Sarepta Therapeutics, a Cambridge, Massachusetts-based biopharmaceutical company focused on RNA-targeted therapeutics for rare neuromuscular diseases, and BioMarin Pharmaceutical, a biotechnology company developing therapies for rare genetic diseases, have executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to BioMarin’s duchenne muscular dystrophy (DMD) patent estate for Exondys 51 (eteplirsen) and all future exon-skipping products.Exondys 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.

Under the license and settlement agreements, Sarepta will make a one-time payment of $35 million to BioMarin and certain additional regulatory and commercial milestone payments for exons 51, 45, 53, and possibly on future exon-skipping products.

For Exon-skipping compounds 51, 45, and 53 and possibly on future exon-skipping products, Sarepta will pay BioMarin 5% of net sales through the end of 2023 in the US. Sarepta will also pay BioMarin 8% of net sales through September 30, 2024 in the European Union and in other countries where certain BioMarin/Academisch Ziekenhuis Leiden patents exist.

BioMarin retains the right to convert the license to a co-exclusive right in the event it decides to proceed with an exon-skipping therapy for DMD.

The effectiveness of the agreements is subject to closing conditions including execution of necessary approvals by Academisch Ziekenhuis Leiden by July 24, 2017.

Source: Sarepta Therapeutics