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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 08, 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 November 8, 2023, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter ended September 30, 2023. 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 8, 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:	November 8, 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 Announces Updated Strategic Plans and Third Quarter 2023 Financial Results

 

 

ROCKVILLE, Md., November 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the third quarter ended September 30, 2023, and recent operational highlights, including a strategic pipeline prioritization and corporate restructuring intended to significantly reduce operating expenses while continuing to support meaningful value generation from the Company’s strong pipeline of AAV Therapeutics.

 

“In the past two months, we reported exciting, positive clinical data from investigational treatments for diabetic retinopathy, and Duchenne, as well as had a very encouraging RMAT meeting with the FDA about expediting the BLA for our treatment for MPS II,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “These milestones demonstrate how our science is supporting avenues to accelerate development, and, today, we are following the data and making necessary decisions to focus our capabilities and resources on these differentiated product candidates which represent opportunities to bring ground-breaking AAV Therapeutics to millions of patients.”

 

 

PIPELINE PRIORITIZATION AND CORPORATE RESTRUCTURING

 

The following key strategic decisions support the pipeline prioritization and corporate restructuring related to product candidates that are differentiated, can be expedited, and support near-term and long-term value generation.

 

 

 

 

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As a result of the portfolio prioritization and corporate restructuring, REGENXBIO anticipates total savings of at least $100 million over the next two years. These anticipated cost savings along with $365 million in cash, cash equivalents and marketable securities as of September 30, 2023 are now expected to fund operations into the second half of 2025.

 

Mr. Mills added, “since our formation, REGENXBIO has been a leader in the field because of our mission to improve lives through the curative potential of gene therapy with our NAV® Technology Platform. As we refocus our capital allocation in a challenging economic environment and position the Company to meet important business goals, I want to express my gratitude for the commitment and dedication exhibited by our colleagues and partners in support of this mission.”

 

 

PROGRAM HIGHLIGHTS AND MILESTONES

 

ABBV-RGX-314: ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).

ABBV-RGX-314 is currently being evaluated in nine ongoing clinical trials, including two pivotal trials, a Phase II bridging study, a Long-term Follow-up study, and a Fellow Eye treatment study in patients with wet AMD, all utilizing subretinal delivery, as well as two Phase II clinical trials in patients with wet AMD and DR, and two corresponding Long-term Follow-up studies, all utilizing in-office suprachoroidal delivery.

 

 

RGX-202: RGX-202 is an investigational one-time AAV therapeutic for Duchenne, using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal 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 MPS II, also known as Hunter syndrome, using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase enzyme.

 

OPERATIONAL UPDATES

 

 

NAV® TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS

 

As of September 30, 2023, 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 $364.5 million as of September 30, 2023, compared to $565.2 million as of December 31, 2022. The decrease was primarily driven by cash used to fund operating activities during the nine months ended September 30, 2023.

Revenues: Revenues were $28.9 million for the three months ended September 30, 2023, compared to $26.5 million for the three months ended September 30, 2022. The increase was primarily attributable to Zolgensma royalty revenues, which increased from $25.2 million for the third quarter of 2022 to $28.4 million for the third quarter of 2023.

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Research and Development Expenses: Research and development expenses were $58.2 million for the three months ended September 30, 2023, compared to $63.3 million for the three months ended September 30, 2022. The decrease was primarily attributable to clinical trial and manufacturing expenses for ABBV-RGX-314 resulting from an increase in development cost reimbursement from AbbVie under our eye care collaboration and was partially offset by an increase in clinical trial expenses for our other lead product candidates.

General and Administrative Expenses: General and administrative expenses were $23.1 million for the three months ended September 30, 2023, compared to $20.9 million for the three months ended September 30, 2022. The increase was primarily attributable to personnel-related costs, expenses for professional services and other corporate overhead costs.

Net Loss: Net loss was $61.9 million, or $1.41 basic and diluted net loss per share, for the three months ended September 30, 2023, compared to a net loss of $75.5 million, or $1.75 basic and diluted net loss per share, for the three months ended September 30, 2022.

 

FINANCIAL GUIDANCE

 

As a result of the portfolio prioritization and corporate restructuring, REGENXBIO anticipates total savings of at least $100 million over the next two years. These anticipated cost savings along with $365 million in cash, cash equivalents and marketable securities as of September 30, 2023 are now expected to fund operations into the second half of 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 ABBV-RGX-314 collaboration.

