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therapy’s effects will last, or whether someone would be eligible for a second treatment if the first one wears off. No safety flags have emerged other than mild immune responses that a short course of steroids can reverse, but side effects from previous generations of gene therapy will likely make investors more skittish than for more established types of medicine.
Significant improvements in more standard types of treatment could also undercut the medical and financial arguments for an expensive, one-time cure. For example, Roche’s Genentech division last month won FDA approval of emicizumab (Hemlibra) for hemophilia A patients whose immune systems reject other medicines—about 20 percent of the hemophilia A population.
Emicizumab could soon be approved for the rest of the hemophilia A population, as well. emicizumab is easier to take, via a subcutaneous injection, than other drugs that require intravenous injection or infusion. And it might work with only one dose a month, compared with standard treatments that require multiple times a week.
Stock Gainers and Losers
The ASH meeting is always a volatile time for biotech stocks. Here are some of the biggest winners and losers coming out of the weekend.
—Spark shares fell 35 percent on the update to its hemophilia A program, as noted above, while BioMarin shares climbed almost 7.5 percent.
—A major question hanging over Bluebird Bio (NASDAQ: BLUE) is whether changes to its manufacturing process would lead to better results in beta-thalassemia and sickle cell disease. So far, so good. At ASH, Bluebird reported early results in a handful of patients suggesting the improvements might be helping. Between its updates in myeloma and other blood diseases, Bluebird shares climbed 18 percent Monday.
—Shares of Blueprint Medicines (NASDAQ: BPMC) climbed almost 23 percent on updates from two Phase 1 studies. At ASH, the company presented data from a study of BLU-285 (avapritinib) in advanced systemic mastocytosis, an abnormal buildup of mast cells, in which 13 of 18 evaluable patients have responded so far. At a separate scientific meeting in Hawaii, Blueprint updated a stomach cancer study in which 5 out of 18 patients responded to BLU-285.
—Seattle Genetics (NASDAQ: SGEN) reported full Phase 3 data for its flagship drug brentuximab vedotin (Adcetris) in first-line classical Hodgkin lymphoma. Investors initially sent the firm’s stock down 10.5 percent Monday amid worries that the generally positive data hid problems in certain key subgroups of patients. Sea-Gen also updated early-stage data from a combination of Adcetris and nivolumab (Opdivo) from Novartis, with a 62 percent complete response rate in 62 patients. The partners have already launched a Phase 3 study in HL patients who have relapsed or haven’t responded to other treatments. Sea-Gen shares closed down more than 6.5 percent Monday.
—Shares of Syros Pharmaceuticals (NASDAQ: SYRS) initially fell almost 50 percent on disappointing data from a Phase 2 trial of experimental SY-1425. Syros has been developing the drug for a genetic subset of patients with acute myeloid leukemia or myelodysplastic syndrome anticipated to be likely to respond to treatment. But just one out of 48 evaluable patients have responded so far. Syros shares regained less than half their losses by market close Monday.
[Updated with end-of-day stock prices.]