Uptick in gene therapy uptake triggers stock bump for beleaguered bluebird
09 May 2024
Gene TherapyDrug Approval
Bluebird bio’s three gene therapies may finally be gaining momentum. The biotech shared in its first-quarter earnings report that, so far this year, 15 new patients have started on one of the multimillion-dollar treatments – and it’s anticipating over 100 starts in total for 2024. Shares of bluebird jumped 15% on the news Thursday.
With a market cap now hovering around $200 million, bluebird has struggled with safety concerns, submission delays, and the rollout of its gene therapy portfolio; at its peak in 2018, the biotech had $1.6 billion in cash on hand. For more analysis, see Vital Signs: The last flight of the bluebird.
However, the company may have turned a corner this year. After scoring FDA approval in December for its one-time sickle-cell disease treatment Lyfgenia (lovotibeglogene autotemcel) – priced at $3.1 million – bluebird has since secured multiple outcomes-based agreements with commercial payers, and the US Centers for Medicare and Medicaid Services (CMS) has proposed to raise reimbursement rates for the therapy.
One new patient has started Lyfgenia this year. Bluebird’s $2.8-million beta-thalassemia gene therapy, Zynteglo (betibeglogene autotemcel), saw 11 new patient starts, and Skysona (elivaldogene autotemcel), a $3-million treatment for cerebral adrenoleukodystrophy, had three.
Approved in 2022, Zynteglo and Skysona carry middling price tags for gene replacement therapies – far less than that of Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics, the most expensive treatment at $4.25 million. For more, see Vital Signs: Shrinkflation hits the gene therapy sector.
Bluebird bio reported net revenues of $18.6 million for the first quarter, a huge jump over its $2.4 million revenues for the year-ago period. While bluebird didn’t provide full-year revenue guidance, it expects between 85 to 105 new patient starts across its three gene therapies.
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