EXHIBIT 99.1

 

REGENXBIO Reports Fourth Quarter and Full-Year 2017 Financial Results and Recent Operational Highlights

 

 

Completed dosing of third cohort in RGX-314 Phase I clinical trial for wet AMD

 

Continuing dosing of second cohort in RGX-501 Phase I/II clinical trial for HoFH

 

Anticipate presenting topline data from RGX-314 and RGX-501 clinical trials in late 2018

 

Expect to initiate dosing in clinical trials for RGX-111 for MPS I and RGX-121 for MPS II in mid-2018

 

$176 million in cash, cash equivalents and marketable securities as of December 31, 2017

 

ROCKVILLE, Md., March 6, 2018 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced financial results for the fourth quarter and full year ended December 31, 2017 and recent operational highlights.

 

“In 2017, we significantly advanced our AAV gene therapy pipeline, which now consists of 12 active clinical stage programs, including four internal programs, as we seek to improve treatment options using our NAV Technology Platform,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We believe 2018 will be a transformative year for REGENXBIO, as we advance our mission of improving lives through the curative potential of gene therapy and expand our leading AAV gene therapy pipeline with our internal lead product candidates and our NAV Technology Licensees’ programs. We look forward to providing updates on our lead product candidates and our NAV Technology Licensees’ programs throughout the year, and are on track to report topline trial data for RGX-314 for wet AMD and RGX-501 for HoFH by year-end.”

 

Recent Operational Highlights

 

 

In February 2018, REGENXBIO announced the completion of dosing of the third cohort in the Phase I clinical trial for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). A total of 18 patients have been treated in the clinical trial to date. REGENXBIO expects to present topline data from the RGX-314 clinical trial in late 2018, which will include both primary and secondary endpoint data.

 

In February 2018, REGENXBIO dosed the second patient in the second cohort, and fifth patient overall, with a single administration of RGX-501 in the Phase I/II clinical trial for the treatment of homozygous familial hypercholesterolemia (HoFH). REGENXBIO expects to present topline data from the RGX-501 clinical trial in late 2018, which will include both primary and secondary endpoint data.

 

Site activation is continuing in the Phase I clinical trial evaluating RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I). Patient recruitment is anticipated to begin in the first quarter of 2018, with the first patient expected to be dosed in mid-2018.

 

Site activation is continuing in the Phase I/II clinical trial evaluating RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II). Patient recruitment is anticipated to begin in the first quarter of 2018, with the first patient expected to be dosed in mid-2018.

 

In January 2018, REGENXBIO and AveXis, Inc. announced an amendment to their license agreement for the development and commercialization of treatments for spinal muscular atrophy (SMA). Under the terms of the amended agreement, REGENXBIO could receive up to $260 million, including an upfront payment of $80 million, $60 million in additional guaranteed annual payments and potential commercial milestone payments of up to $120 million, and AveXis acquired exclusive rights to the entire NAV Technology Platform for the development of treatments for SMA. Additionally, the amended agreement permits assignment by AveXis upon a change of control without REGENXBIO’s consent.

 

In January 2018, REGENXBIO entered into an agreement with FUJIFILM Diosynth Biotechnologies which secures access to dedicated cGMP suite capacity and resources capable of manufacturing REGENXBIO’s lead product candidates at up to 2,000L scale in support of global development and commercialization.

 

 


 

As of December 31, 2017, REGENXBIO’s NAV Technology Platform was being applied in the development of more than 20 partnered product candidates by 10 NAV Technology Licensees. Eight of these partnered product candidates are in active clinical development. Three recent updates from NAV Technology Licensee active clinical programs include:

 

 

In January 2018, AveXis announced that it will initiate screening for remaining patients in the pivotal trial of AVXS-101 for SMA Type I following review of preliminary data from the first three patients. AVXS-101 uses the NAV AAV9 vector.

 

In January 2018, Audentes Therapeutics, Inc. announced positive interim data from the first dose cohort in the Phase I/II clinical trial evaluating AT132 for the treatment of X-linked myotubular myopathy. AT132 uses the NAV AAV8 vector.

 

In February 2018, Audentes Therapeutics announced dosing of the first patient in the Phase I/II clinical trial evaluating AT342 for the treatment of Crigler-Najjar Syndrome. AT342 uses the NAV AAV8 vector.

 

Financial Results

 

Cash, cash equivalents and marketable securities were $176.4 million as of December 31, 2017, compared to $159.0 million as of December 31, 2016. Cash, cash equivalents and marketable securities as of December 31, 2017 exclude the $80 million received from AveXis in connection with the previously announced amendment to the license agreement in January 2018 for the development and commercialization of treatments for SMA.

 

Revenues were $2.0 million and $10.4 million for the three months and year ended December 31, 2017, respectively, compared to $1.7 million and $4.6 million for the three months and year ended December 31, 2016, respectively.

