REGENXBIO Announces FDA Clearance of IND for Phase II Trial of RGX-314 for the Treatment of Diabetic Retinopathy Using Suprachoroidal Delivery

Rhea-AI Summary

REGENXBIO Inc. (Nasdaq: RGNX) announced the FDA's clearance of its Investigational New Drug (IND) application for RGX-314 to treat diabetic retinopathy (DR). This IND activation allows the company to commence a Phase II trial named ALTITUDE in the second half of 2020, targeting around 40 patients. The primary endpoint is to evaluate patient improvement in DR severity after 48 weeks. This advancement is significant as DR is a major cause of blindness, affecting approximately 8 million people in the U.S.

Positive

• FDA clearance for the IND application of RGX-314.
• Initiation of Phase II trial ALTITUDE expected in late 2020.
• Potential for RGX-314 to treat a significant patient population affected by diabetic retinopathy.

Negative

• None.

ROCKVILLE, Md., Aug. 25, 2020 /PRNewswire/ -- 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 the clearance of the Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) to evaluate the suprachoroidal delivery of RGX-314 in patients with diabetic retinopathy (DR). The IND is active, and REGENXBIO plans to begin dosing patients in a Phase II trial, ALTITUDE, in the second half of 2020.

"Diabetic retinopathy is the leading cause of blindness in patients 24 to 75 years of age and affects approximately 8 million people in the United States alone. Without treatment, a large proportion of patients with DR develop proliferative disease and vision threatening complications, including center-involved diabetic macular edema. We believe one-time treatment with RGX-314 can provide sustainable, long-term anti-VEGF delivery to the eye, potentially reducing the severity of DR and preventing vision threatening complications," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We are pleased to advance this program, and look forward to initiating our second clinical trial utilizing suprachoroidal delivery, building upon clinical and preclinical data from the RGX-314 program."

ALTITUDE is a multi-center, open label, randomized, controlled dose-escalation study that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314. The trial is expected to enroll 40 patients with DR across two cohorts. Patients will be randomized to receive RGX-314 versus observational control at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated: 2.5x10¹¹ GC/eye and 5.0x10¹¹ GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.

The primary endpoint of the study is the proportion of patients that improve in DR severity based on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS) at 48 weeks. Other endpoints include safety and development of DR-related ocular complications.

The Company expects to report interim data from this trial in 2021.

About RGX-314
RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an antibody fragment which is designed to inhibit VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss.

About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide. DR affects approximately 8 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 as DR progresses, a large proportion of patients develop vision threatening complications, including diabetic macular edema (DME) and neovascularization that can lead to blindness.  Current treatment options for patients with DR include "watchful waiting", anti-VEGF treatment, retinal laser or surgical treatment. Anti-VEGF treatments have been shown to reduce the severity of DR and prevent vision threatening complications by at least 75% in patients with moderately severe to severe NPDR.

About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

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 and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts:

Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709

Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com

Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com 

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SOURCE REGENXBIO Inc.

FAQ

What is RGX-314 being developed for?

RGX-314 is being developed as a potential one-time treatment for diabetic retinopathy and other chronic retinal conditions.

When will the Phase II trial for RGX-314 begin?

The Phase II trial, named ALTITUDE, is planned to begin in the second half of 2020.

What is the primary endpoint of the ALTITUDE trial?

The primary endpoint is the improvement in diabetic retinopathy severity after 48 weeks of treatment.

How many patients will be enrolled in the ALTITUDE trial?

The ALTITUDE trial is expected to enroll 40 patients with diabetic retinopathy.

What age group does diabetic retinopathy primarily affect?

Diabetic retinopathy primarily affects patients aged 24 to 75 years.