STEAP1

Our analysis of February data reveals key trends in global preclinical-stage biopharmaceutical financing. For contextual understanding, we recommend beginning with the following article. Highlights of Financing and Strategic Collaborations in the Preclinical Biopharmaceutical Sector: February 2025(1) 12.Harbour BioMed and Insilico Medicine Form Strategic Partnership to Advance AI-Driven Antibody Discovery and Development Harbour BioMed and Insilico Medicine have established a strategic partnership aimed at accelerating the development of therapeutic antibodies by integrating artificial intelligence (AI) with antibody research. This collaboration seeks to leverage AI’s potential in antibody applications and target-specific research, aiming for breakthrough advancements in biopharmaceutical innovation. The partnership not only underscores AI’s growing importance in drug discovery but also highlights the transformative power of cross-industry collaboration in driving innovation. According to the agreement, both companies will combine Harbour BioMed’s expertise in antibody development with Insilico Medicine’s AI-driven drug discovery platform. Notably, Insilico Medicine’s Integrated Drug Creation™ platform, which utilizes generative AI and a scalable wet lab system, can significantly reduce the time from data collection to molecular design and experimental validation to approximately six weeks. This highly efficient research process dramatically accelerates drug discovery and facilitates rapid clinical trial progression for promising drug candidates. The initial phase of the collaboration will focus on specific disease targets, although financial terms and drug details have not been disclosed. However, given that similar partnerships in the industry often involve multimillion or even billion-dollar deals, both companies are expected to make substantial investments to ensure the project’s success. Additionally, Insilico Medicine has already achieved significant milestones in collaborations with major pharmaceutical companies, such as AstraZeneca, which suggests that its partnership with Harbour BioMed could generate substantial economic and social value. As AI continues to reshape the healthcare industry, more pharmaceutical companies are exploring ways to integrate AI into their R&D pipelines. The Harbour BioMed-Insilico Medicine collaboration exemplifies this trend, demonstrating how AI-driven solutions can enhance global competitiveness and set a benchmark for industry innovation. Looking ahead, similar cross-sector collaborations are expected to emerge, further propelling advancements in biopharmaceutical research. Despite its immense potential, AI-driven drug discovery still faces challenges, including data privacy concerns, algorithmic transparency issues, and regulatory uncertainties. While AI accelerates research efficiency, stakeholders must work collectively to address these challenges. Through continuous technological innovation and the establishment of robust regulatory frameworks, AI is poised to play an increasingly pivotal role in future drug discovery and development, ultimately providing more effective treatment options for patients worldwide. 13. Epitopea Partners with MSD to Develop RNA-Based Cancer Immunotherapies On February 19, 2025, transatlantic cancer immunotherapy company Epitopea announced a licensing and research collaboration agreement with Merck & Co. (MSD) to identify Cryptigen™ tumor-specific antigens (TSAs) in an undisclosed solid tumor type. These antigens originate from non-coding regions previously considered "junk DNA" and exhibit shared, non-mutated, and aberrant expression across multiple patients. Through this collaboration, the two companies aim to leverage Epitopea's proprietary CryptoMap™ platform to discover novel immunogenic Cryptigen™ TSAs and develop off-the-shelf RNA-based immunotherapies for various refractory cancers. Under the terms of the agreement, Epitopea will use its CryptoMap™ platform to identify and provide novel Cryptigen™ TSAs for predefined tumor types. MSD will have exclusive rights to develop and commercialize any therapeutic products resulting from the collaboration. In return, Epitopea will receive an undisclosed upfront payment and be eligible for milestone payments of up to $300 million per product. This financial arrangement not only highlights MSD’s commitment to the project but also underscores the immense potential of Epitopea’s technology platform. At the core of Epitopea’s technology is its CryptoMap™ platform, which integrates immunopeptidomics, genomics, and bioinformatics pipelines to identify aberrantly expressed tumor-specific antigens hidden within cancer "junk" DNA. This innovative approach opens new avenues for developing highly effective immunotherapies targeting specific tumor types. Furthermore, since these antigens are commonly present across multiple patients, therapies based on them can be applied to a broader patient population, enhancing accessibility and efficacy. This collaboration not only validates Epitopea’s technological capabilities but also positively impacts the entire cancer immunotherapy field. As more partnerships of this kind emerge, the number of therapies targeting newly discovered tumor-specific antigens is expected to grow significantly in the coming years. For MSD, this collaboration strengthens its leadership in immuno-oncology and could yield a series of breakthrough therapies. At the same time, it marks a step forward in the transition from traditional chemotherapy to more personalized and precise cancer treatments. Since its founding, Epitopea has garnered support from top life sciences investors, including Advent Life Sciences, CTI Life Sciences, and Cambridge Innovation Capital. To date, the company has raised over $45 million, providing a strong financial foundation for its R&D activities. Epitopea plans to accelerate the development of its preclinical pipeline as it moves toward becoming a clinical-stage company. With the deepening collaboration with MSD, Epitopea is poised to introduce a range of innovative cancer immunotherapies in the coming years, offering hope to cancer patients worldwide. 