Onco-Summaries: Daily Oncology Updates at a Glance

28/08/2025

TOLREMO Therapeutics' TT125-802 received two fast track designations from the US FDA for NSCLC (Ref)

The US FDA granted two fast track designations to TOLREMO Therapeutics' TT125-802 (CBP/p300 inhibitor) for the treatment of NSCLC:

1. For the treatment of patients with locally advanced or metastatic NSCLC with an EGFR exon 19 deletion or exon 21 L858R substitution mutation, with disease progression on at least one line of prior therapy including an EGFR inhibitor

   
   

2. For the treatment of patients with locally advanced or metastatic NSCLC with KRAS-G12C mutation, with disease progression on at least one line of prior therapy including a KRAS G12C inhibitor

   
   

The designations will support TOLREMO’s clinical development strategy to advance TT125-802 + oncogene-targeting drugs in KRAS- and EGFR-mutated NSCLC.

Vanda Pharmaceuticals' VGT-1849B received the FDA's Orphan Drug Designation for polycythemia vera (Ref)

The US FDA granted the Orphan Drug Designation to Vanda Pharmaceuticals' VGT-1849B (JAK2 inhibitor) for the treatment of polycythemia vera (PV).

• VGT-1849B is an antisense oligonucleotide (ASO) that utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA) peptide nucleic acid, and selectively targets JAK2 mRNA, reducing aberrant levels of JAK2 that may cause hematologic malignancies

  
  

• To note, >95% of PV patients harbor the JAK2 V617F gain-of-function mutation leading to aberrant JAK2 production

D3 Bio's D3S-001 received the FDA Breakthrough Therapy and Orphan Drug Designations for KRAS G12C-mutated cancers (Ref)

The US FDA granted the Breakthrough Therapy Designation to D3 Bio's D3S-001 (KRAS G12C-selective inhibitor) for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic NSCLC who have received prior chemotherapy and immunotherapy but have not been previously treated with a KRAS G12C inhibitor.

• Additionally, D3S-001 also received the Orphan Drug Designation for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic CRC

  
  

• The Breakthrough Therapy and Orphan Drug Designations are based on results from the Phase 1/2 NCT05410145 trial of D3S-001 in patients with advanced solid tumors harboring a KRAS G12C mutation