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Afraxis Enters Collaboration with NIH as Part of Therapeutics for Rare and Neglected Disease Program
Date:9/26/2011

SAN DIEGO, Sept. 26, 2011 /PRNewswire/ -- Afraxis, a San Diego-based biotechnology company developing drugs to treat rare genetic diseases through the modulation of p21-activated kinase (PAK), announced today that it has been selected to participate in the National Institutes of Health's (NIH) Therapeutics for Rare and Neglected Disease (TRND) Program. As part of the program, Afraxis will partner with the NIH to pursue the ongoing development of the company's disease-modifying treatments for Fragile X syndrome. Fragile X syndrome is a rare genetic disorder that is the most common inherited form of mental retardation.

"TRND collaborations help companies focusing on rare and neglected diseases overcome one of the biggest obstacles facing all drug development companies: advancing programs through that 'valley of death' between identification of a promising compound, and clinical stage development when a program becomes more attractive to a big pharma partner," said Jay Lichter, Ph.D., president and CEO of Afraxis. "This is a tremendous opportunity for Afraxis as the NIH is providing access to state-of-the-art laboratory facilities, the expertise of collaborating NIH scientists, and the financial resources to continue our research and development efforts through the initiation of human clinical trials."

Afraxis is developing disease-modifying treatments for Fragile X syndrome and other central nervous system disorders based on a leadership position in the understanding of PAK biology. Fragile X symptoms can range from learning disabilities to more severe cognitive or intellectual disabilities, such as mental retardation. Fragile X is the most common known cause of autism or "autistic-like" behaviors. The disease is caused by a mutation in the FMR gene on the X chromosome and effects approximately one in 3,600 males and one in 4,000 to 6,000 females.

The TRND program is designed to encourage the development of drugs for rare and neglected diseases. The program aims to speed development of new drugs that might otherwise be ignored by industry because they are too early in the development process where the failure rates are high. To make a potential compound attractive to private partners in order to get a treatment to patients, TRND advances a compound through the drug development process to the point where it can be tested in humans. TRND provides all aspects of preclinical development needed to turn promising compounds into potential medicines ready for clinical evaluation. It is anticipated that the average TRND project will take approximately three years.

"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."

The NIH has allocated $50 million to support research and development collaborations between NIH and TRND partner companies in 2011. More than 60 companies submitted proposals to the NIH and four were selected to participate in TRND. Proposals were evaluated on the strength of the data supporting the company's approach, the feasibility of reaching human studies, the medical impact of the treatment and the likelihood that the program would eventually be adopted by a pharma partner that could continue clinical-stage development.

About the Therapeutics for Rare and Neglected Diseases (TRND) Program

The National Institutes of Health (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program, http://trnd.nih.gov is a congressionally mandated program to encourage and speed the development of new drugs for rare and neglected diseases. This unique program creates a drug development pipeline within the NIH and is specifically intended to stimulate research collaborations with academic scientists, non-profit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses. The TRND program provides an opportunity to partner with, and gain access to, drug development scientific capabilities, expertise, and resources in a collaborative environment with the goal of moving promising therapeutics into clinical testing.

About Afraxis

Afraxis, a San Diego-based biotechnology company fully funded by Avalon Ventures, is discovering and developing drugs to treat rare and neglected diseases through the modulation of p21-activated kinase (PAK). Afraxis' initial indication of interest is Fragile X syndrome with expansion into other diseases of the central nervous system, including schizophrenia and autism spectrum disorders. Recent scientific discoveries have linked these disorders to underlying defects in the development and function of specialized structures of the neural synapse, called dendritic spines. The company's lead target is PAK, a protein that regulates the development and activity of dendritic spines, creating potentially disease-modifying therapies rather than symptomatic treatments. Modifying the disease produces beneficial changes at the cellular and behavioral level, creating new hope for patients, their families and their caregivers.  In addition to its impact on dendritic spine biology, PAK is also implicated in cancer, including Neurofibromatosis, a rare form of cancer for which there are no approved drugs. Inhibiting PAK activity has the potential to induce significant anti-tumor activity.

For more information visit the Afraxis Web site: www.afraxis.com


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