Emerging Therapies

San Diego, CA—Ropeginterferon alfa-2b, an investigational, long-acting, mono-pegylated interferon was noninferior to hydroxyurea in achieving complete hematologic response and with superior tolerability to hydroxyurea in the treatment of patients with polycythemia vera, according to the final results of the phase 3 PROUD-PV clinical trial presented by Heinz Gisslinger, MD, Medical University of Vienna, Austria, at the 2016 American Society of Hematology meeting.

San Diego, CA—Chimeric antigen receptor (CAR)-targeting CD22 therapy induced clinical responses and a high rate of complete remission in children and young adults with relapsed or refractory acute lymphoblastic leukemia (ALL), including patients who had received anti-CD19 CAR T-cell therapy, said Terry J. Fry, MD, Hematologic Malignancies Section, Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (NCI), at the 2016 American Society of Hematology meeting. These findings create the opportunity for bispecific or multispecific CAR T-cell targeting.

Washington, DC—The oncology pipeline is bustling and shows no sign of slowing down anytime soon, said Anita Dopkosky, RPh, MS, Director, National Accounts, Walgreens, Pittsburgh, PA, at the Sixth Annual Conference of the Association for Value-Based Cancer Care. She provided an overview of key drugs in the oncology pipeline.

Oral and poster presentations of several promising agents in early- and late-phase clinical trials dotted the program at ASCO 2016. The presentations included studies with positive findings associated with many investigational therapies, including first-in-class therapies such as chimeric antigen receptor (CAR) T-cells for the treatment of patients with B-cell malignancies or acute myeloid leukemia (AML); a vaccine (galinpepimut-S) for the treatment of patients with AML; and a chimeric monoclonal antibody against claudin 18.2 for the treatment of patients with advanced gastric cancers.

The first-in-class antibody-drug conjugate rovalpituzumab tesirine (Rova-T) may be a new treatment option for patients with small-cell lung cancer (SCLC), which has a very poor prognosis and few treatment options. Rova-T is particularly promising in SCLC tumors that overexpress the delta-like (DLL) 3 protein, according to first-in-human study results presented at ASCO 2016.

The oral, investigational, small-molecule BCL-2 inhibitor venetoclax has shown excellent and durable responses in patients with relapsed or refractory chronic lymphocytic leukemia (CLL). All patients in the trial harbored the 17p deletion (del 17p), which signals poor prognosis.

Many presentations at ASH 2015 focused on novel therapies currently in development for the treatment of patients with hematologic malignancies, including a second generation of new agents recently approved by the FDA for a variety of hematologic cancers.

The multikinase inhibitor midostaurin is the first targeted therapy to improve overall survival (OS) in patients with acute myeloid leukemia (AML) and the FLT3 mutation.