One of the most significant obstacles to the application of Elevidys gene therapy developed for Duchenne muscular dystrophy disease is the high anti-AAV antibodies. Hansa Biopharma announces supportive data from treatment with imlifidase prior to the administration of gene therapy for Duchenne muscular dystrophy. Following imlifidase therapy in three patients, pre-existing anti-AAV antibodies decreased to below 1:400 titer, allowing treatment with ELEVIDYS.
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Hansa Biopharma, announced topline results from three patients with Duchenne muscular dystrophy (DMD) treated with Hansa’s imlifidase prior to receiving Sarepta’s ELEVIDYS (delandistrogene moxeparvovec-rokl) in the SRP-9001-104 trial.
Hansa Biopharma reports encouraging results from imlifidase
After one dose of imlifidase, three patients experienced a rapid reduction of IgG antibodies, to levels ≥95% less than baseline. In addition, in these three patients pre-existing anti-AAV antibodies were reduced below a titre of 1:400, which enabled treatment with ELEVIDYS.
The safety profile of imlifidase was in keeping with prior experience and the trial did not generate any new safety signals. Twelve weeks after administration of the gene therapy, patients in the trial demonstrated evidence of AAV-mediated transduction and expression of micro-dystrophin, however with levels lower than seen in other trials with ELEVIDYS. Based on these outcomes, Hansa and Sarepta will discuss appropriate next steps for the program.
What Does Imlifidase Do in Gene Therapies?
Imlifidase is currently being evaluated as a pre-treatment to gene therapy in areas of high unmet need. Many gene therapies are based on the use of Adeno Associated Viruses (AAV) vectors. In some patients the immune system carries antibodies that counteract the gene therapy treatment preventing its success. Pre-treatment with imlifidase prior to AAV-based gene therapy treatment has the potential to inactivate antibodies and thereby enable gene therapy in patients with pre-existing antibodies to AAV-based gene therapies. Currently, it is estimated that NAbs on average prevent 1 in 3 people from benefiting from gene therapy treatments.
What is the SRP-9001-104 Clinical Trial?
SRP-9001-104 is an open-label trial of ambulatory male patients with DMD between four and nine years of age, and with pre-existing antibodies to ELEVIDYS. Up to six patients will be treated with imlifidase followed by ELEVIDYS. All patients included in the trial are currently ineligible to receive ELEVIDYS due to the presence of antibodies targeting AAVrh74, the vector used to deliver Sarepta’s gene therapy treatment.
Learn More: Frequently Asked Questions About Elevidys
