Servier

Oncodesign Precision Medicine reacquires rights to OPM-201 program from Servier after a positive Phase I trial in healthy volunteers, confirming the molecule's safety. OPM aims to develop the program internally for Parkinson's disease treatment.

The pharmaceutical industry must balance patient care with reducing environmental impact, accounting for 4.4% of global CO2 emissions. Strategies include optimizing the value chain, relocating manufacturing, and adopting renewable energy. Breakthrough innovations like AI and eco-design can accelerate drug development and reduce waste. Countries are setting goals for carbon neutrality, with the US aiming for 50% reduction by 2030 and the UK's NHS targeting neutrality by 2040. Collaboration among stakeholders is crucial for achieving these goals.

Digital twins, digital representations of diseases using real-life patient data, offer quicker, innovative solutions to test therapeutic efficacy. They simulate body processes, drug behavior, and identify biomarkers, aiming for personalized treatments. Aitia, a pioneer, uses Gemini Digital Twins for diseases like cancer and neurodegeneration. Benefits include accelerated research, better disease understanding, and reduced development errors.

The IDH market is set to grow rapidly due to expanded indications for approved therapies and increased R&D. Key players like Servier and Bayer are developing novel IDH inhibitors, with IDHIFA (enasidenib) being the first-in-class oral targeted inhibitor for IDH2-mutated AML. IDH inhibitors, including TIBSOVO (ivosidenib) and REZLIDA (enasidenib), are FDA and EMA-approved for various cancers. Vorasidenib, in advanced development, aims to treat IDH-mutant diffuse glioma. The market growth is driven by advancements in research and expanding pipeline of IDH inhibitors.

Three clinical trials from MD Anderson Cancer Center showed significant results with novel triplet therapies for leukemias. The SAVE trial achieved an 82% response rate in advanced AML with KMT2A or NUP98 rearrangements. The IDH1-mutant AML trial showed a 94% overall response rate. The CLL trial achieved 98% undetectable MRD in bone marrow and 100% in blood after 13 cycles.

Despite headwinds, the techbio sector continues to attract significant investment and drive innovation, exemplified by the 2024 launch of Xaira, backed by over $1 billion, aiming to design novel therapeutic proteins using advanced AI. The sector is maturing through strategic consolidation, significant capital deployment, technical diversification, and steady clinical validation, as illustrated by 25 techbio organizations, including notable players like Isomorphic Labs, Generate:Biomedicines, Recursion Pharmaceuticals, and Schrödinger Inc., all leveraging AI for drug discovery. Emerging players such as Archon Biosciences, AION Labs, and Antiverse are also making strides, while established companies like Insilico Medicine and Ginkgo Bioworks continue to expand their platforms and partnerships.

Bio-S, a new production unit for active ingredients derived from living cells, is a significant step in developing new therapeutic solutions. Located at Gidy site, it integrates R&D and CMC expertise to support clinical trials. Bio-S aims to meet medical needs in oncology and neurology, with an investment of 85 million euros. It covers various professions, including biomanufacturing and quality control, and is expected to start producing clinical batches by end of 2025.

The module discusses precision oncology, emphasizing integrated healthcare solutions, genomics testing, and next-generation sequencing (NGS) recommendations for solid tumours. It highlights the importance of structured genomics reports and the transformative impact of clinical trials. The author also explores socio-economic and ethical implications of molecularly-guided treatment options, advocating for multi-stakeholder collaboration.

EFPIA highlights EU's roadmap for precision oncology to address rising cancer burden, despite forecasted mortality rate declines. Stakeholder roundtable in Brussels aims to improve personalized cancer treatment. EU initiatives, including AI-powered cancer care and the DigiONE pilot, aim to advance precision medicine. A gold-standard cancer data management system is necessary for innovative precision medicine access, emphasizing standardized data formats, robust privacy, and professional education.