SEC Filing - Regenxbio Inc

View:

Download DOC

Download PDF

Download XLS

Download XBRL

10-Q

Table of Contents

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q

(Mark One)

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2023

☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from ___ to ___

Commission File Number 001-37553

REGENXBIO Inc.

(Exact Name of Registrant as Specified in its Charter)


Delaware		47-1851754
(State or other jurisdiction of incorporation or organization)		(I.R.S. Employer Identification No.)

9804 Medical Center Drive Rockville, MD		20850
(Address of principal executive offices)		(Zip Code)

(240) 552-8181

(Registrant’s telephone number, including area code)

Not Applicable

(Former name, former address and former fiscal year, if changed since last report)

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 (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.


Large accelerated filer	☒	Accelerated filer	☐
Non-accelerated filer	☐	Smaller reporting company	☐
		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. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

As of November 2, 2023, there were 43,991,980 shares of the registrant’s common stock, par value $0.0001 per share, issued and outstanding.

Table of Contents

REGENXBIO INC.

QUARTERLY REPORT ON FORM 10-Q

FOR THE QUARTERLY PERIOD ENDED SEPTEMBER 30, 2023

TABLE OF CONTENTS


	PART I—FINANCIAL INFORMATION

Item 1.	Financial Statements (Unaudited)	3
	Consolidated Balance Sheets as of September 30, 2023 and December 31, 2022	3
	Consolidated Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended September 30, 2023 and 2022	4
	Consolidated Statements of Stockholders’ Equity for the Three and Nine Months Ended September 30, 2023 and 2022	5
	Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2023 and 2022	7
	Notes to Consolidated Financial Statements	8
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	23
Item 3.	Quantitative and Qualitative Disclosures about Market Risk	33
Item 4.	Controls and Procedures	33

	PART II—OTHER INFORMATION

Item 1.	Legal Proceedings	35
Item 1A.	Risk Factors	35
Item 2.	Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities	35
Item 3.	Defaults Upon Senior Securities	35
Item 4.	Mine Safety Disclosures	35
Item 5.	Other Information	36
Item 6.	Exhibits	37
Signatures		38

Table of Contents

INFORMATION REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act). These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as “anticipate,” “assume,” “believe,” “continue,” “could,” “design,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “objective,” “plan,” “position,” “potential,” “predict,” “project,” “seek,” “should,” or “will,” “would” or by variations of such words or by similar expressions. We have based these forward-looking statements on our current expectations, estimates and assumptions and analyses in light of our experience and our perception of historical trends, current conditions and expected future developments, as well as other factors we believe are appropriate under the circumstances. However, whether actual results and developments will conform with our expectations and predictions is subject to a number of risks, uncertainties, assumptions and other important factors, including, but not limited to:

•

our ability to establish and maintain development partnerships, including our collaboration with AbbVie to develop and commercialize ABBV-RGX-314;

•

our ability to obtain and maintain regulatory approval of our product candidates and the labeling for any approved products;

•

the timing of enrollment, commencement and completion and the success of our clinical trials, including the timing and commencement of our AFFINITY DUCHENNE® clinical trial;

•

the timing of commencement and completion and the success of preclinical studies conducted by us and our development partners;

•

the timely development and launch of new products;

•

the scope, progress, expansion and costs of developing and commercializing our product candidates;

•

our ability to obtain, maintain and enforce intellectual property protection for our product candidates and technology, and defend against third-party intellectual property-related claims;

•

our expectations regarding the development and commercialization of product candidates currently being developed by third parties that utilize our technology;

•

our anticipated growth strategies;

•

our expectations regarding competition;

•

the anticipated trends and challenges in our business and the market in which we operate;

•

our ability to attract or retain key personnel;

•

the size and growth of the potential markets for our product candidates and the ability to serve those markets;

•

the rate and degree of market acceptance of any of our products that are approved;

•

our expectations regarding our expenses and revenue;

•

our strategic pipeline prioritization and corporate restructuring, including plans for advancing our product candidates, the expected charges and cost savings associated with our restructuring and any future cost reduction measures;

•

our expectations regarding our need for additional financing and our ability to obtain additional financing;

•

our expectations regarding the outcome of legal proceedings;

•

our expectations regarding regulatory developments in the United States and foreign countries; and

•

changes in the financial markets and banking system that may affect the availability and terms on which we may obtain financing and our ability to accurately predict how long our existing cash resources will be sufficient to fund our anticipated operating expenses.

Table of Contents

You should carefully read the factors discussed in the sections titled “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” and the factors discussed elsewhere in this Quarterly Report on Form 10-Q, our Annual Report on Form 10-K for the year ended December 31, 2022 and in our other filings with the U.S. Securities and Exchange Commission (the SEC) for additional discussion of the risks, uncertainties, assumptions and other important factors that could cause our actual results or developments to differ materially and adversely from those projected in the forward-looking statements. 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 us or our businesses or operations. Such statements are not guarantees of future performance, and actual results or developments may differ materially and adversely from those projected in the forward-looking statements. These forward-looking statements speak only as of the date of this report. Except as required by law, we disclaim any duty to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Available Information

Our principal offices are located at 9804 Medical Center Drive, Rockville, MD 20850, and our telephone number is (240) 552-8181. Our website address is www.regenxbio.com. The information contained in, or that can be accessed through, our website is not a part of, or incorporated by reference in, this Quarterly Report on Form 10-Q. We file annual, quarterly, and current reports, proxy statements, and other documents with the SEC under the Exchange Act. You may obtain any reports, proxy and information statements, and other information that we file electronically with the SEC at www.sec.gov.

