A biotech company developing several cell and gene therapies for blood cancers and hereditary diseases plans to have data updates by the end of the year for several of its programs, though investment bank analysts were less certain of how much data would be released.
Cambridge, Massachusetts-based bluebird bio said in its third quarter 2019 earnings Thursday that it planned to provide clinical updates for its European Medicines Agency-approved gene therapy Zynteglo (autologous CD34-positive cells encoding betaA-T87Q-globin gene), the investigational gene therapy LentiGlobin and the CAR-T cell therapies bb21217 and bb2121 (idecabtagene vicleucel) in multiple myeloma by the end of this year. The EMA approved Zynteglo in June for transfusion-dependent beta-thalassemia.
Shares of bluebird rose more than 6 percent Friday morning on the Nasdaq following the earnings announcement.
The American Society of Hematology’s annual meeting will be in December in Orlando, Florida, and would seem a likely venue for the presentation of the data. However, Cowen analyst Yaron Werber wrote in a note to investors that there would not likely be data at ASH for the registration-directed Phase II KarMMa study of bb2121, also known as ide-cel, with the full data release more likely in the first half of next year. That surmise was based on bluebird’s earlier announcement that data from KarMMA would be updated by the end of this year, and as such the company may simply announce that the trial was successful, perhaps with some top-line data.
By contrast, Werber wrote, data from the trial of bb21217 likely will be presented at ASH. The therapy is a next-generation CAR-T targeting the antigen BCMA that uses the bb2121 chimeric antigen receptor molecule with a manufacturing process designed to improve CAR-T cell functional persistence.
Werber wrote that the data updates would be key catalysts to drive the company’s stock in 2020. Cowen also confirmed with Celgene – which has a development and commercialization partnership with bluebird – that bb2121 will most likely be filed for Food and Drug Administration approval in the first quarter of next year.
For Zynteglo, the company said it plans to start treating patients with transfusion-dependent beta-thalassemia in early 2020, and it continues to proceed with discussions on value-based payment agreements and qualified treatment center contracts. Werber wrote that it will likely be launched first in Germany, followed by Italy, France, the U.K. and the other E.U. countries. A rolling submission in the U.S. for the therapy will start by the end of this year. The company also anticipates that its Phase III study of LentiGlobin in sickle cell disease will be open and enrolling patients by the end of this year.
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