Listed on the NASDAQ and with market cap of US$1.3 billion, Dutch company uniQure (QURE) is a global leader in gene therapy. Eighteen months ago and at a price of US$450 million in cash upfront, CSL made a major push into the gene therapy space, acquiring the exclusive global licence rights from uniQure to commercialise a treatment for haemophilia B.
“A 54 patient study successfully achieved a secondary endpoint demonstrating statistical superiority in reduction of annualised bleeding rate compared to baseline,” Raymond said.
“Patients also demonstrate durable, sustained increases in FIX activity at 18 months post-infusion.
“CSL is responsible for up to US$1.6 billion in regulatory and commercial milestones as well as royalties on net sales (we estimate 15-25%).
“It is also responsible for clinical development and regulatory activities (we estimate <US$50m).
“The company plans to submit regulatory applications in the US and EU next year, with potential market entry 2023.
“Our view is that it’s great to see one of the largest and first pivotal trial of a gene therapy for patients with haemophilia B report promising results, following prior interim analyses, offering the potential of a long-term treatment option for patients living with the disease.
“However, CSL has yet to fully flush out its commercialisation strategy (as well as pricing) and highlighted at its recent R&D day that a rapid uptake is unlikely given “the need for substantial educational and promotional requirements”,” Raymond said.
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