bluebird bio, Inc. (BLUE - Free Report) announced that the European Commission (EC) has granted conditional marketing authorization to Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), the company’s first gene therapy, for patients aged 12 years or older with transfusion-dependent β-thalassemia (TDT), who do not have a β0/β0 genotype and for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or absent hemoglobin.
Shares of the company increased 20% year to date against the industry’s decline of 0.5%.
Zynteglo was reviewed as part of the European Medicines Agency’s (EMA) Priority Medicines (PRIME) and Adaptive Pathways programs. These programs support the medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.
The conditional marketing authorization is supported by efficacy and durability data from the phase I/II HGB-205 study and the completed phase I/II Northstar (HGB-204) study along with available data from the ongoing phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up LTF-303 study, as of the data cut off on Dec 13, 2018.
Data from phase I/II HGB-205 showed that 75% of patients, who do not have a β0/β0 genotype, achieved transfusion independence, while in the phase I/II Northstar study, 80% achieved the same. In Northstar-2 study, 80% patients achieved transfusion independence.
The approval makes available a gene therapy for certain patients with TDT that has the potential to transform lives by offering the possibility of a transfusion-free future.
The approval will boost sales for the company.
Zacks Rank and Other Stocks to Consider
bluebird currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. (ANIK - Free Report) , Applied Genetics Technologies Corp. and Acorda Therapeutics Inc. . All of them carry a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika’s earnings per share estimates have moved up from $1.22 to $1.28 for 2019 and from $1.21 to $1.33 for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters, with average beat of 72.00%.
Applied Genetics’ loss per share estimates have narrowed from $1.25 to 1 cent for 2019 and from $2.39 to $2.15 for 2020 in the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with average beat of 83.47%.
Acorda’s loss per share estimates have narrowed from $3.84 to $3.59 for 2019 and from $3.32 to $3.09 for 2020 in the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters, with average beat of 79.32%.
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bluebird bio, Inc. (BLUE - Free Report) announced that the European Commission (EC) has granted conditional marketing authorization to Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), the company’s first gene therapy, for patients aged 12 years or older with transfusion-dependent β-thalassemia (TDT), who do not have a β0/β0 genotype and for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or absent hemoglobin.
Shares of the company increased 20% year to date against the industry’s decline of 0.5%.
Zynteglo was reviewed as part of the European Medicines Agency’s (EMA) Priority Medicines (PRIME) and Adaptive Pathways programs. These programs support the medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.
The conditional marketing authorization is supported by efficacy and durability data from the phase I/II HGB-205 study and the completed phase I/II Northstar (HGB-204) study along with available data from the ongoing phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up LTF-303 study, as of the data cut off on Dec 13, 2018.
Data from phase I/II HGB-205 showed that 75% of patients, who do not have a β0/β0 genotype, achieved transfusion independence, while in the phase I/II Northstar study, 80% achieved the same. In Northstar-2 study, 80% patients achieved transfusion independence.
The approval makes available a gene therapy for certain patients with TDT that has the potential to transform lives by offering the possibility of a transfusion-free future.
The approval will boost sales for the company.
Zacks Rank and Other Stocks to Consider
bluebird currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Anika Therapeutics Inc. (ANIK - Free Report) , Applied Genetics Technologies Corp. and Acorda Therapeutics Inc. . All of them carry a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika’s earnings per share estimates have moved up from $1.22 to $1.28 for 2019 and from $1.21 to $1.33 for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters, with average beat of 72.00%.
Applied Genetics’ loss per share estimates have narrowed from $1.25 to 1 cent for 2019 and from $2.39 to $2.15 for 2020 in the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with average beat of 83.47%.
Acorda’s loss per share estimates have narrowed from $3.84 to $3.59 for 2019 and from $3.32 to $3.09 for 2020 in the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters, with average beat of 79.32%.
Today's Best Stocks from Zacks
Would you like to see the updated picks from our best market-beating strategies? From 2017 through 2018, while the S&P 500 gained +15.8%, five of our screens returned +38.0%, +61.3%, +61.6%, +68.1%, and +98.3%. This outperformance has not just been a recent phenomenon. From 2000 – 2018, while the S&P averaged +4.8% per year, our top strategies averaged up to +56.2% per year.
See their latest picks free >>
Zacks Names "Single Best Pick to Double"
From thousands of stocks, 5 Zacks experts each have chosen their favorite to skyrocket +100% or more in months to come. From those 5, Director of Research Sheraz Mian hand-picks one to have the most explosive upside of all.
It’s a little-known chemical company that’s up 65% over last year, yet still dirt cheap. With unrelenting demand, soaring 2022 earnings estimates, and $1.5 billion for repurchasing shares, retail investors could jump in at any time.
This company could rival or surpass other recent Zacks’ Stocks Set to Double like Boston Beer Company which shot up +143.0% in little more than 9 months and NVIDIA which boomed +175.9% in one year.
Free: See Our Top Stock and 4 Runners Up >>
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