Shares of
bluebird bio, Inc
.
BLUE
gained 7.74% on Dec 12 after the company announced new and updated data on its gene therapy programs in beta-thalassemia and sickle cell disease on Dec 10 at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition.
bluebird received approvals for two of its gene therapies, Zynteglo (beti-cel) for transfusion-dependent thalassemia (TDT) and Syskona (eli-cel) for active cerebral adrenoleukodystrophy (CALD) in 2022.
Zynteglo was granted FDA approval in August as a one-time gene therapy customized for treating adults and pediatric patients with beta-thalassemia requiring regular red blood cell (RBC) transfusions across all genotypes.
The updated data presented at ASH demonstrated sustained treatment effects in patients treated with betibeglogene autotemcel (beti-cel) and lovotibeglogene autotemcel (lovo-cel) gene therapies through additional follow-up.
The follow-up data included patients living with beta-thalassemia who require regular RBC transfusions up to 8 years post-treatment (n=3), across ages and genotypes. As of July 2022, 63 patients received beti-cel across four clinical studies and were followed for a median of 52.0 (20.1–101.7) months.
These included two phase III studies that supported FDA approval. In those studies, 90.2% of patients achieved transfusion independence (TI).
Patients who achieved TI produced normal or near-normal levels of total hemoglobin and demonstrated improvements in markers of iron overload and markers of ineffective erythropoiesis. These patients also showed continued improvement in quality-of-life measures through 3 years following treatment in long-term follow-up study, LTF-303.
Moreover, the ability to seek employment or be employed increased to 93% of patients (13/14) from 67% (10/15) at baseline.
The company also presented case studies that threw additional light on investigations into two cases of persistent anemia observed in an adult and pediatric patient in Group C, the pivotal cohort of the HGB-206 study of lovo-cel. Both patients had two α-globin gene deletions (−α3.7/−α3.7), also known as alpha-thalassemia trait. These patients are the only ones in the study with this specific genotype. Integration site analysis and next-generation sequencing showed no evidence of clonal processes (vector-related or otherwise) and the findings are not consistent with an emerging hematologic malignancy. Clinical investigations presented suggest that the alpha-thalassemia trait likely contributed to anemia after lovo-cel infusion. Following these cases, this genotype was added to the exclusion criteria for ongoing studies.
bluebird remains on track to submit a biologics license application (BLA) for lovo-cel in the first quarter of 2023.
Shares of bluebird have declined 20.5% in the year-to-date period compared with the industry’s fall of 18.5%.
Image Source: Zacks Investment Research
The positive long-term data bode well for bluebird which had an eventful 2022. While the therapies promise potential, gene therapies, by nature, are complex. Thus, Zynteglo and Syskona are pretty complex to administer and can only be provided as treatments to patients by qualified treatment centers. The therapies are extremely costly.
Hence, it remains to be seen how these therapies fare in the market.
Zacks Rank and Key Picks
bluebird currently has a Zacks Rank #3 (Hold). Some top-ranked stocks in the overall healthcare sector are
Gilead Sciences, Inc
.
GILD
,
Biogen
(BIIB) and
Vertex Pharmaceuticals
VRTX
. All three companies carry a Zacks Rank #2 (Buy). You can see
the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
.
Earnings estimates for Gilead Sciences have increased 48 cents in the last 60 days to $7.09. Gilead has surpassed earnings estimates in three of the past four quarters with an average positive beat of 0.36%.
Earnings estimates for Vertex Pharmaceuticals have increased 44 cents in the last 60 days to $14.65. Vertex has surpassed earnings estimates in three of the past four quarters with an average positive beat of 3.16%.
Earnings estimates for Biogen have increased 55 cents in the last 60 days to $16.98. Biogen has surpassed earnings estimates in three of the past four quarters with an average positive beat of 7.02%.
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