CSL buys gene therapy

Bill may reach $2.9b for haemophilia B medicine

BIOTECHNOLOGY giant CSL could potentially pay more than $US2 billion ($2.9 billion) for a gene therapy for haemophilia B, which may help people with the blood clotting disorder to stop bleeding.

US company uniQure produces the therapy called etranacogene dezaparvovec, or AMT-061, which could be one of the first to provide longterm benefits to people who suffer from the inherited disease.

The therapy is in phase three trials and promises to increase Factor IX plasma levels in the blood so that recipients have a greatly reduced tendency to bleed.

Haemophilia B can cause internal and external bleeding, and lead to death.

Patients would only need one therapy treatment, rather than on an ongoing basis.

CSL will pay $US450 million ($656 million) upfront to secure the exclusive global rights to commercialise the program.

It will also pay royalties and sales milestone fees.

UniQure in a statement said royalties would range up to about 20 per cent of sales, and the milestone fees would be worth up to $US1.6 billion.

Its chief executive, Matt Kapusta, (left) said the deal would help the business deliver the gene therapy to as many patients as possible.

UniQure will complete the phase three trial and boost manufacturing in order to supply CSL.

This is not the first time CSL has looked to the US to help its gene therapy business.

The company is developing a stem cell gene therapy to treat sickle cell disease and is working with the Seattle Children’s Research Institute.

CSL CEO and managing director Paul Perreault welcomed the acquisition.

“Upon approval, we believe this next-generation therapy would be highly complementary to our existing haemophilia B product portfolio,” he said.

“We hope that it provides patients with an alternate bestin-class treatment option, building on our legacy of delivering lifesaving innovations in haematology.

“Our vision with haemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease.

“With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximise the potential benefit of this therapy.”