According to ForexTV, commercial gene therapy company Spark Therapeutics (“Spark”) recently announced preliminary data from a Phase 1/2 clinical trial evaluating SPK-8016, an investigational gene therapy, for patients with hemophilia A. In addition to determining the safety, efficacy, and tolerability of SPK-8016, the trial is also seeking to identify an appropriate dose. Patients enrolled in the trial are adult males.
SPK-8016
Developed by Spark, SPK-8016 is a novel gene therapy to help patients with no FVIII inhibitors or history of these inhibitors. The data, presented at the 2021 EAHAD Virtual Congress, followed 4 participants with hemophilia A. Participants received a singular 5X1011 vg/kg dose of SPK-8016. Data from the study includes:
- The drug was relatively safe and well-tolerated, with no serious side effects or complications reported.
- None of the 4 patients experienced inhibitor development during or following the trial.
- SPK-8016 reduced the annual infusion rate by 98% and the annual bleeding rate by 85%.
- While following up with patients, many experienced stable and long-lasting Factor VIII activity.
- Patients not taking any other therapies experienced the best response to SPK-8016.
- While being treated, no patients had heightened ALT or AST levels, suggesting that SPK-8016 is not hepatotoxic.
Hemophilia A
Also known as Factor VIII deficiency or classic hemophilia, hemophilia A is an inherited bleeding disorder caused by deficient Factor VIII clotting protein. Because they lack this blood clotting factor, patients with hemophilia A often do not have their blood clot effectively. Thus, bleeding is often excessive, intense, and long-lasting. Because hemophilia A is inherited in a recessive fashion, it mostly affects males. An estimated 1 in every 5,000 births has hemophilia A. Symptoms include:
- Easy bruising and bleeding
- Excessive menstruation or bleeding after birth (if experienced in females)
- Joint pain and inflammation
- Frequent nose bleeds
- Unexplained irritability (in infants)
- Bloody urine or stool caused by digestive and urinary tract bleeding
- Excessive bleeding after trauma, injury, dental work, or surgery
- Muscle pain
Learn more about hemophilia A.
