15 December 2017 No. 3884 Scripscrip.pharmaintelligence.informa.com Pharma intelligence | informa Kymriah (tisagenlecleucel) and Gilead Sciences Inc.’s Yescarta (axicabtagene ciloleucel) for certain blood cancers – and Spark’s gene therapy Luxturna appears poised for approval in the near-term for a rare form of blindness, physicians are anxious to see more treatments that can move the bar closer to a cure. Investors too are rewarding drugs that can deliver the promise of big advances. Wall Street’s reaction to data presented at ASH also shows how important early clinical trial data in even a handful of patients can be a huge catalyst for companies working to bring big breakthroughs to small patient populations. Bluebird bio was handsomely re- warded by Wall Street after presenting Shutterstock: Ervin-Edward Shutterstock: positive data on multiple fronts. The com- pany’s LentiGlobin gene therapy helped patients with transfusion-dependent An ASH That Moves The Ball – β-thalassemia (TDT) achieve freedom from chronic transfusions in a Phase I/II study. The company also presented posi- And The Ticker Price tive updated results from a Phase I trial of JESSICA MERRILL
[email protected] bb2121, an anti-B-cell maturation anti- gen (BCMA) CAR-T cell therapy partnered n a year of notable advances in blood can- 29.4% to open at $221.55 and Blueprint with Celgene Corp., in 21 patients with cer and gene therapy, the American Soci- Medicines Corp.’s stock gained 24.6% to relapsed/refractory multiple myeloma. In Iety of Hematology (ASH) annual meeting open at $89.54. another announcement, Phase I data in Dec.