14. Polymed Partners with Photys Therapeutics for Global Development of HPB-143 On February 18, 2025, Polymed announced a major partnership with Photys Therapeutics, granting Photys the rights to develop, manufacture, and commercialize the IRAK4-targeting protein degrader HPB-143 in all global markets except Greater China and Southeast Asia. In return, Polymed will receive multiple forms of compensation, including upfront and near-term payments, clinical development milestone payments, and sales royalties, while also holding an equity stake in Photys. This collaboration aims to accelerate the development of HPB-143 and expand its global market presence. Compared to the IRAK4-targeting protein degrader KT-474, which is currently in Phase II clinical trials internationally, Polymed’s HPB-143 has demonstrated superior oral bioavailability, reaching 49% in mouse studies. Additionally, it exhibits weaker inhibition of the cardiac ion channel hERG, suggesting improved cardiovascular safety in humans. In multiple mouse disease models, HPB-143 has shown lower effective doses and superior efficacy. As a PROTAC (Proteolysis-Targeting Chimera), HPB-143 specifically degrades the IRAK4 protein by hijacking the intracellular ubiquitin-proteasome system, effectively modulating key signaling pathways involved in inflammatory responses and tumor progression. IRAK4 plays a crucial role in human inflammatory responses and tumorigenesis, making it a highly promising target for protein degraders in treating autoimmune diseases such as atopic dermatitis and hidradenitis suppurativa. The therapeutic demand for these conditions remains largely unmet, with existing treatments often limited by efficacy or side effects. With its unique mechanism of action and superior pharmacological profile, HPB-143 has the potential to provide a safer and more effective treatment option for patients. HPB-143 has already secured approval from both the U.S. FDA and China's National Medical Products Administration (NMPA) to conduct clinical trials. According to Photys Therapeutics' plan, a Phase I clinical study for HPB-143 is set to launch this year, marking a critical step in evaluating the drug's safety, tolerability, and preliminary efficacy. As clinical trials progress, HPB-143 has the potential to become one of the first-in-class IRAK4 degraders to reach the market, offering new hope for patients with autoimmune diseases worldwide. This partnership not only reflects the global recognition of Polymed’s PROTAC drug development platform but also signifies the commitment of both companies to advancing HPB-143’s development through resource integration. Photys Therapeutics, leveraging its proprietary PHICS™ technology and other proximity-based modalities, is dedicated to developing a pipeline of therapies targeting cancer, autoimmune diseases, and cardiometabolic disorders. Through this collaboration, both companies have the opportunity to expand their product portfolios and achieve multiple key development milestones in the coming years. For the broader pharmaceutical industry, this partnership serves as a positive signal of the potential of innovative therapies in addressing unmet medical needs. 15. Perceive Pharma Secures $15 Million Series A Funding to Advance Ophthalmic Neuroprotective Therapy On February 18, 2025, Perceive Pharma, a company focused on developing novel small-molecule ophthalmic therapeutics, announced the completion of a $15 million Series A funding round. The financing was backed by several renowned investment firms, including Deerfield Management, Johnson & Johnson Innovation – JJDC, Inc., Braidwell LP, GV, Retinal Degeneration Fund, and Catalio Capital Management, LP. The funds will be used to advance its glaucoma program to the clinical readiness stage and further expand its research pipeline. One of Perceive Pharma’s core products is PBI-671, a first-in-class drug candidate designed to prevent vision loss caused by glaucoma. All currently approved glaucoma treatments focus on reducing intraocular pressure (IOP), yet approximately half of glaucoma patients continue to experience vision loss despite having normal IOP. PBI-671 is based on a neuroprotective pathway first identified through genomic screening and aims to directly protect retinal cells, offering a potential treatment for patients whose disease continues to progress despite existing therapies. Perceive Pharma's research and development efforts are built on a strong scientific foundation, providing the company with a unique advantage in identifying new therapeutic targets and developing transformative vision-protection therapies. With the successful completion of its Series A financing, the company will accelerate the advancement of its lead therapy to address the medical needs of more than 1.5 million patients worldwide suffering from progressive glaucoma. Additionally, Perceive Pharma plans to explore treatment opportunities for other undisclosed diseases. Notably, Perceive Pharma is part of JLABS @ San Diego, a division of Johnson & Johnson Innovation – JLABS, a global life sciences incubator network. Through this platform, startups gain access to cost-efficient laboratory space, equipment resources, and expert support, helping to accelerate the development of innovative products. Such support is crucial for emerging companies like Perceive Pharma as they navigate critical growth and development stages. 16. Lexenpharm and Sun-Novo Pharmaceutical Join Forces to Revolutionize Asthma and COPD Treatment Recently, Lexenpharm and Sun-Novo Pharmaceutical announced a regional strategic partnership for the development of two innovative drug projects, LXP2311 and LXP0531, in mainland China. Through this collaboration, both companies will leverage their technological expertise to advance the research, development, and commercialization of these breakthrough therapies, bringing new hope to the medical industry. Current COPD treatments primarily rely on short-acting or long-acting monotherapies, which often have limited efficacy and duration of action. To address this challenge, Lexenpharm and Sun-Novo have jointly developed LXP2311 and LXP0531, the world's first ultra-long-acting combination inhalation formulations. In particular, LXP2311, the first ultra-long-acting LABA/LAMA combination therapy, requires only once-daily administration and can be delivered via a standard jet nebulizer, significantly enhancing patient convenience. Meanwhile, LXP0531 is the first ultra-long-acting ICS/LABA combination therapy, recommended for the treatment of asthma and COPD, offering improved ease of use for both physicians and patients. The success of the LXP2311 and LXP0531 projects is supported by five core technologies, including nebulized inhalation formulation and soft mist inhalation technology. These advancements not only enhance drug efficacy and safety but also significantly improve the patient experience. For instance, the application of nano-scale sterile suspension technology and Blow-Fill-Seal (BFS) aseptic technology ensures efficient drug delivery and stability within the body, providing better disease control. These technological innovations are expected to have a substantial impact on improving treatment outcomes for asthma and COPD patients worldwide. Asthma and COPD remain major global public health concerns, with COPD ranking as the second leading disease in China and the third leading cause of death worldwide. It is estimated that COPD results in an annual global economic burden exceeding $4.3 trillion, with China alone bearing a financial burden of $1.36 trillion. Given the severity of this situation, the development of more effective and convenient treatment options is an urgent necessity. The partnership between Lexenpharm and Sun-Novo reflects their commitment to addressing this critical need by providing better therapeutic solutions for patients. 