Onco-Summaries: Daily Oncology Updates at a Glance

02/10/2025

Aminex Therapeutics' AMXT 1501 + DFMO received the FDA Orphan Drug Designation for Neuroblastoma (Ref)

The US FDA granted the Orphan Drug Designation to Aminex Therapeutics' AMXT 1501 (polyamine transport inhibitor) + difluoromethylornithine for the treatment of patients with neuroblastoma.

• Mark Burns, CSO and President, Aminex Therapeutics: "Receiving Orphan Drug Designation for AMXT 1501 in combination with DFMO represents an important milestone in our mission to develop innovative therapies for children with life-threatening cancers. We are committed to working closely with regulators, investigators and patient advocacy groups, to accelerate the clinical development efforts for AMXT 1501 in combination with DFMO for the treatment of neuroblastoma and other childhood and adult tumor types in collaboration with the Beat Childhood Cancer Research Consortium."

First-line maintenance with Zepzelca + Tecentriq received FDA approval for ES-SCLC (Ref)

The US FDA approved Jazz Pharmaceuticals' lurbinectedin (Zepzelca; DNA alkylating agent) + Roche's atezolizumab (Tecentriq; anti-PD-L1) as a maintenance treatment for adults with extensive-stage small cell lung cancer whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.

• The approval is based on results from the Phase 3 IMforte trial in which the combo reduced the risk of disease progression or death by 46% and the risk of death by 27% vs atezolizumab maintenance therapy alone