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CSL licenses uniQure haemophila B gene therapy in $2bn-plus deal

Could free patients from the need for regulator injections with clotting factor drugs

Gene therapy graphic

Dutch biotech uniQure has agreed to license its gene therapy for haemophilia B – currently in a phase 3 registration trial – to CSL Behring for $450m upfront.

The deal – which also includes up to $1.6bn in back-end payments plus royalties – gives CSL global rights to AMT-061 (etranacogene dezaparvovec), which is among the lead candidates in the race to bring a gene therapy for haemophilia B to market.

Under the agreement, uniQure will be responsible for completing the ongoing HOPE-B study of AMT-061, and handling manufacturing of the gene therapy until it can be transferred to Philadelphia, US-based CSL, which is already a prominent player in the haemophilia market with its recombinant clotting factor products.

“Our vision with haemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said CSL’s chief executive Paul Perreault.

“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,” he added.

Last year, uniQure reported phase 2b results with AMT-061 – which aims to replace missing Factor IX in patients with haemophilia B – showing that the therapy raised levels of the clotting factor well above the level needed to reduce bleeding episodes.

If the HOPE-B trial backs up that initial finding with pivotal data on reduced bleeding, AMT-061 could provide a one-shot treatment that would free patients from the need for regulator injections with clotting factor drugs.

uniQure’s closest rival in the race to develop a haemophilia B gene therapy is Roche’s recent acquisition Spark Therapeutics with its SPK-9001 candidate, which is thought to be a few months behind in development, based on the timings of phase 3 readouts.

Roche is also emerging as a force in the haemophilia market thanks largely to the rapid growth of its blockbuster Hemlibra (emicizumab) antibody for haemophilia A.

Meanwhile, Pfizer is also jostling for a podium position in the haemophilia B gene therapy race with its PF-06838435 (fidanacogene elaparvovec), licensed from Spark ahead of the Roche takeover, which is also in phase 3 testing.

In a statement, uniQure said the deal with CSL will “provide…significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington’s disease”. The latter therapy was dosed to the first patients in a clinical trial earlier this week.

Article by
Phil Taylor

25th June 2020

From: Regulatory

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