For the past two days, doctors who treat blood diseases have been meeting at the annual convention of the American Society of Hematology in Atlanta. There’s a lot of innovation coursing through this field. Here’s a guide to companies that are standing out.
Bluebird Bio Hits A Grand Slam
Researchers working with Cambridge, Mass.-based gene therapy startup Bluebird Bio presented three eye-catching studies: of a new kind of cancer-killing cell, called a CAR-T, that is targeted against multiple myeloma; of a gene therapy for beta thalassemia; and of another gene therapy for sickle cell anemia.
CAR-T’s are hot now – Novartis and Gilead Sciences are jockeying for position with their rival CAR-T products – but so far they are limited to types of leukemia and lymphoma. Bluebird’s bb2121, being developed with biotech giant Celgene, targets a protein called BCMA that is found on myeloma cells. Patients had been failed by up to seven previous drugs. Yet 17 of 18 saw their cancer retreat, and in 10, it seemed to disappear – known as a complete response or an unconfirmed complete response.
Results were equally encouraging for a new manufacturing process for Blubird’s treatment for beta thalassemia, another blood disease. All of three patients treated for more than six months were able to go without blood transfusions, and two had normal hemoglobin levels. But the most encouraging news may have been in sickle cell. Bluebird had generated excitement with early results for a single sickle cell patient – and then treatment of seven more patients didn’t show a similar benefit. Now, two patients treated with Bluebird’s new manufacturing process are showing equally positive results. Okay, guys. Let’s see seven more.
BioMarin's Gene Therapy For Hemophilia Impresses
Two different gene therapy products showed promise for potentially curing hemophilia A, the most common form of the genetic disease that causes uncontrolled bleeding. BioMarin, of Novato, Calif., is in the clear lead. Spark Therapeutics presented its own data this morning. A big controversy: how high should levels of clotting factor in the blood get? BioMarin is pushing for higher levels, even with its lower dose; Spark appears to be more conservative. Spark’s data also look volatile, and its timeline is behind BioMarin’s, making it one of the big stock market losers of the meeting, with its shares dropping 20% in early morning trading. Read about BioMarin here, and Spark here.
Roche Roars
The Swiss drug giant is introducing a major potential disruption to the hemophilia market – a new antibody called Hemlibra that mimics the function of the clotting factor VIII. Exactly how much of the hemophilia A market Hemlibra will eventually take is a big question for blood product companies like Shire, Novo Nordisk, and BioVerativ.
But Roche had another big win at the conference: one of the most eye-catching small cancer drug studies presented at the ASH meeting. Polatuzumab vedotin, an antibody-drug conjugate constructed with technology licensed from Seattle Genetics, was tested in patients with diffuse large b-cell lymphoma, the same disease for which Novartis and Gilead are developing CAR-T therapy. When added to the chemotherapy bendamustine and Rituxan, polatuzumab extended overall survival significantly – from 24% of patients at the end of a year for those that just got the standard drugs to 48% for those that received polatuzumab. CAR-T makers want to move earlier in DLBCL. But that could mean they have to compete with drugs like polatuzumab.
Juno Stays In The Game
Juno Therapeutics, which is also working with Celgene, fell behind Novartis and Kite when its lead CAR-T was torpedoed by side effects. Now its developing JCAR017, a mix of t-cells aimed at being safer and more effective. At the highest dose, 13 of 19 patients had a complete response, a 68% complete response rate that compares favorably to any other CAR-T. Of the 67 patients who received any dose, 1% had a severe immune reaction and 15% had neurotoxicity – which also counts as good for CAR-T. Juno says it's going to explore moving earlier in the disease.
BluePrint Prints A Great Number
BluePrint Medicines is one of several companies focused on rare cancer I included in a recent Forbes magazine story. Its avapritinib showed impressive results in systemic mastocytosis: a 72% overall response rate (meaning disease burden shrank) and a 100% disease control rate (the patients didn’t get worse). BluePrint said in a press release that it plans to start a study next year aimed at getting the drug approved. Its shares are up 25%.
