On the 1st of August, RiogenxBio provided a positive update on the interim safety and efficacy data collected from their Phase I/II AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy aged between 1-11 years old.
In patients aged 5.8 and 8.5 who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 77.2%, and 46.5%, respectively, compared to control at three months.
RegenxBio reported that as of July 8, 2024, RGX-202 has been well tolerated with no serious adverse events. All seven patients who completed three-month trial assessments indicate meaningful increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement.
With today’s new, positive data, we are seeing a clear dose response and consistent, robust microdystrophin expression levels across all treated patients in the AFFINITY DUCHENNE trial, and, notably, among the highest levels of microdystrophin expression reported in older ambulatory patients. This data continues to build on the totality of evidence supporting the potential for RGX-202 to be a differentiated, best-in-class treatment for Duchenne. RGX-202 is well positioned to be the next potential gene therapy approved for Duchenne, and, with our commercial-ready, suspension-based manufacturing platform process, we believe we have the capacity to serve the entire market.
Curran M. Simpson, President and CEO, REGENXBIO
I remain encouraged by the biomarker data from the AFFINITY DUCHENNE trial of RGX-202 and am eagerly anticipating the initial functional data from this program. This update is also encouraging for the Duchenne community, which is exploring various treatment options that could influence disease progression.
Aravindhan Veerapandiyan, M.D., Arkansas Children’s Hospital
Key Points:
- Robust microdystrophin expression observed in new data from pivotal dose
- Patients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of control, respectively.
- Consistent high expression of microdystrophin across treated patients in all age groups continues to support plans for accelerated approval
- Expects to initiate pivotal trial in Q4 2024
RGX-202
An investigational one-time AAV Therapeutic designed to deliver a novel microdystrophin gene that includes key regions of the naturally occurring dystrophin gene. RGX-202 is the only gene therapy in development for DMD that encodes for the C-Terminal domain to produce microdystrophin that more closely resembles the naturally occurring dystrophin protein. Preclinical studies have shown the C-Terminal domain to protect the muscle from contraction-induced stress and improve its ability to repair itself.
Further updates
REGENXBIO expects to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
You’re invited! – Our 2024 Annual International Conference will be held on Friday 8th and Saturday 9th November 2024. The conference brings together families, clinicians, researchers and experts for 2 days of sharing knowledge and experience. It is an amazing opportunity to come together as part of the Duchenne community
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