The annual meeting of doctors who treat blood disorders like leukemia and sickle cell is under way. Scientific breakthroughs have made hematology an exciting focus for patients and investors these days, and Saturday’s start of the American Society of Hematology’s yearly conference features encouraging news from developers of cancer-fighting antibodies, cell therapies, and genetic medicines.
Some of the most-anticipated results at the Atlanta meeting are coming from
(ticker: BLUE). The company’s gene therapies have been under development for a long time, and in the last four years its stock has slid from above $200 to under $9 now, leaving a market value of $628 million.
On Saturday, researchers showed that bluebird’s gene therapy can provide long-term freedom from the monthly blood transfusions otherwise needed by those with born with beta-thalassemia. At the meeting on Sunday, researchers will give data updates on bluebird’s therapy for sickle cell, an inherited disorder of red blood cells that has gotten attention from many genetic medicine developers. Other genetic therapies for thalassemia and sickle cell will be discussed by
In bluebird’s thalassemia presentation and an accompanying report in the New England Journal of Medicine, researchers showed that about 90% of those treated in bluebird’s Phase 3 studies achieved transfusion independence. Among 63 patients that bluebird has treated in various studies, some have gone transfusion free for seven years. The initial treatment is a somewhat arduous cell transplant procedure, but the therapy appears safe for adults and children.
Durability and safety are big clinical concerns in gene therapy, said bluebird medical chief Richard Colvin. “The mechanism suggests that it’s a lifetime effect,” he said of the thalassemia treatment. “The transplant integration lasts a lifetime.”
As a genetic medicine pioneer, Cambridge, Mass.-based bluebird has traveled a long road. On Nov. 4, it split off its cancer-treatment pipeline into the separately-traded company
(TSVT)—which is also presenting data at the ASH meeting. With a fast-track designation from the Food and Drug Administration for bluebird’s thalassemia treatment, the company hopes to file for U.S. approval in early 2022. A sickle cell approval application could follow in early 2023; if approved, bluebird’s treatment would probably become the first marketed genetic therapy for sickle cell.
The company previously got approval in Europe for its thalassemia gene therapy, but wasn’t able to agree with national health systems there on a price. Unable to afford the price demanded by governments like Germany’s, bluebird withdrew its therapy from the region.
“It was the last thing that we wanted to do,” bluebird Chief Executive Andrew Obenshain told Barron’s. “We tried everything to stay in Europe.” He’s confident that the American healthcare system will pay for bluebird’s gene therapies. The company has been talking with public and private payers here for three years.
“We’ve been through a lot as a company,” said Obenshain. “Now we’re poised to set the standard in gene therapy for decades to come. We have the largest and deepest ex-vivo gene therapy data set, and we’re excited to make it real.”
Write to Bill Alpert at firstname.lastname@example.org