bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today reported business highlights and financial results for the fourth quarter and full year ended December 31, 2015.
“In 2015 bluebird bio defined an accelerated regulatory path for LentiGlobin in ß-thalassemia and established a powerful reason to believe in LentiGlobin in sickle cell disease, though there is still more to learn as we treat additional patients. We also fully enrolled our Starbeam study of Lenti-D in cerebral adrenoleukodystrophy and made significant advances in building a competitive T cell oncology franchise,” said Nick Leschly, chief bluebird. “In 2016 we are excited to learn even more across all of our programs and continue to innovate and improve. We are particularly looking forward to sharing data from the Starbeam study for the first time and presenting more data with longer follow-up from all three of our LentiGlobin clinical studies.”
- ADVANCED FIRST ONCOLOGY PROGRAM INTO THE CLINIC – Earlier in February, the first patient was infused in the CRB-401 study of bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene has exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies. bluebird bio will receive a $10.0 million option exercise payment from Celgene and is also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.
- PRESENTED UPDATED CLINICAL DATA IN ß-THALASSEMIA FROM HGB-204 AND HGB-205 CLINICAL STUDIES OF LENTIGLOBIN AT ASH – In December 2015, investigators presented data from bluebird bio’s ongoing clinical studies in transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD). Data in patients with TDT from the HGB-204 and HGB-205 studies showed that 100% of patients with non-ß0/ß0 genotypes achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 months to 23.4 months. Patients with the ß0/ß0 genotype all saw reductions in their transfusion needs, ranging from a 33% to 100% reduction.
- PRESENTED CLINICAL DATA IN SICKLE CELL DISEASE FROM HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN AT ASH –Marina Cavazzana, M.D., Ph.D., of Hospital Necker, University Paris Descartes, presented updated data on one patient with SCD from the HGB-205 study, who remained free of transfusions, hospitalizations and acute SCD-related events for more than nine months as of the data cut-off. At the 12-month post-drug infusion follow-up, the proportion of anti-sickling hemoglobin in this patient accounted for 49% (47% HbAT87Q+ 2% HbF) of all hemoglobin production – well above the 30% threshold anticipated to achieve a disease-modifying effect. John Tisdale, M.D., of the National Institutes of Health presented early data from the HGB-206 study, in which two patients had at least three months of post-infusion follow-up. At the three-month post-infusion follow-up for Subject 1301, anti-sickling hemoglobin accounted for 17% of all hemoglobin production (4% HbAT87Q + 13% HbF). At the six-month post-infusion follow-up for subject 1303, anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12% HbAT87Q + 4% HbF).
- PRESENTED PRE-CLINICAL AND MANUFACTURING DATA FROM CAR T ONCOLOGY PROGRAMS AT ASH – bluebird bio scientists presented three posters at ASH, covering critical basic research, translational and manufacturing aspects of the Company’s T cell oncology programs. One poster discussed an important observation made by bluebird bio scientists: culturing anti-BCMA CAR T cells with a PI3K inhibitor generated a product with many of the properties of younger, less differentiated T cells. Consistent with a younger T cell phenotype, this product showed improved in vivo efficacy and persistence in multiple model systems.
- SHARED DATA ON PLATFORM IMPROVEMENTS – In an investor event at ASH, bluebird bio chief scientific officer Philip Gregory, D.Phil., presented data from ongoing research to improve the cell transduction process for LentiGlobin. The presentation showed that in preclinical experiments, adding selected compounds to the transduction process resulted in substantially increased vector copy number and transduction efficiency (i.e. percentage of corrected cells). Importantly, the new process was shown to be robust with similar improvements seen across multiple donors and vector lots.
- ENTERED INTO CAR T LICENSE WITH VIROMED – Signed exclusive license agreement withViromed Co., Ltd., to research, develop and commercialize CAR T therapies using Viromed’s proprietary humanized antibody to an undisclosed cancer target in solid tumors.
Upcoming Anticipated Milestones
- Presentation of interim data from the Starbeam study of Lenti-D in patients with cerebral adrenoleukodystrophy (CALD) at the American Academy of Neurology annual meeting in April 2016
- Update on LentiGlobin process improvements in the second half of 2016
- Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016
- Presentation of updated data from the HGB-204, HGB-205 and HGB-206 studies at the ASH annual meeting in December 2016
Fourth Quarter and Full Year 2015 Financial Results and Financial Guidance
- Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2015were $865.8 million, compared to $492.0 million as of December 31, 2014, an increase of$373.8 million, which was primarily driven by the June 2015 equity financing partially offset by cash used to fund operations.
- Revenues: Collaboration revenue was $1.5 million for the fourth quarter of 2015 and $14.1 million for the year ended December 31, 2015, compared to $6.3 million and $25.0 million in the comparable periods in 2014. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.
- R&D Expenses: Research and development expenses were $35.7 million for the fourth quarter of 2015 and $134.0 million for the year ended December 31, 2015, compared to $20.5 millionand $62.6 million for the comparable periods in 2014. The increase in research and development expenses was primarily attributable to increased employee compensation expense due to increased headcount, in-licensing milestones and fees, and manufacturing and clinical trial-related costs to support our advancing pipeline.
- G&A Expenses: General and administrative expenses were $14.4 million for the fourth quarter of 2015 and $46.2 million for the year ended December 31, 2015, compared to $5.3 millionand $23.2 million for the comparable periods in 2014. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting and facilities-related costs to support our overall growth.
- Net Loss: Net loss was $47.3 million for the fourth quarter of 2015 and $166.8 million for the year ended December 31, 2015, compared to net loss of $19.5 million and $48.7 million for the comparable periods in 2014.
- Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $865.8 million as of December 31, 2015 will be sufficient to fund its current operations through 2018. (Original Source)
Shares of bluebird bio closed today at $52.37, up $0.91 or 1.77%. BLUE has a 1-year high of $197.35 and a 1-year low of $37.40. The stock’s 50-day moving average is $47.56 and its 200-day moving average is $82.75.
On the ratings front, bluebird bio has been the subject of a number of recent research reports. In a report issued on February 17, Roth Capital analyst Elemer Piros reiterated a Hold rating on BLUE, with a price target of $62, which implies an upside of 22.3% from current levels. Separately, on the same day, Leerink Swann’s Michael Schmidt reiterated a Buy rating on the stock and has a price target of $67.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Elemer Piros and Michael Schmidt have a total average return of -36.2% and -2.1% respectively. Piros has a success rate of 11.8% and is ranked #3627 out of 3664 analysts, while Schmidt has a success rate of 45.7% and is ranked #2565.
Overall, 4 research analysts have assigned a Hold rating and 6 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $103.00 which is 103.2% above where the stock opened today.
bluebird bio Inc is a clinical-stage biotechnology company. It is engaged in developing potentially transformative gene therapies for severe genetic and rare diseases and in the field of T cell-based immunotherapy.