Pharnext SA’s PXT3003 treatment for patients with Charcot-Marie-Tooth Disease (CMT) has shown positive interim results in its ongoing Phase III trial.
The results mean the drug could become the first approved treatment in the world for Charcot-Marie-Tooth Disease Type 1A (CMT1A).
CMT is caused by a fault in one of the genes responsible for the development of the peripheral nerves, which causes the nerves to become damaged. Patients suffer from progressive muscle atrophy in both the legs and arms, causing problems with walking, running and balance, as well as abnormal hand functioning.
CMT1A, the most common type of CMT, affects about 150,000 people in Europe and the US, and about 1,500,000 people worldwide.
Today’s results of the PLEO-CMT-FU Period 2 trial into PXT3003 come after 54 months of total trial time, although the study is ongoing. The trial involves 187 patients, and was designed to primarily assess the long-term safety and tolerability of PXT3003.
The trial is assessing data based on the Overall Neuropathy Limitations Scale (ONLS), which measures patients’ functional motor disability.
It was found that, on average, participants improved on ONLS across all patient cohorts. As well as this, patients on placebo during the previous, double-blind phase, have now improved after switching to the drug in this new, open-label phase. It was also found that participants on a high dose of the treatment showed the highest efficacy.
Dr Adrian Hepner PhD, Chief Medical Officer of Pharnext, said: “Although these new data were generated from an open-label study, the findings are consistent with the safety and efficacy results of PXT3003 observed in prior clinical studies in CMT1A.
“In addition, the fact we have just initiated the PREMIER trial in a similar patient population, using the same High Dose of PXT3003 and measuring the same efficacy endpoint ONLS, reinforces our confidence in the potential positive outcome of our ongoing pivotal Phase III study.”
Dr Florian P Thomas PhD, US lead investigator of the PLEO-CMT trial, said: “These new results from the interim analysis of the ongoing open-label Phase III extension study show very promising safety and efficacy data of PXT3003 in CMT1A after more than four years of treatment.
“It reinforces our hope that PXT3003 could be the first treatment approved for patients suffering from this debilitating disease.”
Jack Goddard
This is a syndicated feed from Pharmafile
