The Problem
A substantial investor in a small, publicly traded pharmaceutical development company came to us in a bind. After obtaining promising pre-clinical results with their lead compound, development was languishing. The company had burned through nearly all of its capital conducting three Phase 1 and Phase 2 clinical trials without establishing a meaningful signal. The investors needed to decide whether to invest more capital to rescue the program or to cut bait. They asked us to perform a complete analysis of the company and technology including market opportunity, manufacturing partnerships, pre-clinical and clinical data, and regulatory status and plan.
Our Actions
Our team performed a full company assessment that included:
- Identifying flaws in the company’s clinical trial design for its lead compound that had made it practically impossible to evaluate efficacy.
- Evaluating the R&D budget critically using decades of real-world experience to better project the cash requirements for valuation-driving milestones.
- Reviewing and auditing each essential vendor to evaluate suitability, risks, and fit with company requirements.
- Formulating a regulatory strategy to remove a Partial Clinical Hold that had been placed on each of three open INDs.
Based on this deep dive, the investment group made the decision to re-invest in the company, rescuing the technology. Subsequently, the Company retained our Team on a fractional basis to manage and execute the R&D activities, filling gaps they identified in the management team. Here’s a snapshot of how we helped the client:
- The Company was substantially in arrears with multiple vendors, stalling additional development and preventing initiation of new clinical testing. Leveraging existing relationships and providing credibility to their team, we were able to step in and negotiate payment terms on behalf of the Company, allowing a critical toxicology study to be completed and returning to the company access to its drug product.
- We took over as FDA correspondent, helping the Company obtain a release on the Partial Clinical Hold that limited the Company’s ability to evaluate the lead drug clinically.
- We led the effort to develop a new protocol with a different dosing regimen to better assess efficacy, which was approved by FDA.
- We managed the production of a new batch of the lead drug, coordinating with the API vendor in India and drug encapsulation vendor in the US.
- We facilitated the design and setup of a future Phase II clinical study, including:
- Identifying and vetting sites;
- Obtaining FDA IND approval and central IRB and site IRB approvals;
- Directing and overseeing the contracted CRO to execute the trial;
- Managing packaging and labeling as well as bioanalytical work surrounding the study drug.
Outcome
Based on this successful engagement, the Company was able to regain the confidence of its investors, vendors, and FDA. A clinical program that had been dead in the water was revived.
