MVX-220 for Angelman syndrome earns FDA fast track status
Mavrix Bio preparing to launch first in-human clinical trial of gene therapy
The U.S. Food and Drug Administration (FDA) has granted fast track designation to MVX-220, Mavrix Bio’s investigational gene therapy for Angelman syndrome that is headed into clinical trials.
Fast track designation aims to facilitate the clinical development and regulatory review of therapies intended to treat serious conditions with an unmet medical need.
The status affords the therapy’s developer more frequent interactions with the FDA during the clinical development process. When it comes time to submit a regulatory application, the treatment may be eligible to use regulatory pathways that will speed up the process, including accelerated approval, priority, and rolling review.
“The FDA’s decision to grant Fast Track designation for MVX-220 reflects the urgent need for therapies for individuals living with Angelman syndrome,” Allyson Berent, chief development officer for Mavrix, said in a company press release. “This designation highlights the promise MVX-220 holds as a potential treatment for [Angelman syndrome], and importantly, enables closer collaboration with the FDA and an accelerated path forward.”
Mavrix is now preparing to launch the first in-human clinical trial of MVX-220. The Phase 1/2 ASCEND-AS trial (NCT07181837) will evaluate the safety and efficacy of MVX-220 in 12 children and adults with Angelman syndrome. The FDA gave the green light for the trial’s launch in May.
The company will discuss the study’s design, enrollment criteria, and protocol at a webinar hosted by the Foundation for Angelman Syndrome Therapeutics (FAST) and the Angelman Syndrome Foundation on Oct. 9 at 1 p.m. EST. Registration is available online.
Therapy injected directly into fluid-filled spaces in skull
Angelman syndrome is a rare disorder caused by genetic abnormalities that result in the absence or dysfunction of the UBE3A gene inherited from a person’s biological mother. A loss of activity of this gene, which is important for brain development, leads to serious neurological symptoms.
MVX-220 is a gene therapy designed to deliver a healthy copy of UBE3A to brain cells. The treatment is packaged into a viral carrier that helps it be taken up by human cells.
ASCEND-AS will include people with Angelman syndrome due to various underlying genetic causes, including deletions, uniparental disomy, and imprinting center defects. Initially, it will include a group of adults, ages 18 to 50, and a group of children, ages 4 to 8.
All will receive a single dose of MVX-220, injected directly into the fluid-filled spaces in the skull, which is expected to enable direct delivery to the brain. They will also receive steroid medications before and after the procedure to prevent immune responses against the gene therapy.
For the Angelman syndrome community, Fast Track designation opens up a path toward bringing gene-targeted therapies to patients sooner.
The participants will then be monitored for a total of five years. The main goal is to monitor safety over the first two years. Changes in various Angelman symptoms will be evaluated as secondary outcome measures.
Based on a review of data from the first two groups, a third group of children and adults may later be enrolled. The trial will take place at Rush University Medical Center in Chicago.
FAST supported the early development of MVX-220 at the University of Pennsylvania and launched Mavrix earlier this year to move the treatment into clinical testing. Gemmabio will support Mavrix in its efforts. James Wilson, MD, PhD, whose team originally developed the therapy at the university, founded Gemmabio last year.
“[GemmaBio] has been deeply honored to support MavriX Bio on the MVX-220 development program,” said Wilson, now Gemmabio’s CEO. “For the Angelman syndrome community, Fast Track designation opens up a path toward bringing gene-targeted therapies to patients sooner.”
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