• 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.  

Demo Expansion

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 .

About RGX-314

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.

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 Area 21E from the Securities Exchange Act associated with 1934, because amended. These statements express a belief, expectation or intention and are generally accompanied simply by words that convey projected future activities or final results such as “believe, ” “may, ” “will, ” “estimate, ” “continue, ” “anticipate, ” “assume, ” “design, ” “intend, ” “expect, ” “could, ” “plan, ” “potential, ” “predict, ” “seek, ” “should, ” “would” or by variations of such words or simply by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO’s future operations and clinical trials. REGENXBIO provides based these types of forward-looking claims on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception associated with historical trends, current problems and expected future developments, as nicely as other factors REGENXBIO believes are appropriate under the circumstances. However , whether actual results plus developments will conform along with REGENXBIO’s anticipation and predictions is subject to a number of risks and uncertainties, including the particular timing of enrollment, commencement and completion as well as the success of medical trials conducted by REGENXBIO, its licensees as well as partners, the timing of beginning and conclusion and the achievement of preclinical studies carried out by REGENXBIO as well as its development partners, the timely advancement and launch of new products, the opportunity to obtain and maintain regulatory approval associated with product applicants, the ability to acquire and keep intellectual property protection for product candidates and technology, trends and challenges in the business plus markets within which REGENXBIO operates, the size and growth of possible markets intended for product candidates and the particular ability to serve those markets, the rate and degree of acceptance of item candidates, the impact of the COVID-19 pandemic or even similar public health crises on REGENXBIO’s business, as well as other factors, many of which are usually beyond the particular control of REGENXBIO. Refer to the “Risk Factors” and “Management’s Discussion plus Analysis associated with Financial Condition and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K to get the year ended December 31, 2021 , plus comparable “risk factors” parts of REGENXBIO’s Quarterly Reports on Form 10-Q and some other filings, which have been filed with the U. S. Securities and Trade Commission (SEC) and are available on the particular SEC’s web site at www.sec.gov . All of the forward-looking statements made in this press release are expressly qualified by the cautionary claims contained or referred in order to herein. The particular actual outcomes or advancements anticipated may not be realized or even, even if substantially realized, they may not have the particular expected consequences to or effects upon REGENXBIO or even its businesses or operations. Such statements are not really guarantees of future performance and real results or developments may differ materially from those projected within the forward-looking statements. Readers are usually cautioned not to rely too heavily on the forward-looking claims contained in this press release. These types of forward-looking statements speak only as associated with the date of this push release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or even revise any kind of forward-looking claims, whether as a result of new information, future events or otherwise.

SCS Microinjector ^® is a trademark of Clearside Biomedical, Incorporation. All other trademarks referenced herein are registered trademarks of  REGENXBIO.

Contacts:
Dana Cormack
Corporate Communications
[email protected]  

Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
[email protected]

SOURCE REGENXBIO Inc.