17.DAAN Biotherapeutics and LigaChem Biosciences Enter Exclusive Antibody Licensing Agreement to Advance ADC Therapy Development February 13, 2025, Seoul, South Korea – DAAN Biotherapeutics, a leading innovative drug development company specializing in T-cell receptor (TCR)-based therapies, has announced an exclusive licensing agreement with LigaChem Biosciences. This collaboration aims to develop a novel tumor-targeting antibody to facilitate the advancement and commercialization of antibody-drug conjugates (ADCs). By leveraging their respective technological strengths, the two companies will work together to drive progress in ADC therapies for solid tumor patients. With its cutting-edge next-generation antibody technology, superior target discovery, and antibody development capabilities, DAAN Biotherapeutics has successfully built a proprietary pipeline focused on developing antibodies targeting tumor markers overexpressed in solid cancers. This achievement is supported by its strategic partnership with OmniAb. Additionally, DAAN has developed a platform called TACTIC (Tumor-Activated Conditional T-cell Connector), designed to be a global leader in solid tumor-targeted T-cell activators. These technological advancements provide a strong foundation for the development of ADC therapies. The collaboration between DAAN Biotherapeutics and LigaChem Biosciences represents an effective integration of their technologies and resources, marking a significant milestone in the field of solid tumor treatment. It is expected to accelerate the development of novel ADC therapies and provide patients with more effective treatment options. As the partnership deepens, more research findings are anticipated to translate into practical applications, contributing to the fight against cancer. Looking ahead, DAAN Biotherapeutics plans to continue expanding its collaborations with global pharmaceutical companies, particularly in the ADC sector. Furthermore, the company aims to achieve breakthroughs in T-cell therapies, especially through the development of novel T-cell connectors using its TACTIC platform. Through continuous exploration and innovation, DAAN Biotherapeutics is steadily advancing toward its goal of becoming a global leader in cancer treatment. 18.Xilio Therapeutics Announces Multiple Masked T-cell Engager Programs and Collaboration Agreement with AbbVie February 12, 2025 – Xilio Therapeutics, Inc., a clinical-stage biotechnology company focused on discovering and developing tumor-activated immunotherapies, has announced three wholly-owned preclinical programs targeting prostate-specific membrane antigen (PSMA), claudin 18.2 (CLDN18.2), and six-transmembrane epithelial antigen of the prostate 1 (STEAP1) with masked T-cell engagers. Additionally, Xilio has entered into a collaboration, licensing, and option agreement with AbbVie to leverage Xilio’s proprietary tumor-activated technology platform to develop novel tumor-activated immunotherapies. T-cell engagers are designed to direct immune effector cells against cancer cells by simultaneously binding tumor-associated antigens expressed on the surface of cancer cells and the T-cell receptor complex on T cells, thereby inducing T-cell-mediated tumor cell killing. However, despite their significant therapeutic potential in various advanced solid tumors, the widespread application of T-cell engagers has been limited by toxicity concerns. Xilio is utilizing its clinically validated tumor-activated platform to advance a series of novel masked T-cell engagers with conditionally regulated half-life properties, aiming to achieve potent, localized T-cell activation and tumor cell destruction while improving the therapeutic index. Xilio is currently progressing three wholly-owned preclinical programs, each targeting a distinct tumor-associated antigen: ·PSMA – a promising T-cell engager target for prostate cancer ·CLDN18.2 – a potential T-cell engager target for gastric, pancreatic, esophageal, and lung cancers ·STEAP1 – a viable target for prostate, colorectal, and lung cancers Xilio expects to nominate development candidates and submit Investigational New Drug (IND) applications for these programs in the coming years. Under the terms of the collaboration agreement with AbbVie, Xilio will receive a total upfront payment of $52 million, including $42 million in cash and $10 million in Xilio common stock purchased by AbbVie at a premium. Xilio has granted AbbVie an exclusive option to initiate an initial program for the discovery and development of masked T-cell engagers, with the potential for two additional programs to be launched under the agreement. Moreover, Xilio will be eligible for up to $2.1 billion in additional milestone payments, including option fees, as well as development, regulatory, and commercial sales milestones. Based on its current operational plans, Xilio expects its existing cash and equivalents, combined with the upfront payment from the AbbVie agreement, to fund its operating and capital expenditure needs until the first quarter of 2026. Xilio will hold an investor conference call and webcast on February 12, 2025, to provide further details on this announcement and invite investor participation. These strategic moves not only strengthen Xilio’s financial position but also ensure continued investment in future research and development. 19.Ellipses Pharma Acquires First-in-Class Next-Generation Immuno-Oncology Drug GENA-104 to Accelerate Cancer Treatment Development Ellipses Pharma Limited (Ellipses), a global drug development company dedicated to accelerating cancer treatment through innovative drug development models, announced on February 11, 2025, that it will develop a next-generation immuno-oncology drug aimed at addressing the needs of cancer patients who do not respond to existing checkpoint inhibitors. Ellipses has secured the global rights to ‘GENA-104,’ a first-in-class immuno-oncology monoclonal antibody targeting CNTN4, a recently identified checkpoint protein highly expressed across multiple tumor types. GENA-104 was originally discovered by the South Korea-based biotechnology company Genome & Company (Genome), which specializes in the development of novel cancer-targeting antibodies and antibody-drug conjugates (ADCs). Under the agreement with Genome, Ellipses will assume full responsibility for the future clinical development of GENA-104, designating it as EP0089. The potential of EP0089 as a novel immuno-oncology therapy was highlighted in a landmark study published in Science Immunology in October 2024. The Phase I IND application for EP0089 was approved by the South Korean Ministry of Food and Drug Safety in January 2024. Ellipses plans to initiate Phase I clinical trials in South Korea in 2025 and subsequently expand to the United States and Europe upon obtaining the necessary regulatory approvals. This new therapeutic approach aims to inhibit the CNTN4-APP checkpoint interaction on T cells, thereby enhancing tumor cell destruction, making it particularly promising for cancers resistant to traditional checkpoint inhibitors. This collaboration marks a significant milestone for Ellipses Pharma in expanding its pipeline and represents a crucial advancement in cancer treatment. By acquiring breakthrough candidates such as EP0089, Ellipses reaffirms its commitment to rapidly identifying and developing top-tier therapies to provide new treatment options for patients as quickly as possible. As further research data emerges and clinical trials progress, EP0089 has the potential to transform the treatment landscape for certain refractory cancers, offering new hope to patients worldwide. 