December 9, 2015
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON – Following bluebird bio ’s disappointing report on its gene therapy treatment for hematological diseases including sickle cell and beta-thalassemia major, competitor Acceleron Pharma is surging after announcing strong results for its own blood treatment.
Both companies are developing therapies to treat the blood disorders. On Monday, bluebird revealed mixed results for its gene-therapy treatment, LentiGlobin, works differently for each patient, which means single conclusive results cannot be achieved as some of the patients still required blood transfusions. At the same time, Acceleron announced mid-stage clinical trial results showing its treatment luspatercept increased hemoglobin levels, reduced transfusion burden, improved health-related quality of life measures and had beneficial effects on liver iron concentration in patients with beta-thalassemia. Both companies announced results for their therapies at the American Society of Hematology meeting in Orlando, Fla.
Since announcing the results, Acceleron’s stock has surged, hitting a 52-week high of $45.20 on Monday, the day it announced the data at the ASH meeting. In contrast, bluebird’s stock plunged nearly 34 percent to $55.10 following the release of its data. Bluebird stock is currently trading at $56.54 per share. Since June, bluebird’s from a high of $194.25. Following bluebird’s revelation of mixed results, stock analysts at thestreet rated bluebird’s stock a sell. In contrast, Oppenheimer has rated Acceleron’s stock a buy, the Boston Business Journal reported this morning.
During its presentation at ASH, bluebird said two of the beta-thalassemia major patients using bluebird’s gene therapy LentiGlobin BB305, a treatment for blood disorders including beta- thalassemia major and sickle cell disease, have required transfusions. However, on a positive note, the company said at their most recent follow ups, three months and one month, respectively, “these patients were producing measurable levels of HbAT87Q.”
However, bluebird did show that some patients who required needed frequent and regular blood transfusions have responded positively to the therapy and have been transfusion free for up to two years—showing that gene therapy definitely has promise for some patients.
This weekend’s report may have confirmed some investors’ fears after bluebird released preliminary information in November. Data released in November shows some of the patients treated with the therapy have not required blood transfusions in several months, something the patients typically underwent on a monthly basis. Likewise, in the spring, bluebird released a report showing a sickle cell patient has been transfusion free with no sickle cell hospitalizations for nearly three months after undergoing the therapy and the patient is now producing higher levels of hemoglobin. But, at least two of the three remaining beta-thalassemia patients have needed at least one transfusion and the third is still transfusion dependent, bluebird said in its data. The report indicates the gene therapy treatment is not something that is an effective treatment for every patient.
Acceleron, which is developing luspatercept with Celgene Corporation , said results of its Phase II trial are “very exciting as they show positive effects across a range of clinically challenging complications of beta-thalassemia.”
“A therapy that could potentially treat the anemia, complications of beta-thalassemia, such as iron overload, and improve measures of health-related quality of life would be a huge advance to help address the substantial unmet need of patients with beta-thalassemia,” Antonio Piga, director of Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and coordinating principal investigator of Acceleron’s study, said in a statement.
