Formed in 2003 to develop drugs to help improve neuro-rehabilitation, NeuroHealing Pharmaceuticals Inc. is preparing to move its first product into the clinic, a dopaminergic agent designed to stimulate the recovery of coma patients.
The company recently filed an investigational new drug application to begin a Phase II trial with NH001, a direct-acting dopamine agonist designed to activate post-synaptic dopamine receptors. Open-label trials of the drug already are ongoing outside the U.S., and results are encouraging so far, said Neal Farber, CEO of Newton, Mass.-based NeuroHealing.
"We've treated three patients, and we've seen a very good response," he said.
Work at NeuroHealing focuses on the "repositioning" of existing drugs, Farber told BioWorld Today, a development process that includes looking at new uses, formulations and drug delivery.
"We're very focused on the product story," he added. "The industry is very hungry for product stories these days."
The company's first product, NH001, is aimed at the post-acute, chronic rehabilitation market, which Farber believes has been somewhat neglected by both the pharmaceutical industry and academic research. The drug is expected to benefit patients that are in persistent vegetative states following traumatic brain injuries. A typical patient, Farber said, might be "an 18- to 25-year-old person who gets into a car accident and sustains a traumatic brain injury. The patient is brought to the hospital emergency room, and, very typically, the doctors will do their magic to get the patient breathing and get circulation going."
But, because of the brain injury, the patient either arrives in, or lapses into, a coma. Patients roused after a couple of weeks generally maintain a good prognosis. Those that don't come out of the coma within a month, however, are moved into rehabilitation centers or nursing homes to begin receiving long-term care.
"Doctors at those facilities use methods like sensory stimulation to try to provoke them out of this vegetative state, and there has been some controversy about how much it helps," Farber said. "At NeuroHealing, we feel we can use drug therapy to accelerate or catalyze the patients out of comas."
Since NH001 already has an established safety profile, the company will begin with Phase IIa exploratory studies and move quickly into "full-fledged Phase II studies," to test the product's efficacy, Farber said.
The drug potentially could reach the market in 2009.
NeuroHealing anticipates commercializing and selling the drug itself, since only a small sales force would be needed to accommodate a focused market of coma patients, Farber said.
However, the company likely will look at partnering opportunities for its second product, NH004, an anticholinergic compound aimed at down-regulating secretory glands. That product is being developed as a treatment for sialorrhea, a condition causing excess saliva and drooling that is associated with motor neuron diseases such as Parkinson's disease, cerebral palsy and amyotrophic lateral sclerosis.
"There's not really much these patients can do right now," Farber said, adding that existing treatments consist of "draconian" surgery to remove the salivary glands or injections of Botox, which can successfully block the nerves but also is expensive and requires retreatment every few months.
There are some anticholinergic drugs designed to block those nerve impulses, but most have to be administered orally and might cause systemic side effects that are counteractive to neurology drugs.
"We asked doctors and patients and they said they want something that can be taken on demand and works fast, so they can use it in a social setting, and wears off after an hour or two," Farber said. "So we came up with this anticholinergic that's formulated into an intra-orally-dissolving thin film that goes into the mouth, on top of the salivary glands, and works in about 10 minutes."
He anticipates moving quickly through the clinical process with NH004, which is given on an outpatient basis. Pending clinical and regulatory success, the product also could be launched in 2009.
While NeuroHealing was working on the anti-sialorrhea indication, researchers discovered "a tangential use" for the drug in the dental market, Farber said, specifically in cases in which patients are having dental work done that requires a dry mouth.
"We're in discussions with a couple of dental companies," he added.
The third drug in the company's pipeline is NH02D, for improving the rehabilitation efforts of patients who have suffered strokes or traumatic brain injuries. In those cases, patients could undergo years of rehabilitation before they are able to regain functionality. NH02D is designed to promote neural plasticity.
"In the simplest description," Farber said, NH02D is designed to induce "the good parts of the brain to take over for the injured parts."
"Physiotherapy works on training the brain to take over for the injured parts, but we want to find drugs that will catalyze this process," he added.
NeuroHealing has not yet released a time frame for getting NH02D to the market.
The company, which officially began its operations in early 2004 and has about 10 employees, has relied primarily on funding from three or four principals.
"We're now seeking a Series A of about $10 million," Farber said. That, plus a small ongoing angel round, should "enable us to move into three Phase II trials of all our drugs."