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 7, 2019

 

REGENXBIO Inc.

(Exact Name of Registrant as Specified in its Charter)

 

 

 

(240) 552-8181

(Registrant’s telephone number, including area code)

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 (see General Instruction A.2. below):

Securities registered pursuant to Section 12(b) of the Act:

 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 under the Securities Act of 1933 (17 CFR 230.405) or Rule 12b-2 under the Securities Exchange Act of 1934 (17 CFR 240.12b-2).

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.  ☐

 

---

On August 7, 2019, REGENXBIO Inc. (the “Company”) issued a press release regarding its results of operations and financial condition for the quarter ended June 30, 2019. 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.

(d)Exhibits

 

---

SIGNATURE

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.

 

 

 

EXHIBIT 99.1

 

 

                                                        

 

 

FOR IMMEDIATE RELEASE

 

REGENXBIO Reports Second Quarter 2019 Financial and Operating Results and Additional Positive Interim Phase I/IIa Trial Update for RGX-314 for the Treatment of Wet AMD

 

 

ROCKVILLE, Md., August 7, 2019 (PRNewswire) -- 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 quarter ended June 30, 2019, and recent operational highlights.

 

“We are encouraged by the continued positive interim data from the Phase I/IIa RGX-314 trial and the potential of NAV gene therapy as a one-time treatment for wet AMD, and look forward to sharing six-month results from all five cohorts in the trial in October 2019,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “In our clinical trial for wet AMD, we have seen dose-dependent increases in protein expression levels across cohorts, and 50% of Cohort 3 subjects continued to be free of anti-VEGF injections at 18 months following a single administration of our gene therapy. These findings are particularly encouraging, given the severity of the disease and the high anti-VEGF injection treatment burden of these enrolled patients prior to administration of RGX-314. With feedback from the FDA provided at our recent Type B meeting, we also look forward to our anticipated start of the Phase IIb trial in late 2019.”

 

Mr. Mills added: “In addition to our excitement around RGX-314, the past quarter was highlighted by important milestones for REGENXBIO, including the FDA approval and launch of Zolgensma^®—the first FDA-approved gene therapy based on REGENXBIO’s NAV Technology Platform. We recently announced the expansion of our pipeline using NAV Vectors to deliver therapeutic antibodies, beginning with novel treatments for hereditary angioedema and chronic neurodegenerative diseases; this approach has enormous potential for patients who lack treatments or who are currently underserved by existing therapies. We currently have an internal pipeline of novel gene therapy candidates across large, chronic diseases and rare, monogenic diseases, which positions us to potentially develop meaningful treatments for a broad spectrum of patients with significant unmet needs.”

 

 

 

---

 

Lead Product Candidate Updates

 

Gene Therapy using NAV Vectors for AAV-Mediated Antibody Delivery:

 

 

 

 

 

 

 

 

 

 

Gene Therapy using NAV Vectors for Rare Genetic Diseases:

 

 

 

 

---

 

 

 

 

Other Recent Operational Highlights

 

 

NAV Technology Licensee Program Highlights

 

As of July 31, 2019, REGENXBIO's NAV Technology Platform was being applied in one marketed product, Novartis AG’s Zolgensma, and more than 20 partnered product candidates in development by NAV Technology Licensees. Fifteen of these partnered product candidates are in active clinical development. Over 200 subjects have been treated in clinical trials sponsored by NAV Technology Licensees. REGENXBIO’s NAV Technology Licensees are advancing product candidates in a broad range of therapeutic areas and disease indications. Recent updates from NAV Technology Licensees include:

 

Marketed Products

 

 

Partnered Product Candidates

 

 

 

 

---

 

 

Financial Results

 

Cash, cash equivalents and marketable securities were $449.7 million as of June 30, 2019, compared to $470.6 million as of December 31, 2018. Cash, cash equivalents and marketable securities as of June 30, 2019 include $32.1 million of marketable equity securities of Prevail Therapeutics Inc. REGENXBIO is a co-founder of Prevail and currently owns 2.43 million shares of Prevail common stock. Prior to Prevail’s initial public offering (IPO) in June 2019, REGENXBIO’s equity interests in Prevail had a carrying value of $0.4 million and were included in other assets on the consolidated balance sheet. Upon the completion of Prevail’s IPO in June 2019, the securities were reclassified to marketable securities, and subsequently remeasured at fair value. The decrease in cash, cash equivalents and marketable securities during the six months ended June 30, 2019, was primarily attributable to $53.0 million of net cash used in operating activities during the period, partially offset by the unrealized gain of $31.7 million related to the marketable equity securities of Prevail.

 

Revenues were $7.9 million for the three months ended June 30, 2019, compared to $40.0 million for the three months ended June 30, 2018. The decrease was primarily attributable to $40.0 million of non-recurring license revenue recognized during the three months ended June 30, 2018 under our amended license agreement with AveXis Inc. for the development and commercialization of treatments for SMA. The decrease was partially offset by revenue recognized during the three months ended June 30, 2019 resulting from license options exercised and development milestones achieved by licensees during the period, as well as royalties on net sales of Zolgensma. Commercial sales of Zolgensma commenced in the second quarter of 2019, and we expect royalties to increase in future periods. We are also eligible to receive a milestone payment of $80.0 million from AveXis upon the achievement of $1 billion in cumulative net sales of Zolgensma.

 

Research and development expenses were $29.5 million for the three months ended June 30, 2019, compared to $21.5 million for the three months ended June 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount, laboratory and facilities costs and external expenses associated with conducting clinical trials and manufacturing-related services.

 

General and administrative expenses were $13.4 million for the three months ended June 30, 2019, compared to $8.3 million for the three months ended June 30, 2018. The increase was primarily attributable to personnel costs as a result of increased headcount and professional fees for advisory and other services.

 

Net loss was $1.5 million, or $0.04 basic and diluted net loss per share, for the three months ended June 30, 2019, compared to net income of $10.6 million, or $0.33 basic and $0.30 diluted net income per share, for the three months ended June 30, 2018. Net loss for the three months ended June 30, 2019, includes the unrealized gain of $31.7 million on REGENXBIO’s marketable equity securities of Prevail recognized during the period.

 

Financial Guidance

 

Based on its current operating plan, REGENXBIO expects its balance in cash, cash equivalents and marketable securities to be at least $350 million as of December 31, 2019.

 

Conference Call

 

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. To access the live call by phone, dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 6839996. To access a live or recorded webcast of the call, please visit the Investors section of the REGENXBIO website at www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call.

 

---

 

 

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, research and development activities, preclinical studies, 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, 2018, 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. 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)

 

 

 

 

 

 

---

 

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

(unaudited)

(in thousands, except per share data)

 

 

 

Zolgensma^® is a registered trademark of AveXis.  All other trademarks referenced herein are registered trademarks of REGENXBIO.  

 

###

 

Contacts:

Investors:

Heather Savelle, 212-600-1902

heather@argotpartners.com

 

Media:

David Rosen, 212-600-1902

david.rosen@argotpartners.com