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Martijn de Meulder
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Leiden
The clinical study of the Leiden ProQR into an advanced therapy for an eye condition shows that the drug has no effect. It’s a drama for the biotech company that loses its key asset in development. The news caused the company to lose three quarters of its stock market value on Friday.
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“Today is a sad day for everyone involved in this program,” said ProQR founder and CEO Daniël de Boer during an analyst meeting. “These results are unexpected and disappointing, especially for patients with this condition.” He emphasizes that the company will continue its investigation, but the message will not get through to investors in his Nasdaq-listed company on Friday. The share price collapses by more than three quarters. On Thursday, ProQR was worth almost four hundred million euros, Friday it was less than ninety million euros.
ProQR has been around since 2012 and in recent years has mainly worked on a therapy called Sepofarsen from Leiden and Cambridge in the United States. It should help patients suffering from LCA10 – a rare degenerative eye disease that leads to blindness. But the second- and third-phase clinical efficacy studies now show that it doesn’t help patients. The development of the drug has therefore most likely failed.
That is a heavy blow, also because a similar therapy from competitor Spark did yield results a few years ago. Spark was bought in 2019 by the Swiss pharma giant Roche for four billion euros. Sepofarsen promised to be an improvement on that therapy, especially because it would become more affordable.
Incidentally, this failure does not mean the end of the company located on the Zernikedreef in the Bio Science Park. ProQR still has about two hundred million euros in cash, according to De Boer enough to continue until mid-2024 before he has to raise money from investors again: “We are now going to thoroughly investigate what happened in this study. After that, we will continue to develop our other therapies.” ProQR is working on treatments for three other eye diseases. He wants to develop it further in the coming years, together with its RNA processing platform.
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