 

Total operating expenses were $18.6 million and $86.3 million for the three months and year ended December 31, 2017, respectively, compared to $22.2 million and $69.9 million for the three months and year ended December 31, 2016, respectively.

 

Net loss was $16.0 million and $73.2 million, or $0.51 and $2.45 net loss per basic and diluted common share, for the three months and year ended December 31, 2017, respectively, compared to $19.6 million and $63.0 million, or $0.74 and $2.38 net loss per basic and diluted share, for the three months and year ended December 31, 2016, respectively.

 

Financial Guidance

 

REGENXBIO reiterates that it expects full-year 2018 cash burn to be between $85 million and $95 million, which will support the continued development of its lead product candidate programs. Full-year 2018 cash burn guidance excludes the effect of the upfront payment of $80 million and any other potential consideration that may be received from AveXis in connection with the previously announced amendment to the license agreement in January 2018 for the development and commercialization of treatments for SMA. Subject to this exclusion, full-year 2018 cash burn will be measured as the decrease in cash, cash equivalents and marketable securities from December 31, 2017 to December 31, 2018.

 

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 in multiple therapeutic areas.

 

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,” “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 of REGENXBIO’s clinical trials; the timing and success of preclinical studies and clinical trials 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, 2017, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2018, and comparable “risk factors” sections of REGENXBIO’s Quarterly Reports on Form 10-Q and other filings, which have been filed with the 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. 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, except per share data)

 

 

 

December 31, 2017

 

 

December 31, 2016

 

Assets

 

 

 

 

 

 

 

 

Current assets

 

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

46,656

 

 

$

24,840

 

Marketable securities

 

 

114,122

 

 

 

64,714

 

Accounts receivable

 

 

473

 

 

 

1,032

 

Prepaid expenses

 

 

5,334

 

 

 

1,775

 

Other current assets

 

 

1,412

 

 

 

1,010

 

Total current assets

 

 

167,997

 

 

 

93,371

 

Marketable securities

 

 

15,616

 

 

 

69,412

 

Property and equipment, net

 

 

13,977

 

 

 

9,324

 

Restricted cash

 

 

225

 

 

 

225

 

Other assets

 

 

862

 

 

 

400

 

Total assets

 

$

198,677

 

 

$

172,732

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

 

 

 

Current liabilities

 

 

 

 

 

 

 

 

Accounts payable

 

$

4,832

 

 

$

1,543

 

Accrued expenses and other current liabilities

 

 

9,605

 

 

 

8,126

 

Total current liabilities

 

 

14,437

 

 

 

9,669

 

Deferred rent, net of current portion

 

 

1,211

 

 

 

1,326

 

Total liabilities

 

 

15,648

 

 

 

10,995

 

Stockholders’ equity

 

 

 

 

 

 

 

 

Preferred stock; $0.0001 par value; 10,000 shares authorized,

   and no shares issued and outstanding at December 31, 2017

   and December 31, 2016

 

 

 

 

 

 

Common stock; $0.0001 par value; 100,000 shares authorized

   at December 31, 2017 and December 31, 2016; 31,295

   and 26,477 shares issued and outstanding at December 31, 2017

   and December 31, 2016, respectively

 

 

3

 

 

 

3

 

Additional paid-in capital

 

 

371,497

 

 

 

276,354

 

Accumulated other comprehensive loss

 

 

(715

)

 

 

(33

)

Accumulated deficit

 

 

(187,756

)

 

 

(114,587

)

Total stockholders’ equity

 

 

183,029

 

 

 

161,737

 

Total liabilities and stockholders’ equity

 

$

198,677

 

 

$

172,732

 


 


 

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)

 

 

 

Three Months Ended December 31,

 

 

Years Ended December 31,

 

 

 

2017

 

 

2016

 

 

2017

 

 

2016

 

Revenues

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

License revenue

 

$

2,040

 

 

$

1,665

 

 

$

10,385

 

 

$

4,303

 

Reagent sales

 

 

 

 

 

 

 

 

 

 

 

213

 

Grant revenue

 

 

 

 

 

31

 

 

 

8

 

 

 

73

 

Total revenues

 

 

2,040

 

 

 

1,696

 

 

 

10,393

 

 

 

4,589

 

Expenses

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Costs of revenues

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Licensing costs

 

 

(382

)

 

 

330

 

 

 

1,703

 

 

 

861

 

Costs of reagent sales

 

 

 

 

 

 

 

 

6

 

 

 

98

 

Research and development

 

 

14,170

 

 

 

16,059

 

 

 

57,224

 

 

 

45,482

 

General and administrative

 

 

4,808

 

 

 

5,742

 

 

 

27,229

 

 

 

23,590

 

Other operating expenses (income)

 

 

42

 

 

 

34

 

 

 

116

 

 

 

(102

)

Total operating expenses

 

 

