AI-designed oncology drug candidate IAM1363 showed clinical anti-tumor activity, safety, and tolerability
IAM1363 shown to be active across both HER2-wild-type and HER2-mutated cancers, and in multiple disease indications; responses seen in patients previously treated with T-DXd and tucatinib
IAM1363 brought to clinic in under two years, highlighting the potential of Iambic’s technology platform to rapidly discover and develop differentiated drug candidates
Iambic Therapeutics, a clinical-stage life science and technology company developing novel medicines using its AI-driven discovery and development platform, today announced 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) Congress in Berlin.
"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.
About Iambic
Iambic is a clinical-stage life-science and technology company developing novel medicines using its AI-driven discovery and development platform. Based in San Diego and founded in 2020, Iambic has assembled a world-class team that unites pioneering AI experts and experienced drug hunters. The Iambic platform has demonstrated delivery of new drug candidates to human clinical trials with unprecedented speed and across multiple target classes and mechanisms of action. Iambic is advancing a pipeline of potential best-in-class and first-in-class clinical assets, both internally and in partnership, to address urgent unmet patient need. Learn more about the Iambic team, platform, pipeline, and partnerships at Iambic.ai.
About Iambic’s AI-Driven Discovery Platform
The Iambic AI-driven platform was created to address the most challenging design problems in drug discovery, leveraging technology innovations such as Enchant (multimodal transformer model that predicts clinical and preclinical endpoints) and NeuralPLexer (best-in-class predictor of protein and protein-ligand structures). The integration of physics principles into the platform’s AI architectures improves data efficiency and allows molecular models to venture widely across the space of possible chemical structures. The platform enables identification of novel chemical modalities for engaging difficult-to-address biological targets, discovery of defined product profiles that optimize therapeutic window, and multiparameter optimization for highly differentiated development candidates. Through close integration of AI-generated molecular designs with automated chemical synthesis and experimental execution, Iambic completes design-make-test cycles on a weekly cadence.
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