bluebird bio (BLUE) rides high on its gene therapy approvals – November 22, 2022

biography of the bluebird (BLUE Free Report) is a biotechnology company focused on developing gene therapies for serious genetic diseases and cancer.

The company has had an eventful year so far, although it’s not fully reflected in its price action.

bluebird recently received approval 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) gained FDA approval in August as a personalized one-time gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions across all genotypes.

Syskona received accelerated approval from the FDA in September for the treatment of patients under the age of 18 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 be dependent on verification and description of the clinical benefit in a confirmatory study. Therefore, as a condition of Syskona’s accelerated approval, bluebird will need to provide the FDA with confirmatory long-term clinical data 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 FDA-imposed clinical hold on the development of eli-cel in August 2021 was lifted in September ahead of Syskona’s Biologics License Application (BLA) review.

As of September 30, 2022, BLUE has raised approximately $54.1 million in net proceeds through its At-the-Market (ATM) equity facility. Of this $54.1 million, $46.1 million of 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 restricted cash in the fourth quarter will be sufficient to meet planned operating expenses and capital expenditures in the second quarter of 2023.

Shares of bluebird are down 33.4% in the year-to-date period compared to the industry decline of 33.4%.


Image source: Zacks Investment Research

However, investors should consider the risks involved before considering investing in the stock. Gene therapies, by nature, are complex. Therefore, Zynteglo and Syskona are quite complex to administer and can only be provided as treatments to patients by qualified treatment centers. Therapies are extremely expensive. Zyteglo currently costs $2.8 million, while Syskona is priced at $3 million, making them the most expensive therapies on the market.

The company is also evaluating lovotibeglogene autotemcel (lovo-cel) for the treatment of adult and pediatric patients with sickle cell disease (SCD). bluebird expects to obtain lovo-cel approval in SCD under a fast track in the United States based on data from Group C from the ongoing Phase I/II HGB-206 study. The company is expected to submit a BLA for lovo-cel to the FDA in the first quarter of 2023. The company has also completed enrollment in the Phase III study HGB-210, which will serve as a confirmatory study to gain full approval for lovo-cel in the SCD.

Zack’s 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) e Eton Pharmaceuticals (ETON Free Report), each with a Zacks Rank #2 (Buy). you can see the complete list of today’s Zacks #1 (Strong Buy) stock here.

Immunocore’s 2022 loss-per-share estimates have narrowed from $1.25 to 97 cents over the past 30 days. Loss per share for 2023 narrowed from $1.69 to $1.10 in the same time frame.

Immunocore’s earnings topped estimates in three of the trailing four quarters, while falling short of the same in the remaining occasion. The average earnings surprise for IMCR is 68.34%.

AVEO Pharmaceuticals’ loss-per-share estimates for 2022 have narrowed from 94 cents to 68 cents over the past 30 days. The 2023 earnings estimate held steady at improving from 41 cents to 52 cents over the same time frame.

AVEO’s earnings topped estimates in three of the trailing four quarters, while lacking the same in the bore occasion. The average earnings surprise for AVEO is 19.87%.

Eton Pharmaceuticals’ loss-per-share estimates for 2022 have held steady at 44 cents over the past 30 days. Earnings per share for 2023 have stagnated at a penny over the same time frame.

Eton earnings missed the estimates in three of the four quarter-finals, beating the same on the other occasion. The average earnings surprise for ETON is 115.63%.

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