Laekna Announces IND Approval of LAE120 (a Novel USP1 Inhibitor) for Treatment of Advanced Solid Tumors by FDA

• A novel, allosteric and highly potent USP1 inhibitor, LAE120 exhibits robust tumor inhibitory activity across various xenograft models
• Significantly accelerated the progress of drug discovery leveraging cutting-edge AI models
• Another potential anti-tumor drug candidate, LAE118 (a mutant-selective PI3Kα inhibitor) also in IND-enabling studies

Laekna (2105.HK) announced that the U.S. Food and Drug Administration (FDA) has approved the IND for LAE120, an internally discovered USP1 inhibitor, for the treatment of advanced solid tumors.

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Laekna (2105.HK) announced that the FDA has approved the IND for LAE120, an internally discovered USP1 inhibitor, for the treatment of advanced solid tumors. (Photo: Business Wire)

LAE120 is a novel, allosteric and highly potent USP1 inhibitor, displaying monotherapy potency and combination activity with PARP inhibitor in HRD (homologous recombination deficiency) cancers. It has a unique chemical structure differentiated from all the other disclosed USP1 inhibitors and is expected to induce a different conformational change in USP1. LAE120 shows robust tumor inhibitory activity across various xenograft models such as MDA-MB-436 and K562 as a single agent and exhibits synergistic effect in combination with PARP inhibitors. It also demonstrates good therapeutic windows in GLP long-term toxicology study. Laekna is actively exploring partnerships to accelerate the clinical development of LAE120.

"Leveraging our deep know-how and extensive expertise in drug discovery, Laekna has developed a distinctive portfolio of innovative drug candidates through the close collaboration of our Med Chem, Biology and AIDD (AI-driven Drug Discovery) teams, continuously advancing preclinical drug candidates into clinical stage. Laekna has also significantly accelerated the progress of drug discovery by utilizing cutting-edge artificial intelligence tools," said Dr. Justin Gu, Chief Scientific Officer of Laekna. "We look forward to bringing novel drugs to patients as swiftly as possible," he added.

Advancing Diversified Pipelines

Laekna is actively advancing preclinical drug candidates. In the fourth quarter of 2024, another internally discovered anti-tumor drug candidate, LAE118, a potentially best-in-class, mutant-selective PI3Kαinhibitor, has advanced to IND-enabling study. PI3Kα mutations are prevalent in patients with breast, colorectal, lung, endometrial, and numerous other cancers. However, the first-generation drugs targeting PI3Kα inhibit the wild-type and mutant PI3Kα with equal potency, which raises concerns of tolerability and therapeutic efficacy.

As a novel allosteric inhibitor, LAE118 demonstrates excellent potency and selectivity towards various PI3Kα mutants. With superior anti-cancer efficacy and tolerability than other current PI3Kα inhibitors, LAE118 is potentially the best-in-class pan-mutant-selective PI3Kα inhibitor. Laekna has presented the preclinical characterization of LAE118 at the San Antonio Breast Cancer Symposium (SABCS) in December 2024. LAE118 is in IND-enabling studies and IND is expected to be filed in the fourth quarter of 2025.

Strategic Partnerships to Accelerate Globalization

"We will continue to advance and expand our product portfolio in the therapeutic areas where we have accumulated tremendous experience and extensive know-how," said Dr. Chris Lu, Chief Executive Officer of Laekna. "In November 2024, the Group has entered into a clinical collaboration agreement with Lilly (NYSE:LLY) to support and accelerate global clinical development of LAE102 for the treatment of obesity. We plan to pursue strategic partnerships with global leading pharmaceutical companies to accelerate clinical development and commercialization of our drug candidate assets. We keep advancing and expanding our pipeline and are committed to bringing life-changing medicines to more people around the world," he added.

About Laekna

Stock Code: 2105.HK

Founded in 2016, Laekna is a science-driven, clinical-stage biotechnology company committed to bringing novel therapeutics to patients with cancer, metabolic diseases and liver fibrosis patients around the world.

As of June 30, 2024, Laekna has initiated seven clinical trials for LAE102, LAE002(afuresertib), LAE001 and LAE005 to address unmet medical needs in obesity and cancers.

LAE102 is our internally discovered antibody against ActRIIA. It has been shown in the pre-clinical studies to increase lean mass and decrease fat mass. We've obtained IND approvals from the FDA and the CDE for LAE102 in obesity indication and are advancing the Phase I clinical trial in China.

Blocking Activin-ActRII pathway could promote muscle regeneration and decrease fat mass. Laekna team has accumulated tremendous experiences and deep know-how in this specific field and is developing more drug candidates (LAE103 and LAE123), in addition to LAE102, to maximize the value of targeting ActRII receptors.

In the cancer area, Laekna has built a comprehensive portfolio of drug candidates, covering the treatment of breast cancer, prostate cancer, ovarian cancer and PD-1/ PD-L1 drug-resistant solid tumors. LAE002 (afuresertib) is a potent AKT inhibitor that inhibits all three AKT isoforms (AKT1, AKT2 and AKT3) as well as one of the only two AKT inhibitors in late-stage development for breast and prostate cancer globally. Laekna has commenced the Phase III clinical trial (AFFIRM-205) for LAE002 in patients with HR+/HER2- breast cancer.

Laekna, Inc. (2105.HK) was listed on the Main Board of The Stock Exchange of Hong Kong Limited (the "Hong Kong Stock Exchange") on June 29, 2023.

For more information, please visit: https://www.laekna.com/ or https://www.linkedin.com/company/74110713/

Forward-Looking Statements

This press release may contain certain "forward-looking statements" which are not historical facts, but instead are predictions about future events based on Laekna's current beliefs, assumptions and expectations, commonly identified by words such as "would", "may", "expects", "believes", "plans", "intends", "projects" and other terms with similar meaning. Although we believe that our predictions are reasonable, future events are inherently uncertain and our actual future results or performance may be materially different from what we expect. Accordingly, you are strongly cautioned that reliance on any forward-looking statements is subject to significant known and unknown risks and uncertainties. All forward-looking statements contained herein are qualified by reference to the cautionary statements set forth in this section. All information provided in this press release is as of the date of this press release and are based on assumptions that we believe to be reasonable as of this date, and we do not undertake any obligation to update any forward-looking statement, except as required under applicable law.

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