Adding CAR-T to the shopping cart

How about some exciting, multi-billion dollar deals to spice up the first quarter? First, Sanofi acquired Bioverativ, a hemophilia-focused biopharmaceutical company that spun out of Biogen last February. Since losing patent protection, Sanofi has seen flagging revenue from their flagship Lantus products—which occupy the #4 and #15 spots on IQVIA’s list of Top Medicines by Invoice Spending—and they’re hoping Bioverativ can give their treatment portfolio a boost. Similarly, Celgene boosted their pipeline prospects by acquiring Juno Therapeutics, who have a promising CAR-T candidate expected to be FDA-approved in 2019. Celgene also recently bought Impact Biomedicines, all part of a strategy to preemptively address profit losses when their blood cancer drug Revlimid goes off-patent in a few years.

When blood clotting is a good thing

London researchers have made a rare breakthrough in a rare disease: hemophilia A. In the small-scale clinical study, patients received injections of a missing gene that promotes the production of the factor VIII clotting protein absent in these patients. Results reported in NEJM showed that six of the 7 patients in the high-dose cohort maintained normal levels of factor VIII after a full year. Also amazing, those in the high-dose cohort had an average of 16 bleeding events per year prior to therapy and this was reduced to 1 event per year after therapy. These findings are particularly significant because prior gene therapy studies have shown effectiveness only in hemophilia B sufferers, not hemophilia A, where a different clotting protein is the culprit.