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Nearing gene therapy launch, Bluebird cautions a slower start

  • Bluebird bio, on the cusp of winning European clearance for its experimental gene therapy Zynteglo, detailed commercial plans Thursday that it hopes will provide a template for a pipeline of gene and cell therapies it aims to launch in the coming years.
  • Core to Bluebird’s strategy is an annuity-based payment model for Zynteglo, which treats transfusion-dependent beta-thalassemia. The biotech has previously indicated it hopes to structure payments over five years with potential for later adjustments depending on how patients respond to treatment.
  • In pitching its plans, Bluebird set expectations for modest initial sales of Zynteglo, if approved by the European Commission. “You should take a long-term view,” Chip Baird, Bluebird’s chief financial officer, told attendees at an event the company hosted for Wall Street analysts in New York.

Bluebird doesn’t have many examples to follow.

Three gene therapies for genetic diseases are approved in Europe. Only a handful of patients ever received the first two, Glybera and Strimvelis, while Spark Therapeutics’ Luxturna has yet to gain traction following an approval last November. (Novartis markets Luxturna in Europe.)

Bluebird’s Zynteglo, consisting of reprogrammed patient stem cells, is likely to be next up, having secured the backing of the European Medicines Agency a little over a month ago.

If launched as expected, Zynteglo will face many of the same vexing challenges that its predecessors have faced. Healthcare systems are not set up to easily handle large one-time payments for therapies which are designed to deliver benefits over a lifetime.

Bluebird, like others approaching market with their own gene therapies, hopes to reach agreement with payers on a model that structures payment over time.

In January at the J.P. Morgan healthcare conference, the biotech announced it was discussing spreading payments over five years, with as much as 80% of Zynteglo’s price tied to how well it works. Bluebird has said Zynteglo offers a “lifetime intrinsic value” of more than $2 million, but has not disclosed a potential price.

“The J.P. Morgan presentation opened a lot of doors for us in Europe,” said Andrew Obenshain, Bluebird’s head of Europe, at Thursday’s event.

Bluebird used its analyst event to detail its progress toward that goal. The biotech expects a launch in Germany would occur first, with roll-out in Italy, France and the U.K. to follow in 2020.

“We think there will be variations on a theme in terms of how it works from one country to the next,” said Baird, although he indicated Bluebird hopes to limit payment per patient over the course of five years to a relatively narrow range.

Baird indicated this year’s opportunity in Germany is modest, and noted that product revenue in the near-term would not be “the most telling” indicator on launch progress.

Zynteglo is just the first of a pipeline of cell and gene therapies that Bluebird hopes to launch in the coming years, including therapies for sickle cell disease, multiple myeloma and a metabolic disorder known as adrenoleukodystrophy.

Transfusion-dependent beta-thalassemia (TDT), the disease Zynteglo treats, is a significantly smaller opportunity than sickle cell or multiple myeloma. Bluebird, then, sees launching Zynteglo as testing a model that could be used in the future.

In the U.S., where Bluebird hopes to win approval of Zynteglo by next year, many of the payers covering TDT and sickle cell patients are the same, and the company claims roughly 80% of the relevant healthcare providers overlap.

Reshaping drug payment and reimbursement is no easy task. Bluebird, though, styles itself a pioneer.

“If all we ever do is successfully launch four products for our first four indications, this will be a bellwether biotech,” said Baird.