Pharnext Announces That the DSMB Recommends Continuing the Ongoing Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease T...
September 06 2017 - 10:45AM
Business Wire
Regulatory News:
Pharnext SA (Paris:ALPHA) (FR00111911287 - ALPHA),
a biopharmaceutical company pioneering a new approach to the
development of innovative drugs based on the combination and
repositioning of known drugs, today announced that the independent
Data Safety Monitoring Board (DSMB) has completed its second
pre-specified safety evaluation of PXT3003 in the ongoing PLEO-CMT
Phase 3 clinical trial. Based on a review of safety data from all
randomized patients, the DSMB recommended to continue the PLEO-CMT
study as planned.
PLEO-CMT is a pivotal, multi-center, randomized, double blind,
placebo-controlled, three-arm Phase 3 study that was initiated in
December 2015 and has enrolled 323 patients with mild-to-moderate
CMT1A in 30 sites across Europe, the U.S. and Canada. Diagnosis of
CMT1A has been confirmed genetically through detection of PMP22
gene duplication. Over 15 months, Pharnext will compare in parallel
groups the efficacy and safety of two orally administered doses of
PXT3003 to placebo. Efficacy will be assessed through one primary
endpoint: change in the ONLS score at 12 and 15 months of treatment
to measure improvement of patients’ disability with PXT3003.
Additional secondary outcome measures will be assessed including
functional and electrophysiological endpoints.
The DSMB is an independent body of experts drawn from the fields
of clinical medicine, biostatistics and study methodology,
chartered to provide recommendations to Pharnext upon regular
pre-specified review of the accumulated data during the conduct of
the clinical trial.
“This second positive recommendation from an independent board
of experts reinforces PXT3003’s safety profile, even in patients
who received up to 15 months of treatment. This Phase 3 clinical
trial is highly significant for patients suffering from CMT1A where
only supportive care is available today,” said Daniel Cohen,
M.D., Ph.D., Co-Founder and Chief Executive Officer of
Pharnext. “We believe that our PLEODRUG™ PXT3003, if
successful, has the potential to transform the treatment of CMT1A
in adults. We look forward to completing this clinical trial and
sharing top-line results in mid-2018.”
About PXT3003
PXT3003, Pharnext’s lead PLEODRUG™ in development for the
treatment of Charcot-Marie-Tooth type 1A disease (CMT1A), is a
novel, synergistic, low-dose combination of baclofen, naltrexone,
and D-sorbitol formulated as an oral solution given twice-daily.
PXT3003 has multiple main mechanisms of action: a synergistic
inhibition of PMP22 gene overexpression associated with myelination
improvement, direct nerve protection and additional positive
effects on other cellular types: muscle cells, neuromuscular
junctions and immune cells. PXT3003 obtained positive results in a
Phase II clinical trial in 80 adult patients with CMT1A. In 2014,
the EMA and FDA granted orphan drug designation to PXT3003 for the
treatment of CMT1A in adults. An international pivotal Phase III
trial (PLEO-CMT) in over 300 adult patients with CMT1A is now
underway at 30 sites across Europe, the U.S. and Canada.
About Pharnext
Pharnext is an advanced clinical-stage biopharmaceutical company
founded by renowned scientists and entrepreneurs including
Professor Daniel Cohen, a pioneer in modern genomics. Pharnext has
two lead products in clinical development. PXT3003 is currently in
an international Phase 3 trial for the treatment of
Charcot-Marie-Tooth disease type 1A and benefits from orphan drug
status in Europe and the United States. PXT864 has generated
positive Phase 2 results in Alzheimer’s disease. Pharnext is the
pioneer of a new drug discovery paradigm: PLEOTHERAPY™. The Company
identifies and develops synergic combinations of repositioned drugs
at new optimal lower doses. These PLEODRUG™ offer several key
advantages: efficacy, safety and intellectual property including
several product or composition of matter patents already granted.
The Company is supported by a world-class scientific team.
The company Pharnext is listed on Euronext Growth Stock Exchange
in Paris (ISIN code: FR00111911287).For more information, visit
www.pharnext.com
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PharnextXavier Paoli, +33 (0)1 41 09 22 30Chief
Commercial Officercontact@pharnext.comorInvestor Relations
(Europe)MC Services AGAnne Hennecke, +49 211 529252
22anne.hennecke@mc-services.euorMedia Relations
(Europe)ALIZE RPCaroline CarmagnolMargaux Pronost+33 (0)1 44 54
36 64pharnext@alizerp.comorPharnextRené Goedkoop, +33 (0)1
41 09 22 30Chief Medical Officercontact@pharnext.comorInvestor
Relations (U.S.)Stern Investor Relations, Inc.Matthew Shinseki,
+1 212-362-1200matthew@sternir.comorMedia Relations
(U.S.)Russo PartnersTony RussoScott
Santiamotony.russo@russopartnersllc.comscott.santiamo@russopartnersllc.com+1
212-845-4251+1 718-344-5843orFinancial Communication
(France)New CAPEmmanuel Huynh, +33 (0)1 44 71 20
40pharnext@newcap.eu
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