USP1

Boehringer Ingelheim is paying an upfront fee, of publicly undisclosed size, for global rights to the ALT program.\n Boehringer Ingelheim has slipped another piece of its oncology R&D strategy into place, partnering with Tessellate Bio on a synthetic lethal program in a deal worth more than 500 million euros ($570 million) in biobucks. European biotech Tessellate exited stealth in 2023 with a focus on novel synthetic lethality approaches. PARP inhibitors have shown the potential to shrink tumors by targeting pairs of genes that are essential for the survival of cancer cells. Companies including AstraZeneca and Merck KGaA are studying forms of synthetic lethality beyond the homologous recombination deficiency pathway targeted by PARP drugs. Tessellate has found a largely unclaimed piece of territory, namely tumors that depend on alternative lengthening of telomeres (ALT) for their growth. About 10% to 15% of cancers rely on ALT, according to the biotech. ALT-positive tumors are associated with poor prognosis and a lack of targeted therapies. Boehringer is paying an upfront fee, of publicly undisclosed size, for global rights to the ALT program. Once milestones and other fees are factored in, the value of the agreement could swell to more than 500 million euros.  Telomeres, the caps at the ends of chromosomes, shorten with each cell division and limit the life span of cells. Some cancers, including many sarcomas, gliomas and neuroepithelial tumors, use ALT to maintain telomere length. Tumors’ reliance on ALT creates opportunities to selectively kill cancer cells by targeting the mechanism. Tessellate said it has developed inhibitors of an undisclosed target that helps enable uncontrolled growth of ALT-positive cancer cells. Blocking the target drives DNA damage, replication stress and cell death, the biotech said. When it exited stealth, Tessellate said its lead ALT program targeted the FANCM protein complex. Studies have shown FANCM suppresses DNA replication stress at ALT telomeres. Artios Pharma, another European biotech, has a discovery-stage ALT program, but the industry’s efforts to expand synthetic lethality have largely focused on other pathways to date. AstraZeneca, which helped put PARP inhibitors on the map, is running trials of drug candidates that hit targets including ATR. Other targets include WEE1, USP1 and POLθ.    