20.Alloy Therapeutics Partners with Pfizer to Develop a New Antibody Discovery Platform Boston, February 11, 2025 – Alloy Therapeutics Inc. (Alloy), a biotechnology ecosystem company committed to democratizing access to cutting-edge drug discovery technologies, has announced its latest strategic collaboration with Pfizer. This partnership aims to develop a novel platform designed to enhance Pfizer’s ability to discover highly specific and effective antibodies for challenging targets that current antibody discovery technologies struggle to address. Under the terms of the agreement, Alloy will receive an upfront payment from Pfizer and will be eligible for pre-specified preclinical-to-commercial milestone payments related to products developed using the new platform. Alloy’s ATX-Gx platform has rapidly become the industry standard for fully humanized transgenic mice, utilized by over 170 partners to advance various therapeutic discovery programs. Alloy remains committed to reinvesting its revenue into innovation and has continuously expanded its murine platform by incorporating ATX-GL™, which features a complete human lambda library, and ATX-GKH™, an ultra-immune strain designed to enhance antigen-specific B-cell production and improve IgG class switching. In addition to this collaboration, Alloy and Pfizer have worked together on multiple antibody discovery initiatives, including the use of Alloy’s ATX-CLC platform. This platform enables the expression of common light-chain antibodies with full heavy-chain diversity, facilitating the efficient and modular discovery of bispecific antibodies. 21.Bolt Biotherapeutics and Toray Collaborate on a Novel ISAC Therapy Targeting Caprin-1 On February 11, 2025, Bolt Biotherapeutics announced a global collaboration with Toray Industries to co-develop a therapeutic targeting Caprin-1, a novel cancer target discovered by Toray. The two companies are working together to develop a Boltbody™ immune-stimulating antibody-drug conjugate (ISAC) targeting Caprin-1, intended for use in multiple solid tumor types. Under the existing joint development and licensing agreement, Toray is providing its proprietary Caprin-1-targeting antibody, TRK-950, while Bolt contributes its proprietary linker-payload technology from the Boltbody™ ISAC platform. Caprin-1 is a tumor-specific antigen that is highly expressed on the cell membrane of most solid tumors while exhibiting minimal expression in normal tissues. Additionally, Caprin-1 has been shown to promote tumor growth and metastasis. Toray’s asset, TRK-950, is a monoclonal antibody targeting Caprin-1 and is currently in Phase II development for gastric cancer, validating the antigen’s potential as an ISAC target. TRK-950 functions by binding to and attacking cancer cells expressing Caprin-1, with particularly strong expression observed in gastric cancer and other solid tumors, as well as in metastatic cancer cells and cancer stem cells. Bolt’s Boltbody™ ISAC platform combines the precision of antibodies with the strength of both innate and adaptive immune systems to reprogram the tumor microenvironment and generate potent anti-cancer responses. Each Boltbody™ ISAC candidate comprises a tumor-targeting antibody, a non-cleavable linker, and a proprietary immune-stimulatory payload. This combination activates myeloid cells, initiating a positive feedback loop that releases cytokines and chemokines, attracts additional immune cells, lowers the activation threshold of immune responses, and enhances the number of activated immune system cells in the tumor microenvironment, ultimately promoting a robust immune response. In recent years, Toray has increasingly focused on "Life Innovation," accelerating advancements in medical technology and disease prevention with the goal of alleviating the burden on healthcare professionals and helping people live longer, healthier lives. Oncology has been identified as a key area of focus, and Toray is committed to developing industry-leading therapeutic approaches with the potential to significantly improve patient outcomes. The collaboration with Bolt is part of Toray’s broader mission to create a healthier society through the development of innovative medicines, reflecting the shared commitment of both companies to providing better treatment options for cancer patients across various indications. This partnership builds upon the highly productive interactions between Alloy and Pfizer, further solidifying the companies’ collaboration in antibody discovery and technological innovation. Through joint efforts, the two parties aim to drive scientific progress and accelerate the development of new therapies, ultimately benefiting patients. As the collaboration advances, it is expected to yield additional breakthrough innovations that will contribute to the global healthcare landscape. 22.XtalPi Holdings and Probable Biotechnology Join Forces to Accelerate First-in-Class Drug Discovery On February 10, 2025, XtalPi Holdings announced the completion of a strategic investment in Probable Biotechnology and the establishment of an in-depth research and development collaboration. Together, the two companies will advance the discovery and development of First-in-Class (FIC) drugs targeting novel mechanisms identified by Probable Biotechnology to address significant unmet clinical needs. This partnership aims to leverage XtalPi’s cutting-edge AI and robotics-driven platform alongside Probable Biotechnology’s pioneering research in translational regulation to accelerate drug development. Probable Biotechnology specializes in modulating cellular states through ribosome and kinase methylation switches, developing small-molecule targeted therapies for major diseases such as cancer and inflammation. XtalPi, known for its industry-leading AI and robotics-powered drug discovery platform, integrates high-precision quantum physics-based computational chemistry algorithms with over 300 AI models. This platform enables efficient exploration of large-scale chemical space to design and optimize molecules with both innovation and strong drug-like properties. By partnering with XtalPi, Probable Biotechnology will gain access to powerful computational and experimental tools, significantly enhancing the speed and success rate of its FIC drug development efforts. The first project under this collaboration focuses on small-molecule inhibitors targeting the RAS-MAPK signaling pathway, particularly for pan-cancer indications associated with RAS mutations. These inhibitors have demonstrated the ability to overcome resistance to KRAS-targeted therapies in resistant mouse models. When used in combination with existing standard-of-care therapies, they have shown significant advantages in tumor growth inhibition, prolonged survival, and reduced mortality in preclinical studies. Additionally, Probable Biotechnology is developing a novel small-molecule inhibitor for the treatment of acute pancreatitis. Preclinical data indicate promising pharmacokinetic properties, efficacy, and a favorable safety profile, offering a potential new therapeutic option for patients. By combining Probable Biotechnology’s expertise in translational regulation with XtalPi’s AI-driven drug discovery capabilities, this partnership aims to push the boundaries of innovation in drug development. Through this collaboration, the two companies seek to accelerate the discovery of transformative therapies, bringing hope to patients suffering from challenging diseases worldwide. 