On October 20, 2025 Iambic Therapeutics, a clinical-stage life science and technology company developing novel medicines using its AI-driven discovery and development platform, reported clinical data for IAM1363, a brain-penetrant HER2 small-molecule tyrosine kinase inhibitor (TKI), which showed anti-tumor activity in heavily pretreated patients across multiple disease types, encompassing HER2-amplified, HER2-overexpressing, and HER2-mutant tumors. The data are being presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Berlin.
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"IAM1363 marks one of the first clear demonstrations of a drug candidate with compelling clinical activity and safety from a TechBio company," said Tom Miller, PhD, Co-Founder and CEO of Iambic. "This milestone not only reinforces the potential of IAM1363 as a best-in-class TKI for HER2-driven cancers but also validates the power of our platform to translate model predictions into meaningful clinical impact. With this foundation, we are advancing a robust pipeline of development candidates for both Iambic and our partners, redefining what’s possible in drug discovery and development."
IAM1363 is a potent, irreversible type II HER2 inhibitor, highly differentiated by its target selectivity (>5,000-fold HER2 vs. EGFR selectivity), brain penetrance, pan-mutant activity, and tumor enrichment. Initial clinical data show activity in patients with HER2-mutant NSCLC and HER2-positive breast and gastric cancers, as well as indications lacking approved HER2-directed TKIs or antibodies, such as HER2-mutant renal cell cancer and HER2-amplified NSCLC and ovarian cancer. Best overall response (BOR) data in participants with HER2-targetable alterations dosed at ≥960 mg qd, as assessed by RECIST 1.1 and RANO-BM, showed partial responses in 28% (N=18) with measurable systemic disease and in 33% (N=3) with measurable intracranial tumors.
The data further validate Iambic’s AI platform and clinical execution, with the novel molecule advancing from program start to clinical trial initiation in just two years, and initial proof of concept clinical data now reported just over a year later.
"We are encouraged by IAM1363’s safety and pharmacokinetic profile and, importantly, to see tumor reductions in patients who had exhausted all standard-of-care options," said Neil Josephson, MD, Iambic’s Chief Medical Officer. "These promising data support our plans to evaluate IAM1363 in both monotherapy and combination cohorts."
The Phase 1/1b trial, NCT06253871, is an open-label, multi-center, dose escalation and dose optimization study, designed to evaluate tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of IAM1363 in patients with advanced HER2 cancers. The study, which has multiple sites in the U.S., recently opened in the EU and will continue to expand into additional sites across the U.S. and EU and into the UK and APAC in Q4 2025.
(Press release, Iambic Therapeutics, OCT 20, 2025, View Source [SID1234656844])