REGENXBIO Announces Phase I/II Trial of RGX-202, a Novel Gene … – PR Newswire

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Jan 23, 2023, 07:05 ET
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–  Firm has initiated Part I/II AFFINITY DUCHENNE trial of RGX-202 
–  Firm additionally enrolling newly energetic observational screening research, AFFINITY BEYOND, evaluating AAV8 antibody prevalence in boys with Duchenne 
–  Industrial-scale cGMP materials from the REGENXBIO Manufacturing Innovation Middle for use within the medical trial 
–  RGX-202 is a possible one-time AAV Therapeutic for the remedy of Duchenne and consists of an optimized transgene for a novel microdystrophin and REGENXBIO’s proprietary NAV® AAV8 vector
ROCKVILLE, Md., Jan. 23, 2023 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) as we speak introduced that the Part I/II AFFINITY DUCHENNE trial of RGX-202 for the remedy of Duchenne muscular dystrophy (Duchenne) is now energetic and recruiting sufferers. RGX-202 is designed to ship a transgene for a novel microdystrophin protein that features the useful parts of the C-Terminal (CT) area present in naturally occurring dystrophin. RGX-202 makes use of REGENXBIO’s proprietary NAV® AAV8 vector.
AFFINITY DUCHENNE is a multicenter, open-label dose analysis and dose enlargement medical trial to judge the protection, tolerability and medical efficacy of a one-time intravenous (IV) dose of RGX-202 in sufferers with Duchenne.
Moreover, REGENXBIO is recruiting sufferers within the AFFINITY BEYOND trial, an observational screening research. The first goal is to judge the prevalence of AAV8 antibodies in sufferers with Duchenne as much as 12 years of age. Info collected on this research could also be used to establish potential contributors for the AFFINITY DUCHENNE trial and potential future trials of RGX-202.
“I’m happy that we at the moment are capable of provoke the trial for RGX-202 and in addition start enrollment actions in our AAV8 antibody screening research,” mentioned Kenneth T. Mills, President and Chief Govt Officer of REGENXBIO. “The RGX-202 program is a key piece of our ‘5x’25’ technique to have 5 AAV Therapeutics both in the marketplace or in late-stage growth by 2025. We look ahead to persevering with to work carefully with the Duchenne group as we advance a extremely differentiated product candidate developed with the potential to enhance muscle power and motor perform in boys with Duchenne.”
“Duchenne muscular dystrophy is a devastating illness and there are nonetheless unmet therapeutic wants,” mentioned Aravindhan Veerapandiyan, M.D., a principal investigator within the research and Director of the Complete Neuromuscular Program, PPMD Licensed Duchenne Care Middle, and Co-Director of the Muscular Dystrophy Affiliation Care Middle at Arkansas Kids’s Hospital. “Gene therapies, like RGX-202, have the potential to affect the progressive nature of Duchenne.”
REGENXBIO has manufactured extra medical provide of RGX-202 in its in-house Manufacturing Innovation Middle utilizing the NAVXpress course of platform. Positioned in REGENXBIO’s 132,000 sq. foot headquarters in Rockville, MD, the Manufacturing Innovation Middle is designed to fulfill international medical and business regulatory requirements, and consists of two impartial bulk drug substance manufacturing suites, a remaining drug product suite and built-in high quality management labs. REGENXBIO is one in every of only some gene remedy corporations worldwide with a cGMP facility able to manufacturing at scales as much as 2,000 liters.
Further info will be discovered on for AFFINITY DUCHENNE and AFFINITY BEYOND.
Within the dose analysis section of the trial, six ambulatory, pediatric sufferers (ages 4 to 11 years previous) with Duchenne are anticipated to enroll in two cohorts with doses of 1×1014 genome copies (GC)/kg physique weight (n=3) and 2×1014 GC/kg physique weight (n=3). After an impartial security knowledge assessment for every cohort, a dose enlargement section of the trial could permit for as much as six extra sufferers to be enrolled at every dose stage (for a complete of as much as 9 sufferers in every dose cohort).
The trial design additionally consists of thorough security measures knowledgeable by the Duchenne group and engagement with key opinion leaders, together with a complete, short-term, prophylactic immunosuppression routine to proactively mitigate potential complement-mediated immunologic responses, and inclusion standards primarily based on dystrophin gene mutation standing, together with DMD gene mutations in exons 18 and above. Trial endpoints embrace security, immunogenicity assessments, pharmacodynamic and pharmacokinetic measures of RGX-202, together with microdystrophin protein ranges in muscle, and power and useful assessments, together with the North Star Ambulatory Evaluation (NSAA) and timed perform assessments. Preliminary trial websites are situated within the U.S., with extra websites in Canada and Europe anticipated to observe.
AFFINITY BEYOND is an observational screening research. The first goal is to judge the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in contributors with Duchenne muscular dystrophy. AAV gene therapies are delivered through viral vectors that aren’t identified to trigger illness in people. For AAV gene therapies delivered systemically, it is essential to display screen sufferers for antibodies to the vector. This observational research is meant to assist inform future medical analysis in Duchenne.
