Zou wel eens de ster van de dag kunnen worden.
Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale (‘ONLS’), after 4.5 years of total trial time.
A live conference call and webcast will be held tomorrow, Thursday April 29th at 2:00 p.m. CET
(8:00 a.m. ET)
PARIS, France, April 28, 2021, 6:00 p.m. CET – Pharnext SA (FR0011191287 - ALPHA) (the ‘Company’), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY™ platform, today announces new results from an interim analysis of an ongoing open-label follow-up extension study (‘PLEO-CMT-FU trial’) following the first double-blind, placebo-controlled Phase III study (‘PLEO-CMT trial’) of PXT3003 for the treatment of Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’). There is currently no approved drug treatment for CMT1A.
In January 2020, the company reported interim results suggesting sustained safety and efficacy of PXT3003 in patients with mild-to-moderate CMT1A after 24 months of total trial time (PLEO-CMT and PLEO-CMT-FU Period 1 trials). The new results announced today continue to show sustained treatment benefits for CMT1A patients treated with PXT3003 at High Dose (‘HD’) in the PLEO-CMT-FU Period 2 trial with a data readout at 54 months of total trial time (double-blind + open-label). Highlights from an analysis of available data include:
PXT3003 was safe and well tolerated. Data are consistent with observed safety profile in prior clinical trials.
Data on the Overall Neuropathy Limitations Scale (‘ONLS’), which measures patients’ functional motor disability, are as follows (please refer to the diagram below for graphical illustration):
During PLEO-CMT (double-blind Phase III study), patients treated with placebo on average declined on ONLS while patients treated with PXT3003 on average improved. The best efficacy signal was observed in the cohort of patients treated with PXT3003 HD.