You also may view and download copies of our SEC filings free of charge at our website as soon as reasonably practicable after we electronically file such material with, or furnish it to, the SEC. The information contained on, or that can be accessed through, our website will not be deemed to be incorporated by reference in, and is not considered part of, this Quarterly Report on Form 10-Q. Investors should also note that we use our website, as well as SEC filings, press releases, public conference calls and webcasts, to announce financial information and other material developments regarding our business. We use these channels, as well as any social media channels listed on our website, to communicate with investors and members of the public about our business. It is possible that the information that we post on our social media channels could be deemed material information. Therefore, we encourage investors, the media and others interested in our company to review the information that we post on our social media channels.

As used in this Quarterly Report on Form 10-Q, the terms “REGENXBIO,” “we,” “us,” “our” or the “Company” mean REGENXBIO Inc. and its subsidiaries, on a consolidated basis, unless the context indicates otherwise.

AAVIATE, AFFINITY DUCHENNE, ALTITUDE, ATMOSPHERE, CAMPSIITE, NAV, REGENXBIO and the REGENXBIO logos are our registered trademarks. Any other trademarks appearing in this Quarterly Report on Form 10-Q are the property of their respective holders.

Table of Contents

PART I – FINANCIAL INFORMATION

Item 1. Financial Statements.

REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands, except per share data)


	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; $0.0001 par value; 10,000 shares authorized, no shares issued and outstanding at September 30, 2023 and December 31, 2022		—			—
Common stock; $0.0001 par value; 100,000 shares authorized at September 30, 2023 and December 31, 2022; 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

The accompanying notes are an integral part of these unaudited consolidated financial statements.

Table of Contents

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)


	Three Months Ended September 30,						Nine Months 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

The accompanying notes are an integral part of these unaudited consolidated financial statements.

Table of Contents

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(unaudited)

(in thousands)


	Three Months Ended September 30, 2023
							Accumulated
					Additional		Other						Total
	Common Stock				Paid-in		Comprehensive			Accumulated			Stockholders’
	Shares		Amount		Capital		Loss			Deficit			Equity
Balances at June 30, 2023		43,621	$	4	$	996,239	$	(10,098	)	$	(580,290	)	$	405,855
Vesting of restricted stock units, net of tax		34		—		—		—			—			—
Exercise of stock options, net of tax		6		—		92		—			—			92
Issuance of common stock under employee stock purchase plan		73		—		1,243		—			—			1,243
Issuance of common stock upon private placement, net of transaction costs of $126		257		—		4,874		—			—			4,874
Stock-based compensation expense		—		—		10,219		—			—			10,219
Unrealized gain on available-for-sale securities, net		—		—		—		2,685			—			2,685
Net loss		—		—		—		—			(61,868	)		(61,868	)
Balances at September 30, 2023		43,991	$	4	$	1,012,667	$	(7,413	)	$	(642,158	)	$	363,100


	Three Months Ended September 30, 2022
							Accumulated
					Additional		Other						Total
	Common Stock				Paid-in		Comprehensive			Accumulated			Stockholders’
	Shares		Amount		Capital		Loss			Deficit			Equity
Balances at June 30, 2022		43,171	$	4	$	951,412	$	(14,763	)	$	(306,134	)	$	630,519
Vesting of restricted stock units, net of tax		2		—		—		—			—			—
Exercise of stock options, net of tax		66		—		917		—			—			917
Issuance of common stock under employee stock purchase plan		53		—		1,120		—			—			1,120
Stock-based compensation expense		—		—		10,245		—			—			10,245
Unrealized loss on available-for-sale securities, net		—		—		—		(3,493	)		—			(3,493	)
Net loss		—		—		—		—			(75,484	)		(75,484	)
Balances at September 30, 2022		43,292	$	4	$	963,694	$	(18,256	)	$	(381,618	)	$	563,824

The accompanying notes are an integral part of these unaudited consolidated financial statements.

Table of Contents

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(unaudited)

(in thousands)


	Nine Months Ended September 30, 2023
								Accumulated
					Additional			Other						Total
	Common Stock				Paid-in			Comprehensive			Accumulated			Stockholders’
	Shares		Amount		Capital			Loss			Deficit			Equity
Balances at December 31, 2022		43,299	$	4	$	973,145		$	(15,401	)	$	(441,553	)	$	516,195
Vesting of restricted stock units, net of tax		161		—		(419	)		—			—			(419	)
Exercise of stock options, net of tax		171		—		1,312			—			—			1,312
Issuance of common stock under employee stock purchase plan		103		—		1,826			—			—			1,826
Issuance of common stock upon private placement, net of transaction costs of $126		257		—		4,874			—			—			4,874
Stock-based compensation expense		—		—		31,929			—			—			31,929
Unrealized gain on available-for-sale securities, net		—		—		—			7,988			—			7,988
Net loss		—		—		—			—			(200,605	)		(200,605	)
Balances at September 30, 2023		43,991	$	4	$	1,012,667		$	(7,413	)	$	(642,158	)	$	363,100


	Nine Months Ended September 30, 2022
								Accumulated
					Additional			Other						Total
	Common Stock				Paid-in			Comprehensive			Accumulated			Stockholders’
	Shares		Amount		Capital			Loss			Deficit			Equity
Balances at December 31, 2021		42,831	$	4	$	928,095		$	(2,569	)	$	(161,232	)	$	764,298
Vesting of restricted stock units, net of tax		57		—		(284	)		—			—			(284	)
Exercise of stock options, net of tax		329		—		2,761			—			—			2,761
Issuance of common stock under employee stock purchase plan		75		—		1,742			—			—			1,742
Stock-based compensation expense		—		—		31,380			—			—			31,380
Unrealized loss on available-for-sale securities, net		—		—		—			(15,687	)		—			(15,687	)
Net loss		—		—		—			—			(220,386	)		(220,386	)
Balances at September 30, 2022		43,292