 

CONFERENCE CALL

 

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. EST. A live audio webcast will be available at HERE. Interested parties may also pre-register for the earnings conference call HERE. Once registration is completed, participants will be provided a dial-in number with a personalized conference code to access the call. Those who plan on participating are advised to dial in 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 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

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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 strategic pipeline prioritization and corporate restructuring plan or other cost-saving measures, and expected charges associated with restructuring and any future cost reduction measures, 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, 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.

 

Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.

 

 

 

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REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)

 

		September 30, 2023				December 31, 2022
Assets
Current assets
Cash and cash equivalents		$	53,045			$	96,952
Marketable securities			248,842				267,690
Accounts receivable			28,043				28,082
Prepaid expenses			12,561				13,900
Other current assets			23,347				9,352
Total current assets			365,838				415,976
Marketable securities			62,639				200,560
Accounts receivable, net			1,078				1,504
Property and equipment, net			135,534				141,685
Operating lease right-of-use assets			61,773				65,116
Restricted cash			2,255				2,030
Other assets			4,669				6,397
Total assets		$	633,786			$	833,268
Liabilities and Stockholders’ Equity
Current liabilities
Accounts payable		$	21,859			$	27,213
Accrued expenses and other current liabilities			45,656				46,794
Deferred revenue			442				1,829
Operating lease liabilities			6,639				5,997
Liability related to sale of future royalties			52,750				48,601
Total current liabilities			127,346				130,434
Operating lease liabilities			84,058				88,802
Liability related to sale of future royalties			53,096				89,005
Other liabilities			6,186				8,832
Total liabilities			270,686				317,073
Stockholders’ equity
Preferred stock; no shares issued and outstanding    at September 30, 2023 and December 31, 2022			—				—
Common stock; 43,991 and 43,299 shares issued    and outstanding at September 30, 2023 and    December 31, 2022, respectively			4				4
Additional paid-in capital			1,012,667				973,145
Accumulated other comprehensive loss			(7,413	)			(15,401	)
Accumulated deficit			(642,158	)			(441,553	)
Total stockholders’ equity			363,100				516,195
Total liabilities and stockholders’ equity		$	633,786			$	833,268

 

 

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REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)

 

	Three Months								Nine Months
	Ended September 30,								Ended September 30,
	2023				2022				2023				2022
Revenues
License and royalty revenue	$	28,914			$	26,512			$	68,029			$	81,379
Total revenues		28,914				26,512				68,029				81,379
Operating Expenses
Cost of revenues		12,388				13,094				25,975				41,762
Research and development		58,183				63,313				176,585				179,948
General and administrative		23,083				20,921				69,415				64,071
Other operating expenses		220				229				279				703
Total operating expenses		93,874				97,557				272,254				286,484
Loss from operations		(64,960	)			(71,045	)			(204,225	)			(205,105	)
Other Income (Expense)
Interest income from licensing		56				18				166				265
Investment income		4,660				1,497				8,953				3,357
Interest expense		(1,624	)			(5,954	)			(5,499	)			(18,944	)
Total other income (expense)		3,092				(4,439	)			3,620				(15,322	)
Loss before income taxes		(61,868	)			(75,484	)			(200,605	)			(220,427	)
Income Tax Benefit		—				—				—				41
Net loss	$	(61,868	)		$	(75,484	)		$	(200,605	)		$	(220,386	)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net		2,685				(3,493	)			7,988				(15,687	)
Total other comprehensive income (loss)		2,685				(3,493	)			7,988				(15,687	)
Comprehensive loss	$	(59,183	)		$	(78,977	)		$	(192,617	)		$	(236,073	)
Net loss per share, basic and diluted	$	(1.41	)		$	(1.75	)		$	(4.60	)		$	(5.11	)
Weighted-average common shares outstanding, basic and diluted		43,945				43,251				43,644				43,103

 

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CONTACTS:

 

Dana Cormack

Corporate Communications

Dcormack@regenxbio.com

 

Chris Brinzey, ICR Westwicke

339-970-2843

Chris.Brinzey@westwicke.com

 

 

 

 

 

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