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The power of partnership in small molecule discovery and development

July 10, 2024 by Staff Writer (9 minute read)

Category | Small molecule


Small molecules are foundational to developing new treatments for a wide range of diseases due to their precise interaction with biological targets. However, the journey from lab to patient is not an easy one. Discovering these molecules is a complex process that requires sophisticated technologies and methodologies to identify and optimize potential candidates. And moving a molecule from discovery to development requires seamless integration and coordination to ensure the molecule's therapeutic potential is fully realized. The ability to streamline this process with the right tools and collaborative efforts can significantly enhance the efficiency and success of drug development as well as reduce the overall time to first in human (FIH) trials.

The road to discovery

Drug discovery is the process of identifying new molecular candidates. Advanced high-throughput screening and fragment libraries are essential tools in this process. They enable the rapid identification and optimization of potential drug candidates by testing thousands of compounds quickly and efficiently.

Thermo Fisher Scientific’s Maybridge screening collection is an example. The collection consists of a highly diverse set of more than 51,000 hit-like and lead-like molecules widely acknowledged as a critical tool in screening campaigns. These are individually designed compounds, produced by innovative synthetic techniques. Known for their diversity and quality, utilization of the Maybridge screening compounds and libraries can significantly accelerate drug discovery campaigns and increasing success rates.

For example, the collection was integral to the discovery of several key antituberculosis drugs, including pyrazinamide, which have shown promising results against drug-resistant Mycobacterium tuberculosis strains. This achievement was realized by screening a collection of 1,000 fragments from the Maybridge Ro3 library.

Another compound identified from the Maybridge screening collection led to the development of Olaparib (Lynparza), an FDA-approved drug for ovarian, breast, and prostate cancers. These libraries not only provide a high probability of discovering hits but also ensure that these hits possess drug-like properties, reducing the chances of attrition in the later stages of drug development.

From R&D to cGMP: Bridging the gap

Transitioning from small molecule drug discovery to development involves several challenges, each critical in ensuring the successful progression of a candidate molecule from laboratory research to clinical trials and beyond.

One significant hurdle is scaling up from lab-scale synthesis to higher production volumes needed for clinical trials later. This involves not only increasing the quantity of the compound but also optimizing the synthesis process to maintain quality and consistency at larger scales.

Additionally, robust preclinical testing is essential to assess the safety, efficacy, and pharmacokinetic properties of the candidate molecule before advancing to human trials. This stage requires meticulous planning and execution to generate comprehensive data that can support regulatory submissions.

Furthermore, manufacturing process development and route scouting are crucial steps in this transition. Identifying the most efficient and cost-effective manufacturing processes early on can significantly impact the scalability and commercial viability of the drug candidate. Route scouting, in particular, involves exploring various synthetic routes to identify the most suitable one for large-scale production.

Managing regulatory compliance poses another formidable challenge. Throughout the development process, adherence to regulatory guidelines and requirements is critical to ensuring the safety and efficacy of the drug candidate. This includes compliance with Good Manufacturing Practice (GMP) regulations to guarantee the quality and consistency of the product manufactured for clinical trials and eventual commercialization.

By addressing these challenges comprehensively and proactively, pharma and biotech companies can facilitate a smooth transition from small molecule discovery to development, laying a solid foundation for successful drug development and commercialization endeavors.

Strategic partnerships can help in this endeavor. Full-service contract development and manufacturing organizations (CDMOs) play a pivotal role in coordinating efforts across different phases of drug development. Moving from high-throughput screening and fragment libraries directly into a CDMO environment under a single trusted partner adds further value by ensuring continuity and coherence throughout the drug development process. Working with a single vendor across these critical phases ensures that important steps are not missed and potential risks are identified early. This approach also sets the stage for a long-term partnership based on a deep understanding of project requirements and goals.

For example, once a development candidate has been identified via Thermo Fisher's Maybridge resources, customers seeking a development partner can streamline the progression into API development for preclinical activities by leveraging Thermo Fisher's API drug development and manufacturing services. If successful, this progression can continue seamlessly into clinical development and commercialization.

By executing early-stage drug discovery and preclinical drug development with one partner, customers benefit from seamless integration between these phases, minimizing the risk of information loss or miscommunication during handoffs between multiple providers. Thermo Fisher's expertise across both domains ensures efficient translation of research findings into scalable and compliant manufacturing processes, bridging the gap between synthetic routes optimized for research and those necessary for GMP production. Additionally, this integrated approach saves time, helps understand API critical parameters, and ensures the formulation fit and desired bioavailability for clinical trials.

Leveraging internal synergies to achieve better results

Pharma companies can streamline their small molecule drug development by leveraging the internal synergies of a single, trusted provider for discovery resources and development and manufacturing services. The advantages include:

  • Integrated knowledge and experience, which fosters a deep understanding of each project's intricacies and enables a smoother transition from discovery to development.
  • Time and resource efficiency as well as cohesion achieved through pre-established alignment in approach and objectives.
  • Harmonized quality standards and practices, which ensure comprehensive oversight, meticulous attention to detail, and product and process reliability throughout the drug development journey.
  • Customized solutions and flexibility: A shared view of the specific needs of each project from discovery and early development to scale-up and commercialization better enables internal teams to adapt to changing project needs and timelines.
  • Proactive risk management, which anticipates potential issues early in the process and implements strategies to mitigate them, ensuring a more reliable and efficient development pathway.

Collectively, these benefits can help companies bring innovative, effective therapies to market more efficiently.

Learn how Thermo Fisher’s CDMO services can support your small molecule drug discovery and development projects.

Additional resources

Maybridge screening and fragment libraries

Small-Molecule Drug Discovery