Laekna (2105.HK) has achieved a significant milestone as the U.S. Food and Drug Administration (FDA) grants Investigational New Drug (IND) approval for LAE120, their novel USP1 inhibitor developed for treating advanced solid tumors.
LAE120 represents a breakthrough in cancer treatment as a highly potent, allosteric USP1 inhibitor with a unique chemical structure that distinguishes it from existing inhibitors. The drug has demonstrated impressive efficacy both as a monotherapy and in combination with PARP inhibitors, particularly in cases of homologous recombination deficiency (HRD) cancers.
Robust Preclinical Evidence
The compound has shown significant tumor inhibitory activity across multiple xenograft models, including MDA-MB-436 and K562. Notably, LAE120's favorable safety profile has been confirmed through GLP long-term toxicology studies, establishing promising therapeutic windows for clinical application.
Dr. Justin Gu, Chief Scientific Officer of Laekna, emphasized the company's innovative approach: "Leveraging our deep know-how and extensive expertise in drug discovery, Laekna has developed a distinctive portfolio through close collaboration of our Med Chem, Biology, and AIDD teams. Our utilization of cutting-edge artificial intelligence tools has significantly accelerated the drug discovery process."
Expanding Pipeline with LAE118
In parallel developments, Laekna is advancing LAE118, a potentially best-in-class mutant-selective PI3Kα inhibitor, through IND-enabling studies. This compound addresses a critical need in treating cancers with PI3Kα mutations, which are prevalent in breast, colorectal, lung, and endometrial cancers.
LAE118's selective targeting of mutant PI3Kα, while sparing the wild-type variant, potentially offers superior efficacy and tolerability compared to first-generation inhibitors. The company presented promising preclinical data at the San Antonio Breast Cancer Symposium in December 2024, with plans to file an IND in the fourth quarter of 2025.
Strategic Growth and Partnerships
Dr. Chris Lu, Chief Executive Officer of Laekna, outlined the company's strategic vision: "We will continue to advance and expand our product portfolio in therapeutic areas where we have accumulated tremendous experience." The company recently established a clinical collaboration with Lilly (NYSE:LLY) for LAE102 in obesity treatment, demonstrating its commitment to strategic partnerships for accelerating global clinical development.
Laekna's progress with LAE120 and its expanding pipeline represents significant advancement in developing targeted cancer therapies, particularly for patients with limited treatment options. The company is actively seeking partnerships to expedite LAE120's clinical development and bring innovative treatments to patients worldwide.
