What happened

After big biopharma Astellas (OTC: ALPMY) (OTC: ALPMF) agreed to acquire clinical-stage gene therapy company Audentes Therapeutics (NASDAQ: BOLD) for $3 billion in cash, uniQure's (NASDAQ: QURE) shares have jumped 13% at 12:15 pm EDT on Tuesday.

So what

Although Audentes and uniQure are researching treatments for different indications, both companies are attempting to develop new solutions for genetic disorders by tinkering with genetic code.

Image source: Getty Images.

The potential to permanently fix genetic disorders by modifying a person's genetic makeup sounds like science fiction, but gene therapies have already won FDA approval, and optimism is high that additional gene therapies can also cross the regulatory finish line.

At uniQure, the most advanced gene therapy candidate is etranacogene dezaparvovec for hemophilia B, a blood clotting disorder characterized by an inability to produce clotting factor 9. Because hemophilia B patients are at high risk for life-threatening bleeding events, many require a lifetime of regular clotting factor infusions that can cost well into the six figures annually.

The purpose of uniQure's etranacogene dezaparvovec is to stimulate production of the missing clotting factor by delivering a functional gene to a patient's liver cells. In phase 2b trials, three patients treated with etranacogene dezaparvovec achieved factor IV production of up to 54% of normal, with an average of 45% of normal, at the 36-week mark, removing the need for factor IV transfusions.

Long-term results are also encouraging. None of the five patients receiving a 2x1013 gc/kg dose in phase 1/2 trials has required routine factor IV replacement therapy for up to three years following treatment.

Now what

uniQure is in a footrace with Spark Therapeutics (NASDAQ: ONCE) to complete phase 3 studies that can support an FDA filing for approval. uniQure reported it has fully enrolled its phase 3 study in the third quarter of 2019, ahead of schedule, paving the way for data by the end of 2020. If that data is as strong as the phase 2b results, an application for approval could be filed in 2021.

Whether the potential to reshape hemophilia B treatment is enough to encourage a suitor to step up, as with Astellas' acquisition of Audentes, is anyone's guess. However, it's possible that uniQure's current $2.3 billion market cap doesn't accurately reflect its opportunity to disrupt the billion-dollar hemophilia B marketplace if it can beat Spark and others to the market.

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