Providing clinical due diligence of a novel biologic to treat respiratory disease

Challenge

A pharma company looking to purchase an asset needed a respiratory consultant to conduct clinical due diligence of a Phase II biologic designed to treat a respiratory condition.

Solution

Our respiratory expert examined the biotech company’s:

• investigator brochure
• clinical study reports
• publications from previous clinical trials
• statistical analysis plan
• clinical protocol for the ongoing Phase IIb clinical trial
• correspondence from the European Scientific Working Party concerning potential pediatric development
• correspondence from the FDA (CBER) in response to an IND submission for the Phase IIb protocol

We found that from the information provided, the product at best delivered modest improvement in clinical outcome and had significant toxicity/tolerability concerns. All patients developed anti-drug antibodies with increasing concentrations over time. The data suggested a potentially negative risk/benefit balance in patients with a severe form of the disease where efficacy is likely to be more difficult to demonstrate, and where its use may be associated with a greater risk of more severe adverse events (including anaphylaxis).

Our due diligence team recommended that in-licensing or acquisition of the asset be considered only if the results of the ongoing Phase IIb study provided more compelling evidence than available at the time of the assessment.

Soon after our assessment, the Phase IIb failed to meet its primary endpoint and the program was terminated by the company.