Patients At Last Begin Receiving Vertex-CRISPR and Bluebird Sickle Cell Gene Therapies

 

Last month, Vertex said sickle cell patients had not yet received infusions of its gene therapy Casgevy. That’s now changed, as the company races with bluebird bio’s Lyfgenia.

As of second-quarter earnings on August 1, no patients had received an infusion of Vertex Pharmaceuticals and CRISPR Therapeutics’ sickle cell gene therapy Casgevy. But infusions have since begun, a spokesperson for Vertex confirmed to BioSpace.

Meanwhile, over at peer company bluebird bio, which earned FDA approval of its sickle cell gene therapy Lyfgenia the same day as Casgevy, patients are also beginning to receive infusions of their own edited cells, The New York Times reported on Monday.

These moments mark a breakthrough for sickle cell treatment. Experts expected the uptake of gene therapies to be slow as the process to undergo treatment can take as long as a year. Vertex’s Chief Operating Officer Stuart Arbuckle acknowledged as much in the earnings call last month.

“We have always known that Casgevy offers an enormous advancement for patients. We’ve also consistently communicated that the patient journey, that is the process to go from patient interest all the way to infusion of edited cells, is long and complex. Whilst it’s still early in the launch, we have gained many learnings. Interest level is high among patients, physicians, governments and other stakeholders,” Arbuckle said.

Vertex did not respond to requests for more information as of publication, including on the number of patients receiving Casgevy.

As for bluebird, which was expected to have a faster rollout of Lyfgenia because it already had treatment centers established for its two other FDA-approved gene therapies, the company announced during a second quarter earnings call on August 14 that four patients had undergone cell collection as a first step to Lyfgenia treatment.

When Will Casgevy and Lyfgenia Start Generating Revenue?

At the time of its Q2 earnings call, Vertex had activated 35 centers with a goal of activating 75 around the world. About 20 patients had offered up their cells for collection, putting them “in the funnel” for treatment. Vertex was not specific, but said the patients were coming from all regions where the medicine is approved, which includes the U.S., Europe and the Middle East. Arbuckle noted on the call that the company has also been working to improve reimbursement in all jurisdictions to ensure patients have coverage.

“The treatment process does take time, but we are now even more confident in our view that Casgevy will help large numbers of patients around the world and represents a multibillion-dollar opportunity,” Arbuckle said.

Investors have been eager to know when Casgevy treatments will begin, with analysts peppering executives with questions on the earnings call to try and get any scrap of information. William Blair has estimated that peak sales for the gene therapy could reach $3.6 billion, even with the slow start.

As for Lyfgenia, bluebird bio CFO James Sterling said on the call that investors can expect about two quarters between initial cell collection and revenue recognition. “We are clearly on the right track with accelerating interest in our therapies, and a clear path to translate patient starts into a growing consistent revenue stream over time.”

Sterling noted that patients are scheduling their treatment out many months in advance, often around life events given the time commitment to undergoing gene therapy. “So it’s not a slower start than expected and it’s not a demand issue. Demand is actually strong. It’s simply a timing issue,” he said.

https://www.biospace.com/drug-development/patients-at-last-begin-receiving-vertex-crispr-and-bluebird-sickle-cell-gene-therapies