IPS HEART Granted Orphan Drug Designation in the EU for Becker Muscular Dystrophy

IPS HEART has been awarded Orphan Drug Designation (ODD) in the EU by the EUROPEAN MEDICINES AGENCY for GIVI-MPCs unique ability to create new muscle with 100% full length dystrophin in Becker Muscular Dystrophy (BMD). IPS HEART has now shown GIVI-MPCs can create new human muscle with full length human dystrophin in BMD dystrophic pigs, young and extremely aged Duchenne Muscular Dystrophy (DMD) mice and in a sarcopenia mouse model. The FDA previously awarded GIVI-MPCs Orphan Drug Designation for both BMD & DMD, highlighting the unique platform potential of GIVI-MPCs to reverse both devastating diseases. All current DMD therapeutics initially approved by the FDA through novel biomarkers including exon skipping and gene therapy therapeutics cannot produce 100% full length dystrophin nor can they create any new muscle to replace dead dystrophic muscle.

While we are grateful to the dozens of BMD patients who have emailed us directly for information on our upcoming clinical trials, to date we are initially focused on advancing GIVI-MPCs clinically in a Phase I/II DMD clinical trial until we can secure additional partners to help us advance clinically in BMD. IPS HEART is currently under formal CDA with certain large pharmaceutical companies for a possible partnership that will also allow us to advance GIVI-MPCs clinically in BMD as we have already successfully produced GIVI-MPCs via large scale suspension based bioreactor manufacturing which can supply the large patient population in the future.

About IPS HEART/GIVI-MPCs

GIVI-MPCs derived from pluripotent stem cells form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD. IPS HEART has also developed ISX-9-CPCs, a human iPSC-derived cardiac therapy for treatment of Duchenne cardiomyopathy and heart failure that develops new heart muscle.

About Becker & Duchenne Muscular Dystrophy

BMD is a muscular disease and DMD is a fatal neuromuscular disease.

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