Neuren Pharmaceuticals (ASX:NEU) has received approval from the Food and Drug Administration to conduct a Phase 2 clinical trial of NNZ‐2566 to treat patients with Fragile X Syndrome, expected to commence before the end of 2013.
Fragile X syndrome is the most common inherited cause of intellectual disabilities including epilepsy and autism. It affects 1 out of 4000 males and 1 out of 6‐8000 females, due to a single gene defect on the X chromosome that impacts the FMRP protein responsible for regulating nerve cell synapses.
NNZ‐2566 is a synthetic analogue of a naturally occurring neuropeptide derived from IGF‐1, a growth factor produced by brain cells. In animal models, NNZ‐2566 inhibits neuroinflammation and normalises the function of microglia with consequent improvements in molecular, cellular, anatomic and behavioural outcomes.
Adolescent and adult males will be enrolled in a double‐blind, placebo‐controlled study, which will test two different dose levels of NNZ‐2566.
Preparations for the study are well underway; enrolment is expected to be completed by the end of 2014, with top‐line results announced in the first half of 2015.
This is the fourth Investigational New Drug (IND) approval for NNZ‐2566, adding to the Traumatic Brain Injury, concussion and Rett Syndrome INDs.
The approval of the IND for Fragile X Syndrome reinforces NEU's strategy to expand the use of NNZ‐2566 in both acute and chronic indications.
This supports their approach of bringing a potentially life‐changing medicine to patients whilst maximising value for shareholders, and is testament to the capabilities and expertise of Neuren’s development and regulatory teams.
Currently there are no medicines approved for the treatment of Fragile X Syndrome, representing a substantial market opportunity for Neuren.
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