18,638

 

 

 

22,165

 

 

 

86,278

 

 

 

69,929

 

Loss from operations

 

 

(16,598

)

 

 

(20,469

)

 

 

(75,885

)

 

 

(65,340

)

Other Income

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Investment income

 

 

601

 

 

 

426

 

 

 

2,716

 

 

 

1,938

 

Total other income

 

 

601

 

 

 

426

 

 

 

2,716

 

 

 

1,938

 

Loss before income taxes

 

 

(15,997

)

 

 

(20,043

)

 

 

(73,169

)

 

 

(63,402

)

Income Tax Benefit

 

 

 

 

 

435

 

 

 

 

 

 

435

 

Net loss

 

$

(15,997

)

 

$

(19,608

)

 

$

(73,169

)

 

$

(62,967

)

Other Comprehensive Income (Loss)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Unrealized gain (loss) on available-for-sale securities,

   net of reclassifications and income tax expense

 

 

(161

)

 

 

(886

)

 

 

(682

)

 

 

686

 

Total other comprehensive income (loss)

 

 

(161

)

 

 

(886

)

 

 

(682

)

 

 

686

 

Comprehensive loss

 

$

(16,158

)

 

$

(20,494

)

 

$

(73,851

)

 

$

(62,281

)

Basic and diluted net loss per common share

 

$

(0.51

)

 

$

(0.74

)

 

$

(2.45

)

 

$

(2.38

)

Weighted-average basic and diluted common shares

 

 

31,178

 

 

 

26,476

 

 

 

29,878

 

 

 

26,409

 

 

###


 


 

CONTACT:

 

Investors
Natalie Wildenradt, 646-681-8192
natalie@argotpartners.com

 

Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com

 

 

View differences made from one to another to evaluate Regenxbio Inc.'s financial trajectory

Compare SEC Filings Year-over-Year (YoY) and Quarter-over-Quarter (QoQ)
Sample 10-K Year-over-Year (YoY) Comparison

Compare this 8-K Corporate News to its predecessor by reading our highlights to see what text and tables were  removed  ,   added    and   changed   by Regenxbio Inc..

Continue

Never Miss A New SEC Filing Again


Real-Time SEC Filing Notifications
Screenshot taken from Gmail for a new 10-K Annual Report
Last10K.com Member Feature

Receive an e-mail as soon as a company files an Annual Report, Quarterly Report or has new 8-K corporate news.

Continue

We Highlighted This SEC Filing For You


SEC Filing Sentiment Analysis - Bullish, Bearish, Neutral
Screenshot taken from Wynn's 2018 10-K Annual Report
Last10K.com Member Feature

Read positive and negative remarks made by management in their entirety without having to find them in a 10-K/Q.

Continue

Widen Your SEC Filing Reading Experience


Increased Reading Area for SEC Filings
Screenshot taken from Adobe Inc.'s 10-Q Quarterly Report
Last10K.com Member Feature

Remove data columns and navigations in order to see much more filing content and tables in one view

Continue

Uncover Actionable Information Inside SEC Filings


SEC Filing Disclosures
Screenshot taken from Lumber Liquidators 10-K Annual Report
Last10K.com Member Feature

Read both hidden opportunities and early signs of potential problems without having to find them in a 10-K/Q

Continue

Adobe PDF, Microsoft Word and Excel Downloads


Download Annual and Quarterly Reports as PDF, Word and Excel Documents
Screenshots of actual 10-K and 10-Q SEC Filings in PDF, Word and Excel formats
Last10K.com Member Feature

Export Annual and Quarterly Reports to Adobe PDF, Microsoft Word and Excel for offline viewing, annotations and analysis

Continue

FREE Financial Statements


Download Annual and Quarterly Reports as PDF, Word and Excel Documents
Screenshot of actual balance sheet from company 10-K Annual Report
Last10K.com Member Feature

Get one-click access to balance sheets, income, operations and cash flow statements without having to find them in Annual and Quarterly Reports

Continue for FREE

Intrinsic Value Calculator


Intrinsic Value Calculator
Screenshot of intrinsic value for AT&T (2019)
Last10K.com Member Feature

Our Intrinsic Value calculator estimates what an entire company is worth using up to 10 years of financial ratios to determine if a stock is overvalued or not

Continue

Financial Stability Report


Financial Stability Report
Screenshot of financial stability report for Coco-Cola (2019)
Last10K.com Member Feature

Our Financial Stability reports uses up to 10 years of financial ratios to determine the health of a company's EPS, Dividends, Book Value, Return on Equity, Current Ratio and Debt-to-Equity

Continue

Get a Better Picture of a Company's Performance


Financial Ratios
Available Financial Ratios
Last10K.com Member Feature

See how over 70 Growth, Profitability and Financial Ratios perform over 10 Years

Continue

Log in with your credentials

or    

Forgot your details?

Create Account