On August 5, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2020 and shared recent operational progress (Press release, bluebird bio, AUG 5, 2020, View Source [SID1234562920]).
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"I am incredibly proud of the progress made at bluebird this quarter, and the way in which our employees have continued to execute on behalf of patients in the midst of an ongoing global pandemic," said Nick Leschly, chief bluebird. "It is especially gratifying that despite these challenges, we have continued to treat patients in our clinical studies at levels consistent with prior quarters. Within the quarter, we presented compelling clinical data across three of our core four programs: sickle cell disease, β-thalassemia, and multiple myeloma, and made important progress across all of our programs. In sickle cell disease, we reached an important milestone on our transition to commercial manufacturing process with the successful dosing of the first sickle cell patient using drug product manufactured with suspension-based lentiviral vector. The European launch of ZYNTEGLO continues to progress, with positive ongoing discussions with payers across Europe and we expect to treat our first commercial patients in the second half of this year. Additionally, our multiple myeloma program, partnered with BMS, continues to advance with our submission of the BLA to the FDA and BMS’ validated MAA submission in Europe. With this foundation and additional cash runway, we are confident in our ability to bring our core four programs to patients in the commercial setting by 2022 and grow our sustainable pipeline of transformative gene and cell therapies. All of this progress is made possible by our undaunted bluebirds, who have shown time and again their resourcefulness and ingenuity even under the most challenging of circumstances to bring our therapies to patients."
RECENT HIGHLIGHTS
SUSPENSION LVV MANUFACTURING FOR SCD – Today, bluebird bio announced that it has treated the first sickle cell patient with drug product manufactured with suspension-based lentiviral vector (sLVV). This process is intended to allow for larger scale and more efficient manufacturing of LVV. The company intends to submit data supporting the use of sLVV to the FDA as part of its submission for regulatory approval of LentiGlobin gene therapy for SCD in the second half of 2021.
ELI-CEL ACCELERATED ASSESSMENT – In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted an accelerated assessment to elivaldogene autotemcel (eli-cel, Lenti-D gene therapy) for the treatment of cerebral adrenoleukodystrophy (CALD). The company plans to submit a Marketing Authorization Application (MAA) to the EMA for eli-cel in 2020. Accelerated assessment reduces the timeframe for the EMA to review an MAA to 150 evaluation days rather than 210. The CHMP grants review under the accelerated assessment procedure if the medicinal product is of major interest for public health, especially from the point of view of therapeutic innovation.
NEW ZYNTEGLO QTC – Today, bluebird bio announced that it has contracted with a second qualified treatment center for ZYNTEGLO. The center, in Essen, Germany, is prepared to treat patients with β-thalassemia in 2020.
IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On July 29, 2020, bluebird bio and BMS announced the submission of their Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for idecabtagene vicleucel (ide-cel; bb2121), the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy. This submission provides further details on the Chemistry, Manufacturing and Controls (CMC) module to address the outstanding regulatory requests from the FDA in May 2020 following the original BLA submission.
SCD DATA AT EHA (Free EHA Whitepaper) – On June 12, 2020, bluebird bio presented new data showing a near elimination of sickle cell disease-related vaso-occlusive crises and acute chest syndrome in the phase 1/2 clinical study of bluebird bio’s LentiGlobin gene therapy for sickle cell disease at 25th EHA (Free EHA Whitepaper) Congress. The company plan to submit its BLA to the FDA based on an analysis of clinical data from this study using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint with at least 18 months of follow-up post-treatment with LentiGlobin for SCD. The company continues to plan to submit the U.S. BLA for SCD in the second half of 2021.
TDT DATA AT EHA (Free EHA Whitepaper) – On June 12, 2020, bluebird bio presented new data showing that the majority of evaluable patients across genotypes achieve transfusion independence and maintain it with near-normal hemoglobin levels in phase 3 Studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia) gene therapy presented at EHA (Free EHA Whitepaper) Congress. The company presented data from the Northstar-2 (HGB-207) clinical study of beti-cel in patients with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype and the 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.
IDE-CEL MARKETING AUTHORIZATION APPLICATION (MAA) VALIDATION – On May 22, 2020, BMS announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Applications (MAA) for ide-cel. Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
KARMMA DATA AT ASCO (Free ASCO Whitepaper) – On May 13, 2020, Bristol Myers Squibb (NYSE: BMY) and bluebird announced positive updated results from the pivotal, Phase 2 KarMMa study evaluating the efficacy and safety of the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, ide-cel, in patients with relapsed and refractory multiple myeloma. The data from this study formed the basis of recent regulatory submissions.
EXTENDED CASH RUNWAY – In June 2020, bluebird bio raised approximately $541.5 million in net proceeds through a public equity offering. bluebird bio anticipates that its cash, cash equivalents and marketable securities as of June 30, 2020, together with projected revenue generated under our collaborative arrangements and projected sales of products, will be sufficient to fund operations into 2023 based on the company’s current business plan.
UPCOMING ANTICIPATED MILESTONES
Regulatory
Submission of a Marketing Authorization Application to the European Medicines Agency for eli-cel in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical
Updated data presentation from HGB-206 clinical study in patients with SCD by the end of 2020.
Presentation of ide-cel clinical data from the CRB-401 study in patients with multiple myeloma in 2020, in partnership with BMS.
Updated data presentation from ALD-102 clinical study in patients with CALD by the end of 2020.
Commercial and Foundation Building
ZYNTEGLO first commercial patients treated in Europe in the second half of 2020.
ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
SECOND QUARTER 2020 FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2020 and December 31, 2019 were $1.60 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 $198.9 million for the three months ended June 30, 2020 compared to $13.3 million for the three months ended June 30, 2019. Total revenues were $220.8 million for the six months ended June 30, 2020 compared to $25.8 million for the six months ended June 30, 2019. The increase for both periods was primarily attributable to 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. Deferred revenue under our BMS collaboration will be recognized over time as the associated obligation to provide vector manufacturing through development is satisfied.
R&D Expenses: Research and development expenses were $156.3 million for the three months ended June 30, 2020 compared to $146.5 million for the three months ended June 30, 2019. Research and development expenses were $310.4 million for the six months ended June 30, 2020 compared to $269.2 million for the six months ended June 30, 2019. The increase in both periods was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $68.6 million for both the three months ended June 30, 2020 and June 30, 2019. Selling, general and administrative expenses were $141.9 million for the six months ended June 30, 2020 compared to $128.9 million for the six months ended June 30, 2019. The increase in the six month period was largely attributable to costs incurred to support the Company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $21.5 million for the three months ended June 30, 2020 compared to $195.8 million for the three months ended June 30, 2019. Net loss was $224.1 million for the six months ended June 30, 2020 compared to $360.2 million for the six months ended June 30, 2019.