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In biotech, discovering something that can change medicine might just be the easy part. Getting it from a laboratory bench into a hospital, or pathology clinic can actually present as many, if not more challenges than uncovering the discovery itself.
Imagine a hypothetical ASX junior we’ll call XYZ Bio. Its scientists have discovered a molecule they believe could treat an aggressive cancer. Laboratory results look terrific, management understandably gets excited and investors start reaching for their calculators. A simple rejigging of the pit shell and cut-off grades against today’s much higher gold price could potentially add fresh ounces that were previously considered uneconomic, essentially hiding in plain sight.
XYZ Bio doesn’t have a medicine yet. What it has is a promising scientific proposition.
Between that discovery and the first commercial sale sits one of the toughest obstacle courses in business. Laboratory research, pre-clinical testing, human trials, manufacturing, regulators, intellectual property, mountains of capital, and eventually, convincing doctors and patients the product is actually worth using. And there is no single course through this heady maze.
Drugs, vaccines, diagnostics, medical devices, cell and gene therapies and medical software can all take different routes to market. What they all share however, is the need to progressively turn scientific promise into hard evidence. For investors, knowing exactly where a biotech sits on that journey can be every bit as important as understanding the science itself.
Scientists identify a biological target, develop something designed to influence it and begin testing whether the theory stacks up. That can progress into pre-clinical studies examining activity, dosing, toxicity and how a potential treatment behaves inside a biological system.
On paper, the road from there looks remarkably orderly. But biology frequently has other ideas.
Something can perform beautifully in a laboratory dish or animal model and behave very differently in a human being. That makes building the evidence required to bridge laboratory science and clinical development a critical early hurdle.
NeuroScientific Biopharmaceuticals is currently negotiating that part of the journey through its work across immune-mediated inflammatory and neurological conditions, building the scientific and pre-clinical evidence required to advance its technologies towards clinical application.Interestingly, the company today announced that it was sitting down with the FDA at the end of August where the FDA will review the company’s clinical,scientific and product development plan for its StemSmart product for Crohn’s disease to make sure its up to scratch for a formal Investigational New Drug submission slated for the end of this year.
To be clear, pre-clinical evidence isn’t second-rate evidence. It is answering an important earlier set of questions; whether the biology is sufficiently compelling, whether a potential treatment produces the intended effect and whether the evidence supports taking the next step. The considerably more expensive question of what happens in humans comes later.
Perth-based Argenica Therapeutics has already crossed that divide with ARG-007, its neuroprotective peptide. After extensive pre-clinical work, ARG-007 moved into its first human safety study and then progressed into Phase II testing in acute ischaemic stroke patients.
And the questions being asked are changing along the way.
First, is the scientific proposition worth pursuing? Then, can humans safely tolerate it? Only then can the clinical program start to address the question shareholders ultimately want answered; which is can the treatment improve outcomes in patients?
For a conventional drug, that progression broadly runs through Phase I, Phase II and Phase III trials.
