A challenging aspect of conducting clinical studies is execution of the work associated with bioassays which is magnified in low- and middle-income countries (LMICs). Here one is faced with infrastructure failures in power, availability of water and staffing stability. Shipping samples, a routine activity in high-income countries, is often a major challenge. Working in LMICs requires constant problem solving, extreme attention to details and always having a plan B.

This talk explores the challenges and opportunities in implementing biomarker driven clinical trial assays in oncology from a Diagnostic provider and Biopharma Drug Development viewpoint. We’ll discuss what’s needed to have a test truly ‘fit for purpose’ in clinical trials and how to future proof for a successful CDx.

Researchers have pioneered new ways to target oncogenic drivers – lung cancer alone has more than seven targetable genetic alterations and subtypes. New developments are helping achieve a precision approach for immunotherapy too. PD-L1 offers enrichment – not precision. With mutation burden, microsatellite instability and other immune biomarkers, we may be able to advance treatment. Evolution and real-world adaptation of testing tools will become the new focal point to advance patient care.

The COVID-19 pandemic accelerated advancements in diagnostic, therapeutic, and vaccine development. As a leading provider of biospecimens, BioIVT supported these studies by leaning on our experience and knowledge in biospecimen procurement, donor outreach, IRB-approved collection / processing protocols, and data and sample validation. We will review a case study of BioIVT’s rapid establishment of new collection networks to provide much needed COVID-19 samples to researchers throughout the world.

Drug developers routinely use biomarker endpoints to measure drug exposure, pharmacology, efficacy and safety as critical drug development tools. Attrition due to safety in drug development is a major issue. Safety biomarkers are enablers for risk mitigation and to advance promising molecules to patients. Biomarker research is enabled through precompetitive collaborations between academia, industry and regulators. Case examples and Innovative Medicine Initiative-TransBioLine consortium enabled biomarker development will be presented.

Realizing the vision of Precision Medicine as a key driver of future healthcare involves the work and coordination of multiple institutions and stakeholders. Pharmaceutical companies are key players in this field, and they are constantly developing new business models to accelerate this pace. One of this business models is the development of strategic alliances between key players. We can classify these industry alliances in four big categories: acquisition/equity investments, co-promotion, trial collaborations and R&D/marketing/licensing. We will analyze the most relevant industry alliances and we will offer a vision on what to expect from the new era of Precision Medicine.

The growing number of biomarker-driven therapies that are becoming available to patients highlights the importance of diagnostics in precision medicine. Since the first companion diagnostic was approved in 1998, the regulatory framework has evolved to accommodate advancing technologies, complex indications and molecular signatures. This presentation will provide an overview of the diagnostic regulatory landscape, describe the flexibility over time, and discuss considerations and best practices for drug and diagnostic co-development.

Given rapid growth of precision medicine, the volume of public data on clinical diagnostics and predictive biomarkers in cancer has expanded immensely.  With the Veri platform, Larvol is leveraging text mining, manual curation, and AI to consolidate biomarker data and develop analytics and visualization tools to turn data into insights. 

Burning Rock Dx is the market share leader in China for NGS based precision oncology. We would like to share our perspective of the NGS oncology CDx space in China along with some interesting data and studies we participated in. We have excellent working relationships with the HGRAC/NMPA and can help with commercialization, Day 1 assay availability, and regulatory approvals in China. We also wanted to offer our perspective of how a China clinical trial or study best fits into global trial/study plans and data sharing considerations: TMB as biomarker (HPCA), ctDNA as a predictive and prognostic surrogate marker, cMET exon 14 skipping mutation as a unique profile marker for treatment of NSCLC.

As technologies evolve and regulatory agencies throughout the world change requirements for CDx, pharma companies have to continually adjust their global CDx strategies. In this talk, we will discuss some of the recent changes, challenges, and solutions.

In the change of “one-size-fits-all medicine” to “precision medicine,” many biomarker-driven studies are conducted worldwide. Identification of a candidate biomarker for the purpose of CDx development is key for successful drug and CDx approval. Collecting information such as clinical practice and available testing methods among others from regional teams are also important to develop the CDx strategy as well as testing implementation in each region after drug and CDx launch.

Circulating microRNAs are entering clinical practice as biomarkers for early detection and precision medicine. In this talk, we will share MiRXES' experience in developing and validating a serum microRNA-based diagnostic for gastric cancer early detection and how this experience became the basis for an industry standard that sets out the key considerations for the design, development, and performance evaluation of microRNA-based clinical diagnostic assays. 

As science advances, our technologies and therapies have evolved and data-driven decisions have become the new normal. This introduces an opportunity for clinical trial design to evolve, as well, to better deliver for patients, physicians, and oncology research. A novel trial design will be presented that involves: broad exploratory analyses, to better characterize disease mechanisms and support biomarker-driven patient enrollment on clinical trials, while introducing transparency with physician and patient.

“The Parsortix® system (RUO), from ANGLE, captures a broad range of cancer cells from blood, including both epithelial and mesenchymal phenotypes as well as CTC-clusters. The system is used in many of the world’s leading cancer research centres. ANGLE is now opening two GCLP compliant service labs to provide global support to clinical trials. The service includes enumeration of CTCs, a menu of tumor related biomarkers and custom assay development.”

Comprehensive molecular profiling of cancer cells using genomics has shown that advanced tumors can be very different from one another leading to a new appreciation of cancer individuality, immense genomic complexity and diversity with respect to treatment. Certainly, breakthroughs in immunotherapy and targeted therapies (i.e. RTK, NTRK and checkpoint inhibitors) stress the importance of predictive biomarkers to guide therapy. However, many patients don’t respond or build resistance to treatment requiring comprehensive genomics to understand the individual tumor and guide therapy.

The rapid advancement of immuno-oncology (I-O) therapeutics has necessitated a deeper understanding of the tumor microenvironment (TME) in order to both justify novel I-O combinations and identify patients that may achieve benefit. Biomarkers such as PD-L1 IHC, TMB, and MSI have exemplified the impact biomarkers can have on patient outcomes in I-O monotherapy. Emerging data suggests that combination therapies offer potential to enhance benefit and will further illuminate factors influencing anti-tumor immunity.

Precision medicine has a widespread source of imprecision: preanalytics that impact the quality of patient biospecimens, the sources of all molecular data. Artefactual alterations caused by preanalytics skew data from molecular analyses, making it uninterpretable, misleading or invalid. The College of American Pathologists is addressing this issue through laboratory accreditation, but involvement of all professionals in the specimen quality chain is required in order to resolve this pressing issue.

This talk will discuss a newly formalized consortium, describing the cooperative development of a gene probe set and core gene content shared among numerous academic laboratories, and how this multi-institutional alliance was formed. The initial motivation was reagent cost sharing but evolved into a consortium that is cross-validating assays, troubleshooting and sharing bioinformatics and sample resources to enhance NGS technologies across institutions.

Therapies under development for neurodegenerative diseases employ strategies designed to modify disease processes. Detection of pre-symptomatic disease-related changes will help identify patients for whom treatment will be effective. An overview will be provided of the non-invasive diagnostic/prognostic methods under development for neurodegenerative diseases, with an emphasis on the studies that demonstrated that genomic signatures in the periphery may associate with disease stage, progression, and response to treatment.