23.LEO Pharma and DEBRA Research Collaborate to Advance Treatment for Epidermolysis Bullosa LEO Pharma and DEBRA Research announced a partnership aimed at accelerating the development of treatments for epidermolysis bullosa (EB). This rare genetic disorder causes extremely fragile skin that can blister and develop wounds from minor friction or trauma. The collaboration will not only enhance understanding of EB's pathological mechanisms but also develop new therapeutic strategies to improve patients' quality of life. Epidermolysis bullosa is a group of heterogeneous genetic disorders characterized by mutations in structural skin proteins that lead to varying degrees of skin and mucosal fragility. Current treatments primarily focus on wound care, pain management, and infection prevention, lacking fundamental therapies targeting the underlying cause. For certain severe types of EB, such as dystrophic EB, existing treatments often fail to effectively control symptoms, creating an urgent need for innovative therapeutic approaches. The collaboration between LEO Pharma and DEBRA Research will focus on basic research and clinical trials, exploring various novel therapies including gene therapy. For example, the topical gene therapy B-VEC has shown significant efficacy in promoting wound healing in dystrophic EB patients during Phase 3 clinical trials. Additionally, researchers are investigating other cell-based therapies such as bone marrow transplantation and protein replacement therapy. Through this partnership, both parties hope to discover more effective treatments and accelerate their translation into clinical applications. While the specific financial terms of the collaboration remain undisclosed, it is anticipated that LEO Pharma, as an international pharmaceutical company specializing in dermatology, will provide substantial funding and technical resources for the project. This will help accelerate the translation from laboratory to market, enabling new therapies to benefit patients more quickly. Meanwhile, DEBRA Research, as an organization long committed to EB research and patient support, will contribute its disease-specific expertise and network resources. As the collaboration between LEO Pharma and DEBRA Research deepens, it is expected to yield a series of breakthrough research outcomes in the near future. Beyond directly improving the health of EB patients, this partnership will also enhance the medical community's understanding of genetic skin disorders. More importantly, it demonstrates the tremendous potential of cross-institutional collaboration in addressing rare diseases, providing a replicable model for treating other similar conditions. Ultimately, such collaborations may alleviate the socioeconomic burden on EB patients and their families while improving their quality of life. Summary Looking back at February 2025, we can observe several notable characteristics and trends in financing, collaborations, and licensing activities in the preclinical drug sector. First, capital market enthusiasm has not waned; rather, driven by both policy support and technological advancements, investment in the biopharmaceutical industry has continued to grow. According to statistics, by the end of February, the global biopharmaceutical sector had completed financing activities totaling over $3.2 billion, providing crucial funding support to numerous early-stage R&D companies and helping them bridge the gap from laboratory to market. Second, in terms of international collaborations, Chinese companies are gradually becoming a force to be reckoned with. Whether through technology import or product export, Chinese pharmaceutical firms are actively exploring partnership models with international counterparts to accelerate their development. This is particularly evident in the antibody-drug conjugate (ADC) field, where Chinese companies have performed exceptionally well. A prime example is the collaboration between CSPC Pharmaceutical and Radiance Biopharma. Such cross-border partnerships not only enhance product market competitiveness but also lay a solid foundation for subsequent R&D efforts. How to get the latest progress on drug deals? If you would like to access the latest transaction event information, you can click on the 'Deal' module from the homepage of the Synapse database. Within the Deal module, you can search for global pharmaceutical transaction information using labels such as Drugs, Organization, Target, Drug Type, Deal Date.  Furthermore, you can obtain the original link to the transaction coverage by clicking on the "Deal Name." In the analysis view, you can see the most active assignors, assignees, popular targets, and other dimensions of analysis, as well as the distribution of research and development statuses at the time of the transaction, to help you better understand the search results. The Synapse database also supports the ability to view current transactions from the dimension of "drugs" (by selecting "drugs" from the "Adjust Dimension" dropdown menu above). Targeting transactions involving renowned pharmaceutical companies that are of interest to the industry, such as Merck, Roche, etc., Synapse has identified a group of "leading companies" through drugs that have achieved global sales exceeding 1 billion US dollars in 2022. Transactions involving drugs from these leading companies can be filtered by clicking on the "Leading Company" tag on the left-hand side. In addition to the drug transaction module, you can also view related transaction history on the drug detail page and the institution detail page. Click on the image below to explore new pharmaceutical funding transactions! 

