For biotech success: Start with the end in mind and develop a TPP early

During a recent webinar ICON’s Liz O’Brien, Senior Director, Drug Development and Consulting, and Will Maier, Senior Vice-President, Drug Development Services shared their thoughts on what makes a successful biotech. They were joined by Georg Schragel, Director of Business Development, Genmab. Drawing from their years of experience they offered advice on what biotechs should and shouldn’t do to improve their competitive edge. In this article we offer an overview of the webinar and explain why biotechs should start with the end in mind.

Financial trends

Biotechs increasingly rely on venture funding and brokering deals with pharma companies. These approaches are supplanting IPOs as funding or growth strategies. In Q1 2025, 19 biopharma companies raised venture rounds of $100 million or more.¹ In a crowded, highly competitive marketplace the temptation for biotechs may be to adopt speed and focus on proof of concept (PoC) and efficacy. However, this can lead to problems later in differentiation and demonstrating value to potential partners and investors. Taking time to develop a strategy at the beginning will pay dividends in the long term.

Building for success: Drug development strategy

Biotechs need to develop a target product profile earlier in their drug development. “Focusing only on proof of concept without TPP is like building a bridge to nowhere. You may prove biology, but you may not prove a product,” Maier said. A TPP “defines the value proposition and frames a clinical and commercial vision” he added. While it may be years until your product enters the market, having a TPP is critical. “Your TPP is your North Star,” O’Brien said, “it’s not just a regulatory document, it is a map of all of the inflection points from bench to bedside”. In a crowded market, being able to differentiate and demonstrate value for a payer can give a competitive advantage. Building a lean, strategic TPP helps biotechs to avoid dead ends, drives smart development, and aligns endpoints with FDA, EMA and HTA requirements.

Case study

A small biotech developing a novel kinase inhibitor. The company developed a candidate with the aim of PoC in orphan indication. However, they had no defined differentiation against existing kinase inhibitors. There was no clarity of safety margins or dosing advantages. This resulted in a poorly designed phase 2 with the wrong population, irrelevant endpoints, and misaligned data. There was little acquisition interest and outlicensing had a low valuation. This situation could have been avoided if the biotech developed a TPP earlier.

Find and develop partnerships early on

Another aspect of drug development that biotechs often overlook is a key opinion leader (KOL) strategy. For novel treatments, identifying and working with a respected thought-leader can help to convince regulators to adopt a new surrogate endpoint. Planning for this early in the process and building relationships with relevant KOLs will help with the regulatory process.

Companion diagnostic (CDx) strategies should also be in place if a companion diagnosis is likely to be part of the medical use of the product. Biomarkers are to drug development as GPS is to navigation, therefore a biomarker strategy is integral to biotech success. Biomarker and/or CDx strategy should happen in parallel to your development plan. Partnering with a device company is advisable if a CDx is likely to be part of the regulatory approval. Without the right diagnostic device partner in place there can be time and financial costs.

Common mistakes to avoid

In the rush to develop their PoC some biotechs take short cuts such as skipping pre-IND meetings or going for national advice in Europe for a novel treatment. They mistakenly assume that the national regulator’s advice can act as a surrogate for EMA advice. It’s risky to make this assumption. Investing in getting deep regulatory insights will be worth the time and cost in the long run.

“Be frank and honest about the things that you don’t know,” Schragel said, warning against overselling. It is better to lead with science, even if it means waiting for the relevant data that rather than using vague data. Part of the business case is considering payers’ responses and good data will convince payers also. Other areas that funders are likely to subject to scrutiny are Chemistry, Manufacturing & Controls (CMC), , intellectual property and commercialisation potential

Conclusion

For biotechs in the early stages of drug development having a product on the market can seem a very long way off, and it is. However, to maximise the chances of attracting funding or in/out-licensing agreements biotechs should build their TPP early on. This will guide their strategy and ensure that when they seek funding, they can differentiate their products from the competition and demonstrate value to funders.

Learn more about drug development strategies for biotechs, watch the webinar.

References:

¹ Biopharma Q1 2025: Strategic shifts and market dynamics. J.P. Morgan