Challenge
Our client specialized in the development of proprietary monoclonal antibodies directed towards novel targets for the treatment of immune-mediated and fibrotic disorders, including orphan indications. They had identified a novel pathway that mediated inflammation and fibrosis, and as such had developed proprietary antibodies to target it. For their lead antibody they planned to initiate a Phase IIa proof of concept (POC) repeat dose safety study in patients with Primary Sclerosing Cholangitis (PSC). This was supported by a repeat dose toxicology study conducted in mice and a Phase I, single escalating dose clinical study in healthy volunteers. In order to validate the planned Phase II study and to confirm that the supporting data would allow approval of the trial, the client wished to obtain scientific advice from the regulatory agency.
Solution
Alacrita helped facilitate interactions with the regulator in preparation for the Phase II in the following areas:
- Project management and liaison with the client and the agency
- Input into strategy, communication of mechanisms and timelines
- Review of meeting request letter
- Assistance with preparation of Draft Questions and Company Positions
- Review of briefing document
- Assistance with publishing and submission of briefing package
- Meeting preparation and attendance
- Preparation of meeting minutes and follow-up activities
The Scientific Advice meeting was a success; the trial protocol and supporting data were deemed acceptable by the agency.