0001590877false00015908772023-08-022023-08-02

 

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): August 02, 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. ☐

 

 

---

Item 2.02. Results of Operations and Financial Condition.

On August 2, 2023, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter ended June 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 August 2, 2023 relating to REGENXBIO Inc.’s financial results.
104		The cover page from this Current Report on Form 8-K, formatted in Inline XBRL.

 

 

---

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:	August 2, 2023	By:	/s/ Patrick J. Christmas II
			Patrick J. Christmas II Executive Vice President, Chief Legal Officer

 

---

EXHIBIT 99.1

 

 

 

REGENXBIO Reports Second Quarter 2023 Financial Results and Recent Operational Highlights

 

 

ROCKVILLE, Md., August 2, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the second quarter ended June 30, 2023, and recent operational highlights.

 

“We continue to stay on track advancing our ‘5x’25’ vision to have five gene therapies either on the market or in late-stage development by 2025,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We are executing against a transformational year for REGENXBIO, with multiple clinical milestones and updates anticipated in 2023. The encouraging safety and tolerability data for the high-dose cohorts of our in-office wet AMD and DR programs further validate the potential of the suprachoroidal space for AAV delivery to treat chronic retinal diseases. We successfully reached our goal of enrolling patients in CAMPSIITE clinical trial of RGX-121 for treatment of MPS II and reported initial safety results from the first cohort of the AFFINITY DUCHENNE study and expect to present initial efficacy data later this year. Overall, we’re making excellent progress advancing our ‘5x’25’ strategy to bring leading, ground-breaking AAV Therapeutics to patients in need.”

 

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 age-related macular degeneration (wet AMD), diabetic retinopathy (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).

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 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 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 enzyme.

 

---

 

RGX-111: RGX-111 is an investigational one-time AAV therapeutic for the treatment of severe Mucopolysaccharidosis Type I (MPS I), also known as Hurler syndrome, using the NAV AAV9 vector to deliver the α-l-iduronidase 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 tripeptidyl peptidase 1 (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.

RESEARCH & OPERATIONAL UPDATES

 

New Exon-Skipping Therapy for Duchenne Muscular Dystrophy

 

The RMIC in Maryland is fully operational, producing cGMP bulk substance lots to support programs using the NAVXpress platform process. REGENXBIO is one of only a few gene therapy companies worldwide with a cGMP facility capable of production at scales up to 2,000 liters. The company continues to use the facility to produce commercial-scale cGMP material for its entire clinical pipeline and performance qualification lots to support planned regulatory filings for ABBV-RGX-314 and RGX-121.

NAV TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS

 

As of June 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 $415.4 million as of June 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 first half of 2023.

Revenues: Revenues were $20.0 million for the three months ended June 30, 2023, compared to $32.6 million for the three months ended June 30, 2022. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $28.4 million in the second quarter of 2022 to $19.0 million in the second quarter of 2023.

Research and Development Expenses: Research and development expenses were $59.9 million for the three months ended June 30, 2023, compared to $61.0 million for the three months ended June 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 $23.7 million for the three months ended June 30, 2023, compared to $20.8 million for the three months ended June 30, 2022. The increase was primarily attributable to expenses for professional services and other corporate overhead costs.

Net Loss: Net loss was $72.1 million, or $1.66 basic and diluted net loss per share, for the three months ended June 30, 2023, compared to a net loss of $68.2 million, or $1.58 basic and diluted net loss per share, for the three months ended June 30, 2022.

 

FINANCIAL GUIDANCE

 

REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $415.4 million as of June 30, 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. A live audio webcast will be available at REGENXBIO.COM/INVESTORS. 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, 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, 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.

 

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

 

 

 

---

 

REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)

 

		June 30, 2023				December 31, 2022
Assets
Current assets
Cash and cash equivalents		$	68,600			$	96,952
Marketable securities			251,482				267,690
Accounts receivable			21,380				28,082
Prepaid expenses			15,112				13,900
Other current assets			22,235				9,352
Total current assets			378,809				415,976
Marketable securities			95,302				200,560
Accounts receivable, net			1,143				1,504
Property and equipment, net			138,680				141,685
Operating lease right-of-use assets			62,436				65,116
Restricted cash			2,255				2,030
Other assets			3,833				6,397
Total assets		$	682,458			$	833,268
Liabilities and Stockholders’ Equity
Current liabilities
Accounts payable		$	11,639			$	27,213
Accrued expenses and other current liabilities			50,517				46,794
Deferred revenue			448				1,829
Operating lease liabilities			6,326				5,997
Liability related to sale of future royalties			48,963				48,601
Total current liabilities			117,893				130,434
Operating lease liabilities			85,254				88,802
Liability related to sale of future royalties			67,377				89,005
Other liabilities			6,079				8,832
Total liabilities			276,603				317,073
Stockholders’ equity
Preferred stock; no shares issued and outstanding    at June 30, 2023 and December 31, 2022			—				—
Common stock; 43,621 and 43,299 shares issued    and outstanding at June 30, 2023 and    December 31, 2022, respectively			4				4
Additional paid-in capital			996,239				973,145
Accumulated other comprehensive loss			(10,098	)			(15,401	)
Accumulated deficit			(580,290	)			(441,553	)
Total stockholders’ equity			405,855				516,195
Total liabilities and stockholders’ equity		$	682,458			$	833,268

 

 

---

 

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)

 

	Three Months								Six Months
	Ended June 30,								Ended June 30,
	2023				2022				2023				2022
Revenues
License and royalty revenue	$	19,977			$	32,649			$	39,115			$	54,867
Total revenues		19,977				32,649				39,115				54,867
Operating Expenses
Cost of revenues		9,475				12,951				13,587				28,668
Research and development		59,886				61,008				118,402				116,635
General and administrative		23,698				20,832				46,332				43,150
Other operating expenses		26				391				59				474
Total operating expenses		93,085				95,182				178,380				188,927
Loss from operations		(73,108	)			(62,533	)			(139,265	)			(134,060	)
Other Income (Expense)
Interest income from licensing		40				153				110				247
Investment income		2,127				1,061				4,293				1,860
Interest expense		(1,120	)			(6,860	)			(3,875	)			(12,990	)
Total other income (expense)		1,047				(5,646	)			528				(10,883	)
Loss before income taxes		(72,061	)			(68,179	)			(138,737	)			(144,943	)
Income Tax Benefit		—				—				—				41
Net loss	$	(72,061	)		$	(68,179	)		$	(138,737	)		$	(144,902	)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net		1,524				(2,813	)			5,303				(12,194	)
Total other comprehensive income (loss)		1,524				(2,813	)			5,303				(12,194	)
Comprehensive loss	$	(70,537	)		$	(70,992	)		$	(133,434	)		$	(157,096	)
Net loss per share, basic and diluted	$	(1.66	)		$	(1.58	)		$	(3.19	)		$	(3.37	)
Weighted-average common shares outstanding, basic and diluted		43,531				43,111				43,491				43,028

 

###

 

CONTACTS:

 

Chris Brinzey, ICR Westwicke

339-970-2843

Chris.Brinzey@westwicke.com

 

 

 

---