Additional Phase 2 data for vilastobart, a tumor-activated, Fc-enhanced anti-CTLA-4, in combination with atezolizumab in patients with MSS CRC anticipated in the middle of 2025 Advancing masked T cell engager programs in novel ATACR and SEECR formats for PSMA, CLDN18.2 and STEAP1 Announced collaboration with AbbVie in the first quarter of 2025 to develop novel tumor-activated, antibody-based immunotherapies, including masked T cell engagers WALTHAM, Mass., March 11, 2025 (GLOBE NEWSWIRE) -- Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage biotechnology company discovering and developing tumor-activated immuno-oncology therapies for people living with cancer, today announced pipeline progress and business updates and reported financial results for the fourth quarter and full year ended December 31, 2024. “We started 2025 with multiple promising updates across our pipeline, including encouraging initial Phase 2 combination data for vilastobart, our tumor-activated anti-CTLA-4, in patients with late-line MSS CRC, a difficult to treat, immunologically cold tumor type that is increasing in incidence, particularly in younger people,” said René Russo, Pharm.D., president and chief executive officer of Xilio. “These data included a preliminary 27% objective response rate in patients without liver metastases and continue to support a differentiated safety profile, including a low incidence of immune-related adverse events and only 5% of patients reporting colitis. We look forward to reporting additional data from the ongoing Phase 2 trial in the middle of this year.” Dr. Russo added, “In addition, we are excited to be advancing multiple novel masked T cell engager programs internally and as part of our recently announced collaboration with AbbVie. Each of these programs leverages our clinically-validated, tumor-activation technology, which we believe has great potential to enhance the activity and tolerability of T cell engagers and open up promising new targets within this class of immunotherapies.” Pipeline and Business Updates Vilastobart: tumor-activated, Fc-enhanced, high affinity binding anti-CTLA-4 Vilastobart is an investigational tumor-activated, Fc-enhanced, high affinity binding anti-CTLA-4 monoclonal antibody designed to block CTLA-4 and deplete regulatory T cells when activated in the tumor microenvironment (TME). In January 2025, Xilio announced encouraging initial data from its ongoing Phase 2 clinical trial evaluating vilastobart at a dose of 100 mg once every six weeks (Q6W) in combination with atezolizumab (Tecentriq®) at 1200 mg once every three weeks (Q3W) in patients with metastatic microsatellite stable colorectal cancer (MSS CRC). As of a data cutoff date of January 13, 2025, these data demonstrated a preliminary 27% objective response rate in patients without liver metastases, and responses were accompanied by decreases in levels of serum tumor biomarkers (carcinoembryonic antigen and circulating tumor DNA) as well as improvement in clinical symptoms. In addition, as of the data cutoff date, the combination was generally well-tolerated, with patients experiencing a low incidence of immune-related adverse events and only 5% of patients reporting colitis. These safety data continue to support the potential for vilastobart to be a differentiated next-generation anti-CTLA-4 in combination with PD-(L)1 inhibitors. For more information, read the press release here. Xilio expects to report updated data from the Phase 2 clinical trial in the middle of 2025, including additional response assessments and follow-up. In addition, Xilio continues to enroll patients in Phase 1C (combination dose escalation) evaluating vilastobart at the 150 mg Q6W dose level in combination with atezolizumab at 1200 mg Q3W. Based on the promising initial Phase 2 proof-of-concept data, Xilio plans to seek opportunities for partnering to accelerate and expand further development of vilastobart. XTX301: tumor-activated IL-12 XTX301 is an investigational tumor-activated IL-12 designed to potently stimulate anti-tumor immunity and reprogram the TME of poorly immunogenic “cold” tumors towards an inflamed or “hot” state. In March 2024, Xilio entered into an exclusive license agreement with Gilead Sciences, Inc. (Gilead) related to Xilio’s tumor-activated IL-12 program, including XTX301. In December 2024, Xilio announced preliminary data from its ongoing Phase 1 clinical trial of XTX301 in patients with advanced solid tumors. As of the data cutoff date of November 25, 2024, XTX301 was generally well-tolerated, and no patients experienced a dose limiting toxicity or a dose reduction due to a treatment-related adverse event. In addition, XTX301 showed evidence of sustained interferon gamma signaling without evidence of tachyphylaxis throughout treatment cycles. Tachyphylaxis has historically limited other IL-12 agents. For more information, read the press release here. A maximum tolerated dose has not yet been established, and Xilio continues to enroll patients in Phase 1A monotherapy dose escalation and Phase 1B monotherapy dose expansion of the ongoing Phase 1 clinical trial of XTX301. XTX501: masked PD-1/IL-2 bispecific XTX501 is a novel, tumor-activated bispecific PD-1/IL-2 designed to selectively stimulate PD-1 positive, antigen-experienced T cells and enhance their function. XTX501 incorporates masking designed to overcome IL-2 receptor-mediated clearance and peripheral activity. In preclinical studies, XTX501 demonstrated robust monotherapy activity (including in settings insensitive to PD-1) and tumor-selective pharmacodynamics consistent with its intended mechanism of action. Xilio is currently advancing XTX501 in investigational new drug (IND) application enabling studies and plans to submit an IND application for XTX501 in the middle of 2026. Masked T Cell Engager Programs Xilio is leveraging its proprietary, clinically-validated tumor-activation platform to advance multiple preclinical programs for masked T cell engagers, including wholly-owned programs targeting the tumor-associated antigens for PSMA, CLDN18.2 and STEAP1 and an additional program in collaboration with AbbVie. Xilio’s masked T cell engager programs include bispecific molecules designed using its advanced tumor-activated cell engager (ATACR) format, which consists of a T cell engager with a masked CD3 targeting domain, and tri-specific molecules designed using its selective effector-enhanced cell engager (SEECR) format. The SEECR format builds upon the ATACR format by adding co-stimulatory signaling designed to further enhance potency and T cell activation. PSMA has demonstrated potential as a T cell engager target for prostate cancer. Xilio anticipates nominating a development candidate for its PSMA program in the ATACR format in the third quarter of 2025 and submitting an IND application in the first quarter of 2027. CLDN18.2 has broad potential as a T cell engager target for gastric, pancreatic, esophageal and lung cancers. Xilio anticipates nominating a development candidate for its CLDN18.2 program in the ATACR format in the fourth quarter of 2025 and submitting an IND application in the second quarter of 2027. STEAP1 has broad potential as a T cell engager target for prostate, colorectal and lung cancers. Xilio anticipates nominating a development candidate for its STEAP1 program in the SEECR format in the first half of 2026 and submitting an IND application in the second half of 2027. Corporate Updates In February 2025, Xilio announced a collaboration, license and option agreement with AbbVie leveraging Xilio’s proprietary tumor-activation technology and platform to discover and develop novel tumor-activated immunotherapies, including masked T cell engagers. In the first quarter of 2025, Xilio received $52.0 million in upfront payments from AbbVie, including a $10.0 million equity investment, and Xilio will be eligible to receive up to approximately $2.1 billion in total contingent payments for option-related fees and