Vertex's Casgevy launch progresses as CF business posts impressive growth

While cystic fibrosis has long been Vertex Pharmaceuticals’ bread and butter, the company has been eager to diversify its revenue base in recent years.

As several patients start their treatment journey on the company’s CRISPR-partnered gene-editing cell therapy Casgevy, Vertex is well on its way toward accomplishing that goal.

Following Casgevy’s approval in sickle cell disease (SCD) in December and its subsequent beta thalassemia nod the following month, Vertex is “making strong progress with [authorized treatment center] activation, physician and patient engagement, and payer conversations,” the company’s chief operating officer, Stuart Arbuckle, said on an analyst call Monday.

Aside from treatment center activation, Vertex touched on another important launch aspect during its first-quarter earnings call—cell collection.

As of mid-April, five patients had their cells collected in the three regions where Casgevy is currently approved: The United States, Europe and the Middle East. Arbuckle hailed the milestone as “excellent progress” given the short time since approval, plus the complexity and length of the patient journey on Casgevy.

Five cell collections is “likely a decent first data point,” Mizuho Securities analyst Salim Syed wrote in a note to clients Monday. He said a “somewhat slow and non-linear ramp” early in the drug’s journey is to be expected, citing the potential for collection numbers to start ramping up more significantly in the coming months.

Vertex’s Casgevy collection stat puts the drug ahead of its SCD rival Lyfgenia from bluebird bio, which snared FDA approval simultaneously with Casgevy in December. Bluebird on Monday announced that it’s so far collected just one patient’s cells for its SCD gene therapy.

Ultimately, Vertex will not recognize Casgevy sales until patients are infused, Arbuckle noted on the company’s earnings call. With that in mind, Vertex could begin posting revenues from the gene-edited therapy as early as 2024’s second quarter, followed by a potentially “greater influx of revenue” in the year’s back half, Miles Minter’s team at William Blair wrote in a separate analyst note Monday.

William Blair currently expects Casgevy to generate $84.4 million for the full year.

Vertex is making good progress building out its network of authorized treatment centers (ATCs), too, the William Blair and Mizuho analysts pointed out.

Vertex is planning to open roughly 75 ATCs globally and had nine up and running at the time of Casgevy’s launch, Arbuckle explained. As of Monday, the company’s number of activated treatment centers had crept up to 25 across the U.S., Europe and the Middle East, the COO added. Eighteen of those sites are in the U.S., according to Casgevy’s website.

Bluebird, for its part, boasts around 60 U.S. treatment centers for its gene therapies, Mizuho’s Syed pointed out.

Meanwhile, Minter’s analyst team at William Blair argued in their note that Casgevy’s sales opportunity in Bahrain and Saudi Arabia is currently “underappreciated.”

Arbuckle’s comments largely confirmed that sentiment, with the Vertex COO noting that the number of eligible SCD and beta thalassemia populations in those countries is roughly 23,000 patients, representing a “potentially larger opportunity than even the U.S.”

While much of the attention during Vertex’s earnings call focused on Casgevy, the company has continued to score well with its arsenal of CFTR modulators in cystic fibrosis (CF).

In 2024’s first quarter, Vertex’s product revenues jumped 13% to $2.69 billion, which the company attributed to the continued performance of its triple-combination therapy Trikafta in the U.S., plus strong uptake of the med overseas, where it goes by the name Kaftrio. In particular, Vertex is seeing gains after successfully moving its CFTR drugs into younger patient groups in both the U.S. and abroad.

“We view the company’s top- and bottom-line beats positively as it shows the company is still able to grow its core CF franchise,” the William Blair team commented.

With Casgevy ramping up and Trikafta continuing to hold its own, Vertex is standing by its prior revenue estimate for the full year. For all of 2024, the company expects to generate sales within a range of $10.55 billion to $10.75 billion.