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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): May 03, 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 May 3, 2023, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter ended March 31, 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 May 3, 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:	May 3, 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 First Quarter 2023 Financial Results and Recent Operational Highlights

 

 

 

ROCKVILLE, Md., May 3, 2023 (PRNewswire) -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ended March 31, 2023, and recent operational highlights.

 

“REGENXBIO is well-positioned for a transformative year, with multiple clinical milestones and updates anticipated in 2023. Our goal in partnering our core expertise in gene therapy with AbbVie’s leadership in eye care and their global infrastructure was to expand and advance the reach of our retinal programs for patients in need of a potential one-time gene therapy, and we are pleased to progress this program with a vision of benefiting patients worldwide. Based on the encouraging safety and clinical profiles, we believe ABBV-RGX-314 has the potential to be a highly differentiated product for millions of patients.” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We also continue to make tremendous strides in our rare disease pipeline, advancing clinical trials in Duchenne, Hunter and Hurler syndromes and Batten disease, and we anticipate more updates across each of these programs in 2023. Overall, with an active pipeline of clinical-stage products, we are making excellent progress advancing our ‘5x’25’ strategy to bring leading, ground-breaking AAV Therapeutics to patients in need.”

 

AbbVie Eye Care Collaboration Update

 

REGENXBIO today announced updates from its eye care collaboration with AbbVie to develop and commercialize a potential one-time gene therapy for the treatment of chronic retinal diseases. The updates include the transfer of the Investigational New Drug (IND) applications to AbbVie for all ongoing clinical trials and the expansion of the ATMOSPHERE® and ASCENT™ pivotal trials for the treatment of patients with wet age-related macular degeneration (wet AMD) using subretinal delivery to support new global registration plans. As part of the IND transfers, the investigational gene therapy RGX-314 has been renamed ABBV-RGX-314.

 

As outlined in the original collaboration agreement, beginning in 2023, AbbVie has been responsible for the majority of all development expenses for ABBV-RGX-314. The transfer of the INDs enables AbbVie to serve as lead regulatory party in the U.S. and globally. AbbVie will lead the clinical development and

 

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commercialization of ABBV-RGX-314 globally. AbbVie and REGENXBIO will continue to collaborate on additional trials of ABBV-RGX-314.

 

ABBV-RGX-314 is currently being evaluated in nine ongoing clinical trials in the U.S. and Canada, 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 diabetic retinopathy (DR), and two corresponding Long-term Follow-up studies, all utilizing in-office suprachoroidal delivery.

 

The pivotal program utilizing subretinal delivery is now expected to add new sites in Europe, Japan, and Israel to advance global development plans. Additional clinical trial applications will be submitted to the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency in Japan. The ATMOSPHERE and ASCENT trials are expected to expand enrollment to approximately 540 and 660 patients, respectively. As prospectively allowed in the pivotal trial protocols, the enrollment expansion is also expected to support the increase in power of primary and secondary endpoints to enable additional global regulatory submissions and labelling options. The new global site plans and expanded enrollment targets are expected to support regulatory submissions with the U.S. Food and Drug Agency (FDA) and the EMA in late 2025 through the first half of 2026.

 

Also as previously announced, under the collaboration, REGENXBIO will continue to lead the manufacturing of ABBV-RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of ABBV-RGX-314 for commercial supply outside the U.S. Additionally, cGMP material produced by REGENXBIO’s proprietary NAVXpress™ platform process has been incorporated into the ongoing clinical trials, including the ATMOSPHERE and ASCENT pivotal trials. REGENXBIO shall participate in U.S. commercialization efforts as provided under a mutually agreed upon commercialization plan. REGENXBIO and AbbVie will share equally in profits from net sales of ABBV-RGX-314 in the U.S. and AbbVie will pay REGENXBIO tiered royalties on net sales outside the U.S.

 

Program Highlights and Milestones

 

ABBV-RGX-314: ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for the treatment of wet AMD, DR and other additional chronic retinal conditions. ABBV-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.

 

 

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.

 

 

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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.

 

 

Operational Updates

 

NAV Technology Licensee Program Highlights

As of March 31, 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.

