Generative AI Leap: Insilico Medicine Nominates First Preclinical Candidate in the UAE

For its 30th PCC anniversary, Insilico Medicine nominated ISM0387, the first UAE-based preclinical candidate, jointly announced with the Emirates Drug Establishment (EDE), Abu Dhabi Investment Office (ADIO), and the Department of Health Abu Dhabi (DoH).

● ISM0387 is an MTA-cooperative PRMT5 inhibitor with AI-powered novel structure, showing improved in vitro activity and selectivity, enhanced brain penetrant properties, and robust, dose-dependent efficacy in disease models.

● The discovery process of ISM0387 is wholly local, and it signals that the UAE has evolved into a technology-driven powerhouse with a knowledge-based "Falcon Economy."

● Based on Chemistry42 and its 40+ models, Insilico’s team screened 90 AI-powered novel candidates, considering multiple parameters with a focus on CNS-specific traits, completing the lead discovery phase in just six months.

Drug discovery is famously a “game for the brave”: a high-stakes pursuit that demands immense capital, decades of patience, and unwavering technical fortitude. According to research in Nature, bringing a single drug to market typically costs between $900 million and $2.6 billion, spanning over a decade. Even the early phase, from target identification to candidate nomination, can consume 4.5 years of thousands of molecular screening.

Historically, these barriers have limited pharmaceutical innovation to a handful of resource-rich nations. However, generative AI and foundation models are redrawing these boundaries today. On April 23, 2026, Insilico Medicine ( “Insilico”, HKEX:3696 ), a clinical-stage, generative AI–driven drug discovery company, announced a landmark milestone: the nomination of the UAE’s first-ever developmental candidate. Discovered locally by Insilico’s UAE team using the company’s proprietary Pharma.AI platform, the program completed the early discovery workflow, from molecular design to optimization, within the region.

The nomination also represents Insilico’s 30th AI-supported preclinical candidate (PCC) to date. Witnessed and Supported by the Emirates Drug Establishment (EDE), the Department of Health (DoH) – Abu Dhabi, the Abu Dhabi Investment Office (ADIO) and Masdar City, this milestone signals a major step forward in the UAE’s sovereign biotechnological capabilities and its rise as a global hub for pharmaceutical innovation.

H.E. Saeed bin Mubarak Al Hajeri, Minister of State and Chairman of the Emirates Drug Establishment, said this achievement will strengthen the UAE’s position in global biotech value chains, adding that the country is building knowledge-based capabilities that support its role as an active partner in developing pharmaceutical solutions and expanding its global impact. Al Hajeri added: “This progress demonstrates the maturity of a national model that brings together scientific research with regulatory and investment frameworks. It allows us to turn scientific discoveries into real-world applications faster and supports the development of a system that can compete in high-value sectors.”

For her part, H.E. Dr. Fatima Al Kaabi, Director General of the Emirates Drug Establishment, said “this is not just a research achievement but a clear sign of the country’s growing ability to develop medicines locally, backed by advanced technologies that are improving how discoveries are made, turning data into faster and more accurate development decisions while reducing time and cost.” Her Excellency added that this milestone goes beyond creating new treatments. It reflects the ongoing efforts being made to build a strong national system that is redefining pharmaceutical security, moving from simply ensuring supply to having the capability to develop and sustain it locally, strengthening the healthcare system’s readiness to respond to future challenges.

In May 2025, Insilico launched an ambitious pilot program in the UAE to prove that generative AI could compress years of traditional research into months. The initiative targeted medium-novelty, genetically validated synthetic-lethality targets for solid tumors. To lead this cycle, Insilico deployed a specialized local team of four—comprising two computational chemists, one medicinal chemist, and one translational biologist—tasked with meeting aggressive benchmarks: target finalization by Q3 2025, hit series generation in under 30 days, and lead optimization in less than 6 months.

In less than a year, that vision has become a reality. Insilico has officially nominated ISM0387, an MTA-cooperative PRMT5 inhibitor as the developmental candidate for this program. Featuring a novel structure and exceptional CNS-penetrant abilities, ISM0387 offers a promising new treatment option for patients with Glioblastoma (GBM). Notably, the entire development journey, from molecular design, optimization to preclinical nomination, was conducted locally in the UAE. The program reached this critical milestone in under 12 months.

This milestone also serves as a powerful testament to the maturity of the local biotechnology ecosystem and research infrastructure., reflecting ADIO’s Health, Endurance, Longevity and Medicine (HELM) cluster’s role in attracting and supporting high-value life sciences investments, with a focus on localization, advanced pharmaceutical development and long-term manufacturing potential. Aligned with Abu Dhabi’s broader economic diversification agenda, it highlights the UAE’s strategic transition towards a diversified, technology-led powerhouse—the with a knowledge-based "Falcon Economy."

“For decades, the UAE region has been recognized as a global trade hub; today, we are proving it has evolved into a global science hub," said Alex Aliper, PhD, Co-founder and president of Insilico Medicine. “By delivering ISM0387 as a candidate compound, at world-class standards, in record time, we’re showing how generative AI shifts paradigms, industries, and even development logics. We aren't just using AI to build models; we are using it to build the future of medicine, right here in Abu Dhabi.”

