-Virtual oral presentation will include updated efficacy and safety data from pivotal Phase 2 KarMMa study of ide-cel-

-Biologics License Application under review with U.S. Food and Drug Administration (FDA)-

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 8, 2020-- bluebird bio, Inc. (Nasdaq: BLUE) announced today that updated results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of idecabtagene vicleucel (ide-cel; bb2121), an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, in heavily pre-treated patients with relapsed and refractory multiple myeloma (RRMM) will be presented, in partnership with Bristol Myers Squibb, during the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program beginning on May 29.

Topline results from the KarMMa study were previously disclosed in December 2019. An additional presentation at ASCO20 will include results from a real-world, non-interventional, retrospective study evaluating treatment patterns in heavily pre-treated patients with RRMM compared to outcomes from the KarMMa study.

On Wednesday, May 13 at 5:00 PM ET, accepted abstracts will be available on the ASCO conference website. At that time, the embargo for the data included in the May 29 presentations will lift. The companies plan to issue a data press release at the time of the embargo lift. Video and slide presentations will be available on demand on the ASCO conference website beginning Friday, May 29 at 8:00 AM ET and will remain available for 180 days.

Oral Presentation:

Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): initial KarMMa results

Presenting Author: Nikhil C. Munshi, MD, The LeBow Institute for Myeloma Therapeutics and Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA
Session Title: Hematologic Malignancies- Plasma Cell Dyscrasia
Date & Time: Abstract #8503, Friday, May 29, 2020, 8:00 AM ET

Poster Presentation

KarMMa-RW: A study of real-world treatment patterns in heavily pretreated patients with relapsed and refractory multiple myeloma (RRMM) and comparison of outcomes to KarMMa

Presenting Author: Sundar Jagannath, MD, Mount Sinai Hospital, New York, NY, USA
Session Title: Hematologic Malignancies- Plasma Cell Dyscrasia
Date & Time: Abstract #8525, Friday, May 29, 2020, 8:00 AM ET

About idecabtagene vicleucel (ide-cel; bb2121)

On March 31, 2020, bluebird bio and Bristol Myers Squibb announced the submission of a Biologics License Application for ide-cel to the U.S. Food and Drug Administration (FDA) 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. Ide-cel is the first chimeric antigen receptor (CAR) T cell therapy submitted for regulatory review to target B-cell maturation antigen (BCMA) and for multiple myeloma.

Ide-cel is a BCMA-directed genetically modified autologous CAR T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Bristol Myers Squibb and bluebird bio’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About KarMMa

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adult patients with relapsed and refractory multiple myeloma (RRMM) in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10⁶ CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.
bluebird bio is a trademark of bluebird bio, Inc.

bluebird bio Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations, and projections about our goals, plans and objectives involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, the possibility of unfavorable results from additional clinical trials of ide-cel or from subsequent analysis of existing data from the KarMMa study or existing or new data received from additional ongoing and future studies of ide-cel, that ide-cel may not receive regulatory approval for the indication described in this release and, if approved, whether such product candidate for such indication described in this release will be commercially successful, and that the collaboration with Bristol Myers Squibb may not continue or be successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K for the year ended December 31, 2019, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

Hyperlinks are provided as a convenience and for informational purposes only. Neither Bristol Myers Squibb nor bluebird bio bears responsibility for the security or content of external websites or websites outside of their respective control.

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Investors:
Ingrid Goldberg, 410-960-5022
igoldberg@bluebirdbio.com

Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com

or

Media:
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Source: bluebird bio, Inc.