milestones plus tiered royalties. For more information, read the joint press release here. In December 2024, Xilio announced the appointment of Caroline Hensley as chief legal officer. Year-End and Fourth Quarter 2024 Financial Results Cash Position: Cash and cash equivalents were $55.3 million as of December 31, 2024, compared to $44.7 million as of December 31, 2023. In the first quarter of 2025, Xilio received $52.0 million in upfront payments in connection with the collaboration agreement with AbbVie. License Revenue: License revenue was $1.7 million for the quarter ended December 31, 2024, and $6.3 million for the year ended December 31, 2024, which consisted of revenue recognized under the license agreement and stock purchase agreement with Gilead. No license revenue was recognized for the quarter and year ended December 31, 2023. Research & Development (R&D) Expenses: R&D expenses were $8.8 million for the quarter ended December 31, 2024, compared to $11.7 million for the quarter ended December 31, 2023. R&D expenses were $41.2 million for the year ended December 31, 2024, compared to $52.1 million for the year ended December 31, 2023. The year-over-year decrease was primarily driven by decreased clinical development activities for XTX202, a masked IL-2, as a result of discontinuing further investment in XTX202, decreased spending related to early-stage programs and indirect research and development costs, decreased personnel-related costs and decreased manufacturing costs for XTX301, partially offset by increased clinical development activities for vilastobart and XTX301. General & Administrative (G&A) Expenses: G&A expenses were $6.5 million for the quarter ended December 31, 2024, compared to $6.4 million for the quarter ended December 31, 2023. G&A expenses were $24.8 million for the year ended December 31, 2024, compared to $27.0 million for the year ended December 31, 2023. The year-over-year decrease was primarily driven by decreases in personnel-related costs, professional and consulting fees and directors’ and officers’ liability insurance, partially offset by an increase in legal fees. Net Loss: Net loss was $13.1 million for the quarter ended December 31, 2024, compared to $17.7 million for the quarter ended December 31, 2023. Net loss was $58.2 million for the year ended December 31, 2024, compared to $76.4 million for the year ended December 31, 2023. Financial Guidance Based on its current operating plans, Xilio anticipates that its cash and cash equivalents as of December 31, 2024, together with the $52.0 million in upfront payments received in the first quarter of 2025 in connection with the collaboration agreement with AbbVie, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2026. About Vilastobart and the Phase 1/2 Combination Clinical Trial Vilastobart is an investigational tumor-activated, Fc-enhanced, high affinity binding anti-CTLA-4 monoclonal antibody designed to block CTLA-4 and deplete regulatory T cells when activated in the tumor microenvironment (TME). In 2023, Xilio entered into a co-funded clinical trial collaboration with Roche to evaluate vilastobart in combination with atezolizumab (Tecentriq®) in a multi-center, open-label Phase 1/2 clinical trial. Xilio is currently evaluating the safety of the combination in Phase 1C dose escalation in patients with advanced solid tumors and the safety and efficacy of the combination in Phase 2 in patients with metastatic microsatellite stable colorectal cancer with and without liver metastases. Please refer to NCT04896697 on www.clinicaltrials.gov for additional details. About XTX301 and the Phase 1 Clinical Trial XTX301 is an investigational masked IL-12 designed to potently stimulate anti-tumor immunity and reprogram the tumor microenvironment (TME) of poorly immunogenic “cold” tumors towards an inflamed or “hot” state. In March 2024, Xilio entered into an exclusive license agreement with Gilead Sciences, Inc. for Xilio’s tumor-activated IL-12 program, including XTX301. Xilio is currently evaluating the safety and tolerability of XTX301 as a monotherapy in patients with advanced solid tumors in a first-in-human, multi-center, open-label Phase 1 clinical trial. Please refer to NCT05684965 on www.clinicaltrials.gov for additional details. About Xilio Therapeutics Xilio Therapeutics is a clinical-stage biotechnology company discovering and developing tumor-activated, or masked, immuno-oncology (I-O) therapies with the goal of significantly improving outcomes for people living with cancer without the systemic side effects of current I-O treatments. The company is leveraging its proprietary platform to advance a pipeline of novel, tumor-activated I-O molecules that are designed to optimize the therapeutic index by localizing anti-tumor activity within the tumor microenvironment. Learn more by visiting www.xiliotx.com and follow us on LinkedIn (Xilio Therapeutics, Inc.). Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans, expectations, development timelines and anticipated milestones for Xilio’s programs; the receipt of future contingent payments under Xilio’s collaboration or partnership agreements with Roche, Gilead and AbbVie; the potential benefits of any of Xilio’s current or future product candidates in any indication; the sufficiency of, and the period in which Xilio expects to have, cash to fund its operations, capital expenditure requirements, and development plans and milestones; and Xilio’s strategy, goals and anticipated financial performance, milestones, business plans and focus. The words “aim,” “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “seek,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of important risks, uncertainties and other factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks related to general market conditions and geopolitical uncertainties; risks and uncertainties related to ongoing and planned research and development activities, including initiating, conducting or completing preclinical studies and clinical trials and the timing and results of such preclinical studies or clinical trials; the delay of any current or planned preclinical studies or clinical trials or the development of Xilio’s current or future product candidates; Xilio’s ability to obtain and maintain sufficient preclinical and clinical supply of current or future product candidates; Xilio’s ability to advance multiple early-stage masked T cell engager programs; initial, preliminary or interim preclinical or clinical data or results may not be replicated in or predictive of future preclinical or clinical data or results; Xilio’s ability to successfully demonstrate the safety and efficacy of its product candidates and gain approval of its product candidates on a timely basis, if at all; results from preclinical studies or clinical trials for Xilio’s product candidates may not support further development of such product candidates; actions of regulatory agencies may affect the initiation, timing and progress of current or future clinical trials; Xilio’s ability to obtain, maintain and enforce patent and other intellectual property protection for current or future