The scientific foundation of ISM0387 lies in synthetic lethality, a specific relationship between two genes (known as a synthetic lethal pair) where a mutation or deficiency in either gene alone allows the cell to survive, but the loss of both genes simultaneously is fatal. As a result, the inhibition of Protein Arginine Methyltransferase 5 (PRMT5) enables targeted treatment against cancer cells with deletion of Methylthioadenosine phosphorylase (MTAP), which forms a synthetic lethal pair with PRMT5.

In early 2025, Insilico nominated ISM1745, its first MTA-cooperative PRMT5 inhibitor with best-in-class potential, overcoming the selectivity and toxicity issue for the first generation of inhibitors known to shut down PRMT5 in both healthy and cancer cells. More importantly, the nomination of ISM1745 further validates the abilities of Pharma.AI to break complex real-world drug development challenges, and paves the way for MTA-cooperative PRMT5 inhibitors with broader applications in MTAP-deleted cancers, featuring brain-penetrant properties that are particularly suited for treating central nervous system (CNS) diseases, especially glioblastoma (GBM), the most aggressive brain tumor in adults.

With a recurrence rate exceeding 70%, GBM takes the lives of over 200,000 patients worldwide each year, and accounts for approximately 48% of all primary malignant brain tumors. Luckily, MTAP deletion is detected in up to 40–50% of cases, demonstrating potential of PRMT5 inhibition in this field primarily relying on chemotherapy and radiotherapy as standard of care (SOC).

Discovered at Insilico’s Generative AI and Quantum Computing R&D Center in Abu Dhabi, ISM0387 will potentially expand the “built-in-UAE” innovation to benefit patients all over the globe, demonstrating how HELM enables the translation of advanced research into scalable pharmaceutical innovation and positions Abu Dhabi as an emerging hub for advanced pharmaceutical manufacturing and life sciences exports.

The discovery and optimization process for ISM0387 was powered by Chemistry42, Insilico’s generative chemistry platform integrated with over 40 generative AI models. More specifically, the team utilized the co-crystal structures of known PRMT5 inhibitors, generating 90 compounds with novel scaffolds with AI, which were subsequently evaluated for a balance between binding affinity, synthetic accessibility, and ADMET properties.

To address the specific challenges of Glioblastoma, the AI prioritized CNS-specific engineering, optimizing core parameters such as molecular weight, topological polar surface area (TPSA), hydrogen bond donors, lipophilicity (LogP), and pKa, while simultaneously validating in vitro passive permeability and brain-to-plasma ratios to ensure exceptional blood-brain barrier penetration. The entire lead discovery phase was completed with high efficiency in just six months, identifying four lead compounds with good brain penetration and metabolic stability.

In multiple cancer cell lines, ISM0387 demonstrated improved in vitro activity and selectivity, as well as a promising in vitro safety profile. More importantly, enhanced blood-brain barrier (BBB) penetration properties were reported across animal species, leading to lower efficacious dose projected in humans, highlighting potential in future clinical development.

In orthotopic glioblastoma models, daily administration of ISM0387 at 30 mg/kg robustly suppressed tumor growth over 20 days, in stark contrast to marked tumor progression observed in the vehicle group.

The milestone is the culmination of a strategic journey that began in January 2023 through a partnership with the Abu Dhabi Investment Office (ADIO). Through this partnership, ADIO has supported Insilico Medicine’s establishment and expansion in Abu Dhabi under the HELM cluster, enabling the development of advanced AI-driven drug discovery capabilities and laying the foundation for future pharmaceutical manufacturing and export-oriented growth. Located in Masdar City at the IRENA headquarters, the center hosts a world-class team of 40 specialists dedicated to advancing Insilico’s Pharma.AI platform, aging research, and sustainable chemistry.

Deeply integrated into the UAE’s academic fabric, Insilico has forged research alliances with MBZUAI, Khalifa University, and NYU Abu Dhabi to push the boundaries of generative AI since its UAE site was established. These efforts were honored with the 2024 Health Innovation Trailblazer Award. Further solidifying its local footprint. Started in 2026, Insilico began a strategic partnership with the Emirates Drug Establishment (EDE) to institutionalize pharmaceutical innovation locally. Dr. Sheikha Al Mazrouei, Director of Research and Laboratories Department at the Emirates Drug Establishment, said that the collaboration between the establishment and Insilico allows the use of generative models and reinforcement learning to analyse biological, chemical, and clinical data. This helps in developing drug compounds that are more precise in terms of effectiveness and safety.

With ISM0387 being the 30th PCC nomination, since 2021, Insilico has established a comprehensive portfolio of over 40 assets, with 12 candidates obtaining IND clearance, three reaching Phase II evaluation, among which one Phase IIa clinical trial completed with positive results for the treatment of idiopathic pulmonary fibrosis (IPF). By integrating the technologies of AI and automation, Insilico has demonstrated significant efficiency boost compared to traditional drug discovery methods (often requiring an average of 4.5 years), as announced in the recent key timeline benchmarks for internal programs from 2021 to 2024: the average time to PCC is 12-18 months, with 60-200 molecules synthesized and tested per program.

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