Page 11 - Fall 2020 CMTA Report

have asked the company for
an additional, expanded trial CMTA Announces
using the highest proposed
dose combination. The Collaboration with Pharnext
CMTA has supported
Pharnext with patient advo- On Key Biomarkers for CMT1A
cacy efforts and is providing
biomarkers in preparation he Charcot-Marie-Tooth
for Phase III clinical trials Association and Pharnext SA
(See related story this page.) announced a research collab-
oration September 3 aimed the Inherited Neuropathy Consor-
T at identifying and validating tium.
OVERALL NEXT STEPS potential treatment responsive CMTA Board Chair Gilles
INCLUDE:
CMT1A biomarkers that could be Bouchard said, “[W]e are thrilled
l Development of an in vitro further explored in future clinical Pharnext is focusing its clinical
test model for CMT1A: Our studies. development effort on CMT1A.
alliance partners are constantly Pharnext is an advanced clini- Our collaboration with Pharnext
asking for a “CMT in a dish” cal-stage biopharmaceutical aims to identify potential bio-
model for initial evaluation of company pioneering new markers for CMT1A, which are
potential therapies. Properly approaches to developing innova- crucial to understanding the
done, this could predict likely tive drug combinations based on pathophysiology of the disease
activity in an animal model big genomics data and artificial better, and to evaluate new thera-
where additional issues of drug intelligence. It’s preparing for the peutic agents in future clinical
delivery and metabolism have upcoming Phase III study of its trials.”
to be managed. The current lead drug candidate, PXT3003. Pharnext CEO Dr. David
best available model is a The Paris-based company Horn Solomon said, “This
co-culture in a dish, in which intends to investigate blood sam- exciting collaboration between
nerve cells and Schwann cells ples collected from CMT1A Pharnext and the Charcot-Marie-
isolated from the CMT1A patients with mild to Tooth Association
animal model are put together, moderate cases The collaboration underscores the
thus allowing myelin to be enrolled in the first between Pharnext importance of involv-
formed and used as an out- Phase III study of and the CMTA ing patient advocacy
come measure in the test. PXT3003. Notably, “underscores the organizations in bet-
this collaboration will importance of ter understanding the
l Sanofi has asked for our help evaluate the potential disease and working
in preclinical testing for a new of TMPRSS5, a involving patient to bring new thera-
program opportunity. If we recently identified advocacy pies to CMT1A
take this on, there is potential Schwann cell-specific organizations…” patients.
to launch a new and advanced biomarker in “Through this
effort that they will take CMT1A patients, to confirm if it collaboration, we aim to further
forward and finance. can be used to assess treatment assess blood samples collected
response in future clinical trials. during our first Phase III trial of
l Ensuring the scalability of the
TMPRSS5 is part of a PXT3003 for novel biomarkers
testing resource: The testing broader neurology panel that tests and notably confirm the potential
resource has attracted strong, many additional potential bio- of TMPRSS5 in CMT1A. Results
continued interest and engage- markers and the collaboration of this research collaboration
ment by pharmaceutical between Pharnext and the CMTA might inform the addition of new
alliance partners. As the could lead to the identification of exploratory endpoints in our next
demand increases, the CMTA additional, as yet undescribed bio- Phase III trial of PXT3003 to be
commitment needs to scale up markers for CMT1A. The initiated in Q1 2021. We believe
accordingly. h development of TMPRSS5 this alliance will enable us to

Due to space constraints, genetic evolved from a STAR (Strategy to accelerate our efforts in bringing a
therapy projects for CMT1A will Accelerate Research) collaboration safe and effective therapeutic for
be covered in an upcoming issue. involving CMTA Board members this disease that currently has no
Michael Shy and John Svaren and viable treatment options.” h

FALL 2020 THE CMTA REPORT 11