An inhibitor compound capable of crossing the blood-brain barrier and suppressing tumour growth in preclinical trials has been nominated as the UAE’s first fully AI-discovered drug candidate, marking a concrete step in the country’s push to build an independent pharmaceutical development capability.
The compound, designated ISM0387, was nominated as a preclinical candidate (PCC) by Insilico Medicine in partnership with the Emirates Drug Establishment (EDE). Its mechanism targets the PRMT5 enzyme through synthetic lethality — specifically exploiting the deletion of the MTAP gene found in certain tumour types — which gives it selective activity against cancer cells while reducing effect on healthy tissue.
Animal model data showed measurable tumour suppression at a daily dose of 30 mg/kg over 20 days, with dose-dependent responses that researchers say support its advancement into subsequent clinical phases. Beyond solid tumours, its blood-brain barrier penetration properties make it a candidate for treating central nervous system-related conditions, including aggressive brain cancers that are notoriously resistant to existing therapies.
The discovery phase — from biological target identification to molecular design and optimisation — was completed in approximately six months. During that period, the team generated and evaluated more than 90 molecular candidates using Insilico Medicine’s Chemistry42 platform, which draws on more than 40 generative AI models to process biological and chemical data. The full development timeline, from molecule design to PCC nomination, came in under 12 months. By conventional pharmaceutical standards, the same process typically spans more than a decade and can exceed $1 billion in cost.
Dr. Alex Aliper, Co-founder and President of Insilico Medicine, called the result a turning point: “This achievement marks a turning point in global drug development and shows how effective generative AI can be in improving how drugs are discovered, by reducing time and overcoming traditional challenges.”
He added that what has been achieved in the UAE proves that countries investing in research infrastructure and advanced technologies can produce world-class treatment solutions in record time.
The entire pipeline — from target identification through to candidate nomination — was executed within the UAE, a point EDE officials highlighted as central to the programme’s significance.
Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said the milestone signals more than a research outcome, describing it as evidence of a maturing national capacity to develop medicines locally using technologies that convert data into faster and more accurate development decisions while reducing time and cost.
She added that the effort reflects a broader redefinition of pharmaceutical security — moving away from supply assurance toward local development and sustainability.
Dr. Sheikha Al Mazrouei, Director of Research and Laboratories at EDE, said the collaboration applies generative models and reinforcement learning across biological, chemical, and clinical data to develop compounds with tighter profiles for both effectiveness and safety.
Saeed bin Mubarak Al Hajeri, Minister of State and EDE Chairman, framed the development in terms of the country’s positioning in global biotech value chains. “This progress demonstrates the maturity of a national model that brings together scientific research with regulatory and investment frameworks,” he said. “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.”
EDE’s regulatory frameworks are credited with enabling the flexible research environment in which the programme operated, alongside broader national investments in biotechnology infrastructure and inter-agency collaboration.