SHANGHAI, April 18, 2025 /PRNewswire/ -- Asieris Pharmaceuticals today released its 2024 Annual Report, highlighting a bold and determined journey forward. With a specialty pharma strategy and operational efficiency, the company exceeded its commercialization targets and advanced its innovative products — APL-1702 and APL-1706 (Hexvix®) — toward market launch. R&D efforts also gained strong momentum, with 12 products in the pipeline and 16 ongoing research projects. As of the end of the reporting period, Asieris held approximately RMB 1.89 billion in cash, cash equivalents, and trading financial assets, providing a solid foundation for continued growth. Revenue Tops RMB 200M in 1st Year of Commercialization, with the specialized commercialization platform poised for action 2024 marked Asieris' first full year of commercialization. The company has made strides in refining market strategies and ensuring high-quality execution, while effectively managing costs and expenses. Asieris posted operating revenue of RMB 202 million, demonstrating robust growth and successfully reaching break-even targets in commercial operations. During the reporting period, the company's two commercialized products, Ouyoubi and Dipaite, saw strong uptake, reaching over 1,000 and 500 target hospitals respectively. Sales grew rapidly, with Ouyoubi capturing 27% of the neratinib tablet market and Dipaite securing 12% of the pazopanib tablet market. To further strengthen its presence in key focus areas, the company introduced multiple products and established sales partnerships in women's health and genitourinary (GU) tumors. In March 2025, it licensed-in the eribulin mesylate injection, a novel microtubule inhibitor for the treatment of advanced breast cancer. With this addition, the company's breast cancer portfolio now covers both early and late-stage disease. To further enhance commercialization efficiency, the company has launched its Commercial Operation 2.0 — building on past successes to create a leaner, more agile operating model. Key initiatives include appointing a seasoned Chief Commercial Officer to lead the function end-to-end; streamlining the organizational structure and strengthening commercial capabilities; and strategically expanding the team in line with priority areas and product launch timelines. The company has also set clear five-year business targets, including accelerating growth for existing products, preparing for the commercial rollout of Hexvix® and APL-1702, introducing new products within core therapeutic areas, maximizing synergies across the pipeline, and driving sales efficiency to the next level. Accelerating product launches to build momentum for sales In women's health, APL-1702, a combination of drug and medical device designed for photodynamic non-surgical treatment of cervical high-grade squamous intraepithelial lesions (HSIL), is making steady progress toward market approval. In May 2024, the product's marketing application was officially accepted by the National Medical Products Administration (NMPA). As of the report's release, the Center for Drug Evaluation (CDE), NMPA had completed the first round of technical reviews, covering toxicology, clinical data, biostatistics, clinical pharmacology, and pharmaceutical quality. During the reporting period, the Inspection Center also wrapped up its Good Clinical Practice (GCP) inspection. The company has since received written feedback and is actively coordinating its responses to help expedite the review process and bring APL-1702 one step closer to approval. As a first-in-class treatment set to debut in China, APL-1702 is on track to become the world's first non-invasive therapy for HSIL — supported by robust clinical evidence and validated through an international Phase III trial. Experts note that, with China facing demographic pressures and a rising incidence of cervical intraepithelial neoplasia (CIN) among younger women, surgical excision of the cervix poses increasing challenges — especially during the critical period of fertility preservation. APL-1702 is expected to bridge this critical treatment gap and contribute to the creation of a more fertility-friendly healthcare landscape. To drive the commercialization of APL-1702, the company has hosted eight expert advisory meetings, focusing on Phase III clinical data, the current landscape of HSIL diagnosis and treatment, and unmet clinical needs. These sessions earned strong endorsement from leading gynecological and pharmaceutical experts, who recognized the innovation and clinical value APL-1702 brings to the table. The company has also made meaningful progress in building strategic partnerships with government agencies and leading academic associations. In collaboration with the Cancer Foundation of China, the company unveiled the first real-world showcase of its innovative cervical cancer prevention and control system at the 7th China International Import Expo (CIIE). It also signed medium- to long-term strategic cooperation agreements with both the China Women's Development Foundation and the Cancer Foundation of China during the event. The partnerships are set to roll out in 2025. The Women's Health Division has conducted extensive market and industry research, gaining deep insights into the needs of doctors and patients and broader commercial landscape. Based on this analysis, the team has finalized key launch strategies and mapped out a commercialization roadmap, laying the groundwork for a swift market rollout once approval is secured. Given the relative scarcity of innovation in the gynecological field, APL-1702 stands out as a meaningful breakthrough, backed by solid clinical evidence and proven efficacy. The company will continue to prioritize APL-1702 and its photodynamic drug-device combination platform, in a bid to build a comprehensive, tiered gynecological portfolio. This strategy will center on product iteration and indication expansion, while leveraging synergies across the pipeline to drive broader growth. In urological tumors, after the marketing application for Hexvix® was accepted in November 2023, the company's dynamic and highly skilled team sprang into action to support the review and approval process. As a result, Hexvix® received NMPA approval in November 2024, seven months ahead of the expected timeline. The product has since made history as the first diagnostic imaging agent approved in China for photodynamic blue light cystoscopy in bladder cancer detection. The Oncology Business Unit is fine-tuning the launch strategy for Hexvix®, initially targeting patients with commercial insurance or those willing to pay out-of-pocket. The early rollout will cover the special and international medical departments of top-tier hospitals in major cities, as well as premium foreign-invested and private urology specialty hospitals. As clinical guidelines are released and endorsements from leading urology experts are established, the launch will be paired with the introduction of disposable blue light cystoscopes. Together, these efforts aim to accelerate the adoption of blue light cystoscopy in general hospitals and foster an integrated approach to the diagnosis and treatment of bladder cancer. Focusing on strategic expansion in key areas and fast-tracking high-impact R&D initiatives Aligned with its strategic vision, Asieris has made significant strides in clinical development in 2024, with its global clinical initiatives progressing efficiently. In the fields of women's health, breast cancer, and gynecological tumors, Asieris has made remarkable progress. Building on the strong results of the international multicenter Phase III study of APL-1702, the company received feedback from its communication meeting with the U.S. FDA in December 2024, culminating in an agreement on a new Phase III clinical design to support the U.S. launch of APL-1702. Asieris is now actively seeking international partners to advance the U.S. Phase III clinical trial application. Additionally, APL-1702 is also being investigated for its potential in HPV virus clearance. Meanwhile, the U.S. FDA and China's NMPA approved the Phase I/IIa clinical trial applications for APL-2302 (a USP1 small molecule inhibitor) in advanced solid tumors in October 2024 and January 2025, respectively, with the first patient enrolled in Phase Ia in March 2025. Progress on APL-2501 (CLDN6/9 ADC) is also on track, with its preclinical research and unique linker platform selected for a poster presentation at the 2025 American Association for Cancer Research Annual Meeting (AACR 2025). In the field of urologic oncology, the Phase II clinical trial of oral APL-1202 in combination with tislelizumab as a neoadjuvant therapy for muscle-invasive bladder cancer (MIBC) was successfully completed in September 2024, delivering encouraging efficacy signals. Among protocol-eligible patients, the pathological complete response (pCR) rates were 41% for the combination arm versus 20% for monotherapy. The combination was particularly effective in patients with low PD-L1 expression, where it achieved a pCR rate of 42% compared to just 11% with monotherapy. Meanwhile, APL-2401 — a dual FGFR2/3 small molecule inhibitor — has entered IND-enabling studies, with its findings selected for a poster presentation at AACR 2025. In addition, the investigational new drug (IND) application for APL-1202 in the treatment of free-living amoebae (FLA) infections was approved by the NMPA in June 2024. In January 2025, the company signed an investigational drug supply agreement under the Expanded Access IND Program with the Centers for Disease Control and Prevention (CDC) in the United States for APL-1202, subject to evaluation by CDC experts, to be used for treating FLA infections. Dr. Kevin Pan, Founder, Chairman and CEO of Asieris Pharmaceuticals, commented, "2024 marks our first full year of commercialization. We delivered strong growth across our marketed products and reached a milestone by breaking even in our commercial operations. Our pipeline is entering a critical sprint toward market launch and is set to power the next wave of performance growth. While reinforcing our commercial capabilities, we're also accelerating clinical programs with a strategic focus on women's health and urological oncology. We're building a robust pipeline of first-in-class and highly-differentiated fast-follow therapies, while actively expanding into global markets to unlock the full value of our assets. With relentless innovation and execution, we are confident that Asieris will continue to make a meaningful difference for patients, deliver solid returns to shareholders, and create lasting value for society." Note: As of the date of this publication, APL-1702 has not been approved for the treatment of cervical high-grade squamous intraepithelial lesions (HSIL). This article is intended to disclose the latest development of these products, not to serve as a promotional advertisement for the products. The relevant information is not intended for patients but is provided solely for the reference of healthcare professionals. If you like to learn more about these diseases, please consult healthcare professionals. SOURCE Asieris WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

– 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 March 3, 2025 — 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. 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.” - End – 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.