RGX-202 is designed to ship a transgene for a novel microdystrophin that features the useful parts of the C-Terminal (CT) area present in naturally occurring dystrophin. Presence of the CT area has been proven in preclinical research to recruit a number of key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle harm in dystrophic mice. Further design options, together with codon optimization and discount of CpG content material, could doubtlessly enhance gene expression, enhance translational effectivity and cut back immunogenicity. RGX-202 is designed to assist the supply and focused expression of genes all through skeletal and coronary heart muscle utilizing the NAV AAV8 vector, a vector utilized in quite a few medical trials, and a well-characterized muscle-specific promoter (Spc5-12).
Duchenne muscular dystrophy (Duchenne) is a uncommon genetic dysfunction, brought on by mutations within the gene answerable for making dystrophin, a protein of central significance for muscle cell construction and performance. Duchenne primarily impacts males with roughly 1 in 3,500 to 1 in 5,000 males affected worldwide. The absence of useful dystrophin protein in people with Duchenne leads to cell harm throughout muscle contraction, resulting in cell dying, irritation, and fibrosis in muscle tissues. Preliminary signs of Duchenne embrace muscle weak point that’s typically noticeable at an early age, with analysis sometimes occurring by 5 years of age. Over time, people with Duchenne expertise progressive muscle weak point and ultimately lose the power to stroll. Respiratory and coronary heart muscle tissues are additionally affected, resulting in issue respiration and the necessity for ventilator help, together with the event of cardiomyopathy. There’s presently no treatment for Duchenne.
REGENXBIO is a number one clinical-stage biotechnology firm searching for to enhance lives via the healing potential of gene remedy. REGENXBIO’s NAV Know-how Platform, a proprietary adeno-associated virus (AAV) gene supply platform, consists of unique rights to greater than 100 novel AAV vectors, together with AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Know-how Platform Licensees are making use of the NAV Know-how Platform within the growth of a broad pipeline of candidates, together with late-stage and business packages, in a number of therapeutic areas. REGENXBIO is dedicated to a “5x’25” technique to progress 5 AAV Therapeutics from our inner pipeline and licensed packages into pivotal-stage or business merchandise by 2025.
This press launch consists of “forward-looking statements,” inside the which means of Part 27A of the Securities Act of 1933, as amended, and Part 21E of the Securities Trade Act of 1934, as amended. These statements specific a perception, expectation or intention and are usually accompanied by phrases that convey projected future occasions or outcomes equivalent to “consider,” “could,” “will,” “estimate,” “proceed,” “anticipate,” “assume,” “design,” “intend,” “count on,” “might,” “plan,” “potential,” “predict,” “search,” “ought to,” “would” or by variations of such phrases or by related expressions. The forward-looking statements embrace statements referring to, amongst different issues, REGENXBIO’s future operations and medical trials. REGENXBIO has primarily based these forward-looking statements on its present expectations and assumptions and analyses made by REGENXBIO in gentle of its expertise and its notion of historic traits, present situations and anticipated future developments, in addition to different elements REGENXBIO believes are applicable underneath the circumstances. Nonetheless, whether or not precise outcomes and developments will conform with REGENXBIO’s expectations and predictions is topic to a lot of dangers and uncertainties, together with the timing of enrollment, graduation and completion and the success of medical trials performed by REGENXBIO, its licensees and its companions, the timing of graduation and completion and the success of preclinical research performed by REGENXBIO and its growth companions, the well timed growth and launch of recent merchandise, the power to acquire and keep regulatory approval of product candidates, the power to acquire and keep mental property safety for product candidates and know-how, traits and challenges within the enterprise and markets during which REGENXBIO operates, the scale and development of potential markets for product candidates and the power to serve these markets, the speed and diploma of acceptance of product candidates, the affect of the COVID-19 pandemic or related public well being crises on REGENXBIO’s enterprise, and different elements, a lot of that are past the management of REGENXBIO. Consult with the “Danger Elements” and “Administration’s Dialogue and Evaluation of Monetary Situation and Outcomes of Operations” sections of REGENXBIO’s Annual Report on Type 10-Okay for the 12 months ended December 31, 2021, and comparable “threat elements” sections of REGENXBIO’s Quarterly Studies on Type 10-Q and different filings, which have been filed with the U.S. Securities and Trade Fee (SEC) and can be found on the SEC’s web site at The entire forward-looking statements made on this press launch are expressly certified by the cautionary statements contained or referred to herein. The precise outcomes or developments anticipated might not be realized or, even when considerably realized, they could not have the anticipated penalties to or results on REGENXBIO or its companies or operations. Such statements aren’t ensures of future efficiency and precise outcomes or developments could differ materially from these projected within the forward-looking statements. Readers are cautioned to not rely too closely on the forward-looking statements contained on this press launch. These forward-looking statements communicate solely as of the date of this press launch. Besides as required by legislation, REGENXBIO doesn’t undertake any obligation, and particularly declines any obligation, to replace or revise any forward-looking statements, whether or not because of new info, future occasions or in any other case.
Dana Cormack 
Company Communications 
[email protected] 
Chris Brinzey, ICR Westwicke 
[email protected] 
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