Preliminary Data Available on SPK-8016 for Hemophilia A

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.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email