EXHIBIT 99.1

 

REGENXBIO Reports Second Quarter 2017 Financial Results and Recent Operational Highlights

 

 

Enrollment continues in RGX-314 Phase I clinical trial for wet AMD; interim update anticipated by year-end 2017

 

Enrollment continues in RGX-501 Phase I/II clinical trial for HoFH; interim update anticipated by year-end 2017

 

IND active for third lead product candidate – RGX-111 for MPS I

 

$209 million in cash, cash equivalents and marketable securities as of June 30, 2017

 

ROCKVILLE, Md., August 8, 2017 (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 second quarter ended June 30, 2017 and recent operational highlights.

 

“We continue to progress our lead product candidates, most notably with the continued enrollment in our Phase I clinical trial evaluating RGX-314 for the treatment of wet AMD and in our Phase I/II clinical trial evaluating RGX-501 for the treatment of HoFH. The active IND for RGX-111 for the treatment of MPS I – our third clinical-stage lead product candidate – is another pipeline achievement that demonstrates our strong internal capabilities and commitment to the development of a robust clinical pipeline of gene therapy product candidates in many diseases,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We continue to advance our internal and partnered gene therapy product candidates, leveraging our proprietary NAV Technology Platform to demonstrate the curative potential of one-time administrations of gene therapy. We look forward to sharing more pipeline updates, including interim updates from the RGX-314 Phase I clinical trial for wet AMD and the RGX-501 Phase I/II clinical trial for HoFH, by year-end 2017.”

 

Recent Operational Highlights

 

 

REGENXBIO initiated dosing in the Phase I clinical trial evaluating RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Patient recruitment and enrollment is continuing and REGENXBIO expects to provide an interim update from the RGX-314 clinical trial by the end of 2017.

 

 

Patient recruitment and enrollment is continuing in the Phase I/II clinical trial evaluating RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH). REGENXBIO expects to provide an interim update from the RGX-501 clinical trial by the end of 2017.

 

 

REGENXBIO today announced that the Investigational New Drug (IND) application for the Phase I clinical trial of RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) is active. Site activation in this planned multi-center, open-label, multiple-cohort, dose-escalation trial is underway to support recruitment and patient enrollment, with the first patient expected to be dosed in the first half of 2018.

 

 

REGENXBIO plans to file an IND application for RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) in the second half of 2017, which will incorporate feedback received from the U.S. Food and Drug Administration (FDA) in its review of the IND application for RGX-111.

 

 

REGENXBIO has completed production of investigational product for all four of its lead product candidates in an amount which is expected to supply planned clinical trials through 2018 and has established a supply chain to ensure effective distribution to REGENXBIO’s planned clinical trial sites worldwide.

 

 

As of June 30, 2017, REGENXBIO’s NAV Technology Platform was being applied in the development of more than 20 partnered product candidates by nine NAV Technology Licensees. Eight of these partnered product candidates have advanced to clinical stages of development.

 

 

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In June 2017, AveXis, Inc. announced alignment with the FDA on its Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. AveXis announced that it plans to initiate the pivotal trial later in the third quarter of 2017. AVXS-101 uses the NAV AAV9 vector.

 

 

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Also in June 2017, REGENXBIO and AveXis entered into an exclusive worldwide license agreement for AveXis to develop and commercialize gene therapy treatments using the NAV AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and amyotrophic lateral sclerosis (ALS).


The following information was filed by Regenxbio Inc. (RGNX) on Tuesday, August 8, 2017 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-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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