On February 23, 2021 bluebird bio, Inc. (NASDAQ:BLUE) reported financial results and business highlights for the fourth quarter and full year ended December 31, 2020 and shared recent operational progress (Press release, bluebird bio, FEB 23, 2021, View Source [SID1234575464]).
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"For over ten years, bluebird bio has been pioneering gene therapies for patients with rare diseases and cancer, and our work continues with the analysis of the recent safety events identified in our HGB-206 study in patients with sickle cell disease. We have seen the transformative benefit of these therapies and are conducting a scientific and medical investigation to best inform our path forward on behalf of the patients we hope to serve with LentiGlobin," said Nick Leschly, chief bluebird. "While this work is ongoing, we remain focused on the upcoming PDUFA action due date for ide-cel for multiple myeloma next month, the potential EU approval of eli-cel for CALD mid-year and the planned US filings for β-thalassemia and CALD later in the year. Through these challenges, we are keeping patients at the center and were very excited to reach a major milestone of the first commercial infusion of ZYNTEGLO in Germany earlier this month."
RECENT HIGHLIGHTS
COMPANY
CLINICAL HOLD OF PHASE 1/2 AND PHASE 3 STUDIES OF LENTIGLOBIN GENE THERAPY FOR SICKLE CELL DISEASE AND TEMPORARY SUSPENSION OF MARKETING OF ZYNTEGLO IN EUROPE – On February 16, 2021, bluebird bio announced that due to a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia and a SUSAR of myelodysplastic syndrome in our HGB-206 clinical study, the FDA has placed our clinical studies of LentiGlobin for SCD on clinical hold. We are investigating these events and plan to continue to work closely with the FDA in their review of these events. In addition, we are also engaged with the EMA in discussions regarding our proposed development plans for LentiGlobin for SCD in Europe. In light of these SUSARs, we have temporarily suspended marketing of ZYNTEGLO in Europe. Additionally, the EMA has paused the renewal procedure for ZYNTEGLO’s conditional marketing authorization while the EMA’s pharmacovigilance risk assessment committee reviews the risk-benefit assessment for ZYNTEGLO and determines whether any additional pharmacovigilance measures are necessary.
INTENT TO SEPARATE – On January 11, 2021, bluebird bio announced its intent to separate its severe genetic disease and oncology businesses into two independent, publicly traded companies (bluebird bio and "Oncology Newco"). The separation is expected to be completed by year-end 2021 and it is anticipated that the spin out of Oncology Newco is to be tax-free to shareholders, subject to receipt of a favorable Internal Revenue Service (IRS) ruling.
Following the separation, bluebird bio intends to focus on delivery of its Core 3 therapies in β-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease, expand access and reimbursement for our commercial product, ZYNTEGLO, in Europe and continue to explore innovative tools and technologies to ultimately bring these transformative medicines to more patients.
Following the separation, Oncology Newco plans to support commercial success of ide-cel and continued development of bb21217, deliver on the oncology pipeline of cellular therapies with a focus on non-Hodgkin’s lymphoma, acute myeloid leukemia, next-generation multiple myeloma and solid tumors and advance next generation product cycling engine with an overarching goal of 1-2 investigational new drugs (INDs) in each of the years 2021 and 2022.
At the time of separation, bluebird bio plans to capitalize each business with sufficient capital to achieve value creating milestones and intends to provide additional financial details closer to the date of separation.
TRANSFUSION DEPENDENT β-THALASSEMIA
FIRST PATIENT TREATED WITH ZYNTEGLO GENE THERAPY – bluebird bio is announcing today that a patient was treated earlier this month with the first commercial infusion of ZYNTEGLO [betibeglogene autotemcel (beti-cel)]. The patient was treated at a Qualified Treatment Center (QTC) in Germany.
TDT DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio presented new data showing that all patients that achieved transfusion independence continue to remain free from transfusions up to six years in the ongoing long-term follow-up study (LTF-303) of beti-cel gene therapy in patients with TDT at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The company also presented updated efficacy and safety results for pediatric patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies.
SICKLE CELL DISEASE
FIRST PATIENT TREATED IN CONFIRMATORY HGB-210 STUDY –bluebird bio is announcing today that within the first quarter, the first patient was infused in HGB-210, a Phase 3 confirmatory study of LentiGlobin gene therapy (bb1111) for adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).
SCD DATA AT ASH (Free ASH Whitepaper) – On December 7, 2020, bluebird bio presented new data showing a complete elimination of severe vaso-occlusive events (VOEs) through 24 months of follow-up in patients who had a history of at least four severe VOEs and at least six months follow-up in Group C of its ongoing Phase 1/2 HGB-206 study of bb1111 for patients with SCD at the 62nd ASH (Free ASH Whitepaper) Annual Meeting.
