Success Story: Series A Biotech Company
There were three questions:
1. Would their lead compound be efficacious in an animal model developed at Massachusetts General Hospital (MGH)?
2. Could a novel imaging technology developed at MGH be used to monitor treatment response with their lead compound?
3. Could their lead compound be radio-labeled with a positron emitter to enable human whole body pharmacokinetics and potentially target occupancy and target quantification studies?
How we helped
The i3 met with the company’s senior team to understand their needs and provide guidance on how imaging could be incorporated into their development process. We identified three key objectives: compound efficacy validation in an MGH animal model; therapy monitoring using MGH imaging technology; radiolabeling of their lead compound, and IND enabling studies for the labeled compound.
The i3 created a multi-tiered project plan that was tranched with clear milestones and executed a service agreement within one month of first contact. We built a team of three MGH principal investigators to execute the studies and the i3 managed the project.
The efficacy validation and therapy monitoring studies were executed on time, with full reports delivered to the company. We utilized a mouse model developed at MGH, with ex vivo biochemical and histological analyses to characterize the model. A quantitative MRI protocol was utilized to monitor therapeutic response. We developed chemistry to radiolabel the lead compound.
During the radiolabeling project, we needed to overcome a limitation in the synthetic procedure in order to synthesize material with sufficient specific activity for human use, and identified a catalyst that could enable the chemistry. We coordinated a materials transfer agreement with a third party academic site to obtain the catalyst, and delivered a radiochemical synthesis that was compatible with human use.
The radio-labeled compound was used to assess specific binding and whole body pharmacokinetics using positron emission tomography (PET) imaging in rodent models
This project ran for a six-month period with multiple outcomes:
• A study report describing treatment effects of an experimental drug in a mouse model of disease along with positive and negative controls.
• An imaging method to noninvasively monitor treatment response.
• A robust radiochemical synthesis of the lead compound that produced >10 mCi of product.
• Whole body pharmacokinetics and target specificity of the lead compound.
• For each tranche of the project a complete report was delivered to the company, and all projects were performed on time, on budget.