Study Medication

What is NNZ-2591?

NNZ-2591 is a synthetic analog of a peptide involved in the regulation of Insulin-like Growth Factor-1 (IGF-1), a growth hormone that occurs naturally in the brain. IGF-1 contributes to the growth and maturation of brain cells and the connections between them (synapses) and plays an important role in repairing damaged neuronal cells and improving synaptic plasticity.

NNZ-2591 comes as a ready-to-drink strawberry-flavored liquid and can be taken from a dosing cup or syringe which will be provided by your study team. The medication needs to be taken on an empty stomach, though sips of water can be taken after dosing.

NNZ-2591 is not yet approved by the FDA. This clinical trial is to study its safety, tolerability, and efficacy.

Why could NNZ-2591 be helpful for Pitt Hopkins syndrome?

The objective of treating with NNZ-2591 is to target the genetic deficiency that causes Pitt Hopkins syndrome. NNZ-2591 mimics the actions of the natural peptide, correcting impairments in synapse form and function by reducing neuroinflammation and regulating the available amount of IGF-1 in the brain.

In a mouse model which resembles features of Pitt Hopkins syndrome in humans, treatment with NNZ-2591 normalized all the deficits in the mice, including eliminating seizures, while having no effect on healthy mice.

What are the possible side effects of NNZ-2591?

NNZ-2591 has been studied in a Phase I trial of healthy adult volunteers dosed for 7 days. The following side effects were seen.

All side effects resolved within a couple of days and whilst participants continued treatment. No medical assistance was needed and there were no lasting effects.

You need to make sure you tell your study doctor if you notice any side effects. This is important for assessing the safety of the study medication.