Could a successful HIE trial and drug registration be the ‘back...

Could a successful HIE trial and drug registration be the ‘back door’ into the treatment of TBI, stroke and all the other hypoxic related brain pathologies?

This has certainly crossed my mind. In the same way that successful use of NNZ-2591 in multiple neurodevelopmental disorders that overlap with autism provides a strong case for its applicability in autism, successful use of NNZ-2591 in HIE in neonates would inspire confidence in its potential benefit in other global hypoxic-ischemic brain injuries, caused by events such as cardiac arrest, stroke and TBI.

Clearly, if the intention was to treat TBI or stroke, IV administration of NNZ-2591 would be more appropriate than oral dosage. Also, in acute HIE in neonates, where the bubs are still in ICU, I would think that IV administration would be more likely. An oral dosage form might subsequently be used in the chronic setting.

In the preclinical model of hypoxia-ischemia in adult rats, NNZ-2591 was administered both intracerebroventricularly and subcutaneously.

And although there is a specific patent for an oral formulation of NNZ-2591, the patent for NNZ-2591 in autism, for example, makes claim for administration via multiple routes including oral, intravascular, subcutaneous, infusion and intraspinal.

Also, I note that earlier this month, Neuren filed two new patent applications for a process for preparing NNZ-2591. There are no details yet so it's unclear whether this is relevant.

Neuren, a relative minnow in the world of pharma, has stated that its focus is on developing treatments for rare neurodevelopmental disorders which emerge in early childhood.

However, a much larger pharma with interest in NNZ-2591 could certainly be interested in the drug’s much broader potential application, especially if there is compelling clinical data which supports this.