Bluebird shares more data on its breakthrough gene therapy trials, and it continues to impress
- Bluebird Bio on Thursday shared more data from an ongoing clinical trial of patients with beta-thalassemia and severe sickle cell disease being treated with the company's gene therapy LentiGlobin. And so far, the results continue to impress.
- Bluebird reported that two patients with the rare and devastating blood disorder beta-thalassemia did not require their standard blood transfusions at 14 months and 11 months after being treated with the therapy.
- Furthermore, a sickle cell disease patient stopped receiving his or her regular blood transfusions after day 88 of treatment with LentiGlobin. That patient was being weaned off of the transfusions as early as day 37. Earlier this week, BioPharma Dive reported that Bluebird had struck a deal with U.S. and European regulators mapping a pathway to regulatory approval for its pioneering therapy.
In a note, analysts from JP Morgan heralded the sickle cell results in particular. "The SCD patient is showing everything we would want to see at this point to demonstrate highly promising proof-of-concept," wrote the analysts.
It's important to recognize that "proof-of-concept" is the prudent term for Bluebird's successes so far. The trials that the closely-watched biotech has been running have involved patient pools as small as four.
However, earlier this week, the company announced that it had struck deals with the FDA and the EMA to map out an accelerated approval pathway. Under the terms of that deal, Bluebird will have to run two additional trials beyond the ones that are already underway that will be structured as 15-patient open-label studies in adults, adolescents, and children. Regulatory filing would be based on eliminating those trial participants' need for blood transfusions for one year.
The data that the company released on Thursday is one indicator that they may just achieve that endpoint.