product candidates; Xilio’s need to obtain additional cash resources to fund its operations beyond the first quarter of 2026, including to advance its pipeline of tumor-activated I-O molecules; the impact of international trade policies on Xilio’s business, including U.S. and China trade policies; and Xilio’s ability to maintain its collaboration or partnership agreements with Roche, Gilead and AbbVie. These and other risks and uncertainties are described in greater detail in the sections entitled “Risk Factor Summary” and “Risk Factors” in Xilio’s filings with the U.S. Securities and Exchange Commission (“SEC”), including Xilio’s most recent Quarterly Report on Form 10-Q and any other filings that Xilio has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Xilio’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Xilio explicitly disclaims any obligation to update any forward-looking statements. This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release. TECENTRIQ is a registered trademark of Genentech USA, Inc., a member of the Roche Group. Investor and Media Contact Scott Young Vice President, Investor Relations and Corporate Communications investors@xiliotx.com XILIO THERAPEUTICS, INC. Condensed Consolidated Statements of Operations and Comprehensive Loss (In thousands, except share and per share data) (Unaudited) (1) Operating expenses include the following amounts of non-cash stock-based compensation expense: (2) Weighted average common shares outstanding, basic and diluted, includes prefunded warrants to purchase common stock and excludes shares of restricted common stock that were not vested as of the applicable period.

Also entered into collaboration and option agreement with AbbVie to develop novel tumor-activated immunotherapies, including masked T cell engagers, with $52.0 million in upfront payments Extended anticipated cash runway into the first quarter of 2026 Company to host investor conference call and webcast today at 8:30 am ET WALTHAM, MA, USA I February 12, 2025 I Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage biotechnology company discovering and developing tumor-activated immuno-oncology therapies for people living with cancer, today announced three wholly-owned preclinical programs for masked T cell engagers targeting prostate-specific membrane antigen (PSMA), claudin 18.2 (CLDN18.2) and six-transmembrane epithelial antigen of prostate 1 (STEAP1). In addition, as announced earlier today, Xilio entered into a collaboration, license and option agreement with AbbVie leveraging Xilio’s proprietary tumor-activation technology and platform to discover and develop novel tumor-activated immunotherapies, including masked T cell engagers. Under the collaboration, Xilio will receive $52.0 million in upfront payments and be eligible for additional option-related fees and milestones, as well as tiered royalties. “Today, we are excited to announce the next phase of Xilio’s tumor-activation platform with multiple internal masked T cell engager programs, as well as a multi-program collaboration with AbbVie,” said René Russo, Pharm.D., president and chief executive officer of Xilio. “T cell engagers have demonstrated meaningful clinical activity, yet many promising targets for solid tumors remain out of reach due to toxicity limitations. Leveraging our clinically-validated masking technology, we aim to lead the next wave of innovation in cancer immunotherapy by developing masked T cell engagers designed to improve tolerability through tumor-selective activation. In addition, we are able to incorporate co-stimulatory signaling into our T cell engager designs with the goal of further enhancing T cell activation, persistence and anti-tumor activity. We look forward to collaborating with AbbVie as we advance our pipeline of novel tumor-activated immunotherapies.” Masked T Cell Engager Programs T cell engagers are designed to redirect immune effector cells against cancer cells by simultaneously binding to a specific tumor-associated antigen expressed on the cancer cells and the T cell receptor complex on T cells, resulting in T cell-mediated killing of tumor cells. To date, T cell engagers have demonstrated significant promise as cancer immunotherapies in a variety of advanced solid tumors, but their potential has been limited by toxicity. Xilio is leveraging its proprietary, clinically-validated tumor-activation platform to advance a pipeline of wholly-owned, novel masked T cell engager molecules with conditional half-life modulation designed to enable potent, localized T cell activation and tumor cell destruction together with an improved therapeutic index. These programs include bispecific molecules designed using Xilio’s advanced tumor-activated cell engager (ATACR) format, which consists of a T cell engager with a masked CD3 targeting domain, and tri-specific molecules designed using Xilio’s selective effector-enhanced cell engager (SEECR) format. The SEECR format builds upon the ATACR format by adding co-stimulatory signaling designed to further enhance potency and T cell activation. Xilio is currently advancing three wholly-owned preclinical programs for masked T cell engager molecules targeting the following tumor-associated antigens: PSMA, CLDN18.2 and STEAP1. Anticipated milestones beyond the first quarter of 2026 are subject to obtaining sufficient additional capital. Collaboration, License and Option Agreement with AbbVie Under the terms of the collaboration, license and option agreement announced earlier today with AbbVie: Financial Guidance Based on its current operating plans, Xilio anticipates that its existing cash and cash equivalents, together with the upfront payments under the agreement with AbbVie, will be sufficient to fund its operating expenses and capital expenditure requirements into the first quarter of 2026. Investor Conference Call Information Xilio will host a conference call and webcast today (Wednesday, February 12, 2025) at 8:30 am ET. Viewers can access the webcast by using this link . Listeners who require dial-in access should register here to receive a unique PIN and information to join the call. Listeners are encouraged to join at least 15 minutes prior to the scheduled start time. The webcast will also be accessible under “Events & Presentations” in the Investors & Media section of the Xilio Therapeutics website at https://ir.xiliotx.com . A replay of the webcast will be archived on the website for 30 days following the presentation. About Xilio Therapeutics Xilio Therapeutics is a clinical-stage biotechnology company discovering and developing tumor-activated immuno-oncology (I-O) therapies with the goal of significantly improving outcomes for people living with cancer without the systemic side effects of current I-O treatments. The company is using its proprietary platform to advance a pipeline of novel, tumor-activated clinical and preclinical I-O molecules that are designed to optimize the therapeutic index by localizing anti-tumor activity within the tumor microenvironment. Learn more by visiting www.xiliotx.com and follow us on LinkedIn ( Xilio Therapeutics, Inc .). SOURCE: Xilio Therapeutics