Brief thoughts and impressions from the American Society of Hematology (ASH) annual meeting.
1. San Diego weather is really, really, really nice, especially since I hear it's snowing in Boston.
2. Spark Therapeutics (ONCE) won the weekend battle in hemophilia B gene therapy. The highly active Padua variant used in Spark's gene therapy delivered impressive factor IX activity levels that look durable out to seven months and up to one year in a tiny number of patients. The two patients with immune reactions will be watched closely, particularly as the course of steroids used to treat them are stopped. Factor IX is a nice biomarker but patient outcomes -- stopping bleeds and lifting need rescue rescue Factor IX replacement -- is what really matters.
3. Uniqure's (QURE) enterprise value of $15 million means nobody is giving the company credit for its hemophilia B gene therapy, even though the data presented this weekend were generally fine. Sure, Uniqure's FIX numbers from a less active wild-type gene didn't look as visually impressive as what Spark showed, but the Uniqure patients had more severe baseline hemophilia and still showed meaningful reductions in bleeds and less use of rescue Factor IX. They're turning severe hemophiliacs into mild hemophiliacs, so what's that worth? The Street likes to pick immediate winner and losers, but with hemophilia gene therapy still in early innings, don't be surprised to see wild swings in sentiment.
4. Celgene (CELG) , per usual, does good ASH meeting. BCMA is the hot new immunotherapy target for multiple myeloma, and as you'd expect, Celgene is all over it with various approaches, including Bluebird Bio's (BLUE) CAR-T. The partnership with Acceleron Pharma (XLRN) to expand development of luspatercept into new MDS patient groups and myelofibrosis seems important but underappreciated. Revlimid still rules.
5. Global Blood Therapeutics (GBT) continues to show consistent and durable hemoglobin responses to GBT440 with 700 mg and 900 mg doses in the phase II sickle cell disease study. Safety looks clean, most importantly with no hypoxia signal. The first patients in the well-designed phase III study will start in January. The development of the patient reported outcome (PRO) scale to assess overall benefit, and not just vaso-occlusive crises, in sickle cell patients (as a key secondary endpoint) is really smart drug development and will rally patient groups to the company's side. All good news, right? Well, Global Blood shares fall because nothing the company says or shows, data-wise, seems to be enough.
8. Sorry, if I didn't mention your favorite stock. I can't cover everything.