4 Massachusetts Biotechs Making Waves at American Society of Hematology
December 6, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN DIEGO – There has been a lot of positive news coming out of the American Society of Hematology meeting in California, with a number of Massachusetts companies making waves with data from key trials.
The four companies, highlighted by the Boston Business Journal, are Karyopharm Therapeutics , bluebird bio , Novartis AG and Agios Pharmaceuticals .
1. Novartis
Swiss drugmaker Novartis , the fourth largest employer in Massachusetts, announced experimental CAR-T therapy CLT019 sent 82 percent of patients’ blood cancer into remission, according to interim data presented Saturday at the American Society of Hematology. That data puts Novartis in line to hit its 2017 goal to seek regulatory approval for its pediatric leukemia CAR-T therapy, CTL019. The global Phase II study found that 82 percent of pediatric patients, 41 out of 50, achieved complete remission or complete remission with incomplete blood count recovery at three months following infusion of CLT019.
2. Karyopharm Therapeutics
Cambridge-based Karyopharm announced full results from its Phase II trial of multiple myeloma drug, selinexor. Data showed an overall response rate of 21 percent, which analysts told the Journal was “solid activity.” Karyopharm plans to double the size of the 78 patient Phase II study for its next trial in hopes of clearing the way for regulatory filing in 2018, the Journal said.
3. bluebird bio
During a presentation at ASH, bluebird bio provided updates on LentiGlobin, its treatment for sickle cell anemia and beta-thalassemia major. The company said the treatment proved positive for some patients with transfusion-dependent beta-thalassemia and one patient with severe sickle cell disease, were benefiting from the drug. That data came on the heels of early data from its Phase I multiple myeloma treatment for patients who have not responded to previous treatments. Data from that drug, which is being co-developed by Celgene , indicated that all patients in the second and third cohorts being dosed showed some positive responses. Data from that trial was not presented at ASH, but at the 28th EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium in Munich.
4. Agios Pharmaceuticals
On Sunday, Agios Pharmaceuticals presented data on two molecules, AG-248 and AG-519, for the treatment for pyruvate kinase deficiency. The Journal said data showed both molecules “showed sustained activity.” Both drugs were well tolerated and benefitted patients, the company said in a release. PK deficiency often requires blood transfusions.
Although not included in the Journal’s report, Lexington, Mass.–based Shire also presented positive data at ASH for its Phase III study of vonvendi for Von Willebrand disease. The company announced that data shows the trial met its primary endpoint of controlling bleeding and blood loss during surgical procedures. The company plans to submit data for regulatory review by the U.S. Food and Drug Administration.