Nordic Life Science 1
PHOTO PETER BRINCH by treating “root-cause” I thi
nk that we will achieve a better effect, not only when it comes to glucose regulation but also, as the scientific literature indicates, we may see a positive effect on the secondary diseases of diabetes. For example, on cardiovascular diseases, which cause people with diabetes type 2 to die ten years prematurely. Imagine being able to prevent that. That would be cool and obviously very meaningful!” XEN-D0501 is a synthetic small molecule that was in-licensed in 2016. Prior to in-licensing, it had been found to have a good safety profile in other (non-diabetic) patient groups. “The tablet is very small and simple to swallow. Another advantage is that it is relatively cheap to produce, which means that it may be possible to price it quite low, so a lot of diabetics can afford it. This is very important to me because my goal is to help to treat this growing group of diabetics who are currently not receiving adequate treatment,” says Dorte X. Gram. n the eight phase I and II clinical studies that have been performed so far there have not been many side effects and in the two diabetes studies the company themselves have performed, no serious side effects have been registered. “In our most recent study, which was a pilot phase II study, we were also able to show that patients who received XEN-D01501 for one month had a statistically significant better insulin secretion than other patients with diabetes type 2 who received placebo treatment,” says Dorte. Diabetes has increased fourfold over the past 25 years, and according to the WHO, it is a worldwide pandemic. Approximately 90% of all diabetics suffer from type 2 diabetes, whilst approximately 10% suffer from type 1 diabetes. Last summer Pila Pharma was listed on Nasdaq First North GM in Stockholm with the goal to finance the road ahead towards phase IIb data, where they aim to demonstrate that XEN-D0501 has a good effect on blood sugar regulation after three months treatment without contemporary unacceptable side effects. “First, we aim to perform a three month-long preclinical safety assessment study in animals, and then, in about a year, move on to three month tests in humans. Phase IIb is our first great big interim goal, which might trigger a collaboration with a larger pharma company regarding the development through phase III and registration and marketing globally,” says Dorte. “The ultimate goal is to help treat the over 500 million people suffering from diabetes. They and their families will suffer a sad fate if the disease is not treated. There are long lead times within drug development, but slowly but surely we will reach our goal!” she concludes. NLS Dorte X. Gram, founder and CEO, Pila Pharma I