Bluebird Bio is making progress on the launch of its three approved gene therapies but appears to be trailing Vertex in its competition to attract patients to their respective treatments for sickle cell disease (SCD).
While Bluebird has collected cells from four patient starts to start its SCD therapy Lyfgenia, Vertex reported earlier this month that it had recorded approximately 20 patient starts for its SCD treatment Casgevy. Both gene therapies were approved on the same day in December last year.
Vertex also picked up FDA approval in January for Casgevy to treat patients with transfusion-dependent beta thalassemia (TDT). While Vertex’s start figure for Casgevy includes patients with either of the diseases, it is expected that a majority of the 20 who have undergone cell collection are those with SCD.
With the results, Bluebird's stock price tumbled by 18% Wednesday morning.
During a conference call Wednesday, Bluebird’s chief commercial and operating officer Tom Klima reminded investors that Vertex’s figure is for worldwide patient starts while Bluebird’s is limited to the U.S.
He also said that the company expects Lyfgenia patient starts to accelerate in the second half of this year.
“With Lyfgenia, there’s simply a longer path to patient starts. This includes going through the payer approval process but also the steps to clinical readiness within the patient community. It’s really just a timing thing,” Klima said. “Additionally, we are seeing patients scheduling many months in advance. They’re scheduling around life events. It’s not a slower start than expected and it’s not a demand issue. Demand is actually strong.”
Bluebird said it expects to recognize revenue from Lyfgenia by the end of this year as payments don’t begin until a patient is infused.
Overall with its three gene therapies—including TDT treatment Zynteglo and cerebral adrenoleukodystrophy (CALD) therapy Skysona, both of which were approved in 2022—Bluebird has registered 27 patient starts, with 19 coming for Zynteglo and four from Skysona. Three months ago, Bluebird reported 14 patient starts, with 11 for Zynteglo and three for Skysona.
By the end of the year, Bluebird expects to have conducted 85 cell collections for its three gene therapies. Three months ago, the company projected between 85 and 105 patient starts for 2024. More than 40 patients have been scheduled for cell collection by the end of this year, with more expected to sign on.
One metric where Bluebird leads Vertex is in the scaling up of qualified treatment centers. Bluebird has more than 70 up and running for administration of Lyfgenia and Zynteglo, compared to 35 for Vertex, which added 10 sites during the second quarter.
For the second quarter, Bluebird reported revenue of $16.1 million, which fell short of Leerink Partners’ projection of between $23.5 million and $26.6 million. It also was down sequentially from $18.6 million. The company added that it expects revenue to decline in the third quarter and rebound in the fourth.
Bluebird also reported “significant progress” with access to Lyfgenia as more than half of Medicaid-insured individuals with SCD in the U.S. live in a state that has affirmed coverage through a preferred drug list or published coverage criteria. Additionally, nearly 20% of Medicaid-insured individuals with SCD in the U.S. live in a state that has already completed prior authorization approval for use of the treatment by at least one resident.
Bluebird expects its cash and cash equivalents of $193 million to fund operations into the second quarter of 2025. At the end of the first quarter, the company had $264 million in cash and cash equivalents.
Bluebird also announced that it has renegotiated its loan agreement with Hercules Capital and is eligible to receive two future tranches totaling $50 million, contingent upon the achievement of patient start and product delivery milestones.
“We are not surprised to see shares down sharply as slower-than-anticipated cell collections for Lyfgenia put near-term tranches out of reach and renegotiated terms require additional financing,” Leerink Partners analysts wrote in a Wednesday note to clients.