BioMarin just announced interim results from a continuing Phase 1/2 study for hemophilia A patients, testing a gene therapy known as BMN 270.
Hemophilia A is a rare bleeding disease that ultimately results from faulty blood clotting mechanisms. There are approved orphan drugs for using preventatively in patients, there is no known cure for hemophilia as of yet, which is of great need in the medical community.
BioMarin is trying to change this with BMN 270, as the therapy is a vector that is engineered to rehabilitate plasma concentrations of Factor VIII to a normal level. In doing so, BMN 270 essentially cures the patient of hemophilia A.
This phenomenon is being proven to work, and the interim results showed that patients dosed with BMN 270 had an increase in Factor VII and also experienced a significant reduction in number of bleeding episodes. This trend was observed in both patients given a small dose, and in those given a higher dose.
In fact, the results were so encouraging that the company is planning to begin a Phase 3 clinical program just later this year. This program will consist of 2 distinct Phase 3 studies, one with each dosage. They are scheduled to begin in the last few months of 2017 – approximately 100 patients will be enrolled.
Usually, there are requirements for patients to enroll in these clinical trials, and even after a therapy has been approved, not everyone meets the qualifications for treatment. However, with BMN 270, The President of Worldwide R and D at BioMarin, Dr. Hank Fuchs, said that the company expects the vast majority (90 percent) of total patients would qualify as eligible for BMN 270 treatment, since there is a low level of pre-existing immunity to AAV5.
