Exscientia

AI is revolutionizing drug discovery, from designing molecules to predicting protein structures, significantly reducing costs and timelines. AI-derived drugs have shown higher success rates in clinical trials, with companies like Exscientia and Insilico Medicine leading the charge. Key AI technologies include machine learning, deep learning, NLP, and generative models, transforming stages from target identification to clinical trials. Challenges include data quality, validation, and integration, but the future promises more personalized medicine and effective treatments.

Morgan Stanley's Vikram Purohit maintains Buy rating on Axsome Therapeutics (AXSM) with $115.00 target, citing Auvelity's strong MDD treatment performance, $254M sales post-launch, and potential $1B by 2027. Mizuho Securities also reiterates Buy with $106.00 target.

AI accelerates drug discovery by identifying candidates faster, reducing costs, and shortening timelines. It excels in early stages, chemical synthesis, clinical trials, and precision medicine, enabling quicker development of treatments for previously untreatable diseases.

AI-powered Recursion Pharmaceuticals (RXRX) hit a milestone with FDA approval for REC-1245, targeting RBM39 in advanced cancers. The company plans to merge with Exscientia (EXAI) in 2025 and has partnerships with Bayer (BAYZF) and Sanofi (SNY), with an average 12-month target price of $10.14 for RXRX stock.

AI revolutionizes drug discovery and development, accelerating research, reducing costs, and increasing the probability of drug success in Phase 2 clinical trials. AI tools, including generative AI, enable rapid exploration of chemical spaces and multi-objective optimization of drug properties. AI-focused biotechs have already advanced several drug candidates into clinical trials in record time. AI also enhances clinical trial operations and regulatory affairs, promising faster, cheaper, and better-value drug development. The second part of the series will explore the financial benefits and the impact on the Probability of Success (PoS) in Phase 2 trials.

AI in drug design is delivering results, with companies like DeepCure, VeriSIM Life, Iktos, and Exscientia leveraging AI to target previously undruggable proteins, reduce animal testing, and predict clinical trial outcomes. DeepCure uses a multiparametric approach to design drugs with specific requirements, while VeriSIM Life combines AI with mathematical models to predict clinical success. Iktos employs AI-driven retrosynthesis and robotics for faster molecule synthesis, and Exscientia focuses on learning-based drug design to create innovative compounds.

Recursion Pharmaceuticals' AI platform identified a drug candidate for solid tumors and lymphoma, gaining FDA approval for a phase 1/2 trial in under 18 months. The potential market exceeds 100,000 patients in the U.S. and EU. The trial will start in Q4 2024, targeting RBM39 in advanced HR-proficient cancers.

Exscientia presented at ELRIG Drug Discovery 2024 on using AI and robotics for drug discovery, noting an 80% Phase I success rate for AI-designed molecules. The company's system focuses on flexibility rather than high-throughput screening, aiming to increase productivity without replacing human roles.