RGX-121 is a gene therapy designed to deliver a functional copy of the IDS gene to the central nervous system via the NAV AAV9 vector. Credits: Natali _ Mis/Shutterstock.com
RegenxBio
has announced that the pivotal dose level from its multiphase study of RGX-121 has demonstrated positive long-term data for the treatment of patients with mucopolysaccharidosis type II (MPS II) or Hunter syndrome.
As per the 3 September press release, patients who received a pivotal dose of RGX-121, described as dose level 3, had a median reduction of cerebrospinal fluid (CSF) heparan sulfate D2S6 levels of 85%. These levels were found to be approaching normal measures and were reportedly sustained for up to two years. RGX-121 is a gene therapy designed to deliver a functional copy of the IDS gene to the central nervous system via the NAV AAV9 vector.
The biotech is on track to submit a rolling biologics license application (BLA) through the accelerated approval pathway in Q3 2024. RegenxBio intends to use CSF D2S6 as a surrogate endpoint to predict clinical benefit.
The Phase I/II/III CAMPSIITE study (NCT03566043) is an open label study comprised of two parts. Part 1 marked the Phase I/II single arm dose escalation study evaluating RGX-121 in 16 pediatric participants with neuronopathic MPS II over a period of 24 weeks. Assessing safety was the primary objective during the study period, after which participants continued to be evaluated for safety and efficacy for up to 104 weeks.
Part 2 is the single arm pivotal expansion evaluating 30 pediatric patients at various points over 24 months. Primary endpoints of part 2 included CSF GAG biomarker levels, as measured by D2S6, and neurodevelopmental function as determined by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL).
See Also:
Ocaliva approval in Europe withdrawn following data re-assessment
UK NHS launches vaccine programme for RSV
The Rockville, Maryland-based biotech noted that as of 5 January, RGX-121 was found to be well tolerated in the 25 patients who received treatment across all phases of the study.
MPS II is a rare, X-linked recessive disorder characterised by a deficiency in lysosomal enzyme iduronate-2-sulfatase (IDS), which leads to a build-up of large sugar molecules called glycosaminoglycans in body tissues.
According to GlobalData’s consensus forecasts, RGX-121 is anticipated to generate global sales of $460m in 2030. GlobalData is the parent company of
Pharmaceutical Technology
.
Presenting data earlier in the year at the 20th Annual WORLDSymposium, RegenxBio shared that CAMPSIITE
achieved its primary endpoint
, with the findings being consistent with that found in the dose-finding portion of the study.
“The data continue to support that by restoring the gene missing in boys with Hunter syndrome, RGX-121 changes the course of disease and has the potential to significantly improve both vital brain function and the systemic manifestations of this devastating disease,” said CMO Dr. Steve Pakola in the press announcement.
Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.
Editorial content is independently produced and follows the
highest standards
of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
Free Whitepaper
Cell and gene therapies: Pipe dream to pipeline
The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead.
Thank you.
You will receive an email shortly. Please check your inbox to download the Whitepaper.
By Cytiva Thematic
By downloading this Whitepaper, you acknowledge that GlobalData may share your information with
Cytiva Thematic
and that your personal data will be used as described in their
Privacy Policy