This week, gene therapies from Novartis AG, Gilead Sciences Inc. and Juno Therapeutics Inc. took center stage at the American Society of Hematology conference in Atlanta.
The companies are developing medicines that belong to a new class of therapy. The treatments, known as chimeric antigen receptor T-cell, or CAR-T cell, therapies, work by manipulating a patient's own disease-fighting T-cells and then infusing them back into the body to fight blood cancers.
Gene therapies offer new promises
Patients who responded to Novartis' Kymriah, the first CAR-T cell therapy, approved by the U.S. Food and Drug Administration for relapsed or refractory diffuse large B-cell lymphoma, continued to respond six months after the drug was first administered.
Among 46 patients with at least six months of follow up after receiving treatment, 30% had no detectable sign of cancer, while another 7% achieved a partial response.
Follow-up data on a study of Gilead Sciences' CAR-T cell therapy Yescarta in patients with diffuse large B-cell lymphoma resistant to treatment showed "encouraging" durability and depth of response. Results showed that 42% of patients continued to respond to therapy, including 40% who were in complete remission, or showing no evidence of disease.
In a separate trial, patients treated with Gilead's CAR-T cell therapy KTE-C19 experienced a 71% response rate in acute lymphoblastic leukemia patients whose disease is refractory to or has relapsed following standard chemotherapy or hematopoietic stem cell transplantation.
Complete tumor remission was seen in 17 of the 24 patients being treated with the Gilead therapy.
Juno Therapeutics' CAR-T cell therapy JCAR017 reduced tumors in 74% of patients in a phase 1 trial for relapsed or refractory aggressive B-cell non-Hodgkin lymphoma. Patients in the trial were divided into two groups. In the core group, 68% of patients showed no signs of the disease.
Fortress Biotech Inc. unit Mustang Bio Inc.'s CAR-T cell drug MB-102 was found to be safe and well-tolerated in a phase 1 study to treat blastic plasmacytoid dendritic cell neoplasm and acute myeloid leukemia in two separate arms.
In a phase 3 trial, patients who received Kyowa Hakko Kirin Co. Ltd.'s mogamulizumab lived for 7.7 months without the disease worsening, versus 3.1 months for Merck & Co. Inc.'s Zolinza, or vorinostat. Among 372 patients enrolled in the study, 28% of those who received mogamulizumab achieved a reduction in their tumor size, compared to 4.8% of those treated with vorinostat.
Data presented at the meeting shows that two new therapies for hemophilia A, Roche Holding AG's Hemlibra and BioMarin Pharmaceutical Inc.'s experimental gene therapy valoctocogene roxaparvovec, eliminated bleeding events entirely, significantly changing the sometimes daily treatment regimens for the disease.
Wedbush Securities Analyst Liana Moussatos wrote in a Dec. 14 research note that valoctocogene roxaparvovec has the potential to become a single-treatment cure.
A combination of Karyopharm Therapeutics Inc.'s selinexor with available treatments led to high durable response rates in an ongoing phase 1b/2 study of patients with heavily pretreated multiple myeloma.
Final analysis of a phase 3 study of Amgen Inc.'s Kyprolis in combination with two other drugs showed the treatment extended life for patients with multiple myeloma whose cancer had relapsed after earlier treatment or did not respond at all.
Results from the trial showed that Kyprolis, or carfilzomib — when added to dexamethasone and Celgene Corp.'s Revlimid, or lenalidomide — cut the risk of death in patients by 21%.
Johnson & Johnson scores wins in blood cancer
Johnson & Johnson's immunotherapy Darzalex produced a significant response in over half of the patients treated for a type of intermediate or high-risk smoldering multiple myeloma — a precursor to multiple myeloma — in a phase 2 trial.
Darzalex is already approved as a monotherapy to treat multiple myeloma patients who have received at least three prior lines of therapy.
In another trial, a combination of Darzalex and Takeda Pharmaceutical Co. Ltd.'s Velcade, melphalan and prednisone reduced the risk of disease progression or death by 50% in newly diagnosed myeloma patients.
Promising responses were seen in a study evaluating GlaxoSmithKline plc's GSK2857916 in patients with previously treated multiple myeloma as 60% of the patients responded to the drug and lived for 7.9 months without the disease worsening.
Adaptimmune Therapeutics plc's NY-ESO SPEAR T-cell therapy showed promising efficacy and acceptable safety in a pilot study of multiple myeloma patients with overall response rates of 76% at 100 days and 44% at one year, and a median predicted overall survival of about three years.
Agios Pharmaceuticals Inc.'s leukemia drug ivosidenib showed promising responses in patients with relapsed or refractory acute myeloid leukemia and an isocitrate dehydrogenase-1 mutation in an ongoing phase 1 trial.
A combination of Immune Design Corp..'s G100 and Merck's Keytruda helped reduce the tumor in 39% of follicular non-Hodgkin lymphoma patients. This would compare to 15% for those who only received G100.
BeiGene Ltd.'s investigational drug zanubrutinib in combination with tislelizumab led to a 40% reduction in disease activity in patients with B-cell malignancies in an ongoing phase 1b trial.
Preliminary data from Actinium Pharmaceuticals Inc.'s phase 2 trial of Actimab-A to treat patients with acute myeloid leukemia showed that 69% of patients saw a reduction in their disease.
Affimed NV's AFM13 drug and Merck's Keytruda were well-tolerated in patients with relapsed or refractory Hodgkin lymphoma in an ongoing phase 1b study.
The combination treatment is being investigated as a salvage therapy for patients who have failed prior standard treatments like brentuximab vedotin.
Daiichi Sankyo Co. Ltd.'s blood thinner edoxaban met its primary goal in a phase 3b trial, by demonstrating non-inferiority for the composite outcome of first recurrent venous thromboembolism, a condition in which blood clots form in the deep veins of the leg and can travel in the circulation and lodge in the lungs.
Paving way for sickle cell cure
Bluebird Bio Inc. presented promising results of its sickle cell treatment at the meeting. Data from an ongoing phase 1/2 trial showed that patients treated with the LentiGlobin gene therapy experienced rising hemoglobin levels in the first six months.