According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that are being developed to treat hemophilia. Furthest along is SPK-8011. Preliminary data from a Phase I/II clinical trial suggests that this investigational therapy could be effective in treating symptoms of hemophilia A. The company has also released information related SPK-8016, in development for treating hemophilia A in patients who have developed inhibitors to factor replacement therapy, and SPK-9001, which is in development for hemophilia B.
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.
Preliminary data indicates that SPK-8011 is capable of curtailing annual bleeds by an impressive 97 percent. The therapy is intended for one-time use and is delivered intravenously. Another good sign is that patients do no appear to have developed any antibodies against the therapy that could prevent it from helping them. There were three different dosing levels tested in the this trial and all three showed significant signs of improvement.
SPK-8016 will soon be tested in a Phase I/II clinical trial of its own. This trial is expected to involve 30 participants and will be similar in approach to the SPK-8011 trial.
Additionally, the company announced that it has officially transitioned SPK-9001 over to Pfizer as part of an agreement between the two companies. Pfizer will now continue the development of this treatment. Phase II data indicates that this experimental gene therapy can reduce annual bleeding by 98 percent in hemophilia B patients.