REGENXBIO Provides Year-End 2017 Corporate Update
- Initiated third cohort dosing of RGX-314 Phase I clinical trial for wet AMD -
- Initiated second cohort dosing of RGX-501 Phase I/II clinical trial for HoFH -
- Anticipate completing dosing and presenting topline data from RGX-314 and RGX-501 trials in late 2018 -
- Anticipate initiating dosing in clinical trials for MPS I and MPS II in first half 2018 -
- Ended 2017 with greater than
“In 2017,
Lead Product Candidate Clinical Trial Updates
- RGX-314 for the treatment of wet age-related macular degeneration (wet AMD)
-- As ofDecember 31, 2017 , 13 participants had been treated with a single administration of RGX-314 in the dose-escalation clinical trial, with no reported drug-related serious adverse events (SAEs).
-- An independent Data Safety and Monitoring Board (DSMB) granted clearance to proceed to dosing a third cohort based on their assessment of the safety and tolerability data of the first and second cohorts.
-- As ofDecember 1, 2017 , the first and second cohorts (six participants in each) had received a single administration of RGX-314 at doses of 3 x 10^9 and 1 x 10^10 genome copies (GC)/eye, respectively, and had been followed for an average of 20 weeks and six weeks, respectively. Additionally, the first patient in the third cohort was subsequently treated at a dose of 6 x 10^10 GC/eye.
-- Treatments have been administered at four sites inthe United States . The procedure has been completed via automated delivery by each of the trained surgeons using a vitrectomy machine within an hour or less in most cases.
-- The primary endpoint for our ongoing Phase I clinical trial of RGX-314 is safety. Based on the 12 patients dosed as of December 1, 2017, we have observed RGX-314 to be generally well-tolerated. There were no drug-related SAEs, and all drug-related adverse events (AEs) were assessed as mild or moderate.
-- There have been no observed immune responses, drug-related ocular inflammation, or any post-surgical inflammation beyond what is expected following routine vitrectomy.
-- Evidence of dose-dependent RGX-314 protein expression, as measured by enzyme-linked immunosorbent assay (ELISA) at approximately four weeks after administration of RGX-314, has been noted in subjects treated to date in the first and second dosing cohorts.
-- For further details on the RGX-314 Phase I trial, enrollment criteria and eligibility, please contact patientadvocacy@regenxbio.com or visit https://clinicaltrials.gov/ct2/show/NCT03066258. - RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH)
-- As ofDecember 31, 2017 , three participants had been treated with a single administration of RGX-501 in the dose-escalation clinical trial, with no dose limiting toxicities reported, and a fourth participant was recently treated.
-- An independent DSMB granted clearance to proceed to dosing a second cohort based on their assessment of the safety and tolerability data from the first dosing cohort.
-- As ofDecember 1, 2017 , the first three participants had received a single administration of RGX-501 at an initial dose of 2.5 x 10^12 genome copies (GC)/kg body weight and had been followed for an average of 29 weeks. A fourth patient was recently infused at a dose of 7.5 x 10^12 GC/kg body weight.
-- All treatments have been administered at theUniversity of Pennsylvania and infusion time has averaged less than two hours.
-- The primary endpoint for our ongoing Phase I/II clinical trial of RGX-501 is safety. Based on the three patients dosed as ofDecember 1, 2017 , we have observed RGX-501 to be generally well-tolerated.
-- One subject in the first cohort experienced an SAE within 24 hours of dosing of hypotension associated with a mild inflammatory response, which resolved within a few hours of onset. The nature and time frame of the SAE is distinct from expected and known immune responses to AAV therapy. The subject recovered quickly from the event without significant sequalae. All other AEs to date have been mild to moderate with no determined relationship to drug product.
-- For further details on the RGX-501 Phase I/II trial, enrollment criteria and eligibility, please contact patientadvocacy@regenxbio.com or visit http://www.clinicaltrials.gov/ct2/show/NCT02651675. - RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I)
-- Site activation is continuing in the Phase I clinical trial evaluating RGX-111 for the treatment of MPS I.
-- Patient recruitment is anticipated to begin in the first quarter of 2018, with the first patient expected to be dosed in the first half of 2018. - RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)
-- InDecember 2017 ,REGENXBIO announced that the Investigational New Drug (IND) application for the Phase I/II clinical trial of RGX-121 for the treatment of MPS II 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.
Other Operational Highlights
In 2017,
Anticipated 2018 Milestones
- RGX-314 for the treatment of wet AMD
-- Complete enrollment in the Phase I clinical trial in the first half of 2018.
-- Present topline data from the Phase I clinical trial in late 2018. - RGX-501 for the treatment of HoFH
-- Complete enrollment in the Phase I/II clinical trial in mid-2018.
-- Present topline data from the Phase I/II clinical trial in late 2018. - RGX-111 for the treatment of MPS I
-- Begin enrollment in a Phase I clinical trial in the first half of 2018.
-- Present interim update from the Phase I clinical trial in late 2018. - RGX-121 for the treatment of MPS II
-- Begin enrollment in a Phase I/II clinical trial in the first half of 2018.
-- Present interim update from the Phase I/II clinical trial in late 2018.
In addition,
NAV Technology Licensee Program Highlights
As of
Clinical Development of NAV Technology Platform
- AVXS-101 (
AveXis, Inc. ), which uses the NAV AAV9 vector for the treatment of spinal muscular atrophy (SMA):
-- Announced the first patient has been dosed in the pivotal trial of AVXS-101 for the treatment of SMA Type 1.
-- Announced plans to immediately initiate a Phase I clinical trial of AVXS-101 for the treatment of SMA Type 2 via the intrathecal (IT) route of administration. - AT132 (
Audentes Therapeutics, Inc. ), which uses the NAV AAV8 vector for the treatment of X-linked myotubular myopathy:
-- Announced completed enrollment of the first cohort in the Phase I/II clinical trial evaluating AT132 for the treatment of X-linked myotubular myopathy.
-- OnJanuary 4, 2018 , Audentes announced positive interim data from the first cohort. - DTX301 (
Ultragenyx Pharmaceutical Inc. ), which uses the NAV AAV8 vector for the treatment of ornithine transcarbamylase (OTC) deficiency:
-- Announced completed enrollment of the first cohort in the Phase I/II clinical trial evaluating DTX301 for the treatment of OTC deficiency. SHP654 (Shire plc ), which uses the NAV AAV8 vector for the treatment of hemophilia A
-- Announced theFDA had awarded Orphan Drug Designation to its gene therapy candidateSHP654 for the treatment of hemophilia A.
Expansion of NAV Technology Platform
- In
August 2017 , we grantedPrevail Therapeutics Inc. a license for the use of the NAV AAV9 vector to develop gene therapy product candidates for the treatment of Parkinson’s disease and other related neurodegenerative disorders. - In
June 2017 , we grantedAveXis, Inc. a new license for the use of the NAV AAV9 vector for the development and commercialization of gene therapies for Rett syndrome and amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene.
Financial Guidance
As of
About REGENXBIO Inc.
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.
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