Sparks Gene Therapy For Hemophilia B Looks To Change Up The Game
Recently, Spark Therapeutics (ONCE) reported that it had achieved positive results from its phase 1/2 study using SPK-9001 to treat patients with Hemophilia B. The most notable item from this form of treatment was able to reduce routine infusions of Factor IX concentrates.
Recently, Spark Therapeutics (ONCE) reported that it had achieved positive results from its phase 1/2 study using SPK-9001 to treat patients with Hemophilia B. The most notable item from this form of treatment was able to reduce routine infusions of Factor IX concentrates. That means that SPK-9001 has the potential to change the way these patients are treated. I feel that Spark has been doing well with this clinical product, and thus the reason why I remain bullish on Spark after the latest results.
Phase 1/2 Data
This phase 1/2 study recruited a total of 15 patients with Hemophilia B who were treated with SPK-9001. The most notable item that was revealed was that patients had discontinued routine infusions of Factor IX. It is imperative that these patients get a routine infusion. That’s because without Factor IX, they lack the ability for normal clotting. That’s important, because otherwise bleeding episodes last for a long period of time. For the first 10 patients from week 12 through week 52 of follow up it was shown that Factor IX activity level ranged from 14.3% to 76.8 %. In the other three patients, the Factor IX activity level ranged from 38.1% to 54.5%. I must say that after treatment with SPK-9001 these percentages are good. That’s because in order for Hemophilia B patients to have Normal plasma levels of FIX they must be at 50% or higher. However, that doesn’t mean those who are slightly under have the severe form of Hemophilia B. This is the breakdown of Hemophilia B severity:
- Mild Hemophilia B - This is where FIX in the blood stands in the range of 6% to 49% - This is where bleeding only occurs after a severe injury or trauma to the body. Other than that, the bleeding is not that bad in this situation.
- Moderate Hemophilia B - This is where FIX in the blood stands in the range of 1% to 5% - This is where bleeding occurs on every injury, and it is also the point of where spontaneous bleeding happens as well
- Severe Hemophilia B - This is where FIX in the blood stands in the range of < 1% - This is where an injury always causes bleeding, and in this case frequent spontaneous bleeding episodes especially in the joints and muscles occur
Reference:https://seekingalpha.com/article/4177706-sparks-gene-therapy-hemophilia-b-looks-change-game
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