Regenxbio Reports Additional Positive Interim Phase 1/2a Trial Update for RGX-314 for the Treatment of Wet AMD

Source: Regenxbio

As part of its quarterly results reporting, Regenxbio provided an update on its phase 1/2a  for RGX-314 for the treatment of wet AMD.

“We are encouraged by the continued positive interim data from the phase 1/2a RGX-314 trial and the potential of NAV gene therapy as a one-time treatment for wet AMD, and look forward to sharing 6-month results from all five cohorts in the trial in October 2019,” Kenneth T. Mills, President and Chief Executive Officer of Regenxbio, said in a company news release. “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 2b trial in late 2019.”

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

  • RGX-314 for the Treatment of Wet AMD
    • As of July 31, 2019, 42 subjects across five dose cohorts have been treated in the phase 1/2a trial of RGX-314. RGX-314 continues to be well-tolerated across all five dose cohorts, with no drug related serious adverse events (SAEs) reported.
    • Dose-dependent increases in RGX-314 protein expression levels, as measured from aqueous samples by electrochemiluminescence immunoassay (ECL) at approximately 1 month after administration of RGX-314, have been observed across all doses.
    • 50% of subjects (3/6) in Cohort 3 continue to remain injection-free at 18 months.
    • Regenxbio expects to present long-term clinical data from Cohort 3 out to 18 months and interim data from Cohort 4 and Cohort 5 in October 2019.
    • Following the Type B meeting with the U.S. Food and Drug Administration (FDA) held in July 2019, Regenxbio remains on track to initiate a Phase IIb trial for wet AMD in late 2019.
  • RGX-314 for the Treatment of Diabetic Retinopathy (DR)
    • Regenxbio is on track to file a new Investigational New Drug (IND) application for a phase 2 trial evaluating RGX-314 in subjects with DR in the second half of 2019.
  • Hereditary Angioedema (HAE)
    • Regenxbio continues work on the HAE program and expects to provide an update in early 2020 on the preclinical studies, as well as plans for entering clinical trials.
  • Neurodegenerative Disease
    • Regenxbio, in collaboration with Neurimmune AG, has initiated work to jointly develop novel therapies using AAV vectors to deliver human antibodies targeting abnormal tau and will provide program updates as the collaboration advances. 

Gene Therapy using NAV Vectors for Rare Genetic Diseases:

  • RGX-501 for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
    • Dosing resumed and recruitment continues in the Phase I/II clinical trial evaluating RGX-501 for the treatment of HoFH.
    • REGENXBIO expects to report interim data from Cohort 2 with corticosteroid prophylaxis from the Phase I/II clinical trial in the second half of 2019.
  • RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
    • Recruitment, screening, and dosing continue in the first of two dose cohorts in the Phase I/II clinical trial evaluating RGX-121 for the treatment of MPS II.
    • REGENXBIO expects to present an interim data update from the Phase I/II clinical trial in the second half of 2019.
  • RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
    • REGENXBIO expects to file an IND or foreign equivalent for the first-in-human clinical trial evaluating RGX-181 for the treatment of CLN2 in the second half of 2019.
  • RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I)
    • Recruitment, screening and additional site activations are ongoing in the Phase I clinical trial evaluating RGX-111 for the treatment of MPS I.

Other Recent Operational Highlights

  • In May 2019, Regenxbio announced its plans to construct a current good manufacturing practice (cGMP) production facility in Rockville, Maryland. The new cGMP production facility will be integrated into Regenxbio’s previously announced new 139,000 square foot headquarters, for which construction is currently underway, and will allow for production of NAV Technology-based vectors at scales up to 2,000 liters using Regenxbio’s platform suspension cell culture process. The facility will be designed to meet global regulatory requirements and is expected to be operational in 2021.

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

  • In May 2019, Novartis announced the FDA approval of Zolgensma as a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age. Novartis has stated that it anticipates regulatory approvals of Zolgensma in Japan and Europe in the second half of 2019. Regenxbio earned a $3.5 million milestone payment upon Zolgensma’s approval in the United States and is eligible to receive an additional $80 million milestone payment upon the achievement of $1 billion in cumulative net sales of Zolgensma. Regenxbio will receive tiered royalties on sales of Zolgensma up to a low double-digit percentage. Zolgensma uses the NAV AAV9 vector. 

Partnered Product Candidates

  • In July 2019, Regenxbio announced that it granted a non-exclusive worldwide license for its NAV AAV9 vector to Pfizer Inc. for the development and commercialization of gene therapy for the treatment of Friedreich’s ataxia, the most common hereditary ataxia. In return, Pfizer will provide Regenxbio with an upfront payment, and Regenxbio has the potential to receive ongoing fees, development and commercial milestone payments and royalties on net sales of products incorporating the NAV AAV9 vector.
  • In July 2019, Abeona Therapeutics, Inc. announced positive data from its ongoing phase 1/2 clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA).  ABO-102 uses the NAV AAV9 vector.
  • In June 2019, Rocket Pharmaceuticals, Inc. announced it dosed the first subject in its Phase I clinical trial of RP-A501 for the treatment of Danon disease. RP-A501 uses the NAV AAV9 vector.


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