- Insilico Medicine nominates ISM0387 as the company’s 30th self-developed preclinical candidate compound (PCC), and the first candidate drug fully discovered in the UAE. This announcement was jointly released by Insilico Medicine, the Emirates Drug Establishment (EDE), the Abu Dhabi Investment Office (ADIO), and the Abu Dhabi Department of Health (DoH).
- ISM0387 is an MTA-cooperative PRMT5 inhibitor with a novel molecular structure designed by AI. It demonstrates favorable in vitro activity and selectivity in preclinical studies, along with optimized brain penetration properties; it also shows potent efficacy in disease models with a clear dose-dependent trend.
- The entire discovery process for this candidate drug was completed locally in the UAE, marking the region’s transformation into a technology-driven frontier breakthrough and a knowledge-empowered “Falcon Economy.”
- Based on Chemistry42 and its over 40 AI models, the Insilico Medicine team first used AI to generate 90 innovative candidate molecules, then screened them across multi-dimensional parameters, with a focus on central nervous system (CNS)-related features. The lead compound discovery phase took only 6 months.
Shanghai and Abu DhabiApril 24, 2026 /PRNewswire/ — Drug discovery has long been called a “game for the brave,” requiring immense investment, lengthy timelines, and extremely high technical barriers. Research published in *Nature* shows that developing a new drug from R&D to market typically costs between $900 million and $2.6 billion, with cycles often exceeding 10 years. Even in the early stages, from target identification to candidate drug nomination, it usually takes about 4.5 years, involving the screening and validation of thousands of molecules.
For a long time, the extremely high barriers in the pharmaceutical industry have concentrated drug innovation in a few resource-rich countries. Now, generative artificial intelligence and foundation models are changing this landscape. On April 23, 2026, Insilico Medicine (03696.HK), a clinical-stage biotechnology company driven by generative AI, announced a milestone achievement: successfully nominating the first preclinical candidate compound (PCC) developed locally in the UAE. This project was completed by Insilico Medicine’s UAE team using the company’s proprietary Pharma.AI platform, with the entire R&D process from molecular design to optimization conducted locally in the UAE.
This is also the 30th AI-driven PCC nominated by Insilico Medicine to date. The achievement was announced with the support and presence of the Emirates Drug Establishment (EDE), the Abu Dhabi Department of Health (DoH), the Abu Dhabi Investment Office (ADIO), and key members of the local biomedical ecosystem, marking a critical step for the UAE in building independent biotechnology capabilities and further solidifying its position as a global pharmaceutical innovation hub.
His Excellency Saeed bin Mubarak Al Hajeri, Minister of State for the UAE and Chairman of the Emirates Drug Establishment, stated that this achievement will strengthen the UAE’s position in the global biotechnology value chain. He added that the UAE is building knowledge-based capabilities to support its role as an active partner in developing drug solutions and expanding its global influence. He further noted: “This progress reflects a maturing national model that integrates scientific research with regulatory and investment frameworks. It enables us to more quickly translate scientific discoveries into practical applications and supports building a system capable of competing in high-value fields.”
Her Excellency Fatima Al Kaabi, Director General of the Emirates Drug Establishment, stated, “This is not just a scientific achievement but a clear indication that the UAE is continuously enhancing its local drug R&D capabilities with the support of advanced technologies. These technologies are transforming discovery methods, turning data into faster, more accurate R&D decisions while reducing time and costs.” She added that this milestone is not only about creating new treatment options but also reflects ongoing efforts to build a robust national system that redefines “drug security”: shifting from merely ensuring supply to possessing the capacity for local R&D and sustained supply, thereby improving the healthcare system’s preparedness for future challenges.
Insilico Medicine team and partners participate in ribbon-cutting ceremony
AI Innovation Empowers: UAE Reshapes Global Research Landscape
In May 2025, Insilico Medicine launched a pilot project in the UAE to validate the acceleration potential and reproducibility of generative AI, aiming to leverage a new ecosystem to compress drug R&D, which typically takes years, into just a few months. The project focused on synthetically lethal targets in solid tumors that are both innovative and genetically validated. To advance this project, Insilico Medicine assembled a local team of four, including two computational chemists, one medicinal chemist, and one translational biologist. The team set clear and tight goals: complete target validation by Q3 2025, generate hit compound series within 30 days, and finish lead compound optimization within 6 months.
