Therapeutic window is a very crucial issue in drug development. It is a well-defined range of serum concentration of the drug at which the desired effect occurs. Below this concentration there is little or no effect, and above – there is too high toxicity. This concentration window is usually very narrow, and drugs that fail do so due to high toxicity and severe adverse effects. One of the fresh examples is cavosonstat:
“Nivalis Therapeutics Inc. (NVLS -54.40%) shares fell 56% in the extended session on Monday after the drug developer said its cystic fibrosis drug failed to reach endpoints in a clinical trial. Nivalis shares, which had been briefly halted, fell to $2.73 after hours. The company said a mid-stage clinical study of its drug cavosonstat did not significantly improve lung function in cystic fibrosis patients when compared to a placebo” (more info here)
In simple words it means that the therapeutic concentration of the drug in this study was not reached. Why?
Because of safety reason – in a clinical trial the dose of a drug is chosen from the safety perspective, i.e. the dose cannot be escalated limitlessly due to dramatically increasing adverse effects. Cavosonstat is a drug candidate for treatment of cystic fibrosis – a pulmonary disease, and what if this drug could be targeted selectively to lung tissue? The answer is obvious – the outcome of the clinical trial probably would be much more promising if the exposure of the rest of the body to the toxic action of the drug would be substantially lowered. Is it possible? The answer is again obvious – yes.
But how can it be achieved pure technically?
Please, welcome BeloGal® platform! According to our in vitro and in vivo investigations, BeloGal® platform increases concentration of a drug in lung tissue about 10 times compared to the parent drug, regardless of indication. This opens a new tremendous opportunity for development of novel medicinal products for treatment of all possible respiratory/pulmonary diseases: cystic fibrosis, COPD, pneumonia, TB, candidosis, asthma, lung cancer, etc.
I would just wish the developers of new medicinal products do not repeat the mistake of Nivalis Therapeutics and jeopardize both the drug development process and patient safety by not using the best tools available today in the form of the drug-delivery BeloGal® platform. Patients really need superior medicinal products.