 

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Financial Results

 

Cash Position: Cash, cash equivalents and marketable securities were $473.5 million as of March 31, 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 first quarter of 2023.

 

Revenues: Revenues were $19.1 million for the three months ended March 31, 2023, compared to $22.2 million for the three months ended March 31, 2022. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $21.5 million in the first quarter of 2022 to $16.1 million in the first quarter of 2023.

 

Research and Development Expenses: Research and development expenses were $58.5 million for the three months ended March 31, 2023, compared to $55.6 million for the three months ended March 31, 2022. The increase was primarily attributable to personnel costs as a result of increased headcount and laboratory and facilities costs, driven primarily by the activation of the REGENXBIO Manufacturing Innovation Center in mid-2022.

 

General and Administrative Expenses: General and administrative expenses were $22.6 million for the three months ended March 31, 2023, compared to $22.3 million for the three months ended March 31, 2022.

 

Net Loss: Net loss was $66.7 million, or $1.53 basic and diluted net loss per share, for the three months ended March 31, 2023, compared to a net loss of $76.7 million, or $1.79 basic and diluted net loss per share, for the three months ended March 31, 2022.

 

Financial Guidance

 

REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $473.5 million as of March 31, 2023, 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 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. 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.

 

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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 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, 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)

 

		March 31, 2023				December 31, 2022
Assets
Current assets
Cash and cash equivalents		$	70,091			$	96,952
Marketable securities			261,726				267,690
Accounts receivable			18,861				28,082
Prepaid expenses			15,521				13,900
Other current assets			23,003				9,352
Total current assets			389,202				415,976
Marketable securities			141,709				200,560
Accounts receivable, net			1,300				1,504
Property and equipment, net			141,573				141,685
Operating lease right-of-use assets			63,726				65,116
Restricted cash			2,030				2,030
Other assets			8,290				6,397
Total assets		$	747,830			$	833,268
Liabilities and Stockholders’ Equity
Current liabilities
Accounts payable		$	23,283			$	27,213
Accrued expenses and other current liabilities			31,709				46,794
Deferred revenue			1,311				1,829
Operating lease liabilities			6,303				5,997
Liability related to sale of future royalties			49,728				48,601
Total current liabilities			112,334				130,434
Operating lease liabilities			86,992				88,802
Liability related to sale of future royalties			77,382				89,005
Other liabilities			5,983				8,832
Total liabilities			282,691				317,073
Stockholders’ equity
Preferred stock; no shares issued and outstanding    at March 31, 2023 and December 31, 2022			—				—
Common stock; 43,465 and 43,299 shares issued    and outstanding at March 31, 2023 and    December 31, 2022, respectively			4				4
Additional paid-in capital			984,986				973,145
Accumulated other comprehensive loss			(11,622	)			(15,401	)
Accumulated deficit			(508,229	)			(441,553	)
Total stockholders’ equity			465,139				516,195
Total liabilities and stockholders’ equity		$	747,830			$	833,268

 

 

 

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REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)

 

		Three Months Ended March 31,
		2023				2022
Revenues
License and royalty revenue		$	19,138			$	22,218
Total revenues			19,138				22,218
Operating Expenses
Cost of revenues			4,112				15,717
Research and development			58,516				55,627
General and administrative			22,634				22,318
Other operating expenses			33				83
Total operating expenses			85,295				93,745
Loss from operations			(66,157	)			(71,527	)
Other Income (Expense)
Interest income from licensing			70				94
Investment income			2,166				799
Interest expense			(2,755	)			(6,130	)
Total other income (expense)			(519	)			(5,237	)
Loss before income taxes			(66,676	)			(76,764	)
Income Tax Benefit			—				41
Net loss		$	(66,676	)		$	(76,723	)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net			3,779				(9,381	)
Total other comprehensive income (loss)			3,779				(9,381	)
Comprehensive loss		$	(62,897	)		$	(86,104	)
Net loss per share, basic and diluted		$	(1.53	)		$	(1.79	)
Weighted-average common shares outstanding, basic and diluted			43,451				42,944

 

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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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