Five months out from their twin FDA approvals in sickle cell disease (SCD), who’s winning the race to treat patients with their respective gene therapies—bluebird bio or partners Vertex Pharmaceuticals and CRISPR Therapeutics?
It’s not an easy question to answer. While Vertex and CRISPR have started cell collection in five patients, bluebird has begun the process with just one. On the flip side, however, while Vertex and CRISPR have activated 25 qualified treatment centers (QTCs), bluebird has opened 64 QTCs for business.
The battle got interesting on Monday of this week. As Vertex announced its first-quarter results, bluebird's therapy took center stage in a New York Times story detailing the first cell collection for Lyfgenia. The patient is 12-year-old Kendric Cromer from Washington, D.C., “a solemn and shy adolescent,” who is likely on his way to becoming a poster child for the treatment.
Thursday, when bluebird conducted its first-quarter conference call, CEO Andrew Obenshain called the first cell collection for the $3.1 million therapy a “monumental milestone."
Conversely, Vertex and CRISPR have been relatively low-key about the patient starts for their $2.2 million one-time therapy. Since Casgevy (exa-cel) is approved in three regions, laying the groundwork is more challenging. The companies have 75 QTCs planned in the U.S., Europe and the Middle East. Of the 25 that are activated, 18 are in the U.S., with those sites mapped out on a website for the treatment. At least one patient in each of the regions is among the five who have undergone cell collection.
At bluebird, much of the company's work to lay the launch groundwork is complete. The roster of 64 established QTCs is “unparalleled compared to others in the field,” bluebird's chief commercial and operating officer Tom Klima said on Thursday's call.
“More than half of our Lyfgenia QTCs have communicated to us that they are actively evaluating patients for gene therapy initiation and one quarter of our centers are in the prior-authorization process for one or more patients,” Klima said.
The treatment start-up process is time consuming and includes steps ranging from the initial patient consultation, enrollment in the company’s support program and reimbursement negotiations with payers. During this period, the patient undergoes a two-month “wash-out period,” Klima said, for hydroxyurea, a process that enlargens and rounds out the red blood cells.
Bluebird's 64 centers handle the administration of both Lyfgenia and the company's beta-thalassemia treatment Zynteglo (beti-cel). Six of the centers also are activated to administer Skysona (evi-cel), bluebird’s medicine for cerebral adrenoleukodystrophy (CALD). Zynteglo and Skysona won FDA approvals in August of 2022 and September of 2022, respectively.
“We have established a substantial QTC footprint very quickly following Lyfgenia’s approval,” Klima said. “We were able to quickly transition these centers for Lyfgenia due to both our learned experience of setting up these centers for Zynteglo and the strong relationships we’ve built with these centers.”
Klima added that “almost half” of the QTCs have come online since Lyfgenia’s approval and that the company’s focus has now shifted from establishing the QTCs to serving patients.
While bluebird expects to realize its first revenues from Lyfgenia in the third quarter of this year, Vertex and CRISPR said they expect to begin booking revenue in the second quarter.
In all, bluebird has started 15 patients on its three gene therapies, all this year, with 11 collections for Zynteglo and three for Skysona. By the end of this year, the company expects to have between 85 and 105 new patient starts.
The company reported revenue of $18.6 million for the first quarter and has $264 million cash on hand. Bluebird’s cash runway, which includes achieving expected revenue milestones, runs through the first quarter of 2026, chief financial officer Chris Krawtschuk said.