Pharnext drug shows promise in neurological disease with no treatment

(Reuters) - Using technology gleaned from the mapping of the human genome, privately held French drug developer Pharnext SAS is a step closer to providing a treatment for a rare neurological disorder called Charcot-Marie-Tooth (CMT) disease, for which no pharmaceutical therapy exists.

The company on Wednesday said its three-drug combination, currently known as PXT-3003, led to a 14.4 percent improvement compared with placebo on a scale used to evaluate disability of the upper and lower limbs in an 80-patient mid-stage study.

The inherited nerve disorder, caused by over expression of a gene, is characterized by a progressive muscle atrophy of the extremities, leading to problems in walking, running and balance, as well as abnormal hand function.

CMT patients currently receive only supportive care for the disease including orthotics, leg braces, physical therapy or surgery. About 5 percent of CMT patients with the most common 1A form of the disease end up in a wheelchair. There are no approved drugs for the disorder.

Pharnext’s data represents “improvement beyond the stabilization of the disease,” Chief Executive Daniel Cohen said.

Cohen, who was one of the researchers involved in the mapping of the human genome, drew on that experience to develop the company’s so-called pleotherapy technology, which identifies the best combination of available medicines to fight several targets underlying a particular disease. The technology is akin to broad spectrum drugs that attack a virus or bacteria by several means.

“This is the extension to neurology, using genomics, to find out what we should combine together to fight the disease,” Cohen told Reuters.

Genomic mapping, which identifies all manner of genetic mutations, allows for the creation of an inventory of all possible molecular impairments associated with disease.

PXT-3003 consists of mini-doses of three approved drugs - baclofen, naltrexone and sorbitol. This approach allows for a significantly shorter developmental timeline as it involves medicines whose safety is already well established.

The company, founded in 2007, plans to initiate a late-stage study of about 300 patients next year to confirm the positive results. There are about 100,000 patients with CMT 1A in the United States and Europe.

Pharnext is also evaluating the drug combination in other neurological disorders, including Lou Gehrig’s disease (ALS) and Alzheimer’s.

Reporting by Natalie Grover in Bengaluru; additional reporting by Bill Berkrot in New York; Editing by Cynthia Osterman