Why bluebird bio Got More Attention Than Others at JPM 2018

Published: Jan 12, 2018 By

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Plenty of companies had interesting news to share at the J.P. Morgan Healthcare Conference in San Francisco this week, and bluebird bio received more attention than some. It might just be possible that this clinical-level company is moving toward being a commercial-level company in the next year or so. One of the company’s announcements was it expected to file three applications for regulatory approval by the end of 2019.

Todd Campbell, writing for The Motley Fool, notes that 2017 was a big year for gene therapy, with two approvals for two cancer gene therapies and one for an eye disease, showing that after decades of research, the technology was starting to pay off. “The validation of this approach suggests that bluebird bio could be in a perfect position to ride a wave of rising demand for these treatments because it boasts one of the most comprehensive gene therapy research pipelines,” he wrote.

The first candidate for approval is bluebird’s LentiGlobin. The company 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). Bluebird expects to file for regulatory approval for those in 2019.

Its bb2121 is likely the product investors are most excited about. Bluebird licensed the tech to Celgene Corp., and this CAR-T therapy has been outstanding in trials so far. It presented data at the American Society of Hematology (ASH) meeting in December 2017 from a Phase I trial in late-stage relapsed/refractory multiple myeloma. In the study, 94 percent of patients on the therapy achieved an objective response, and 89 percent achieved at least a very good partial response.

Campbell notes, “Celgene is already the market share Goliath in multiple myeloma treatment, generating over $10 billion in annualized sales in the indication. Its Revlimid is the top-selling first-line therapy and its Pomalyst is a fast-growing third-line therapy. If bb2121 gets the nod, there’s no partner better than Celgene to position it to win market share in the fourth-line setting.”

Bluebird isn’t relying on just those three products, either. Reuters noted, “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.”

But of the big three products, if approved, analysts and the company project each of them could deliver “nine figures in annual sales,” Campbell says, “and over time, each could have 10-figure peak sales potential.”

But is it a good investment? Should you be buying up shares of bluebird bio? Campbell writes, “This is still a clinical-stage company, however, so there’s plenty of risks associated with buying shares. Investors have already driven share prices to new highs on optimism, so disappointment in these trials would likely cause a big drop in market cap. Nevertheless, I believe that gene therapy will revolutionize treatment for many diseases, and bluebird bio is one of the best gene therapy stocks investors can buy.”

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