Tweaks to bluebird bio's Gene Therapy Approach May be Working
Bluebird bio announced it will be presenting four oral presentations and seven posters at the 59th Annual Meeting of the American Society of Hematology (ASH).
Bluebird is making forays into the CAR-T immuno-oncology market, although it has yet to bring a product to market. Its late-stage candidates include Lenti-D and LentiGlobin. The company is often cited as a potential acquisition target, with a market cap of around $6.4 billion. It was most recently mentioned as a potential target of Celgene, should Celgene decide to go on a shopping spree.
The company’s presentations will focus on its LentiGlobin candidate in patients with transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). It will also focus on its bb2121 product candidate in patients with relapsed/refractory multiple myeloma.
“This year at ASH, we have the opportunity to share updated data across our clinical studies in severe genetic diseases and cancer, and to provide a look at some of the preclinical work that will inform the next phase of clinical development at bluebird,” said David Davidson, the company’s chief medical officer, in a statement. “The new data in sickle cell disease suggest that the changes made to the HGB-206 protocol and to our manufacturing process are having a favorable impact on the engraftment of the gene-modified stem cells. The two patients treated in Group B have consistently higher DP VCN and in vivo VCN than Group A patients, and patient 1313 has the highest Month 3 HbA(T87Q) level seen to date in the study. We plan to share updated clinical data on these patients at ASH. Additionally, we are very encouraged by the emerging profile of plerixafor mobilization in patients with sickle cell disease. Early data show a more favorable safety profile and substantial improvement in the collection of CD34+ cells compared to bone marrow harvest, suggesting that plerixafor may offer a more effective and less burdensome means to collect stem cells in patients with sickle cell disease.”
The company also released its third-quarter financial results today. In addition to outlining the ASH presentations, the company reported a cash position of $1.1 billion as of Sept. 30, 2017, compared to $884.8 million as of Dec. 31, 2016. Total revenue was $7.7 million for the quarter compared to $1.6 million from the third quarter of 2016. The increase is based on revenue recognition for the bb2121 license and manufacturing services deal with Celgene and for out-licensing with Novartis Pharma. In addition, in August, the U.S. Food and Drug Administration (FDA)approved Novartis’ Kymriah, and Bluebird expects to see royalty revenue beginning the fourth quarter of this year.
Research and development expenses were $61.5 million for the third quarter, down from $64 million for the same period in 2016. The decrease was attributed to less platform-related expenses related to a one-time $15 million upfront license payment expensed in the third quarter of 2016, and was partially offset by increased employee-related expenses caused by increased headcount.
“2017 has been a year focused on execution,” said Nick Leschly, whose title is “chief bluebird,” in a statement. “In January, we outlined a set of goals intended to bring us closer to our 2022 vision of becoming the gene therapy products company. I’m pleased to say that we have accomplished many of these goals.”
Those include data presentations at American Society of Clinical Oncology (ASCO), European Hematology Association (EHA) and in the New England Journal of Medicine, the first patient dosing in its Phase I multiple myeloma trial of bb2121, and moving the anti-BCMA CAR-T program, bb21217, into the clinic.