Page 20 - 2021 Spring CMTA Report - Special Research Edition

PHARNEXT ANNOUNCES

PHASE 3

CLINICAL TRIALS FOR 1A

Recruiting for 1A Clinical Trials Underway

Pharnext announced Phase III
clinical trials for its PXT3003 drug
for CMT1A February 21. The
international, multi-center,
randomized, double-blind,
placebo-controlled study is designed
to evaluate PXT3003 versus placebo
in male and non-pregnant female
subjects with genetically confirmed
CMT1A of mild-to-moderate severity,
aged 16 to 65 years.
PXT3003 is a novel, fixed-dose
synergistic combination of baclofen,
naltrexone and sorbitol formulated as
an oral solution. The three individual
components of PXT3003 were
selected to downregulate the
overexpression of PMP22 protein,
leading to improvement of neuronal
signaling in dysfunctional peripheral
nerves. Visits will take place at screening,
Pharnext is an advanced baseline (day 1), and months 3, 6, 9,
clinical-stage biopharmaceutical 12, and 15. Randomization will occur
company pioneering new approaches at the baseline (day 1) visit. Telephone unused study medication, as part
to developing innovative drug contacts will take place at weeks 2 of study drug compliance at visits at
combinations based on big genomics or 3, month 1 and 2, and then monthly months 3, 6, 9, 12, and 15.
data and artificial intelligence. between subsequent in-person visits. The primary outcome measure
The CMTA is collaborating with A safety follow-up visit will be (mONLS) and the 10-Meter Walk Test
Pharnext on a project aimed at conducted at month 16. (10mWT), along with the Columbia
identifying and validating potential Subjects will receive in-clinic dosing Suicide Severity Rating Scale
treatment-responsive CMT1A of study medication at visits on day (C-SSRS) will be evaluated at
biomarkers that could be further 1 and months 6, 12, and 15. Study each post-screening visit. A safety
explored in future clinical studies. medication will be dispensed for follow-up visit will take place 30 days
The current clinical trial will be outpatient dosing on day 1 and (month 16) after the active treatment
conducted in approximately 48 sites months 3, 6, 9, and 12. During period ends (month 15). A Data
worldwide. Genetically confirmed outpatient dosing, subjects must Safety and Monitoring Board (DSMB)
CMT1A subjects will be screened and complete a study medication diary will meet on a scheduled basis
randomized in a 1:1 ratio to receive using an application on their tablet, throughout the study to review safety
either oral PXT3003 or a matching phone or computer. The diary will data and will reconvene on an ad
placebo daily for 15 months. A total be evaluated, along with returned hoc basis as necessary.
of approximately 350 subjects will
be enrolled.

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