On November 4, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the third quarter ended September 30, 2020 and shared recent operational progress (Press release, bluebird bio, NOV 4, 2020, View Source [SID1234569859]).

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"While 2020 continues to present unprecedented challenges, bluebird has continued to advance our innovative cell and gene therapy programs. Looking to 2021 and beyond, this is a catalyst-rich period for bluebird as we are on the cusp of multiple approvals in the U.S. and EU," said Nick Leschly, chief bluebird. "In the near term, we look forward to collaborating with FDA to find innovative approaches to help advance these complex therapies. We know that the role of a pioneer in science is never easy, but we are driven by the patients we hope to help. As always, I would like to thank our birds for their passion and resiliency, and for their relentless commitment to the people we serve. The way our employees continue to show up is incredibly inspiring."

RECENT HIGHLIGHTS

SICKLE CELL DISEASE

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – Today, bluebird bio announces confirmation of its general agreement with the U.S. Food and Drug Administration (FDA) that the clinical data package required to support a BLA submission for LentiGlobin for sickle cell disease (bb1111) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. bluebird bio is also announcing today that it has reached general agreement with FDA on its path to transition to commercial manufacturing using an analytical comparability strategy, including suspension-based lentiviral vector (sLVV). These developments meaningfully de-risk the bb1111 program and bring clarity on the path to approval. However, FDA requested the use of drug product manufactured from sickle cell disease (SCD) patient cells in addition to healthy donors as well as commercial lentiviral vector to demonstrate drug product comparability. Given this feedback, alongside COVID-19 related shifts and contract manufacturing organization COVID-19 impacts, bluebird is adjusting its submission timing to late 2022. The company looks forward to continuing to work with the Agency to find an innovative approach to reviewing the CMC portion of a BLA submission and address the high unmet need in sickle cell disease.
LENTIGLOBIN FOR SICKLE CELL DISEASE PRIME DESIGNATION – On September 23, 2020, bluebird bio announced that its investigational treatment for SCD, LentiGlobin for SCD gene therapy, was granted eligibility to the Priority Medicines (PRIME) program by the European Medicines Agency (EMA). The EMA’s PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly.
TRANSFUSION DEPENDENT β-THALASSEMIA

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – Today, bluebird bio announces that based on continued and ongoing discussions with the FDA in the context of bluebird bio’s Fast Track and Breakthrough Therapy designations, the company intends to seek approval for all patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes). The company remains on track to complete the rolling BLA submission for betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia) in mid-2021.
HGB-212 FINAL INFUSION – Today, bluebird bio announces the completion of treatment in the ongoing phase 3 Northstar-3 (HGB-212) clinical study of beti-cel in patients with transfusion-dependent β-thalassemia who have a β0/β0 genotype or IVS-I-110 mutation.
MULTIPLE MYELOMA

CRB-402 FINAL INFUSION – Today, bluebird bio announces the completion of treatment in the ongoing Phase 1 study (CRB-402) of bb21217, an investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy being studied in patients with relapsed/refractory multiple myeloma (R/RMM).
IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) ACCEPTANCE AND PRIORITY REVIEW – On September 22, 2020, bluebird bio and BMS announced that the U.S. FDA has accepted for Priority Review their BLA for idecabtagene vicleucel (ide-cel; bb2121), the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.
CEREBRAL ADRENOLEUKODYSTROPHY

ELI-CEL MARKETING AUTHORIZATION APPLICATION (MAA) VALIDATION – On October 2, 2020, bluebird bio announced that the EMA has accepted its MAA for elivaldogene autotemcel (eli-cel, Lenti-D). Validation of the application confirms the submission is sufficiently complete to begin the EMA’s centralized review process.
ELI-CEL DATA AT EBMT – On August 29, 2020, bluebird bio presented new data suggesting durability of response and a strong safety profile post eli-cel gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 46th EBMT annual meeting. Long-term results from the Phase 2/3 Starbeam study of eli-cel, showed that eighty-seven percent of patients are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up and there were no reports of graft failure, graft rejection, or GVHD.
COMPANY

NEW BOARD APPOINTMENT – On August 11, 2020, bluebird bio announced the appointment of Denice Torres to its Board of Directors.
UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

SCD: The company plans to complete the BLA submission to the U.S. FDA for LentiGlobin for SCD in 2022.
TDT: The company is on track to complete a rolling BLA submission to the U.S. FDA for beti-cel in mid-2021. This submission will include all patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
Multiple Myeloma: The FDA has set a PDUFA goal date of March 27, 2021 for the approval of ide-cel (bb2121) in patients with relapsed and refractory multiple myeloma.
CALD: The company is on track to complete the BLA submission to the U.S. FDA for eli-cel in mid-2021.
Clinical

Today, bluebird bio announces its intention to present bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2020.
bluebird bio plans to present ide-cel clinical data from the ongoing CRB-401 study in patients with multiple myeloma by the end of 2020, in partnership with BMS.
bluebird bio plans to present updated data from the ongoing HGB-206 clinical study in patients with SCD by the end of 2020.
Commercial and Foundation Building

ZYNTEGLO first commercial patients treated in Europe by the end of 2020.
THIRD QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2020 and December 31, 2019 were $1.44 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 $19.3 million for the three months ended September 30, 2020 compared to $8.9 million for the three months ended September 30, 2019. Total revenues were $240.0 million for the nine months ended September 30, 2020 compared to $34.7 million for the nine months ended September 30, 2019. The increase for the three month period was primarily driven by an increase in ide-cel license and manufacturing services revenue and an increase in research and development revenue under our agreement with BMS. The increase for the nine months period was primarily driven by the recent 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 $140.4 million for the three months ended September 30, 2020 compared to $151.4 million for the three months ended September 30, 2019. Research and development expenses were $450.9 million for the nine months ended September 30, 2020 compared to $420.6 million for the nine months ended September 30, 2019. The decrease for the three month period was primarily driven by a decrease in manufacturing costs. The increase for the nine month period was primarily driven by an overall increase in costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $68.0 million for the three months ended September 30, 2020 compared to $66.3 million for the three months ended September 30, 2019. Selling, general and administrative expenses were $209.9 million for the nine months ended September 30, 2020 compared to $195.2 million for the nine months ended September 30, 2019. The increase for both periods was largely attributable to costs incurred to support the Company’s ongoing operations and growth of its pipeline.
Net Loss: Net loss was $194.7 million for the three months ended September 30, 2020 compared to $206.0 million for the three months ended September 30, 2019. Net loss was $418.8 million for the nine months ended September 30, 2020 compared to $566.3 million for the nine months ended September 30, 2019.
CONFERENCE CALL DETAILS

bluebird bio will hold a conference call to discuss business updates and third quarter 2020 financial results on Wednesday, Nov 4 at 4:30PM ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 545-6725

To access the live webcast of bluebird bio’s presentation, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source Replays of the webcast will be available on the bluebird bio website for 90 days following the event.