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Pharnext rare disease drug scores in late-stage study, teeing up applications at the FDA and EMA

French drug developer Pharnext SAS says their crucial late-stage study has come through with significant results for a new drug to address a rare neurological disease that has so far eluded treatment. The company’s stock (EPA: $ALPHA) soared 25% Tuesday morning after it announced that the experimental treatment for Charcot-Marie-Tooth (CMT) disease was compelling enough to apply for FDA and EMA approval.

CMT is an inherited disease caused by gene mutations that result in peripheral nerve damage, and is characterized by the progressive muscle wasting of the extremities as well as sensory loss. Currently, patients are treated with orthopedic devices, physical therapy and pain killers. There exist no treatments that directly target the disease, and the most common 1A form of the disease affects at least 125,000 people in the US and EU.

The experimental treatment, PXT-3003, is comprised of three approved drugs — baclofen, naltrexone and sorbitol. In the Phase III trial, two doses of the drug were tested against a placebo in patients with mild-to-moderate CMT type 1A over a 15-month period. The main goal of the study, which enrolled 323 patients aged 16 to 65 years, was to get at least a 0.3 reduction in a scale measuring patient disability called Overall Neuropathy Limitation Scale (ONLS).

Daniel Cohen

Data showed a mean reduction of 0.4-point on the ONLS (95% CI [0.1,0.6], p=0.008) in the higher dose group compared to the placebo. Secondary endpoints confirmed the superiority of the higher dose, in particular demonstrating improvement on a 10-meter walk test with a reduction of 0.5 sec (95%CI [0.1,0.9], p=0.016), Pharnext added.

In the trial, 87 patients were given a placebo, 93 patients were given the lower dose and 55 patients got the higher dose. The lower number of patients given the higher dose is due to unexpected formulation/stability issues, the company said, adding that the treatment was found to be safe and well tolerated.

Under the stewardship of its CEO Daniel Cohen, who was one of the researchers instrumental in mapping the human genome, Pharnext has developed its technology based on a similar model. Its pleotherapy platform identifies the ideal combination of available medicines to fight targets underlying disease.

Pharnext plans to file for marketing approval first with the FDA, Cohen told Endpoints News, and expects the drug will officially hit the market by 2020, but intends to make the drug available to patients sooner via early-access programs. His approach to pricing is also strikingly different.

“After many discussions with payers, we’re thinking of pricing it at 10,000 euros in Europe, and around 40,000 – 50,000 dollars in the US, as suggested by the payers themselves,” he said.

Cohen has long suggested that he is against the standard industry approach to rare drug pricing, in which treatments for rare diseases typically exceed $100,000 per patient per year.

PXT-3003, which has procured orphan drug designations in the US, EU and China, is also being evaluated in an ongoing follow-up extension study that’s expected to produce results in the second half of next year, and a pediatric study is set to commence in the first half of 2019. Meanwhile, Pharnext’s second drug in development, PXT864, is being evaluated as a treatment for Alzheimer’s and Lou Gehrig’s disease.


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