Taking research from the bench to the bedside is an infamous graveyard in biotech. Researchers from Charité and their colleagues have a plan to help startups survive.
“Many basic research approaches currently fail far too late in their development,” write researchers from Charité University Hospital, based here in Berlin, in collaboration with colleagues from the University of Utah, Salt Lake City. These false positives drain research budgets that are already strained, and the failures in translating basic research have come to represent what the biotech industry describes as ‘The Valley of Death.’
What’s to be done? The researchers outlined a 12-point strategy to help mitigate the risks embedded in drug development, published in Cell: Trends in Biotechnology. We’ve cherrypicked the important points for startups to consider.
Bring your basic research closer to clinical and business development groups, as their expertise will help you build a bridge across the valley of death. You might also consider patient advocacy groups as partners: a notable example included in the paper lies in the approval of Kalydeco, which was developed from research funded by the Cystic Fibrosis Foundation and became the centerpiece of a $3.3B deal with Vertex Pharmaceuticals.
From the start, you should have an early market assessment of clinical needs and product targets, as well as a regulatory strategy. Though these analyses are ‘unconventional’ in academia, write the authors, it’s important to “fine-tune preclinical and basic research hypotheses earlier in the process to provide a clearer path to a suitable clinical solution.”
The paper also highlights the need for a milestone-based strategy, which the authors say is a rarity in academia. While researchers in this setting might pale in the face of a Gantt chart, a successful company typically has a map with checkpoints that include evidence-based criteria for decision-making about if or how to proceed.
So-called ‘fast-failure routes‘ have become popular as a way to swiftly cull expensive or ineffective programs. However, the authors caution about proceeding too zealously down such a road, noting that “many candidates may show efficacy only after modifying outcome measures or integrating suitable biomarkers into their clinical testing” — it might be too early to know for sure if your candidate is deadweight.
It may go without saying, but it’s worth reiterating: be absolutely sure your intellectual property (IP) is solid, because it will be the foundation of your enterprise. In filing a patent, you are guaranteeing exclusive access to the technology, and without that, your leg to stand on in the marketplace is shaky. The academic tendency is to sacrifice attention to IP in favor of inventing and rapidly publishing, so the authors highlight patents as particularly worth investment as you translate research from the lab bench to commercialization.
Basic science is an inherently conservative process and the tendency might be to rely on existing models, even if they aren’t the best fit for your candidate. So, you might consider finding creating one tailored to your program before it advances. Bear in mind that entire companies are built around this concept, but this means that they can help you find one — perhaps a 3D cell coculture or an organoid system.
When it comes time to move to in vivo models, you might find that one for your target indication doesn’t exist, especially if you’re working in Type I diabetes, osteoarthritis or stroke. It might therefore be worth exploring alternatives, but make sure they’re accepted by regulators — involve them as early as possible in the discussion.
Entrepreneurs, what are your learning experiences? Comment below!
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