bluebird bio (BLUE – Free Report) is a biotechnology company focused on developing gene therapies for severe genetic disorders and cancer.
The company has had an eventful year so far, though it is not fully reflected in its price movement.
bluebird recently received approvals for two of its gene editing therapies, Zynteglo (beti-cel) for transfusion dependent thalassemia (TDT) and Syskona (eli-cel) for active cerebral adrenoleukodystrophy (CALD).
Zynteglo (beti-cel) 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.
Syskona received accelerated approval from the FDA in September for treating patients younger than 18 years with CALD. It is the second ex-vivo lentiviral vector gene therapy approved by the FDA in the United States.
Syskona’s continued approval may depend upon verification and description of clinical benefit in a confirmatory study. Thus, as a condition of Syskona’s accelerated approval, bluebird will have to provide confirmatory long-term clinical data to the FDA from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years and from commercially treated patients.
The clinical hold imposed by the FDA on the eli-cel development in August 2021 was lifted in September before the review of Syskona’s biologics license application (BLA).
As of Sep 30, 2022, BLUE raised approximately $54.1 million in net proceeds through its At-the-Market (ATM) equity facility. Of this $54.1 million, $46.1 million in net proceeds were realized in the third quarter. The company expects cash, cash equivalents and marketable securities and the anticipated release of $40 million of the restricted cash in the fourth quarter to be sufficient to meet planned operating expenses and capital expenditures into the second quarter of 2023.
Shares of bluebird have declined 33.4% in the year-to-date period compared with the industry’s fall of 33.4%.
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However, investors should consider the risks before considering investing in the stock. 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. Zyteglo currently costs $2.8 million, while Syskona is priced at $3 million, making them the most expensive therapies in the market.
The company is also evaluating lovotibeglogene autotemcel (lovo-cel) for treating adult and pediatric patients with sickle cell disease (SCD). bluebird expects to seek approval for lovo-cel in SCD under an accelerated pathway in the United States based on data from Group C of the ongoing phase I/II HGB-206 study. The company is expected to file a BLA for lovo-cel with the FDA in first-quarter 2023. The company also completed enrolment in the phase III HGB-210 study, which will serve as a confirmatory study to seek full approval for lovo-cel in SCD.
Zacks Rank and Key Picks
bluebird currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the same sector include Immunocore (IMCR – Free Report) , sporting a Zacks Rank #1 (Strong Buy) and AVEO Pharmaceuticals (AVEO – Free Report) and Eton Pharmaceuticals (ETON – Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.
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