A novel way of developing new pharmaceuticals
The Swedish drug discovery and development company Axcentua Pharmaceuticals has seen a most interesting development since its inception in 2007. The company develops small-molecule drugs derived from natural compounds through a unique process, by Axcentua called Crystal re-engineering, which, when applied to natural compounds, transforms a bioactive compound into an experimental medicine for clinical testing that can be developed into a novel medicine.
CEO and Co-founder Stefan Rehnmark gives several examples of known natural compounds that possess huge potential as drug candidates, but are limited by poor pharmaceutical properties.
Soybeans, for example, contain the natural compound Genistein that in several preclinical studies has been shown to sensitize tumor cells to chemotherapy and kill cancer cells. Genistein, with its multi-targeted actions, has also been shown to have beneficial effects on other diseases, making this compound an interesting candidate as a novel medicine.
Transforming natural compound
However, even though natural compounds are a rich source of substances for drug development, their use as potential drugs are in many cases limited by poor pharmaceutical properties. It has also been difficult to obtain patent on known natural compounds, further limiting the development of interesting natural compounds into new drugs.
By using Crystal re-engineering, it is possible to transform natural compounds with known medicinal properties into candidate drugs and receive patent protection on the new crystalline forms, Stefan adds.
Axcentua applied Crystal re-engineering to Genistein and identified several new crystalline forms with significantly improved properties. One of the new crystalline forms, AXP107-11, was selected for further development and is currently in clinical trials.
AXP107-11 was developed from bench to clinic in only two years showing that crystal re-engineering is a unique strategy for accelerated drug development. Patent protection of the crystalline form, the crystallization process, composition of matter, and therapeutic use of AXP107-11 is secured through a recent US patent.
An additional advantage of the process is lower risk of failure in clinical trials because the effects of natural compounds are more widely documented, unlike those of synthetic compounds, which are used by most major drug companies. This translates into an accelerated drug development process, leading to dramatic savings time-wise.
“Our development process is the result of innovation. The company thrives on that idea. There is so much potential and if our patent strategy holds, the sky is the limit,” Stefan continues.
Proceeding with clinical trials
Clinical trials are now underway at the Karolinska University Hospital giving AXP107-11 in combination with chemotherapy to patients with pancreatic cancer.
Axcentua recently received an Orphan Designation from the European Medicines Agency for the development of AXP107-11 for the treatment of Sanfilippo disease, a rare genetic disorder leading to mental retardation and early death, and are currently preparing for clinical trials in this small patient population.
“Our goal is to develop new effective treatments for diseases with unmet medical needs, however, even though Crystal re-engineering speeds up the development process, the time for a new drug to reach the market is still quite long”, Stefan concludes.