D3 Bio Receives FDA Breakthrough and Orphan Drug Designations for KRAS G12C Inhibitor
Friday, August 29, 2025
D3 Bio has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational drug D3S-001 for the treatment of adults with KRAS G12C-mutated non-small cell lung cancer (NSCLC).
KRAS mutations are among the most common cancer drivers, found in up to 30% of tumours. The KRAS G12C mutation occurs in around 12% of NSCLC cases and 3–4% of CRC cases. Patients with these mutations often experience aggressive disease and limited benefit from current treatments such as chemotherapy and immunotherapy.
D3S-001 is a next-generation KRAS G12C inhibitor designed for strong and selective binding to the mutant protein. It blocks the switching mechanism between active and inactive forms of KRAS G12C, preventing tumour growth. Preclinical studies have shown full target engagement at clinically relevant doses, high potency, and central nervous system penetration. The drug is currently being tested as both monotherapy and in combination regimens in a global Phase II trial involving NSCLC, CRC, and other solid tumours.
The designation applies to patients with locally advanced or metastatic disease who have previously received chemotherapy and immunotherapy but have not been treated with a KRAS G12C inhibitor.
In addition, D3S-001 has been awarded Orphan Drug Designation for the treatment of adults with KRAS G12C-mutated colorectal cancer (CRC).
The designations are supported by data from an ongoing Phase 1/2 trial (NCT05410145) assessing D3S-001 in patients with advanced solid tumours carrying the KRAS G12C mutation.
Early results show encouraging and durable responses according to RECIST criteria, alongside a favourable safety profile.
Source: prnewswire.com
