New Zealand-based biotech Neuren Pharmaceuticals has announced impressive preclinical results in the Fragile X mouse model with two of their lead compounds, NNZ-2566 and NNZ-2591.

These compounds are examples of a new class of drugs based on insulin-like growth factors (IGF-1). IGF analogs are currently considered the most promising approach for treating Rett Syndrome, a fatal genetic disorder that affects only girls, and one of the other leading genetic models for the study of autism (along with Fragile X).

IGF-1 (source: Wikipedia)
IGF-1 (source: Wikipedia)

The surprising news is that FRAXA researchers have found that this treatment strategy works even better in Fragile X knockout mice than in Rett syndrome mice!

FRAXA’s strategy is to find and target the critical bottlenecks which block the way to development of treatments. In this case, a small pharma company with interesting compounds approached FRAXA to get it tested in Fragile X. The Drug Validation Initiative, or DVI, a testing facility in Chile funded by FRAXA, was able to very quickly take the compound and test it in Fragile X mice, in a “blinded” fashion.

The next step is to organize clinical trials of this new medication for people who have Fragile X. Stay tuned…

Neuren Pharmaceuticals announcement

FRAXA DVI: drug testing facility