bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today reported business highlights and financial results for the second quarter ended June 30, 2015.
“The first half of this year was a period of exceptional progress for bluebird bio across all fronts, and we made significant strides toward our goal of delivering transformative gene therapy products to change the lives of patients,” said Nick Leschly, chief bluebird. “We gained general agreement from U.S. and E.U. regulators on the path forward for LentiGlobin in beta-thalassemia major, and presented early data in severe sickle cell disease that supports the potential for LentiGlobin to make a meaningful difference for these patients. We also spoke for the first time in greater detail about our strategy to build an independent, differentiated immuno-oncology business.”
- SICKLE CELL DISEASE (SCD) AND BETA-THALASSEMIA DATA PRESENTATION AT THE 20TH CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION – Presented positive data from two patients with beta-thalassemia major and the first patient with severe sickle cell disease ever treated with our LentiGlobin BB305 product candidate. As of May 2015, Subjects 1201 and 1202 with beta-thalassemia major remained transfusion-independent for 16 and 14 months, respectively. Subject 1204 with severe SCD demonstrated increasing HbAT87Q production at six months’ follow-up and was free of transfusions for more than three months. At the six-month visit post-drug product infusion, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) in the patient with SCD accounted for 45 percent of all hemoglobin production. As of May 2015, the patient with SCD had no hospitalizations for sickle cell complications post-transplant, despite weaning of transfusions. LentiGlobin BB305 was well-tolerated, with no drug product-related adverse events observed as of the May 2015 data cut-off.
- LENTIGLOBIN BETA-THALASSEMIA GLOBAL REGULATORY STRATEGY – Announced plan to pursue conditional approval of our LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major in the E.U. through the Adaptive Pathways Pilot Program based on data from the ongoing Northstar and HGB-205 studies and plan to pursue accelerated approval in the U.S. based on our planned HGB-207 and HGB-208 studies. Completed NIH RAC review of HGB-207 and HGB-208 study protocols in adult and adolescent patients with beta-thalassemia major and pediatric patients with beta-thalassemia major, respectively.
- IMMUNO-ONCOLOGY STRATEGY – Announced strategy to build a broad T cell-based immuno-oncology portfolio based on our immuno-oncology, gene therapy clinical development and lentiviral vector manufacturing expertise and genome editing capabilities. Revised Celgenecollaboration to focus exclusively on anti-BCMA product candidates in multiple myeloma, initiated a strategic collaboration with Kite Pharma focused on second-generation T cell receptor (TCR) therapies in HPV-associated cancers and entered into an exclusive license agreement with Five Prime Therapeutics around chimeric antigen receptor (CAR) T cell therapies against an undisclosed cancer target for hematologic malignancies and solid tumors.
- CHIEF SCIENTIFIC OFFICER – Hired Philip Gregory, D. Phil., as Chief Scientific Officer. Formerly Chief Scientific Officer and Senior Vice President, Research at Sangamo BioSciences, Dr. Gregory brings extensive expertise in the fields of genome editing and gene therapy.
- STRENGTHENED BALANCE SHEET – Raised $477.2 million in net proceeds in an equity financing in June 2015. Our cash, cash equivalents and marketable securities are sufficient to fund our operations through 2018, based on the company’s current business plan. Proceeds from the equity financing will fund advancement of our immuno-oncology programs, development of a commercial infrastructure to support a potential conditional commercial launch of LentiGlobin in Europe, expansion of manufacturing capabilities to support ongoing and anticipated development and commercial efforts, and initiation of clinical studies of LentiGlobin in adult, adolescent and pediatric subjects with beta-thalassemia major.
Second Quarter 2015 Financial Results and Financial Guidance
- Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2015 were$936.4 million, compared to $492.0 million as of December 31, 2014, an increase of $444.4 million, which was primarily driven by the June 2015 equity financing.
- Revenues: Collaboration revenue was $4.9 million for the second quarter of 2015 compared to $6.3 million for the second quarter of 2014. Collaboration revenue is primarily comprised of the amortization of deferred revenue related to our collaboration agreement with Celgene.
- R&D Expenses: Research and development expenses were $44.3 million for the second quarter of 2015, compared to $13.9 million for the same period in 2014, an increase of $30.4 million. The increase in research and development expenses was primarily attributable to an$11.0 million increase in stock-based compensation expense, of which $8.5 million is non-recurring, a $10.7 million increase in one-time in-license milestones and fees, and an increase in expenses necessary to support the advancement of our clinical and pre-clinical programs.
- G&A Expenses: General and administrative expenses were $10.7 million for the second quarter of 2015, compared to $5.7 million for the same period in 2014, an increase of $5.0 million. The increase in general and administrative expenses was primarily attributable to a$3.5 million increase in employee- and contractor-related costs to support our overall growth.
- Net Loss: Net loss was $51.8 million for the second quarter of 2015, compared to net loss of$1.5 million for the second quarter of 2014.
- Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $936.4 million as of June 30, 2015 will be sufficient to fund its operations through 2018, based on the company’s current business plan. (Original Source)
Following the second-quarter update, shares of bluebird bio are trading at $155, down $11.51 or 6.91%. BLUE has a 1-year high of $197.35 and a 1-year low of $29.73. The stock’s 50-day moving average is $164.49 and its 200-day moving average is $138.43.
On the ratings front, bluebird bio has been the subject of a number of recent research reports. In a report issued on July 28, Piper Jaffray analyst Joshua Schimmer reiterated a Buy rating on BLUE, with a price target of $213, which implies an upside of 27.5% from current levels. Separately, on July 13, Roth Capital’s Debjit Chattopadhyay reiterated a Buy rating on the stock and has a price target of $192.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Joshua Schimmer and Debjit Chattopadhyay have a total average return of 11.5% and 31.4% respectively. Schimmer has a success rate of 61.3% and is ranked #248 out of 3727 analysts, while Chattopadhyay has a success rate of 64.9% and is ranked #17.
The street is mostly Bullish on BLUE stock. Out of 7 analysts who cover the stock, 7 suggest a Buy rating . The 12-month average price target assigned to the stock is $206.40, which represents a potential upside of 23.6% from where the stock is currently trading.
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.