uniQure Granted 2 New Patents Covering Gene Therapy Approach for Hemophilia B

A gene therapy approach acquired by uniQure was recently covered in two new patents, one issued in the United States and one in Canada, broadly covering ways of treating bleeding disorders that include hemophilia B, the company announced in a press release.

The therapy has a nucleic acid encoding the hyperactive Factor IX (FIX) Padua variant — a factor IX protein carrying a leucine (an amino acid, or building block of proteins) at the R338 position, often called Padua mutant or FIX-Padua. The patent cover the use of adeno-associated virus (AAV) gene therapy with FIX-Padua to treat hemophilia B and other disorders.

A potential treatment using this gene therapy approach, called AMT-060, is in an ongoing Phase 1/2 study (NCT02396342) in hemophilia B patients.

The newly issued U.S. patent in the second such award in a group of patents that uniQure acquired from the approach’s inventor, Paolo Simioni, a hemophilia expert at the University of Padua, Italy. The earlier U.S. issued patent covers compositions of FIX-Padua nucleic acids (DNA or RNA) and polypeptides (proteins) and their therapeutic uses.

Canada granted uniQure a patent (2,737,094) covering FIX-Padua nucleic acids for use in gene therapy and FIX-Padua polypeptides for use in FIX replacement therapy in February. Similar patents are now being sought in Europe.

“We are pleased with the ongoing progress related to our FIX-Padua patent portfolio,” Jonathan Garen, chief business officer at uniQure, said in the release. “Our new U.S. patent … , further confirms the patentability in the U.S of Professor Simioni’s groundbreaking invention.  We believe this patent significantly strengthens our intellectual property portfolio and covers the use of the FIX-Padua variant in AAV gene therapy to treat hemophilia B.”

Trial data released in January showed that AMT-060 is to be safe and effective in treating severe and moderately severe hemophilia B for up to two years. Overall, adult patients required fewer doses of replacement therapy and showed a marked decrease in spontaneous bleeding rates.

These results provided a basis for uniQure’s also advancing another investigational gene therapy, AMT-061, for hemophilia B.  A Phase 2 clinical trial (NCT03489291) is set to begin enrolling three severe and moderately severe hemophilia B patients soon at two sites in the U.S.

AMT-061 and AMT-060 are identical therapies FIX-Padua except for a small variation in the gene sequence for FIX. AMT-061  has been designated a breakthrough therapy by the U.S Food and Drug Administration, and was given PRIME designation by the European Medicines Agency.

Like the FDA’s breakthrough therapy status, PRIME offers incentives to support the development of medicines targeting diseases with an unmet medical need.