted net loss per share, for the year ended December 31, 2022, compared to net income of $127.8 million, or $3.01 basic and $2.91 diluted net income per share, for the year ended December 31, 2021

 

Financial Guidance

 

REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $565.2 million as of December 31, 2022 to fund its operations into 2025. This cash runway guidance is based on the Company’s current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under our RGX-314 collaboration.

 

Conference Call

 

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

 

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 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a “5x’25” strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.

 

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, clinical

 

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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, 2022, 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.

 

 

 

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REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)

 

		December 31, 2022				December 31, 2021
Assets
Current assets
Cash and cash equivalents		$	96,952			$	345,209
Marketable securities			267,690				112,230
Accounts receivable, net			28,082				32,439
Prepaid expenses			13,900				18,752
Other current assets			9,352				10,196
Total current assets			415,976				518,826
Marketable securities			200,560				391,907
Accounts receivable, net			1,504				2,262
Property and equipment, net			141,685				131,547
Operating lease right-of-use assets			65,116				60,904
Restricted cash			2,030				2,030
Other assets			6,397				6,428
Total assets		$	833,268			$	1,113,904
Liabilities and Stockholders’ Equity
Current liabilities
Accounts payable		$	27,213			$	11,387
Accrued expenses and other current liabilities			46,794				76,111
Deferred revenue			1,829				3,333
Operating lease liabilities			5,997				1,752
Liability related to sale of future royalties			48,601				37,889
Total current liabilities			130,434				130,472
Operating lease liabilities			88,802				84,929
Liability related to sale of future royalties			89,005				133,460
Other liabilities			8,832				745
Total liabilities			317,073				349,606
Stockholders’ equity
Preferred stock; no shares issued and outstanding    at December 31, 2022 and 2021			—				—
Common stock; 43,299 and 42,831 shares issued and    outstanding at December 31, 2022 and 2021, respectively			4				4
Additional paid-in capital			973,145				928,095
Accumulated other comprehensive loss			(15,401	)			(2,569	)
Accumulated deficit			(441,553	)			(161,232	)
Total stockholders’ equity			516,195				764,298
Total liabilities and stockholders’ equity		$	833,268			$	1,113,904

 

 

 

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REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

(unaudited)

(in thousands, except per share data)

 

		Three Months								Years
		Ended December 31,								Ended December 31,
		2022				2021				2022				2021
Revenues
License and royalty revenue		$	31,345			$	398,655			$	112,724			$	470,347
Total revenues			31,345				398,655				112,724				470,347
Operating Expenses
Cost of revenues			12,783				23,058				54,545				51,833
Research and development			62,505				47,978				242,453				181,437
General and administrative			21,210				22,040				85,281				79,333
Credit losses (recoveries)			—				(8,102	)			—				(2,569	)
Other operating expenses (income)			(7,382	)			85				(6,679	)			333
Total operating expenses			89,116				85,059				375,600				310,367
Income (loss) from operations			(57,771	)			313,596				(262,876	)			159,980
Other Income (Expense)
Interest income from licensing			77				19				342				719
Investment income			2,026				311				5,383				6,825
Interest expense			(4,310	)			(6,500	)			(23,254	)			(26,277	)
Total other income (expense)			(2,207	)			(6,170	)			(17,529	)			(18,733	)
Income (loss) before income taxes			(59,978	)			307,426				(280,405	)			141,247
Income Tax Benefit (Expense)			43				(13,403	)			84				(13,407	)
Net income (loss)		$	(59,935	)		$	294,023			$	(280,321	)		$	127,840
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net			2,855				(1,284	)			(12,832	)			(2,209	)
Total other comprehensive income (loss)			2,855				(1,284	)			(12,832	)			(2,209	)
Comprehensive income (loss)		$	(57,080	)		$	292,739			$	(293,153	)		$	125,631
Net income (loss) per share:
Basic		$	(1.38	)		$	6.87			$	(6.50	)		$	3.01
Diluted		$	(1.38	)		$	6.67			$	(6.50	)		$	2.91
Weighted-average common shares outstanding:
Basic			43,296				42,774				43,152				42,438
Diluted			43,296				44,084				43,152				43,913

 

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Contacts:

Dana Cormack

Corporate Communications

dcormack@regenxbio.com

 

Investors:

Chris Brinzey, ICR Westwicke

339-970-2843

Chris.brinzey@westwicke.com

 

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