NHGRI Collaboration

New Zealand Pharmaceuticals Announces Collaboration with the NHGRI to Develop DEX-M74 as a Treatment for Hereditary Inclusion Body Myopathy (HIBM)

Palmerston North, New Zealand, May 19, 2011 — New Zealand Pharmaceuticals Ltd (NZP), announced today that it has entered into a collaboration with the National Human Genome Research Institute (NHGRI), part of the US National Institutes of Health (NIH) located in Bethesda, MD, to take the investigational Hereditary Inclusion Body Myopathy (HIBM) therapeutic, DEX-M74, through pre-clinical development followed by initial clinical trials with HIBM patients lead by NHGRI’s William Gahl MD PhD. Two other NIH groups will contribute to the research, including the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the NIH Clinical Center.

DEX-M74 is intended to reduce or halt the progression of HIBM, a debilitating condition of limb muscle tissue. In order to rapidly bring DEX-M74 to the people suffering from this debilitating disorder NZP exclusively licensed related Intellectual Property from the NHGRI. DEX-M74 is believed to be the leading clinical-stage therapeutic that directly targets HIBM, also known as Distal Myopathy with Rimmed Vacuoles (DMRV), Quadriceps Sparing Myopathy (QSM) and GNE related muscle disease.

HIBM is an ultra-rare disorder of the distal (limb) muscle tissue afflicting thousands of people clustered around the world but currently most commonly diagnosed in the USA, Japan and Israel. HIBM causes progressive muscle wasting that may begin in teenagers and can lead to very severe disability within 10 - 20 years.

TRND and NZP are currently collaborating to complete all pre-clinical development activities needed for the Food and Drug Administration (FDA) to review an investigational new drug (IND) application for DEX-M74. Once the IND goes into effect, clinical studies in HIBM patients will be conducted at the NIH Clinical Center by Dr. Gahl’s group, which is part of the intramural research program at NHGRI.

As a new congressionally mandated program, TRND is designed to advance preclinical and early clinical development of new drugs for rare and neglected diseases. The program also drives forward adoption of new technologies and treatment paradigms which improve product development efficiency for these diseases. TRND has formed numerous collaborations with researchers from academia and industry to work on specific rare or neglected diseases.

 “We are delighted to have the opportunity to work with the NIH,” said Andy Lewis, NZP’s Chief Executive Officer. “DEX-M74 represents an attractive development opportunity in our view because its mechanism of action has been thoroughly studied and animal model studies indicate that DEX-M74 offers a real opportunity for an effective treatment to delay or halt the progression of HIBM. We are pleased that the NHGRI and TRND chose to collaborate with us to move it into the clinic.”

DEX-M74 is one of the first molecules to enter development in the TRND program and the NZP-NHGRI joint research project was selected in 2010 to be a one of only five TRND Pilot Projects ( The US Congress funded TRND to speed the development of new medications that might otherwise be ignored by industry because they are too early in the development process or would not be sufficiently profitable. TRND provides drug development expertise and resources required to produce compounds that can be tested in people.

“TRND collaborates with researchers and companies with an aim of translating promising molecules into potential new drugs for rare and neglected diseases,” said NHGRI’s John McKew PhD, Chief of TRND’s Therapeutic Development Branch. “This is a difficult phase of drug development associated with high failure rates for any molecule. The outcome can be uncertain.”

 If the pre-clinical development and safety testing goes well enough for the FDA to issue an IND, tests of the HIBM treatment will quickly move to the NIH Clinical Center for Phase I/II trials with human volunteers.

“Patients with HIBM suffer a great deal from the disorder and the research group is committed to realizing this new treatment. Based on our own research and collaborative research relationships, we believe there is value in exploring DEX M74 as a potential treatment for HIBM patients,” said Dr Gahl, Head of the Human Biochemical Genetics Section of the NHGRI and the principal investigator for the planned clinical trials.

Scientific papers on HIBM and a scientific description of the disease can be found at:

Additional information about HIBM is available at:



Note: References to the National Human Genome Research Institute and the National Institutes of Health, its programs or its staff, should not be viewed as an endorsement or implied endorsement of NZP, its products or services.

About New Zealand Pharmaceuticals Limited

New Zealand Pharmaceuticals Ltd (NZP; is a manufacturing company, located in Palmerston North, New Zealand, dedicated to the supply of essential pharmaceutical intermediates and ingredients for therapeutic use. It is wholly owned by NZP Holdings Ltd, a company that also owns Dextra Laboratories Ltd, located at Reading, UK, ( which provides a comprehensive service in the custom synthesis, analysis and manufacture of carbohydrates from bench to cGMP API manufacture. The Company’s drug development program (DEX-M74) is targeted to the treatment of HIBM. NZP is working with TRND to facilitate the rapid completion of the pre-clinical development program for DEX-M74 and will support Dr Gahl’s human clinical trial early in 2012.

This press release contains certain statements that may be forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, including statements relating to the product portfolio, pipeline and clinical programs (collectively the “Products”) of New Zealand Pharmaceuticals Ltd (the “Company”), the market opportunities for the Products, the potential effectiveness of the Products based on the interpretation of past and/or planned pre-clinical or clinical data and the Company’s goals and objectives. Planned clinical trials are subject to FDA and other regulatory reviews prior to commencement. These statements are subject to numerous risks and uncertainties.