On May 11, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2020 and provided an operational update (Press release, bluebird bio, MAY 11, 2020, View Source [SID1234557450]).

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"We remain grounded in our core values and priorities: our patients, our people, our community and our business," said Nick Leschly, chief bluebird. "This quarter, we successfully submitted the ide-cel BLA with our partners at BMS and I am pleased to announce today that we have alignment with FDA on our clinical data package and filing path for LentiGlobin for sickle cell disease, which accelerates our planned base case filing timeline into 2021. Additionally, we are amending our co-promotion/co-development agreement with BMS to enable both companies to focus their efforts on efficient commercialization of ide-cel and generate non-dilutive capital for our business. Lastly, after a rigorous review of all operational plans to reflect COVID-19 uncertainties and recent program shifts, we have prioritized our core four programs to drive ide-cel launch and three filings in 2021 for CALD, TDT and SCD and continue to drive forward high potential pipeline assets. This prioritization effort and operational review has led to significant efficiencies and spend reduction across our company and will extend our cash runway into 2022. The fundamentals of our business remain sound and our newly revised operating plan enables us to execute on the 2022 vision while putting us on a path towards financial sustainability. Our team remains excited and committed to our mission with the belief we will emerge from a very tough period in history stronger than ever to deliver for patients. Thank you birds!"

SCD REGULATORY PATH

Today, bluebird bio announced general agreement with FDA that the clinical data package required to support a BLA submission for LentiGlobin for sickle cell disease (SCD) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint and at least 18 months of follow-up post drug product infusion. Globin response will be used as a key secondary endpoint. The company anticipates additional guidance from FDA regarding the commercial manufacturing process, including suspension lentiviral vector. The company is planning to seek an accelerated approval and expects to submit the U.S. Biologics Licensing Application (BLA) for sickle cell disease in the second half of 2021. bluebird bio plans to present updated data from the HGB-206 study at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting.

Additionally, to enhance its strategic, clinical and commercial manufacturing platform, bluebird bio has entered into expanded relationships with two subsidiaries of Hitachi Chemical Co., Ltd. (Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma GmbH). These agreements will support late-stage and commercial drug product manufacturing of LentiGlobin for SCD in both the United States and Europe, expand commercial drug product manufacturing capacity for ZYNTEGLO (betibeglogene autotemcel) in Europe, and expand clinical and commercial manufacturing capacity for Lenti-D for cerebral adrenoleukodystrophy (CALD) in Europe.

FINANCIAL AND BUSINESS UPDATES

IDE-CEL ROYALTY MONETIZATION AND BCMA RELATIONSHIP AMENDMENT

Today, bluebird bio announced in a separate press release that it has amended its existing co-promotion/co-development agreement with Bristol Myers Squibb (BMS) to enable the companies to focus their efforts on efficient commercialization of idecabtagene vicleucel (ide-cel; bb2121) in the U.S., the companies’ lead investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, currently in review with the FDA. The companies will continue to share equally profits and losses in the U.S. Under the terms of the amended agreement, BMS will buy out its obligations to pay bluebird bio future ex-U.S. milestone and royalty payments for ide-cel and bb21217, the companies’ second BCMA-directed CAR T immunotherapy, for a one-time upfront payment of $200 million. bluebird bio is currently in the process of building out and qualifying its wholly-owned manufacturing facility in Durham, North Carolina for the production of lentiviral vector (LVV) to support the U.S. commercial market for ide-cel and for bluebird bio’s pipeline. Over time, BMS will assume responsibility for manufacturing of LVV outside the U.S. In partnership with BMS, bluebird bio is planning to present updated ide-cel clinical data from the Phase 2 KarMMa study at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting.

REVISED BUSINESS PRIORITIES AND OPERATING PLAN

Given the ongoing impact of the COVID-19 global pandemic and recent shifts in regulatory timelines, bluebird bio has undertaken a comprehensive business review with the goal of ensuring the ability to achieve its 2022 vision with a path towards financial sustainability. Under the revised business priorities and operating plan, bluebird remains on track for potential regulatory approval and commercial launch for ZYNTEGLO, ide-cel, Lenti-D for CALD, and LentiGlobin for SCD by 2022.

Through this comprehensive business review, bluebird bio has prioritized key research and development programs and has made a number of changes to the future cost structure relative to the prior long-range plan, including:

Reduced investment in selling, general and administrative expenses, including a deferred investment in building a U.S. commercial organization, reduced facilities and IT infrastructure, and other cost-reduction measures.
Prioritized investment in R&D expenses, including an indefinite pause of the HGB-211 clinical study in SCD patients at high risk of stroke, adjustment to the timing of investment in ongoing clinical studies to reflect COVID-19 related delays in enrollment, reduction or elimination of investment in certain preclinical programs, and other cost-reduction measures.
Nick Leschly, chief bluebird, will decline nearly 100% of his salary for the next 12 months. Similarly, additional members of the bluebird bio senior leadership team and all members of the Company’s Board of Directors will forgo 20% of their salaries or Board cash retainers for the next 12 months. All will receive a grant of restricted stock units equal to 80% of the value of the released cash compensation, which will vest over one year.
In total, these changes are expected to result in over $500 million of net cash savings through 2022 compared to the prior long-range plan. As a result, bluebird bio expects cash, cash equivalents, and marketable securities of $1.02 billion as of March 31, 2020, together with projected revenue generated under collaborative arrangements, projected sales of products and cash inflows associated with the amended agreements with BMS, to fund the revised operating plan into 2022. bluebird bio expects to continue to drive additional savings through rigorous prioritization and focus on expenses, real estate optimization, and exploration of additional sources of funding to further strengthen its financial position.

PREVIOUSLY DISCLOSED RECENT HIGHLIGHTS

IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On March 31, 2020, bluebird bio and BMS announced the submission of their BLA to the U.S. FDA for ide-cel, the companies’ lead investigational BCMA-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The BLA submission includes results from pivotal KarMMa study evaluating ide-cel in a heavily pre-treated patient population with relapsed and refractory multiple myeloma.
COVID-19 IMPACT – On March 26, 2020, bluebird bio provided an assessment of the impact of the COVID-19 pandemic and outlined steps the company has taken to ensure the safety of its patients and employees, while working to ensure the sustainability of its business operations as this unprecedented situation continues to evolve. Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months and expects timing shifts to vary by clinical trial and by program.
UPCOMING ANTICIPATED MILESTONES

Regulatory
Submission of a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical
Presentation of ide-cel clinical data from the KarMMa study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting later this month, in partnership with Bristol-Myers Squibb.
Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes at the 2020 Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Updated data presentation from HGB-206 clinical study in patients with SCD at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.
Updated data presentation from ALD-102 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.
FIRST QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2020 and December 31, 2019 were $1.02 billion and $1.24 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $21.9 million for the three months ended March 31, 2020 compared to $12.5 million for the three months ended March 31, 2019. The increase was primarily attributable to an increase in ide-cel license and manufacturing service revenue under our agreement with BMS, as well as an increase in royalty revenue.
R&D Expenses: Research and development expenses were $154.1 million for the three months ended March 31, 2020 compared to $122.6 million for the three months ended March 31, 2019. The increase was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $73.2 million for the three months ended March 31, 2020 compared to $60.3 million for the three months ended March 31, 2019. The increase 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 $202.6 million for the three months ended March 31, 2020 compared to $164.4 million for the three months ended March 31, 2019.
CONFERENCE CALL DETAILS

bluebird bio will hold a conference call to discuss business updates and first quarter 2020 financial results on Monday, May 11 at 8:00AM 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 335-5158.