Rapid Proof-of-Concept for Novel Drug Treatment for Rare Metabolic Disease

Case Study

Emerging US biotech company requiring integrated program management of outsourced Investigational New Drug applications (IND)-enabling preclinical studies for first lead compound
Assistance with documentation for filing IND
Testing human safety and potential efficacy of novel small molecule drug
Financing dependent on expeditious development and linked to development milestones

Integrated project team led by Celerion program director supported by experienced program manager
Assisted the client with planning of drug development program and managed all outsourced studies
Provided regulatory guidance, facilitated PreIND meeting with the FDA, and prepared IND documentation
Developed and validated challenging bioanalytical assays in time for IND-enabling toxicology studies and first-in-human study
Biomarker assays developed at academic institutions adapted within Celerion labs, optimized, and validated for deployment in clinical studies
Phase I Multiple Ascending Dose (MAD) study design incorporated biomarker of target enzyme activity
Samples collected and processed at multiple clinical sites with biomarker assays performed at centralized bioanalytical lab
Coordinated complex sample collection and processing logistics
Provided flexible scheduling of biomarker analyses to support multi-site proof-of-concept Phase II studies

Completely outsourcing drug development program enabled the client to focus internal resources on Discovery needs
Preclinical work completed and IND submitted within challenging investor-driven timelines
Biomarker results from Phase I MAD study demonstrated proof-of-concept, enabling the client to raise additional financing
Biomarkers translated from lab bench to clinic with “fit-for-purpose” assay validation; qualified assays available for the client to carry forward to Phase II and III
Knowledge acquired from program was leveraged by the client to facilitate even faster drug development of follow-on molecules