Chiesi dumps uniQure’s hemophilia B gene therapy

Chiesi's research center in Parma, Italy

Chiesi has cut its ties to uniQure’s hemophilia B gene therapy. The split gives uniQure full rights to AMT-060 but leaves it without a partner to cofund R&D as it closes in on the start of a pivotal trial.  

Italian drugmaker Chiesi picked up the rights to commercialize AMT-060 in certain markets in 2013 as part of a deal that also gave it a piece of Glybera, the gene therapy that made history by coming to market in Europe only to flop commercially. Chiesi backed out of the Glybera agreement earlier this year and has now completed its split from uniQure by terminating the hemophilia B pact.

Amsterdam, the Netherlands-based uniQure framed the termination as it reacquiring the rights to AMT-060, rather than Chiesi dumping the program. But as the deal will see money transfer from Chiesi to uniQure and the former stated a shift in priorities prompted it to sever ties to AMT-060, it seems clear the Italian drugmaker wanted to exit the agreement.


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That leaves uniQure facing the prospect of taking AMT-060 into a pivotal trial without the financial support of a partner. Chiesi and uniQure have evenly shared R&D costs since 2013. The loss of the support of Chiesi will add $3 million to uniQure’s outlay this year, although the Dutch biotech still thinks it has enough cash to take it into 2019.

After a trying time on public markets dotted with stock drops following unfavorable comparisons to Spark Therapeutics’ rival hemophilia B program, uniQure is less well equipped to raise more money than in the past. But uniQure CEO Matthew Kapusta spun the regaining of full rights to the gene therapy as a boost for the company.

“We believe uniQure is better positioned to accelerate the global clinical development plan, maximize shareholder return on our pipeline and take advantage of new potential opportunities related to the program,” Kapusta said in a statement.

If the “potential opportunities” are to include a deal covering AMT-060, uniQure must persuade a potential partner of the merits of its asset. UniQure has sought to focus attention on the durable clinical benefits associated with AMT-060 but investors have fixated on Spark’s clear advantage in terms of Factor IX activity. 

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