Case Study: Rapid Proof-of-Concept for Novel Drug Treatment for Rare Metabolic Disease
Consultancy.
Customized Program Planning.
Scientific Input
Need
- 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
Approach
- 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
Benefits
- 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