In less than a year, this vision has become a reality. Insilico Medicine officially nominated ISM0387, an MTA-cooperative PRMT5 inhibitor, as the development candidate (DC/PCC) for this project. ISM0387 features a novel molecular structure and excellent blood-brain barrier penetration properties, offering potential new treatment options for glioblastoma multiforme (GBM) patients. Notably, the entire process from molecular design and optimization to preclinical candidate nomination was completed locally in the UAE, taking less than 12 months.
This achievement also reflects the rapid maturation of the UAE’s local biotechnology ecosystem and research infrastructure, demonstrating the role of the Abu Dhabi Investment Office’s (ADIO) Health, Endurance, Longevity, and Medicine (HELM) cluster in focusing on localization, advanced pharmaceutical R&D, and long-term manufacturing potential, as well as attracting and supporting high-value life science investments. Aligned with Abu Dhabi’s economic diversification blueprint, this project showcases the UAE’s transformation from a global trade and energy hub to a diversified, technology-driven economy, solidifying the region’s knowledge-based “Falcon Economy.”
His Excellency Badr Al-Olama, Director General of the Abu Dhabi Investment Office, stated that this milestone marks a structural shift in Abu Dhabi’s life science ecosystem, from supporting research development to providing commercially viable, globally scalable pharmaceutical innovations. This is precisely the vision behind ADIO’s launch of HELM, and ADIO will continue to support companies like Insilico Medicine, aiming to produce and export more high-value therapeutic drugs to benefit patients worldwide.
Dr. Alex Aliper, Co-founder and President of Insilico Medicine, stated, “For decades, the UAE has been a vital global trade hub, and today, it is emerging as a major center for global scientific innovation. The nomination of ISM0387 as a candidate drug in record time, meeting world-class standards, demonstrates how generative AI can reshape R&D paradigms, industrial logic, and innovation pathways. We are not just building models with AI; we are advancing the future of medicine in Abu Dhabi.”
New Options for Cancer Treatment: Synthetic Lethality Mechanism and PRMT5 Inhibitor Breakthrough
The development of ISM0387 is based on the synthetic lethality mechanism, where a cell can survive if either of two genes is mutated or deleted individually, but cannot survive when both are inactivated. Since protein arginine methyltransferase 5 (PRMT5) and methylthioadenosine phosphorylase (MTAP) form a pair of synthetically lethal targets, inhibiting PRMT5 can selectively kill cancer cells with MTAP deletion.
In early 2025, Insilico Medicine announced the nomination of its first MTA-cooperative PRMT5 inhibitor, ISM1745. This candidate overcomes the selectivity and toxicity issues of first-generation PRMT5 inhibitors, which inhibit PRMT5 in both healthy and cancer cells, and has “best-in-class” potential. The successful nomination of ISM1745 further validated the Pharma.AI platform’s ability to tackle complex drug R&D challenges and laid the foundation for a PRMT5 inhibitor with blood-brain barrier penetration, offering innovative solutions for central nervous system diseases, particularly with significant potential in glioblastoma multiforme (GBM).
Glioblastoma multiforme is a highly aggressive adult brain tumor, accounting for approximately 48% of all primary malignant brain tumors, with a recurrence rate exceeding 70%. Over 200,000 patients worldwide die from this disease annually, yet current standard of care (SOC) still primarily relies on chemotherapy and radiotherapy. Meanwhile, 40% to 50% of GBM cases exhibit MTAP deletion, indicating significant potential for PRMT5 inhibition strategies in this area.
ISM0387’s early discovery work was completed at Insilico Medicine’s Generative AI and Quantum Computing R&D Center in Abu Dhabi. This innovation, nurtured locally in the UAE, demonstrates the role of the HELM cluster in translating cutting-edge research into scalable pharmaceutical innovations, positioning Abu Dhabi as an emerging hub for advanced pharmaceutical manufacturing and life science exports.
AI-Driven CNS Expansion: Brain-Penetrant Molecular Design Completed in 6 Months Using Chemistry42
The discovery and optimization of ISM0387 were completed using Insilico Medicine’s proprietary generative chemistry platform, Chemistry42, which integrates over 40 generative AI models. Specifically, based on the co-crystal structure of known PRMT5 inhibitors, the research team used AI to generate 90 candidate compounds with novel scaffolds, systematically evaluating them against key metrics such as binding affinity, synthetic accessibility, and ADMET properties.
