Page 8 - 2021 Summer CMTA Report

CMTA-STAR Awards $1.1 Million in Grants

To Researchers Prepping for CMT1X, CMT2A Clinical Trials

he Charcot-Marie-Tooth Association Dr. Michael Shy at the University of
TStrategy to Accelerate Research Iowa and John Svaren, PhD, at the
(CMTA-STAR) announced $1.1 million in University of Wisconsin will use a
grants March 23 aimed at helping top $601,407 grant for a project aimed at
CMT scientists prepare for clinical trials developing outcome measures, plasma
for CMT1X and CMT2A. biomarkers and a nanostring platform
for skin biopsy in 60 CMT1X patients.
Because CMT typically advances CMT1X currently affects about 15
slowly, measuring progression percent of all diagnosed cases of CMT.
during a multi-year clinical trial is
challenging. Biomarkers measure Another grant of $559,555 will enable
changes over shorter periods of time Shy and Svaren to begin a project
than self-reported measures like taking the same state-of-art approach
clinical outcome assessments so that to prepare for clinical trials in CMT2A,
researchers can quickly and precisely which affects about 5 percent of
measure whether a treatment or drug all patients with CMT and up to 25
had a positive impact on neuropathy. A percent of patients with CMT2.
shortened timeline keeps costs down
for companies interested in investing in The findings from the two projects will
be critical in advancing clinical trials in
CMT research. CMT1X and CMT2A.
The latest grants will fund two studies For more information on how to
that will collect patients’ natural
history data over a two-year period. get involved in these studies,
please visit www.cmtausa.org.

Interim Analysis Shows Sustained

Benefits of PXT3003 for CMT1A Patients

harnext SA announced April 28 new continue to show sustained treatment
Presults suggesting that PXT3003 benefits for CMT1A patients treated
has a good safety profile and sustained with PXT3003.
efficacy in the treatment of CMT1A Adrian Hepner, MD, PhD, chief medical
after 4.5 years of total trial time.
officer of Pharnext, said: “Although
According to the company, the new these new data were generated from
results are from an interim analysis an open-label study, the findings are
of an ongoing open-label follow-up consistent with the safety and efficacy
extension study after the first double- results of PXT3003 observed in prior
blind, placebo-controlled Phase III clinical studies in CMT1A.”
study of PXT3003 for the treatment
of CMT1A. Florian P. Thomas, MD, PhD, and U.S.
lead investigator of the PLEO-CMT
Based in France, Pharnext is an trial, said: “These new results from the
advanced late-stage clinical biophar- interim analysis of the ongoing open-
maceutical company pioneering new label Phase III extension study show
approaches to developing innovative very promising safety and efficacy data
drug combinations based on big ge- of PXT3003 in CMT1A after more than
nomics data and artificial intelligence. four years of treatment. It reinforces
our hope that PXT3003 could be the
In January 2020, the company first treatment approved for patients
reported interim results suggesting suffering from this debilitating disease.”
sustained safety and efficacy of
PXT3003 in patients with mild-to- More information about enrollment in
moderate CMT1A after 24 months the PXT3003 study can be found at
of total trial time. The new results www.premiercmt1a.com/.

8 THE CMTA REPORT SUMMER 2021