Bluebird bio stock drops as SCD gene therapy Lyfgenia trails Vertex

Bluebird bio experienced a significant decline in its stock value, with shares plummeting by 23% on Wednesday. This drop followed the announcement of its second-quarter results, which highlighted a lackluster uptake of its gene therapy for sickle-cell disease (SCD), Lyfgenia (lovotibeglogene autotemcel). The company disclosed that only four patients had begun treatment with Lyfgenia. This was notably lower than the 20 patient starts recorded for Vertex Pharmaceuticals' competing CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel).

Both Lyfgenia and Casgevy received approval from the FDA on the same day last December. Since then, Casgevy has also been approved for the treatment of beta thalassemia in the United States. Despite these challenges, Bluebird bio remains optimistic about its overall portfolio performance. The company projects that approximately 85 patients will commence cell collections for its three FDA-approved therapies by the end of 2024. These therapies include Lyfgenia, Zynteglo for beta thalassemia, and Skysona for cerebral adrenoleukodystrophy.

In a prior estimate made in May, Bluebird bio had anticipated between 85 to 105 new patient starts across its trio of gene therapies. Up to this point, the company has reported 27 patient starts, with 19 for Zynteglo, four for Skysona, and the aforementioned four for Lyfgenia.

Tom Klima, Bluebird bio's chief commercial and operating officer, addressed the disparity in patient starts between Lyfgenia and Casgevy. He explained that the longer path to patient starts for Lyfgenia is due to several factors. These include navigating the payer approval process and achieving clinical readiness within the patient community. Klima emphasized that the slow start is not due to a lack of demand. "It's really just a timing thing," he said. "It's not a slower start than expected and it's not a demand issue. Demand is actually strong."

Bluebird bio's optimism about its portfolio performance suggests that while the initial uptake of Lyfgenia has been slower compared to Casgevy, there is confidence in the therapy's long-term potential. The company’s projections for new patient starts and the ongoing patient starts for its other therapies indicate a robust demand and confidence in their gene therapy offerings.

In summary, while Bluebird bio has faced challenges with the initial uptake of Lyfgenia, the company remains hopeful about the future performance of its gene therapy portfolio. The slower start for Lyfgenia compared to Casgevy is attributed to procedural and readiness factors rather than demand issues. The company’s projections for the future suggest that they expect to overcome these initial hurdles and see increased uptake of their therapies moving forward.