Emerging Therapies

Two large, phase 3 clinical trials demonstrated that targeting B-cells can have a significant impact on disease progression in patients with relapsing multiple sclerosis.

Although multiple sclerosis (MS) is dominated by a progressive phase of the disease, few therapies are available to modify clinically defined progression. There is a significant unmet need for treatments targeting the delayed neurodegenerative components of the disease, said Gavin Giovannoni, MBBCh, PhD, Chair of Neurology, Blizard Institute of Cell and Molecular Science, Queen Mary University of London, England, at the 2016 American Academy of Neurology annual meeting.

A pivotal phase 3 clinical trial demonstrated that dichlorphenamide (Keveyis) significantly reduced the rate and severity of hypokalemic episodes in patients with periodic paralysis, a rare muscle disease affecting children and young adults. In addition, a parallel phase 3 clinical trial with dichlorphenamide showed a similar treatment effect in patients with hyperkalemic periodic paralysis but failed to demonstrate significance because of recruitment issues.

Entrectinib, an investigational potent oral inhibitor of tyrosine kinases, ROS1, and ALK proteins, achieves rapid and durable responses in patients with a range of advanced or metastatic solid tumors harboring NTRK, ROS1, or ALK gene fusions.
The investigational oral androgen receptor drug ODM-201 has significant antitumor activity with a favorable safety profile in men with metastatic castration-resistant prostate cancer, according to a pooled analysis of 2 early-phase clinical trials.
The investigational biosimilar (LA-EP2006) to the reference drug pegfilgrastim (Neulasta) has demonstrated similar results in patients with breast cancer who are receiving the regimen known as TAC (docetaxel, doxorubicin, and cyclophosphamide), according to data from the multicenter, randomized, double-blind PROTECT 2 trial.
Patients with previously treated metastatic urothelial cancer had response rates that exceeded historical standards when treated with an investigational immunotherapeutic agent, updated results of a large phase 2 clinical trial showed. Treatment with the PD-1 ligand 1 (PD-L1) inhibitor atezolizumab led to an overall response rate of 15% in 311 patients, including a 26% rate among patients who had the highest levels of PD-L1 expression. Historical data have demonstrated response rates of about 10% for second-line therapy and beyond.
Elderly patients with Philadelphia-negative B-cell acute lymphoblastic leukemia (ALL) have overall poor outcomes with current therapies. Results of a new study presented at ASH 2015 suggest that frontline treatment with the investigational antibody-drug conjugate inotuzumab ozogamicin in combination with deintensified chemotherapy is a good option for older patients with this disease.
Chimeric antigen receptor (CAR) T-cell therapy has been striking in various hematologic malignancies, and for the first time this treatment approach is being evaluated in multiple myeloma.
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.
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  • Value-Based Care in Myeloma
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