FDA Approves Laekna's IND for LAE120 to Treat Advanced Solid Tumors

On March 3, 2025, Laekna (2105.HK) announced a significant milestone with the U.S. Food and Drug Administration's approval of the Investigational New Drug (IND) application for LAE120. This innovative drug, a potent USP1 inhibitor developed internally by Laekna, is designed for the treatment of advanced solid tumors. LAE120 stands out due to its novel allosteric mechanism and potent activity, both as a monotherapy and in combination with PARP inhibitors, particularly in cancers exhibiting homologous recombination deficiency (HRD). Its unique chemical structure distinguishes it from other known USP1 inhibitors, suggesting it may induce different conformational changes in the target protein. Impressively, LAE120 has demonstrated robust tumor suppression in a variety of xenograft models, including MDA-MB-436 and K562, and shows a synergistic effect when used with PARP inhibitors. Moreover, long-term toxicology studies indicate a favorable therapeutic window for LAE120, underscoring its potential as a safe and effective cancer treatment. Laekna is actively seeking partnerships to expedite the clinical development of this promising candidate.

Dr. Justin Gu, Laekna's Chief Scientific Officer, highlighted the company's strategic use of advanced artificial intelligence in drug discovery, which has significantly sped up the development process. By leveraging AI and the expertise of its Med Chem, Biology, and AI-driven Drug Discovery (AIDD) teams, Laekna is rapidly moving preclinical drug candidates into clinical trials. Dr. Gu expressed optimism about the company's ability to bring innovative drugs to patients quickly.

In addition to LAE120, Laekna is making progress with other promising drug candidates. Notably, LAE118, another internally developed anti-tumor candidate, has progressed to IND-enabling studies. LAE118 is a mutant-selective PI3Kα inhibitor, potentially best-in-class, that targets prevalent PI3Kα mutations found in various cancers, including breast, colorectal, lung, and endometrial cancers. Unlike first-generation PI3Kα inhibitors, which affect both wild-type and mutant forms equally and pose tolerability issues, LAE118 is designed to selectively inhibit cancer-associated mutants, showing superior efficacy and tolerability. The preclinical characteristics of LAE118 were presented at the San Antonio Breast Cancer Symposium in December 2024, with an IND filing anticipated in late 2025.

Laekna is also focused on strategic partnerships to boost its global clinical development efforts. In November 2024, the company entered into a collaboration with Lilly (NYSE: LLY) aimed at advancing the clinical development of LAE102, an investigational treatment for obesity. Laekna is committed to further expanding its product pipeline through strategic collaborations with leading pharmaceutical firms, with the goal of accelerating the development and commercialization of its innovative drug candidates.

Founded in 2016, Laekna is a biotechnology company dedicated to developing novel therapeutics for cancer, metabolic diseases, and liver fibrosis. As of mid-2024, the company had initiated multiple clinical trials for various drug candidates, including LAE102, LAE002 (afuresertib), LAE001, and LAE005, addressing unmet medical needs in obesity and cancer. LAE102, an antibody targeting ActRIIA, has shown promise in preclinical studies and is currently in Phase I trials for obesity in China, following IND approvals from both the FDA and China's CDE. By targeting the Activin-ActRII pathway, Laekna aims to promote muscle regeneration and reduce fat mass, leveraging its deep expertise in this area to develop additional promising candidates such as LAE103 and LAE123.

In the realm of cancer treatment, Laekna has developed a comprehensive portfolio targeting various cancers, including breast, prostate, ovarian, and drug-resistant solid tumors. LAE002 (afuresertib), a potent AKT inhibitor in late-stage development, is undergoing Phase III trials for HR+/HER2- breast cancer, showcasing the company's commitment to addressing critical needs in oncology. Laekna continues to advance its diverse pipeline, with a steadfast commitment to delivering transformative medicines to patients worldwide.