MAGENTA COLLABORATION – On December 4, 2020, bluebird bio and Magenta Therapeutics announced an exclusive clinical trial collaboration to evaluate the utility of MGTA-145, in combination with plerixafor, for mobilization and collection of stem cells in patients with SCD. The combination approach has the potential to achieve safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to ultimately improve outcomes associated with stem cell transplantation. The companies will co-fund the clinical trial and Magenta will retain all rights to its product candidate.
MULTIPLE MYELOMA
CRB-401 DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio and Bristol-Myers Squibb presented longer-term results showing ongoing deep and durable responses in the original Phase 1 study (CRB-401) of the companies’ investigational B-cell maturation antigen (BCMA) directed chimeric antigen receptor (CAR) T cell therapy, idecabtagene vicleucel (ide-cel), in patients with relapsed and refractory multiple myeloma at the 62nd ASH (Free ASH Whitepaper) Annual Meeting.
BB21217 DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio and Bristol-Myers Squibb presented updated safety and efficacy results showing promising response rates and durability from the ongoing Phase 1 study (CRB-402) of bb21217, an investigational BCMA-directed CAR T cell therapy in patients with relapsed and refractory multiple myeloma at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. The company announced the study has completed enrollment and follow-up is ongoing as data continue to mature and the durability of response at the recommended phase 2 dose is assessed.
UPCOMING ANTICIPATED MILESTONES
Regulatory Outlook
Multiple Myeloma: The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021 for the approval of ide-cel (bb2121) in patients with relapsed and refractory multiple myeloma.
SCD: The company is investigating the recently-reported safety events and plans to continue to work closely with the FDA in their review of these events to provide an update on the Company’s development plan and timeline for submission for regulatory approval.
TDT: The company is on track to complete its rolling BLA submission to the U.S. FDA for beti-cel in mid-2021, contingent upon successful resolution of any U.S. FDA concerns applicable to the program arising out of the recently-reported safety events in the SCD program. This submission is anticipated to include patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
CALD: The company is on track to complete its BLA submission to the U.S. FDA for elivaldogene autotemcel (eli-cel) in mid-2021. The company plans to receive European approval for eli-cel in patients with cerebral adrenoleukodystrophy (CALD) in mid-2021.
Clinical Updates and Milestones
Updated data from ongoing clinical study in patients with SCD by the end of 2021.
Updated data from ongoing clinical studies in patients with TDT in mid-2021.
Updated data from ongoing clinical studies in patients with CALD in mid-2021.
bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2021.
Submission of 1 – 2 investigational new drug (IND) applications by the end of 2021.
FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2020 and December 31, 2019 were $1.27 billion and $1.24 billion, respectively. The increase in cash, cash equivalents and marketable securities is primarily a result of proceeds received from the May 2020 public offering of the Company’s common stock and a one-time upfront payment received in connection with the Company’s amended collaboration with BMS, partially offset by cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $10.7 million for the three months ended December 31, 2020 compared to $10.0 million for the three months ended December 31, 2019. Total revenues were $250.7 million for the twelve months ended December 31, 2020 compared to $44.7 million for the twelve months ended December 31, 2019. The increase for the three-month period was primarily driven by an increase in royalty and other revenue. The increase for the twelve-month period was primarily driven by the amended BMS collaboration and monetization for ex-U.S. milestones and royalties from ide-cel and bb21217, with the majority of the revenue recognized relating to ide-cel license and manufacturing services.
R&D Expenses: Research and development expenses were $137.1 million for the three months ended December 31, 2020 compared to $161.8 million for the three months ended December 31, 2019. Research and development expenses were $588.0 million for the twelve months ended December 31, 2020 compared to $582.4 million for the twelve months ended December 31, 2019. The decrease for the three-month period was primarily driven by a decrease in manufacturing costs, partially offset by increased costs incurred through the amended BMS collaboration. The increase for the twelve-month period was primarily driven by an overall increase in costs incurred to advance and expand the company’s pipeline, partially offset by a decrease in manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses were $77.0 million for the three months ended December 31, 2020 compared to $76.2 million for the three months ended December 31, 2019. Selling, general and administrative expenses were $286.9 million for the twelve months ended December 31, 2020 compared to $271.4 million for the twelve months ended December 31, 2019. The increase for both periods was largely attributable to increased headcount and costs incurred to support the Company’s ongoing operations and growth of its pipeline, partially offset by a decrease in costs related to commercial readiness activities due to delays during 2020 as a result of the COVID-19 pandemic.
Net Loss: Net loss was $199.9 million for the three months ended December 31, 2020 compared to $223.3 million for the three months ended December 31, 2019. Net loss was $618.7 million for the twelve months ended December 31, 2020 compared to $789.6 million for the twelve months ended December 31, 2019.