MELBOURNE, Australia, March 24, 2017 (GLOBE NEWSWIRE) -- Neuren Pharmaceuticals (NEU.AX) has reported top-line results for its Phase 2 clinical trial in girls with Rett syndrome aged 5 to 15.  Rett syndrome is a serious and life threatening condition caused by a gene mutation, for which there are currently no approved treatments.

Neuren’s trial was a double-blind, randomized, placebo controlled study conducted in 82 subjects at 12 sites in the United States, led by clinicians experienced in the diagnosis and treatment of Rett syndrome and supported by Rettsyndrome.org.

The highest dose of trofinetide achieved statistically significant clinical benefit compared with placebo for each of three syndrome-specific efficacy measures, the Rett Syndrome Behaviour Questionnaire (p=0.042), the Clinical Global Impression of Improvement (p=0.029) and the Rett Syndrome Domain Specific Concerns (p=0.025).  The improvement increased through to the time that treatment ceased.  This suggests that further benefit may be achieved with longer treatment duration.

These results provide strong evidence of biological activity of the high dose across multiple symptom areas, indicating the potential for disease modification rather than simply addressing isolated symptoms.  In addition, trofinetide was well tolerated and had a good safety profile in these younger subjects, with no dose-limiting effects observed.

Neuren now intends to discuss with the US Food and Drug Administration plans for a pivotal trial commencing in 2018 using the Rett Syndrome Behaviour Questionnaire as a primary efficacy measure, supported by the Clinical Global Impression of Improvement as a key secondary efficacy measure.

Walter Kaufmann, MD, Ravenel Boykin Curry Chair of Genetic Therapeutics and Director of the Center for Translational Research at the Greenwood Genetic Center, was an investigator for this trial and was also the original principal investigator for the clinical trial of IGF-1 in children with Rett Syndrome at Boston Children’s Hospital.  Dr Kaufmann commented:

“The outcome of this trial is very encouraging.  Safety, the primary goal, was achieved.  As important and with broad implications, there was a clear clinical improvement covering several common symptoms in Rett syndrome, which are known to impair the quality of life of girls affected by the disorder.  The variety of improved symptoms suggests that trofinetide is a drug that targets mechanisms underlying the disorder rather than a symptomatic medication.  Similar to the previous adult trial, the results are particularly significant because of the relatively short duration of the trial.  The impact of the study goes beyond the suggested efficacy of trofinetide, since it shows the potential of neurobiologically-based drugs for the treatment of Rett syndrome and other neurodevelopmental disorders.”

Steve Kaminsky, PhD, Chief Science Officer of Rettsyndrome.org commented:

“These pediatric study results are very exciting.  The data suggest that trofinetide is having a positive change on a number of challenges of Rett syndrome.  We at Rettsyndrome.org are very proud to have supported this game-changing study, believing that the best is yet to come.” 

Neuren’s Executive Chairman Dr Richard Treagus commented:

“This was a profoundly important study for all Rett families, neuroscience research and for Neuren.  These are deeply encouraging results that build on the clinical data generated from our first Rett syndrome study.  Taken together, this data provides a strong basis to move forward with the remaining steps in trofinetide's development.  We will discuss the results of this trial and our future plans with the FDA Division of Neurology as soon as possible and also provide the safety data to the Division of Psychiatry for consideration in our Fragile X syndrome development program.  Neuren is grateful to the girls, families, clinical experts and the Rettsyndrome.org team, all of whom made a very significant contribution towards the completion of this clinical trial.”

About trofinetide

Trofinetide is a synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1, a growth factor produced by brain cells.  In animal models, trofinetide exhibits a wide range of important effects including inhibiting neuroinflammation, normalizing the role of microglia, correcting deficits in synaptic function and regulating oxidative stress response.  Trofinetide is being developed both in intravenous and oral formulations for a range of acute and chronic conditions.  The most advanced program is for Rett syndrome, supported by Rettsyndrome.org.  Both the Rett syndrome and Fragile X syndrome programs have been granted Fast Track designation by the US Food and Drug Administration (FDA) and have orphan drug designation in both the United States and the European Union.

About Neuren

Neuren Pharmaceuticals Limited (Neuren) is a biopharmaceutical company developing new therapies for neurodevelopmental disorders, neurodegenerative diseases and acute brain injury.  Neuren presently has a clinical stage molecule, trofinetide, in Phase 2 clinical trials as well as NNZ-2591 in pre-clinical development.  Neuren is listed on the Australian Securities Exchange.

Forward-looking Statements
This announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.

For more information, please contact: 
Dr Richard Treagus, Executive Chairman: rtreagus@neurenpharma.com ; +61 417 520 509
Larry Glass, Chief Science Officer: lglass@neurenpharma.com ; +1 301 758 2987