REGENXBIO Reports Third Quarter 2022 Financial Results and Recent Operational Highlights

ROCKVILLE, Md., Nov. 3, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the third quarter ended September 30, 2022, and recent operational highlights.

"REGENXBIO has seen continued progress since our last earnings update, with encouraging data updates across four of our key gene therapy programs that make up our '5x'25' strategy," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We shared multiple updates for our lead program, RGX-314, that is being developed in collaboration with AbbVie, including new long-term follow-up data for RGX-314 subretinal delivery that supports our anticipated 2024 BLA filing. We also shared new Phase II data for RGX-314 suprachoroidal delivery in both wet AMD and diabetic retinopathy. We are expanding these trials in both indications as we continue developing this novel approach to delivering gene therapy with the potential to expand patient access to the in-office setting. Within our rare disease pipeline, we also announced new data for RGX-121, our gene therapy for the treatment of MPS II, and we continue enrolling in the pivotal CAMPSIITE trial that we expect to support a planned BLA filing in 2024 using the accelerated approval pathway. We remain well funded to execute upon our goals and look forward to keeping you updated on the advancement of our programs as we finish the year and look ahead to 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).

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.

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.

Operational Updates

NAV Technology Licensee Program Highlights

As of September 30, 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 $617.0 million as of September 30, 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 nine months ended September 30, 2022.

Revenues: Revenues were $26.5 million for the three months ended September 30, 2022, compared to $30.8 million for the three months ended September 30, 2021. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $30.3 million for the third quarter of 2021 to $25.2 million for the third quarter of 2022. Zolgensma royalty revenues for the nine months ended September 30, 2022 were $75.1 million for the nine months ended September 30, 2022, compared to $66.9 million for the nine months ended September 30, 2021.

Research and Development Expenses: Research and development expenses were $63.3 million for the three months ended September 30, 2022, compared to $47.9 million for the three months ended September 30, 2021. The increase was 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 $20.9 million for the three months ended September 30, 2022, compared to $21.0 million for the three months ended September 30, 2021.

Net Loss: Net loss was $75.5 million, or $1.75 basic and diluted net loss per share, for the three months ended September 30, 2022, compared to a net loss of $58.4 million, or $1.37 basic and diluted net loss per share, for the three months ended September 30, 2021.

Financial Guidance

Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $617.0 million as of September 30, 2022 to fund its operations into 2025.

Conference Call

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 8:30 a.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, 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, 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, 2021, 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.

REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)

September 30, 2022

December 31, 2021

Assets

Current assets

Cash and cash equivalents

$

118,544

$

345,209

Marketable securities

263,874

112,230

Accounts receivable, net

32,549

32,439

Prepaid expenses

14,824

18,752

Other current assets

6,848

10,196

Total current assets

436,639

518,826

Marketable securities

234,594

391,907

Accounts receivable, net

1,803

2,262

Property and equipment, net

140,906

131,547

Operating lease right-of-use assets

59,471

60,904

Restricted cash

2,030

2,030

Other assets

8,350

6,428

Total assets

$

883,793

$

1,113,904

Liabilities and Stockholders' Equity

Current liabilities

Accounts payable

$

25,210

$

11,387

Accrued expenses and other current liabilities

44,462

76,111

Deferred revenue

5,903

3,333

Operating lease liabilities

3,608

1,752

Liability related to sale of future royalties

44,365

37,889

Total current liabilities

123,548

130,472

Operating lease liabilities

84,673

84,929

Liability related to sale of future royalties

103,084

133,460

Other liabilities

8,664

745

Total liabilities

319,969

349,606

Stockholders' equity

 Preferred stock; no shares issued and outstanding
at September 30, 2022 and December 31, 2021

—

—

 Common stock; 43,292 and 42,831 shares issued
and outstanding at September 30, 2022 and
December 31, 2021, respectively

4

4

Additional paid-in capital

963,694

928,095

Accumulated other comprehensive loss

(18,256)

(2,569)

Accumulated deficit

(381,618)

(161,232)

Total stockholders' equity

563,824

764,298

Total liabilities and stockholders' equity

$

883,793

$

1,113,904

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,

2022

2021

2022

2021

Revenues

License and royalty revenue

$

26,512

$

30,773

$

81,379

$

71,692

Total revenues

26,512

30,773

81,379

71,692

Operating Expenses

Cost of revenues

13,094

14,105

41,762

28,775

Research and development

63,313

47,855

179,948

133,459

General and administrative

20,921

21,030

64,071

57,293

Credit losses and other

229

5,131

703

5,781

Total operating expenses

97,557

88,121

286,484

225,308

Loss from operations

(71,045)

(57,348)

(205,105)

(153,616)

Other Income (Expense)

Interest income from licensing

18

117

265

700

Investment income

1,497

5,535

3,357

6,514

Interest expense

(5,954)

(6,709)

(18,944)

(19,777)

Total other income (expense)

(4,439)

(1,057)

(15,322)

(12,563)

Loss before income taxes

(75,484)

(58,405)

(220,427)

(166,179)

Income Tax Benefit (Expense)

—

—

41

(4)

Net loss

$

(75,484)

$

(58,405)

$

(220,386)

$

(166,183)

Other Comprehensive Loss

Unrealized loss on available-for-sale securities, net

(3,493)

(30)

(15,687)

(925)

Total other comprehensive loss

(3,493)

(30)

(15,687)

(925)

Comprehensive loss

$

(78,977)

$

(58,435)

$

(236,073)

$

(167,108)

Net loss per share, basic and diluted

$

(1.75)

$

(1.37)

$

(5.11)

$

(3.93)

Weighted-average common shares outstanding, basic and diluted

43,251

42,629

43,103

42,324

Contacts:

Dana Cormack
Corporate Communications
dcormack@regenxbio.com

Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.brinzey@westwicke.com

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SOURCE REGENXBIO Inc.