This morning at the United European Gastroenterology week Cour Pharmaceuticals provided successful results of their Phase 2a test of their drug.
Cour Pharmaceutical describes their drug as, ” a biodegradable nanoparticle encapsulating gliadin proteins – the major component of dietary gluten.” The nanoparticle is ignored by the immune system and delivered to the gut. Once in the gut the nanoparticle releases a macrophage that acts as a “vacuum cleaner” sucking up the gliadin particles, destroying them, and escorting the debris out of the system. Pretty cool.
Cour Pharmaceuticals did a small Phase 2a study with 34 patients. Treatments were administered intravenously (IV) on day 1 and day 8 of the trial. Participants were then given 12g of gluten (that is the equivalent of about 5 slices of bread) for three days and then an additional 6g of gluten (about 2 slices of bread) for an additional 11 days. Then they directly tested the gluten related T-cell activation in the patients and noticed the drug blocked the T-cell activation. Patients treated with the drug demonstrated a 90% reduction in immune inflammation compared to the untreated patients.
T-cells are what cause reactions in celiac disease. So, if this drug blocked the T-cell activation, that is f&^king amazing!
In the study there were 34 participants. Only 28 completed the study and the other 6 dropped out due to gluten related symptoms. Side effects in those that completed the study such as nausea, headache, backache, and stomach ache were reported at a greater rate than those on placebo. So, the drug may have some unpleasant side effects.
Cour Pharmaceuticals has just sold the license for the drug to Takeda pharmaceuticals to develop and commercialize the drug. Takeda is a giant pharmaceutical company that is heavily involved in several drugs currently under development for celiac disease. These guys are committed to finding a cure.
Couple of things to remember and not to rain on any parades, but….this was a very small study. About as small as the initial Phase 1 Nexvax study and we all know how that ended up. This drug seems to have more side effects than Nexvax did. I’m okay with side effects if the stupid thing works, right? This might also be an ongoing drug to take rather than one or two doses. So, we might be looking at a lifetime of medication. Again, a cure and I have to go to the MD monthly to get an IV, sign me up!
The good news is that a MAJOR pharmaceutical company has taken this product into its machine. That is AMAZING and makes this drug much more viable than a small company trying to do it on its own.
This is very, very exciting news in the celiac community and hopefully, a giant leap forward in the search for a cure! This might be what we have been looking for. Woo Hoo!!
Here is a link to excerpts from the presentation with results. It is very technical but on page 2 they talk about side effects. Page 4 they talk about how the drug protected against villous atrophy. Page 6 they demonstrate that the CD4 and CD8 T-cells were not activated in patients on the study drug.