To address the therapeutic needs of glioblastoma multiforme, the team optimized parameters critical for CNS drug development during molecular design, including molecular weight, topological polar surface area, hydrogen bond donor count, lipophilicity, and pKa. They also assessed the compounds’ ability to cross the blood-brain barrier using indicators such as in vitro passive permeability and brain-to-plasma ratio. The entire lead compound discovery phase was completed in just 6 months, ultimately identifying 4 lead compounds with favorable brain penetration and metabolic stability.
In multiple tumor cell lines, ISM0387 demonstrated favorable in vitro activity, selectivity, and in vitro safety profiles. More importantly, the compound showed strong blood-brain barrier (BBB) penetration in various animal models, suggesting potential efficacy at lower doses in humans, providing a positive basis for subsequent clinical development.
In an orthotopic glioblastoma model, daily administration of 30 mg/kg ISM0387 for 20 consecutive days significantly inhibited tumor growth, while the control group showed marked tumor progression.
From Rooting to Fruiting: Settling in Masdar City, Gaining Local Recognition
This milestone achievement stems from a strategic partnership between Insilico Medicine and the Abu Dhabi Investment Office (ADIO) initiated in January 2023. Based on this collaboration, Insilico Medicine, with ADIO’s support, became a member of the HELM cluster, establishing and expanding the Middle East’s leading AI-driven biotechnology R&D center in Masdar City, Abu Dhabi. This has driven the development of advanced AI-powered drug discovery capabilities and laid the foundation for future pharmaceutical manufacturing and export-oriented growth. The center, located at the IRENA headquarters in Masdar City, brings together 40 experts focused on cutting-edge areas such as the Pharma.AI platform, aging research, and sustainable chemistry.
Since establishing a presence in the UAE, Insilico Medicine has continuously integrated into the local research ecosystem, forming research partnerships with renowned institutions such as Mohamed bin Zayed University of Artificial Intelligence (MBZUAI), Khalifa University, and New York University Abu Dhabi (NYU Abu Dhabi) to jointly promote the application and development of generative AI in scientific research. In recognition of its achievements, Insilico Medicine received the “Health Innovation Pioneer” award in 2024.
To further consolidate its local R&D presence, Insilico Medicine established a strategic partnership with the Emirates Drug Establishment (EDE) in 2026 to promote and institutionalize local pharmaceutical innovation capabilities. Previously, Dr. Sheikha Al Mazrouei, Director of the Research and Laboratory Department at the Emirates Drug Establishment, stated that through collaboration with Insilico Medicine, both parties can use generative models and reinforcement learning to analyze biological, chemical, and clinical data, thereby helping to develop candidate drug compounds with greater precision in efficacy and safety.
Following the nomination of ISM0387 as the 30th preclinical candidate compound, Insilico Medicine’s R&D pipeline, built since 2021, encompasses over 40 projects. Among these, 12 candidate drugs have received clinical trial approvals, 3 projects have reached Phase II clinical evaluation, and a Phase IIa clinical trial for idiopathic pulmonary fibrosis (IPF) has been completed with positive results. Compared to the typical 4.5-year timeline for traditional early-stage drug R&D, Insilico Medicine nominated 20 preclinical candidate compounds between 2021 and 2024, with an average time from project initiation to PCC nomination of 12–18 months, synthesizing and testing approximately 60–200 molecules per project.
About Insilico Medicine
Insilico Medicine is a global pioneer biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery and drive innovation in life sciences, empowering people to live longer, healthier lives. On December 30, 2025, the company was listed on the Main Board of the Hong Kong Stock Exchange under the stock code 03696.HK.
Using its proprietary Pharma.AI platform and advanced automated biology laboratories, Insilico Medicine is providing innovative drug solutions for unmet disease areas such as fibrosis, oncology, immunology, pain, obesity, and metabolic disorders. Additionally, Insilico Medicine continues to expand the application of Pharma.AI into diverse fields, including advanced materials, agriculture, nutritional products, and veterinary medicine. For more information, please visit the website www.insilico.com