A one-time hemophilia A treatment from Pfizer (PFE) and Sangamo Therapeutics (SGMO) showed promising results in a small, early-stage study, pointing to a possible future in which patients might have a handful of curative options for the rare bleeding disorder.
In an ongoing 10-patient trial, the treatment boosted the production of factor VIII, a protein that is key to forming blood clots and is lacking in people with hemophilia A, according to data presented Saturday. Three patients getting the highest dose — the one Pfizer and Sangamo plan to use in future trials — saw their factor VIII levels returned to normal, and bleeds were reduced to zero. A fourth patient was dosed too recently to draw any conclusions.
Pfizer and Sangamo have less than six months of data on patients who got the highest dose, and the treatment’s effects on factor VIII could wane over time. But the companies said their early results suggest the treatment could eventually compete with more advanced one-time therapies developed by BioMarin (BMRN) and Spark Therapeutics (ONCE).