Last Thursday, uniQure announced the immediate resignation of its CEO, Dan Soland. After only 9 months on the job, this comes as a shock – Soland claims that his decision is solely due to family reasons.

uniqure logouniQure‘s position as a leader in gene therapy seems to be crumbling. Adding to troubles with product delivery, the company’s CEO has just bailed without warning. UniQure has announced that it won’t be hiring a new CEO until it finishes implementing a new corporate strategy. Matt Kapusta, its current CFO, will be the interim CEO for now.

The company generated a lot of hype with Glybera, a treatment for lipoprotein lipase deficiency (LPLD) that became the first gene therapy to ever be approved worldwide back in 2012. However, the high price of the treatment (€1M) and the difficulty of prescribing it meant that it has only been used once. In the long term, the price of Glybera is cost-effective, but a one-off payment of €1M is tough for most individual payers.

Pricing gene therapies can prove to be a difficult subject. These treatments have the potential to cure a disease for life in only one dose. This is awesome news for the patient, but for a pharma company, it means it won’t have a stable income. The development of Glybera cost uniQure a massive €100M, which will be tough to offset given that LPLD is a very rare disease affecting only 1 in 1 million people. This strategic flop coupled with the difficulties in replacing former CEO Jörn Aldag, who told us about the fantastic job he was doing this year at Refresh, place uniQure in rather troubled waters.

Figure 1.

Figure 1. Glybera set a record price of €1M in 2014

The focus of uniQure now is the development of a gene therapy for hemophilia B, a genetic blood clotting disorder that appears in 1 out of 20,000 male births. In July, the company announced successful results for their candidate AMT-060 in Phase I/II trials. As Kapusta commented last week, uniQure plans to advance this treatment into a pivotal trial to aim for early approval.

The future of the company seems to rely now on the results of their hemophilia treatment. With a target disease with higher prevalence and a new strategy in the works, the company may overcome the current downward trend of their stock price. Indeed, their stock has shot back up since the new appointment on Friday, so investors don’t seem too worried.

However, they have a strong competitor in Spark Therapeutics; so far its results in hemophilia clinical trials are better, so much so that its candidate, SPK-9001, has already attained FDA approval for accelerated development. How long will uniQure be able to hang on to its position at the top of gene therapy?

Featured image credit: Sergey Nivens/
Figure 1 credit: Ravital/

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  • It’s not Kaputsa (2x) but Kapusta. uniQure’s portfolio is broader than hemophilia B. Also contains programs for Sanfilippo B and Parkinson.

    • Thanks for the correction, it’s now updated. We know they have more programs but decided to focus on Hemophilia B because they emphasized their efforts into this program in the press release.