Biotech Daily Neuren: ‘NNZ-2566 Phase II Safety, Efficacy For Rett Syndrome’

Biotech Daily
Wednesday November 12, 2014
Daily news on ASX-listed biotechnology companies
Neuren: ‘NNZ-2566 Phase II
Safety, Efficacy
For Rett Syndrome’
Neuren says its phase II trial of NNZ-2566 for Rett syndrome “exceeded ... expectations”,
and would lead to applications for orphan drug and breakthrough therapy designation.
Neuren published detailed results showing that the two trial doses of 35mg/kg and
70mg/kg twice daily were safe and well-tolerated, with no drug related serious adverse
events and that the higher dose met several of the efficacy secondary endpoints.
The company said the next step would be to submit applications to the US Food and Drug
Administration for both orphan drug and breakthrough therapy designation.
Neuren said it expected to meet with the FDA by April 2015 to discuss the trial results and
the requirements for the further development of NNZ-2566 in Rett syndrome.
The company said that there were no approved medicines for the treatment of Rett
syndrome, which was a severe genetic neurological disorder.
Neuren said its trial was the first multi-site, sponsor-led clinical trial in Rett syndrome and
the first trial in an adolescent and adult population.
Harvard Medical School professor of neurology and Boston Children’s Hospital Rett
syndrome program director Prof Walter Kaufmann, who was not involved in the trial, said
that the outcome was “very promising in terms of both safety and clinical improvement”.
“It opens not only the possibility of successful treatment of adults with Rett syndrome, but
also of early interventions modifying the course of the disease,” Prof Kaufmann said.
Neuren said that 53 subjects aged 16 to 45 years completed the double-blind placebocontrolled trial.
The company said that efficacy was measured across six core measures in four efficacy
domains, the analysis plan was pre-specified and submitted to the FDA before the data
was unblinded and compared clinical responses in the core measures for each subject
individually, as well as the mean clinical responses for each treatment group.
Neuren said that evidence of clinical benefit in the group-level analysis required
improvement in at least two core outcome measures from two different efficacy domains,
with no clinically significant worsening in all other core endpoints and this was exceeded
at day 26 in the higher dose group, with three measures from three different efficacy
domains achieving the target, the motor-behavior assessment change index, the clinical
global impression of improvement and the caregiver top three concerns.
The company said that the 70mg/kg dose group achieved a benefit in the subject-level
efficacy analysis.
Neuren said the probability of the results being chance rather than a drug effect was
determined as 2.3 percent (p = 0.023) by permutation testing, which the FDA had
accepted in previous trials in other therapeutic areas, including for the development of a
product recently granted breakthrough therapy designation.
Neuren executive chairman Dr Richard Treagus said the trial results “exceeded our
expectations and we look forward to discussing with the FDA the remaining requirements
to develop NNZ-2566 for the treatment of Rett syndrome”.
In a teleconference Dr Treagus said that given the safety profile the company had “the
ability to contemplate going to a higher level dose” possible for a longer treatment period.
Dr Treagus said that the data showed “a clear separation between drug and placebo”.
University of Alabama professor of paediatric neurology, trial investigator Prof Alan Percy
said that the results “suggest a very promising proof of concept as we continue on the
pathway to develop a disease-altering treatment for girls and women with Rett syndrome”.
“Not only was this short-term trial managed successfully, but also the data analyses were
conducted in a very robust fashion,” Prof Percy said.
Neuren climbed as much as 6.7 cents or 85.9 percent to 14.5 cents before closing up 4.2
cents or 53.85 percent at 12 cents with 50.6 million shares traded.
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