NeuroHealing Pharmaceuticals Receives Grant from the FDA Office of Orphan Drugs for Clinical Development of NH001 to Treat Patients in a Vegetative and Minimally Conscious State
July 16, 2008 Business News(PRLEAP.COM) NeuroHealing Pharmaceuticals, a company developing products for individuals with chronic brain injuries, today announced that the Food and Drug Administration (FDA) has awarded the company a three-year grant of $1,044,000 to assist in the clinical development of NH001. NH001 is under clinical development to improve the functional outcome of patients in a vegetative state or minimally conscious state following a severe traumatic brain injury. The product has demonstrated promising results in an open label Phase IIa trial and has received authorization from the FDA to begin a double blind Phase II clinical trial, under an open IND. NH001 received Orphan Drug Status from the FDA in 2006.
"This is a significant milestone and accomplishment for NeuroHealing," commented Elkan Gamzu, PhD, acting Head of Clinical Trials at NeuroHealing. "We are very pleased to receive this support from the FDA as it represents a nice validation of the promise of NH001. This grant will provide the initial funding to begin the clinical program at the Spaulding Rehabilitation Hospital, a Harvard Medical School affiliated hospital and one of the leading rehabilitation facilities in the U.S., and will enable the continued development of this treatment which may offer patients, their families and physicians a new therapeutic option to help brain injured patients in an altered consciousness state."
Dr. Ross Zafonte, Earle P & Ida S Charlton Chair of the Department of Physical Medicine & Rehabilitation at Harvard Medical School, and Vice President of Medical Affairs for Spaulding Rehabilitation Hospital, Boston will be the principal investigator of this FDA funded study.
About The Office of Orphan Products Development
The Office of Orphan Products Development (OOPD) of the FDA is dedicated to promoting the development of products that demonstrate promise for the treatment of rare diseases or conditions. OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. The OOPD administers the major provisions of the Orphan Drug Act (ODA), which provides incentives for sponsors to develop products for rare conditions. The ODA has been very successful — more than 300 drugs and biological products for rare diseases have been brought to market since 1983. The OOPD also funds the development of orphan products through its grants program for clinical studies. Applications are reviewed by panels of outside experts and are funded by priority score.
About Coma, Vegetative State and Minimally Conscious State
Each year in the U.S. 50,000 to 75,000 patients survive a severe TBI but remain in an altered consciousness state – such as coma, vegetative state (VS) and minimally conscious state (MCS) – for an extended period of time. These 'altered consciousness states' are characterized by the lack of awareness and lack of interaction with the environment. Diffuse axonal injury, a neural pathology occurring in post-TBI patients, is associated with a disruption of dopamine pathways. There is evidence suggesting that dopamine agonists may have a role in regaining consciousness, facilitating rehabilitation and improving outcome.
In the acute stage, the main medical efforts are focused on stabilizing vital signs, internal homeostasis and intra-cranial pressure. After the patient reaches stability, current therapy is based on sensory stimulation. There are no approved drug treatments for post-traumatic patients who remain in a vegetative state or minimally conscious state.
NeuroHealing's NH001 therapy is a potent dopaminergic agonist with well-established human safety data, appropriate pharmacokinetic profile and convenient route of administration. It is a direct acting dopamine agonist on both D1 and D2 dopamine receptors. It rapidly crosses the blood brain barrier and reaches a concentration in the brain eight times higher than that in plasma. Because dopaminergic function appears to be of importance in executive central nervous system function, such as arousal and memory, NH001 has been designed to help regain consciousness, accelerate recovery and improve the functional outcome of patients from a coma, vegetative or minimally conscious state following a traumatic brain injury.
NeuroHealing previously presented findings of the response to NH001 treatment in eight patients with a traumatic brain injury who were in a vegetative or minimally conscious state for 1-4 months and neurologically stable for weeks prior to beginning of NH001 treatment. Seven of the eight patients had previously received, and failed to respond, to commonly used stimulant drugs, such as l-dopa. The responses to NH001 were rapid and observed in the first 2-4 weeks. Five of seven patients showed a progressive and continuous improvement with clinically significant changes in consciousness, functional recovery and reached an independent walking ability. The responses were maintained after NH001 discontinuation. NH001 treatment was well tolerated.
About NeuroHealing Pharmaceuticals
NeuroHealing Pharmaceuticals, Inc. is a clinical stage private company developing therapies to improve the neuro-rehabilitation process and functional outcome for patients who have suffered debilitating brain injuries. Clinical stage programs include: NH001 (phase II), a dopaminergic agent to help post traumatic brain injury patients to emerge from a coma, vegetative or minimally consciousness state; NH004 (phase II), an anticholinergic agent in a convenient intra-orally muco-adhesive dissolving film to help treat motor neuron disease patients who suffer from sialorrhea (drooling); and NH02D, a novel mechanism compound to accelerate the rehabilitation outcomes of stroke patients who remain with chronic motor and cognitive disabilities.
NeuroHealing Pharmaceuticals, Inc.
Daniel Katzman, President