Neuroblastoma study results show reduced pain

Apeiron Biologics announced the publication of a clinical study of Qarziba (dinutuximab beta) in high-risk neuroblastoma patients in the December issue of mAbs. The data demonstrate that dinutuximab beta (together with IL-2) given by long-term infusion is associated with a substantially reduced pain and thus could be delivered in an outpatient setting. The single-center study, without a control group, showed a best response rate of 40% and a significantly prolonged survival compared to matched historical controls. In detail, the results are as follows: a best response rate of 40.5% (15/37; 5 CR, 10 PR), 4-year (4 y) Progression-free (PFS) of 33.1% (observation 0.1- 4.9 y, mean: 2.2 y) and a 4 y overall survival (OS) of 47.7% (observation 0.27 – 5.20 y, mean: 3.6 y). Survival of the entire cohort (53/53) and the relapsed patients (29/53) was significantly improved compared to historical controls. The study also found a lower frequency of neuropathic pain with Long Term Infusion vs. Short Term Infusion (37.7% vs. 51.8%, respectively).

The study results were a key component of Apeiron’s Marketing Authorisation Application for dinutuximab beta to the European Medicines Agency, which was granted in May, 2017. The drug has orphan status in the EU, and orphan designation in the U.S.

See: "Tolerability, response and outcome of high-risk neuroblastoma patients treated with long-term infusion of anti-GD2 antibody ch14.18/CHO." Ina Mueller et al. mAbs Published online: 05 Dec 2017 https://doi.org/10.1080/19420862.2017.1402997