Thursday, March 14, 2013

Neuren Pursues New Drug for RTT

Pursuing the first Investigational New Drug (IND) for Rett syndrome:
Neuren Pharmaceuticals announces a program to the FDA about a proposed Rett syndrome clinical trial

This month Neuren Pharmaceuticals Ltd held a pre-IND meeting with the FDA Division of Neurology Products to discuss clinical development plans for the NNZ-2566 Rett Syndrome program. The company requested the meeting to seek input and guidance from the FDA for the first clinical trial in older patients with Rett syndrome. The Investigational New Drug application is the precursory step before starting a clinical trial. Other than FDA staff, participants in the meeting included:
  • Dr. Daniel Glaze, Professor, Baylor College of Medicine, and Medical Director of the Blue Bird Circle Rett Center at Texas Children’s Hospital
  • Dr. Jeffrey Neul, Associate Professor, Baylor College of Medicine, and Assistant Medical Director of the Blue Bird Circle Rett Center at Texas Children’s Hospital
  • Dr. Mike Snape, Chief Scientific Officer, Autism Therapeutics Ltd
  • Dr. Steven Kaminsky, Chief Science Officer, International Rett Syndrome Foundation
  • Dr. Joseph Horrigan, Assistant Vice President and Head of Medical Research, Autism Speaks
  • David Zuchero, Chesapeake Regulatory Group (Neuren’s US regulatory agent)
  • Larry Glass, Neuren CEO

What is NNZ-2566?

NNZ-2566 is a drug that Neuren developed with the Department of Defense to treat mild Traumatic Brain Injury (mTBI) and has been shown to have great success in mTBI animal models. Neuren also tested NNZ-2566 in Rett mice and it has significant effects on lengthening neurons and maintaining synaptic connections in the Rett mice. These are very exciting studies that suggest there is a potential to increase the plasticity (or rewiring) of the Rett brain to reverse Rett symptoms.

Why is this exciting for the Rett community?

Neuren Pharmaceuticals has joined with the Baylor College of Medicine to test NNZ-2566 in a proposed Phase I and Phase IIa clinical trial. This proposed trial, along with the IGF-1 trial at the Children’s Hospital in Boston, are trials designed to investigate whether these compounds can reverse some of the symptoms of Rett syndrome and modify the progression of the disorder in our Rett children. This announcement portrays the importance of partnerships between industry, academia, and private foundations like IRSF to accelerate bringing forward clinical trials specific to Rett syndrome.
For more information concerning their announcement please see the Neuren press release at
If you have questions about this program please contact Paige Nues, Director of Family Support, or Jennifer Endres, Family Resource Manager, at The International Rett Syndrome Foundation (email:
Source: Internationa Rett Syndrome Foundation

Phase 2 Clinical Trial Study of IGF-1

IRSF Announces Funding to Boston Children’s Hospital for the Phase 2 Clinical Trial Study of IGF-1 for Rett Syndrome

The International Rett Syndrome Foundation (IRSF) is pleased to announce the approval of funding to Dr. Walter Kaufmann and the Boston Children’s Hospital (CHB) for the second phase of the IGF-1 clinical trial for treatment of Rett syndrome.
The project entitled “A Phase 2b placebo-controlled crossover study of rhIGF1 (mecasermin [DNA] injection) for treatment of Rett syndrome and development of Rett-specific novel biomarkers of cortical and autonomic function” was submitted under IRSF’s Translational Research ANGEL (Advanced Neurotherapeutic Grant of Excellence) grant award mechanism. The application had undergone an extensive, rigorous review by a distinguished scientific review committee, and a programmatic review by Dr. Steve Kaminsky, IRSF Chief Science Officer, who moved to recommend the application for funding. The IRSF Board of Directors (comprised mainly of parents of children with Rett syndrome) unanimously voted to approve funds in the amount of $600,000.
Prior support from IRSF and Autism Speaks allowed CHB and then Principal Investigator (PI), Dr. Omar Khwaja to successfully complete the Phase 1 study that involved the safety and tolerability of IGF-1 use in patients diagnosed with Rett syndrome. The Phase 2 study will continue to investigate the efficacy of IGF-1 in a larger cohort of Rett patients (30) in a double-blind placebo-controlled cross-over trial.
IRSF is excited to work with Dr. Kaufmann who is now the PI heading this study and is also the Director of the Rett Syndrome Program at CHB. By supporting this project, IRSF continues to accelerate and develop treatments for Rett syndrome.
For questions about this Phase 2 Trial, please contact Theresa Trapilo of CHB for more information.

Source: International Rett Syndrome Foundation