Ambitious Bluebird Seeks Three Drug Approvals by 2019

At the J.P. Morgan Healthcare Conference in San Francisco, bluebird bio announced that it expected to file three applications for regulatory approval by the end of 2019. As yet, the company has no products on the market, so the applications, if approved, would mark the company’s shift into a commercial company.

The first candidate is its LentiGlobin candidate. It expects to submit to the European Medicines Agency (EMA) for transfusion-dependent beta thalassemia this year.

The other two candidates are Lenti-D in cerebral adrenoleukodystrophy (CALD) and a CAR-T candidate, bb2121 in multiple myeloma (MM). They expect to file for regulatory approval for those in 2019.

Although the company presented a number of studies at the American Society of Hematology (ASH) meeting held in December 2017, one of the standouts was results from its Phase I trial of bb2121 in patients with late-stage relapsed/refractory multiple myeloma. Although the primary focus of the study was safety, the efficacy results were dazzling, with 94 percent of patients on the therapy achieving an objective response, and 89 percent achieving at least a very good partial response.

"To see these types of responses after one treatment with bb2121 in a heavily pre-treated patient population is very promising, and we are hopeful that CAR-T therapy with bb2121 may become an important therapy in the fight against multiple myeloma, which remains an insidious and incurable diseases," said James Kochenderfer, primary investigator in the study, and physician with the Center for Cancer Research at the National Cancer Institute in Bethesda, Maryland, in a statement at the time.

And these aren’t the only compounds bluebird is developing, Reuters notes, "Beyond those three, bluebird is also developing a promising treatment for sickle cell disease, a potentially life-shortening inherited blood disorder that causes severe pain and often leads to repeat hospitalizations. The company plans to discuss appropriate endpoints for future sickle cell clinical trials with health regulators and will provide an update on the program at the end of the year, the CEO said."

Meanwhile, the company and its chief executive officer, Nick Leschly, are looking ahead to possible approvals and the tricky topic of pricing. At the meeting, Leschly said, "If you have a life-long value on a patient, you can’t expect a payer to pay that all up front at one time. How do we address that?"

Some of the focus was on Medicaid Best Price regulations and problems matching upfront payments and long-term value. "If you want to get creative there [in pricing], you have got to take down some barriers," Leschly said at the meeting. "We want to recognize value over time. We want to share risk and we need the participants in the system to play ball with us on that."

One does have to wonder when insurers and patients will revolt over some of these cutting-edge therapies’ prices. For example, the first CAR-T therapies approved are from Novartis and Gilead Sciences and are priced at $475,000 and $373,000, respectively, for one-time treatments. It’s hard to put a price on what seem to be almost miracle cures for certain types of cancer, but these companies have, and the insurers are complicit in agreeing to pay for them. The economics of the pricing are complex and to a certain extent make sense, given the development costs and that CAR-T therapies are unique to each patient, but from the perspective of a patient and the general public, the prices are dizzyingly high.

Spark Therapeutics is evaluating a pricing scheme, an odd sort of pay-performance approach, where it will charge $425,000 per eye for its Luxturna (voretigene neparvovec). The gene therapy is for a rare form of hereditary blindness caused by a mutation in the RPE65 gene, and only affects a few hundred individuals in the U.S. The company has set up several different pricing plans, one, with New England-based Harvard Pilgrim, bases the price on outcomes.

BioPharmaDIVE notes, "That bluebird is thinking about these problems now is a positive sign. Such forethought should help the company down the road if all goes according to plan. But delivering a pricing strategy that pleases investors as much as it wins over payers remains a high bar to clear."