New Zealand-Based Biotech Neuren Pharmaceuticals Has Announced Impressive Preclinical Results in the Fragile X Mouse Model with Trofinetide.
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).
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.
Studies are continuing at Neuren.
IGF-1 (source: Wikipedia)