ann: neuren completes enrolment of second pati

Neuren completes enrolment of second patient cohort;
provides update on NNZ-2566 program


"SYDNEY, Australia, 21 November 2011: Neuren Pharmaceuticals Limited (ASX:NEU) announced
today that enrolment of the second cohort in the INTREPID-2566 traumatic brain injury (TBI) trial has
been completed. Review of safety data by the Data Safety and Monitoring Committee has been
finalized and the third cohort has been opened for enrolment. There were no Serious Adverse
Events (SAEs) in Cohort 2 patients reported as being drug-related.
As part of Cohort 3, implementation of the protocol approved under Exception from Informed
Consent (EFIC) provisions is in progress. EFIC facilitates study execution by allowing enrolment of
patients for whom it is not possible to obtain informed consent from a legally authorized
representative. EFIC is restricted to situations in which the condition is life-threatening, immediate
treatment is required and there is no alternative treatment available. The first phase of EFIC
implementation is a program of community consultation and public disclosure at each participating
site to inform the community and seek feedback on the trial. This process is well underway.
Neuren is also developing an oral form of NNZ-2566 to treat patients who have had a concussion, a
milder type of head injury than the TBI being targeted with the intravenous (IV) form of the drug.
Concussions are more than four times as common as moderate or severe TBI, frequently occurring
in people participating in sports and as a result of falls and motor vehicle accidents. The oral version
of NNZ-2566 is a liquid produced by dissolving the same powder used to make the IV form in a
water-based solution. The additional toxicology and pharmacokinetic studies in animals that are
required to initiate human trials have been completed and showed the oral form to be safe with
minimal side effects. Preparations for a Phase I safety and pharmacokinetic study in healthy
volunteers have been finalized and the study to be undertaken in Australia is planned for early 2012.
A Phase II trial in concussion patients is expected to start in mid-2012.
In addition to concussion, the Company has begun development of the oral form of NNZ-2566 for
Rett Syndrome, a very severe, physically disabling disease and is considered one of the autism
spectrum disorders. There is no approved drug for Rett Syndrome which occurs in approximately 1
of 10,000 female children worldwide. Preliminary results with NNZ-2566 in an animal model of Rett
Syndrome were promising. A Phase IIa protocol to establish proof of principle in Rett Syndrome
patients has been developed. We plan to file an IND for the Rett Syndrome study in the second half
of 2012 and to initiate the clinical trial in late 2012. The Company believes that Rett Syndrome will
qualify for Orphan Disease and Fast Track designation under US FDA regulations.
Commenting on the NNZ-2566 program, Larry Glass, Neuren’s CEO, said: “We are pleased that
Cohort 2 was completed successfully and that NNZ-2566 appears to be well-tolerated in TBI
patients. We are also gratified that the changes instituted in the study have improved the pace of
enrolment and remain confident that enrolment will continue to accelerate in Cohort 3 with the
inclusion of female as well as younger and older patients and as EFIC is implemented. Progress
with the oral formulation has been excellent and we are excited about the new clinical programs that
we believe will significantly increase the value of the NNZ-2566 franchise.”"