- RGX-314 continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events
- Patients treated with RGX-314 in Cohorts 1-3 demonstrated clinically meaningful improvements in disease severity and less disease worsening versus observation control at six months
- 20% associated with patients achieved ≥2-step DRSS improvement vs. 10% inside control
- 54% of patients accomplished any DRSS improvement vs . 20% in control
- 0% of patients worsened ≥2 steps versus 20% in charge
- No meaningful differences in safety outcomes at 6 months for individuals who are NAb positive
- Phase II trial expanded to include higher third dose level, with patients stratified by DRSS levels across cohorts and all receiving short-course prophylactic ocular steroids following RGX-314 administration
ROCKVILLE, Md. , Nov. 3, 2022 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX ) today announced additional positive interim data from the particular ongoing Phase II ALTITUDE ® trial associated with RGX-314 for the treatment of diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME) using in-office suprachoroidal delivery. The data were presented in the 55 th Annual Retina Society meeting in Pasadena, CA by Lejla Vajzovic , MD, FASRS, Associate Professor of Ophthalmology plus Director associated with Duke Vitreoretinal Fellowship Program, Vitreoretinal Surgery and Disease, Department of Ophthalmology, Duke University School of Medicine. RGX-314 is being investigated as a potential one-time gene therapy for the remedying of wet age-related macular degeneration, DR, and other chronic retinal conditions.
“We are usually pleased with these positive interim results which demonstrate a clinically significant improvement in disease intensity versus observational control, along with more than 50% associated with patients dosed with RGX-314 in Cohorts 1-3 seeing improvement through baseline in their DRSS scores, ” said Steve Pakola , M. D., Chief Medical Officer of REGENXBIO. “RGX-314 continues to be well-tolerated across cohorts, and we look forward to the particular expansion associated with this trial to further evaluate the potential of RGX-314 for sufferers with DOCTOR. ”
“I am encouraged simply by the positive results observed inside the HÖHE trial of RGX-314, inch said Dr. Vajzovic. “A one-time, in-office injection associated with RGX-314 gene therapy could potentially provide critical improvement in DR severity and reduce the risk of vision threatening complications. Early intervention is key in slowing disease progression for DOCTOR patients, and RGX-314 has the possible to offer an important treatment option to address the current unmet need for these types of patients. I anticipate viewing additional results from Cohorts 4 plus 5 of the ALTITUDE test. ”
“DR is the leading cause of blindness in working-age adults globally, and there is a significant requirement for new treatment options, ” said Michael Robinson , MD, Vice President, Global Therapeutic Area Head Eye Care at AbbVie. “Progressing RGX-314 and the particular ALTITUDE demo with the aim of addressing unmet needs for patients with retinal diseases is part of AbbVie’s commitment to advancing vision care. ”
Data Summary and Safety Update from Phase II ALTITUDE Test of RGX-314 for the Treatment of DR Using Suprachoroidal Delivery
ALTITUDE is a multi-center, open-label, randomized, controlled, dose-escalation trial evaluating the efficacy, safety and tolerability of suprachoroidal delivery associated with RGX-314 using the SCS Microinjector ® in individuals with the DR diagnosis of moderately severe or severe nonproliferative diabetic retinopathy (NPDR) or even mild proliferative diabetic retinopathy (PDR). Individuals in Cohort 1 received RGX-314 at a dose level of 2. 5×10 11 genomic copies per eye (GC/eye) (D1). Patients within Cohorts 2 and 3 received RGX-314 at an increased dose degree of 5×10 eleven GC/eye (D2). Patients in Cohorts 1-3 did not receive prophylactic corticosteroid therapy before or after administration of RGX-314.
As of October 17, 2022 , RGX-314 was reported to end up being well tolerated in Cohorts 1-3. Five serious undesirable events were reported, none of which were considered drug related. For the total group of Cohorts 1-3 (n=50), common ocular treatment-emergent adverse occasions in the particular study vision through six months had been predominantly mild and included conjunctival hemorrhage, conjunctival hyperemia and episcleritis. In addition, three sufferers had intraocular inflammation (IOI), all moderate and resolved on topical corticosteroids. There were no meaningful variations in safety results observed with regard to patients who are neutralizing antibody (NAb) good. Best Corrected Visual Acuity remained stable in Cohorts 1-3 through six months.
At 6 months, patients treated with RGX-314 exhibited clinically meaningful improvements inside disease severity versus statement control as measured by the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (DRSS). Specifically:
- 20% (D1: 40%, D2: 11%) attained ≥2-step DRSS improvement vs . 10% in control
- 54% (D1: 60%, D2: 51%) achieved any DRSS improvement versus 20% in charge
- 0% (D1: 0%, D2: 0%) worsened ≥2 steps vs. 20% in control
A ≥2-step enhancement in DRSS at 12 months has been accepted like a pivotal endpoint by the U. S. Food and Drug Administration regarding DR clinical trials.
REGENXBIO introduced today that the ALTITUDE trial continues to be expanded to incorporate a higher third dosage amount of 1×10 twelve GC/eye. The trial will be currently enrolling two new cohorts (Cohorts 4 and 5) at the third serving level. Cohorts 4 plus 5 are enrolling individuals stratified simply by DRSS levels, with sufferers in Cohort 4 having moderately severe or serious NPDR DRSS levels associated with 47-53 and patients in Cohort 5 having slight to moderate PDR DRSS levels of 61-65. Patients in these cohorts will get short-course, prophylactic ocular corticosteroids following RGX-314 to evaluate the ability to prevent or reduce the occurrence of the particular mild intraocular inflammation seen to date. Patients will be enrolled in these cohorts regardless of baseline AAV8 NAb status.
Data presented today is available on the “Presentations plus Publications” section of the particular REGENXBIO website at www.regenxbio.com .
RGX-314, being developed in collaboration with AbbVie, is being investigated as a potential one-time therapy for wet AMD, diabetic retinopathy, along with other chronic retinal conditions. RGX-314 consists of the NAV ® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which usually new, leaky blood vessels grow and contribute to the particular accumulation associated with fluid in the retina.
REGENXBIO is improving research within two separate routes of administration associated with RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO offers licensed certain exclusive rights towards the SCS Microinjector ® through Clearside Biomedical, Inc. in order to deliver gene therapy treatments to the particular suprachoroidal area from the attention.
About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of eyesight loss inside adults between 24 and 75 years of age worldwide. DOCTOR affects approximately 10 million people in the United States alone. The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR) and while DR progresses, a large proportion of patients develop vision threatening problems, including diabetic macular edema (DME) plus neovascularization that will can lead to blindness. Chronic, frequent treatment with anti-VEGF agents has been shown to improve DR intensity and minimize danger of development to eyesight threatening complications by > 70%. However, the majority of DOCTOR patients without having vision threatening complications are usually not given anti-VEGF due to the unsustainable therapy burden of frequent injections in the eye.
About REGENXBIO Inc.
REGENXBIO is usually a leading clinical-stage biotechnology company seeking to improve lives through the particular curative potential of gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene shipping platform, consists of exclusive rights in order to more compared to 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technologies Platform Licensees are applying the NAVIGATION Technology Platform in the development of the broad pipeline of candidates, including late-stage and commercial programs, within multiple therapeutic areas. REGENXBIO is committed to a “5x’25” strategy to progress five AAV Therapeutics from our internal pipeline plus licensed programs into pivotal-stage or commercial products by 2025.
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SCS Microinjector ® is a trademark of Clearside Biomedical, Incorporation. All other trademarks referenced herein are registered trademarks of REGENXBIO.
SOURCE REGENXBIO Inc.