What happened
Shares of ProQR Therapeutics (PRQR -0.49%) were down 31% Wednesday morning after the company reported 2022 full-year earnings and updated the status of its pipeline. The biotech stock is up more than 174% over the past year, but down more than 32% so far in 2023.
So what
ProQR is a clinical-stage biopharmaceutical company that focuses on RNA-based therapies to treat rare genetic diseases, especially inherited eye diseases. ProQR released its fourth-quarter and full-year earnings before the market opened on Wednesday, along with an update regarding its pipeline.
The biggest reason for the decline was the update, showing that two of its programs appear to be a little slow getting out of the gate. The company said AX-0810, targeting cholestatic disorders that cause a buildup of bile acids in the liver and can lead to liver failure, and AX-1412, to lower the chance of cardiovascular disease, are not expected to enter clinical development until late 2024 or early 2025. The company has eight programs in its pipeline but none in late-stage trials.
ProQR's financials were also a concern. The company, which doesn't have any revenue yet, said it had 94.8 million pounds (roughly $117 million) in cash, and it lost 64.9 million pounds in 2022, roughly $80.1 million. The company said that was enough to fund operations into 2026.
Now what
There's still a lot of enthusiasm for ProQR because its Axiomer RNA technology uses a cell's editing machinery, called ADAR, to make specific edits in RNA to reverse a mutation or modulate protein expression to treat diseases.
The biggest concern is the company's small cash position and slow development timeline. Fortunately, the company has a partnership with Eli Lilly and is actively looking for other partnerships to allow it to continue operations.
