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.

 

 


The following information was filed by Regenxbio Inc. (RGNX) on Tuesday, March 6, 2018 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-K Annual Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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