bluebird Inc. (BLUE - Free Report) announced new data from ongoing phase III studies onits investigational betibeglogene autotemcel (formerly LentiGlobin for β-thalassemia gene therapy). The data showed that pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia (TDT) achieved and maintained transfusion independence with hemoglobin (Hb) levels that are nearnormal.
TDT is a severe genetic disease caused by mutations in the β-globin gene that results in significantly reduced or absent adult hemoglobin (HbA).
A total of 60 pediatric, adolescent and adult patients across genotypes of TDT were treated with betibeglogenein the phase I/IINorthstar (HGB-204) and HGB-205 studies, and the phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies as of Mar 3, 2020.
The results showed that 89% of the evaluable patients (17/19) with transfusion-dependent β-thalassemia did not have a β0/β0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207
Data from exploratory analyses of HGB-207 showed improved markers of blood cell production and bone marrow function in patients who achieved transfusion independence.
Of the total, 85% of patients (11/13) with a β0/β0 genotype or IVS-I-110 mutation in HGB-212 had been transfusion-free for at least sevenmonths.
Shares of the company have decreased 27.2% year to date against the industry’s rise of 4.4%.
The company also announced new data from its ongoing phase I/II HGB-206 study of lentiGlobin gene therapy for adult and adolescent patients with sickle cell disease (SCD). The study showed a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS).
In Group C of HGB-206, 25 patients were treated with LentiGlobin for SCD and had up to 24.8 months of follow-up.
The data showed 99.5% reduction in annualized rate of VOC and ACS in Group C patients with a history of VOCs and ACS (n=14) who had at least six months follow-up. At up to 24 months, there were no reports of serious VOC or ACS in Group C patients (n=18) with at least six months follow-up.
Group C patients with at least six months follow-up continued to produce consistent levels of gene therapy-derived anti-sickling hemoglobin (HbAT87Q) at up to 24 months, reducing levels of abnormal sickle hemoglobin (HbS)
Key markers of hemolysis approached near-normal levels in Group C patients, supporting the potential of LentiGlobin for SCD to modify the underlying pathophysiology of the disease.
The company is also developing its Lenti-D product candidate for cerebral adrenoleukodystrophy (CALD), a rare, hereditary neurological disorder, which can often be fatal. The FDA granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with CALD, which should expedite the development and review of the therapy. Lenti-D also enjoys orphan drug status in the Unites States and Europe. It was also granted Rare Pediatric Disease designation by the FDA for the treatment of adrenoleukodystrophy.
Zacks Rank and Other Stocks to Consider
bluebird currently carries a Zacks Rank #2 (Buy).
Some other top-ranked stocks in the biotech sector are AkceaTherapeutics Inc. (AKCA - Free Report) , Axcella Health Inc. (AXLA - Free Report) and Aytu BioScience Inc. (AYTU - Free Report) , all carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Akcea’s loss per share estimates have narrowed from $2.07 to $1.37 for 2020 and from $2.46 per share to $1.85 for 2021 in the past 60 days.
Axcella’s loss per share estimates have narrowed from $1.14 to 79 cents for 2020 and from $3.30 per share to $3.03 for 2021 in the past 60 days.
Aytu’s loss per share estimates have narrowed from $1.26 to 12 cents for 2020 in the past 60 days.
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