Shares of Spark Therapeutics, Inc. (NASDAQ: ONCE), a biotech developing gene therapies, fell hard this morning after the company reported hemophilia trial data that bodes well for rival BioMarin Pharmaceutical Inc. (NASDAQ: BMRN). Spark Therapeutics stock has fallen about 40% as of 11:01 a.m. EST on Monday.
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Spark's stock surged when SPK-8011 produced some encouraging data earlier this year. It's giving a lot of its gains back today after the company released data that suggests a potential competitor from BioMarin might have the upper hand from this point forward.
SPK-8011 is a one-time gene therapy that aims to help hemophiliacs produce necessary clotting factors. Although Spark's candidate, which it's developing in partnership with Pfizer (NYSE: PFE), helped knock back dangerous uncontrolled bleeds to essentially zero, investors are troubled by a spotty clotting factor production benefit.
In short, one patient receiving a high dose of Spark's SPK-8011 produced clotting factors as high as 12% of normal while a patient receiving a low dose produced clotting factors as high as 24% of normal.
BioMarin's candidate hasn't been measured in a head-to-head trial against Spark's so we need to take any comparisons with a big grain of salt. That said, the differences look pretty big. BioMarin's valoctocogene roxaparvovec (formerly BMN 270) helped three patients with a 48-week observation produce clotting factors at an average of 49% of normal.
While the future of Spark's fledgling hemophilia program seems even less certain now than it did a few days ago, the company could launch its first commercial-stage drug in the new year. The U.S. Food and Drug Administration is expected to announce an approval decision for the company's Luxturna as the first treatment for a rare cause of blindness in the weeks ahead.
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