Dutch pharmaceutical company ProQR Therapeutics now has two key patents protecting its first-in-class ribonucleic acid (RNA)-based oligonucleotide QR-010 for the treatment of cystic fibrosis (CF) in the United States and Europe. The patents are valid until at least July 2033.
The new U.S. patent, announced April 3, covers methods of targeting the F508del mutation in the CF transmembrane conductance regulator (CFTR), using RNA oligonucleotides to restore the dysfunctional CFTR protein. The F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the defective CFTR protein. QR-010 binds to the defective CFTR mRNA and restores CFTR function.
ProQR adds this U.S. patent to an equivalent European patent granted last year. Topline data from a previous trial showed that QR-010 restored CFTR function in CF patients with two F508del mutations — encouraging researchers to further develop the candidate.
QR-010 is now the focus of another Phase 1b clinical trial (NCT02532764) of 64 CF patients with two F508del mutations. The trial is still recruiting patients in the United States, Canada and Europe, with topline data to be reported later this year.
“We are very pleased with the granting of these key pieces of intellectual property (IP) in our large and expanding IP estate, protecting QR-010 and the broader technology beyond that,” Rene Beukema, general counsel and chief corporate development officer of ProQR, said in a press release. “We pursue a very aggressive patent strategy as our 16 patent families, along with six in-licensed patent estates, provide multiple layers of protection for our novel products and technologies.”
ProQR plans to present data on QR-010 at the European Cystic Fibrosis Society Conference in June. The therapy — which enjoys orphan drug designation in the United States and Europe, and since July 2016, fast-track status in the United States — is designed for self-administration via an optimized eFlow Nebulizer that directs the drug directly into the lungs.