Nature Medicine：AI赋能研发原创新药2a期临床数据积极，肺纤维化患者迎来新希望

Core Viewpoint - The article discusses the transformative impact of artificial intelligence (AI) on the pharmaceutical industry, particularly in drug discovery and development, highlighting the first AI-discovered drug Rentosertib for idiopathic pulmonary fibrosis (IPF) and its promising clinical trial results [1][2][3]. Group 1: AI in Drug Development - AI is rapidly reshaping the pharmaceutical landscape, offering unprecedented opportunities to accelerate drug development and market entry [1]. - Despite the growing application of AI, very few drugs empowered by AI have entered clinical trials, indicating a gap between AI potential and practical outcomes [1]. - The use of AI in drug development can significantly reduce time and costs, with the potential to discover candidate compounds more quickly and comprehensively [9][28]. Group 2: Rentosertib Clinical Trial Results - Rentosertib, the first drug entirely discovered and designed by AI, showed good safety and tolerability in a phase 2a clinical trial, with an average improvement in forced vital capacity (FVC) of 98.4 mL compared to a decline of 20.3 mL in the control group [2][8][18]. - The trial involved 71 IPF patients across 22 centers, assessing safety, tolerability, pharmacokinetics, and FVC changes over 12 weeks [15][16]. - The results indicate Rentosertib's potential anti-fibrotic and anti-inflammatory effects, warranting further research in larger trials [2][18]. Group 3: Traditional Drug Development Challenges - Traditional drug development is characterized by high risks, long development cycles, and significant costs, with an average expenditure of $2-3 billion and a timeline of 10-15 years to bring a new drug to market [7]. - Approximately 90% of candidate drugs fail during clinical trials, underscoring the urgent need for effective therapies for diseases like IPF, which currently has limited treatment options [7][8]. Group 4: Future Outlook - The successful clinical data for Rentosertib marks a milestone in AI-driven drug development, providing hope for IPF patients and indicating a shift in the pharmaceutical industry towards AI as a new engine for drug discovery [24][28]. - The continuous advancements in generative AI are expected to redefine the "Moore's Law" of the pharmaceutical industry, enhancing the efficiency and effectiveness of drug development processes [24][28].