bluebird bio, Inc. (BLUE) Presents New Data from Northstar (HGB-204) and Northstar-2 (HGB-207) Studies of LentiGlobin™

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bluebird bio Presents New Data from Northstar (HGB-204) and Northstar-2 (HGB-207) Studies of LentiGlobin™ Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia at Annual Congress of the European Hematology Association

bluebird bio, Inc. (BLUE) announced that new data from the completed Phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent β-thalassemia (TDT) and any genotype, and its ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of LentiGlobin™ investigational gene therapy in patients with TDT and non-β0genotypes, will be presented in an oral session on June 16 at the 23rdAnnual Congress of the European Hematology Association by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesù of Rome, Italy.

“The maturing data from HGB-204 and HGB-207 suggest that one-time treatment with LentiGlobin may address the underlying genetic cause of TDT. With our refined manufacturing process, the majority of patients with TDT and non-β00 genotypes are transfusion-free and producing total hemoglobin at normal or near-normal levels,” said David Davidson, M.D., chief medical officer, bluebird bio. “We are on track to submit a marketing authorization application in the European Union later this year, and we continue to work closely with clinical investigators and regulatory authorities to complete our ongoing clinical trials and bring this important treatment option to patients as soon as possible.”

People with TDT need regular blood transfusions to survive, but chronic transfusions carry risks, including unavoidable iron overload that can result in multi-organ damage and shortened life expectancy. Eliminating or reducing the need for transfusions may reduce the long-term complications associated with TDT and current standards of care.

“Consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels in patients indicate that LentiGlobin manufacturing refinements have resulted in improved gene therapy characteristics and may enable sustained transfusion independence for a great majority of patients,” said Professor Locatelli, the lead investigator of the Northstar-2 study. “Further, we are now seeing more than three years of data from the Northstar study indicating that LentiGlobin therapy may enable long-term transfusion independence in the majority of patients with non-β00 genotypes. These results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.”

LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β00 Genotypes: Updated Results from Northstar-2 (Abstract #1510)

Presenter: Franco Locatelli, M.D., Ph.D., Professor of Pediatrics, University of Pavia, Italy and Director, Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
Oral Session: Cell and Gene Therapy: Clinical Results
Date & Time: Saturday, June 16, 2018, 11:45 a.m. – 12:00 p.m. CEST (5:45 a.m. EDT)
Location: Room A8

The safety and efficacy of LentiGlobin in patients with TDT were evaluated in the Phase 1/2 Northstar study (HGB-204). To further improve clinical results, a refined manufacturing process was used to produce LentiGlobin drug product (DP). The aim of the ongoing Phase 3 Northstar-2 study (HGB-207) is to evaluate the efficacy and safety of LentiGlobin DP with manufacturing refinements in patients with TDT and non-β00genotypes. Data from Northstar and Northstar-2 will be included in the oral presentation.

Northstar-2 (HGB-207) results include (as of May 15, 2018):

  • 11 patients had been infused with LentiGlobin and the median follow-up was 8.5 months (range: 0.3 – 16.2 months).
  • 7 of 8 patients are producing ≥ 7.6 g/dL of HbAT87Q and are maintaining total hemoglobin levels of 11.1 – 13.3 g/dL by 6 months.
  • These 7 patients with ≥ 6 months follow-up remain transfusion free for 4.7 – 15.1 months.
  • For the 11 study participants, the median DP vector copy number (VCN) was 3.4 (range: 2.4 – 5.6) copies/diploid genome, and the median proportion of transduced CD34+ cells was 82% (range: 53 – 90%).
  • The safety profile of LentiGlobin to date is similar to that observed in the Northstar study, and consistent with myeloablative conditioning with single-agent busulfan. No grade 3 or higher DP-related adverse events (AE) have been observed.
  • All study participants remain enrolled in the trial, and there have been no reports of graft-versus-host disease (GvHD).

Northstar (HGB-204) results include (as of March 7, 2018):

  • All 18 patients have completed the primary two-year study and are continuing into the long-term follow-up study LTF-303.
  • 8 of 10 patients with non-β00 genotypes were transfusion independent for a median of 33 months as of last follow-up.
  • For the 18 study participants, the median DP VCN was 0.7 (range: 0.3 – 1.5) copies/diploid genome, and the median proportion of transduced CD34+ cells was 32% (range: 17 – 58%).
  • The safety profile of LentiGlobin DP continues to be consistent with myeloablative conditioning with single-agent busulfan. There have been no reports of GvHD and no deaths on the study.
  • One serious AE of HIV infection was reported 23 months after infusion. HIV was contracted from typical exposure and is not related to treatment with LentiGlobin. This was confirmed by two laboratory tests that differentiate between HIV and the lentivirus used in LentiGlobin.

Conference Call & Webcast Information
bluebird bio will host a conference call and live webcast at 8:00 a.m. EDT on Friday, June 15, 2018. To access the live webcast, please visit the “Events & Presentations” page within the Investors and Media section of the bluebird bio website at http://investor.bluebirdbio.com. Alternatively, 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 4678706. A replay of the webcast will be available on the bluebird bio website for 90 days following the call.

About the Northstar (HGB-204) Study
The completed Phase 1/2 Northstar study was an open-label, single-dose, non-randomized, multi-center study conducted in the United States, Australia and Thailand. It was designed to evaluate the safety and efficacy of LentiGlobin in increasing hemoglobin production and eliminating or reducing transfusion dependence in subjects with transfusion dependent beta-thalassemia. The study treated 18 adults and adolescents who are being followed to evaluate safety and efficacy post-LentiGlobin infusion. For more information on the Northstar study, please visit www.northstarclinicalstudies.com or clinicaltrials.gov using identifier NCT01745120.

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