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bluebird (BLUE) Down Despite FDA Nod to Gene Therapy for SCD
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bluebird bio, Inc. (BLUE - Free Report) obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (“SCD”) in patients ages 12 and older who have a history of vaso-occlusive events (“VOEs”).
The FDA approved lovo-cel under the brand name Lyfgenia.
SCD is a group of inherited blood disorders affecting approximately 100,000 people in the United States. It is a complex and progressive genetic disease that generally leads to unpredictable and debilitating VOEs.
Lyfgenia works by genetically modifying a patient’s blood stem cells to produce HbAT87Q. This gene-therapy-derived hemoglobin functions similarly to hemoglobin A, which is the standard adult hemoglobin produced in people not affected by SCD. Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient.
The therapy’s label is based on data from patients from the phase I/II HGB-206 study. Safety data supporting the application includes data from 54 patients who initiated stem cell collection. Efficacy of the gene therapy was backed by data from 36 patients in the phase I/II HGB-206 Group C study following enhancements to the treatment and manufacturing processes made through the course of the clinical development program.
Data showed 32 patients were evaluable for the endpoints of complete resolution of VOEs and severe VOEs in the 6-18 months post-infusion, including eight adolescent patients. Severe vaso-occlusive events were resolved in 94% of patients in this cohort and 88.2% experienced no VOE at all.
However, shares of bluebird were down 40.5% despite receiving the FDA’s approval for lovo-cel. This is primarily because of the boxed warning of hematologic malignancy issued with Lyfgenia’s label.
Some patients developed blood cancer when being treated with Lyfgenia.
Shares of bluebird have plunged 58.7% year to date compared with the industry’s decline of 20.4%.
Image Source: Zacks Investment Research
Lyfgenia was granted Priority Review in June 2023. bluebird, however, did not receive a Rare Pediatric Disease Priority Review Voucher as part of the review. The therapy was also previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy designation and rare pediatric disease designation.
Patients treated with Lyfgenia in bluebird-sponsored clinical studies will be monitored for a total of 15 years through a long-term safety and efficacy follow-up study.
We remind investors that the FDA approved bluebird’s gene therapy Zynteglo for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions on Aug 17, 2022 and Skysona for treating early, active cerebral adrenoleukodystrophy on Sep 16, 2022.
bluebird has made significant progress in the launch of Zynteglo, with 16 patient starts (cell collections) for individuals suffering from beta-thalassemia.
The first commercial infusion for Syksona was completed in March 2023. Cell collection has been completed for six patients to be treated with Skysona. bluebird continues to anticipate 5-10 patient starts for Skysona this year.
Lyfgenia marks the second FDA approval for bluebird for an inherited hemoglobin disorder.
Concurrently, the FDA approved Vertex Pharmaceuticals (VRTX - Free Report) and CRISPR Therapeutics’ (CRSP - Free Report) exagamglogene autotemcel, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of SCD in patients 12 years and older with recurrent VOCs.
Exagamglogene autotemcel was approved under the brand name Casgevy, making it the first FDA-approved treatment to utilize a type of novel genome editing technology. Vertex will make a $200 million milestone payment to CRISPR following the FDA’s approval of Casgevy, which will be capitalized and amortized to the cost of sales.
However, both Vertex and CRISPR Therapeutics were down like bluebird despite FDA approval.
The long-term safety of both therapies remains unknown and hence, investors seem wary. Per the FDA, patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.
Entrada’s loss per share estimate for 2023 narrowed from $2.07 to 9 cents in the past 60 days. The same for 2024 narrowed from $2.35 to $2.04 during the same period.
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bluebird (BLUE) Down Despite FDA Nod to Gene Therapy for SCD
bluebird bio, Inc. (BLUE - Free Report) obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (“SCD”) in patients ages 12 and older who have a history of vaso-occlusive events (“VOEs”).
The FDA approved lovo-cel under the brand name Lyfgenia.
SCD is a group of inherited blood disorders affecting approximately 100,000 people in the United States. It is a complex and progressive genetic disease that generally leads to unpredictable and debilitating VOEs.
Lyfgenia works by genetically modifying a patient’s blood stem cells to produce HbAT87Q. This gene-therapy-derived hemoglobin functions similarly to hemoglobin A, which is the standard adult hemoglobin produced in people not affected by SCD. Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient.
The therapy’s label is based on data from patients from the phase I/II HGB-206 study. Safety data supporting the application includes data from 54 patients who initiated stem cell collection. Efficacy of the gene therapy was backed by data from 36 patients in the phase I/II HGB-206 Group C study following enhancements to the treatment and manufacturing processes made through the course of the clinical development program.
Data showed 32 patients were evaluable for the endpoints of complete resolution of VOEs and severe VOEs in the 6-18 months post-infusion, including eight adolescent patients. Severe vaso-occlusive events were resolved in 94% of patients in this cohort and 88.2% experienced no VOE at all.
However, shares of bluebird were down 40.5% despite receiving the FDA’s approval for lovo-cel. This is primarily because of the boxed warning of hematologic malignancy issued with Lyfgenia’s label.
Some patients developed blood cancer when being treated with Lyfgenia.
Shares of bluebird have plunged 58.7% year to date compared with the industry’s decline of 20.4%.
Image Source: Zacks Investment Research
Lyfgenia was granted Priority Review in June 2023. bluebird, however, did not receive a Rare Pediatric Disease Priority Review Voucher as part of the review. The therapy was also previously granted orphan drug designation, fast track designation, regenerative medicine advanced therapy designation and rare pediatric disease designation.
Patients treated with Lyfgenia in bluebird-sponsored clinical studies will be monitored for a total of 15 years through a long-term safety and efficacy follow-up study.
We remind investors that the FDA approved bluebird’s gene therapy Zynteglo for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions on Aug 17, 2022 and Skysona for treating early, active cerebral adrenoleukodystrophy on Sep 16, 2022.
bluebird has made significant progress in the launch of Zynteglo, with 16 patient starts (cell collections) for individuals suffering from beta-thalassemia.
The first commercial infusion for Syksona was completed in March 2023. Cell collection has been completed for six patients to be treated with Skysona. bluebird continues to anticipate 5-10 patient starts for Skysona this year.
Lyfgenia marks the second FDA approval for bluebird for an inherited hemoglobin disorder.
Concurrently, the FDA approved Vertex Pharmaceuticals (VRTX - Free Report) and CRISPR Therapeutics’ (CRSP - Free Report) exagamglogene autotemcel, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of SCD in patients 12 years and older with recurrent VOCs.
Exagamglogene autotemcel was approved under the brand name Casgevy, making it the first FDA-approved treatment to utilize a type of novel genome editing technology. Vertex will make a $200 million milestone payment to CRISPR following the FDA’s approval of Casgevy, which will be capitalized and amortized to the cost of sales.
However, both Vertex and CRISPR Therapeutics were down like bluebird despite FDA approval.
The long-term safety of both therapies remains unknown and hence, investors seem wary. Per the FDA, patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.
Zacks Rank and A Stock to Consider
bluebird currently has a Zacks Rank #3 (Hold). A better-ranked stock in the overall healthcare sector is Entrada Therapeutics (TRDA - Free Report) , sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Entrada’s loss per share estimate for 2023 narrowed from $2.07 to 9 cents in the past 60 days. The same for 2024 narrowed from $2.35 to $2.04 during the same period.