Pharnext Amends the Protocol of the International Pivotal Phase 3 Trials of PXT3003 for Charcot-Marie-Tooth Disease Type 1A
September 18 2017 - 11:00AM
Business Wire
- No safety concern and PXT3003 development
milestones remain unchanged -
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 an amendment in the
protocol of the ongoing Phase 3 clinical program (PLEO-CMT and
PLEO-CMT-FU studies) of PXT3003 for Charcot-Marie-Tooth disease
type 1A (CMT1A) in adults to address a stability issue in the high
dose formulation of PXT3003.
PLEO-CMT, a pivotal, multi-center, randomized, double blind,
placebo-controlled, Phase 3 study, completed enrollment of 323
patients with mild to moderate CMT1A in 30 sites across Europe, the
U.S. and Canada in December 2016. Patients have been randomized to
receive during 15 months either the placebo or one of two doses of
PXT3003: dose 1 (5 mL) or dose 2 (5 mL) with dose 2 equal to twice
the dose 1. According to the protocol, all patients were supposed
to continue treatment in a 9-month extension study (PLEO-CMT-FU),
whilst placebo patients were randomized to dose 1 or dose 2 of
PXT3003.
Overtime, after 12 months, a stability issue emerged in some
batches of the high dose formulation (dose 2). This finding has
raised no safety concern, but to ensure that the high dose patients
get full exposure to the dose 2 level, Pharnext decided to switch
these patients to receive double the amount of dose 1 (2 X 5 mL) in
the 9-month open label extension study (PLEO-CMT-FU). Patients from
the placebo and dose 1 arms in the 15-month double blind PLEO-CMT
study will continue the Phase 3 clinical trial as planned: then,
these patients will have the opportunity to continue treatment with
PXT3003 in the PLEO-CMT-FU extension study for 9 months.
Main PXT3003 development milestones remain unchanged: adaptive
design and futility analysis still planned by the end of 2017,
results of the PLEO-CMT trial still expected in the second half of
2018, most likely in Q3. The statistical analysis plan will take
the amendment into consideration. The data will form the basis of
the submission package for market approval in the first quarter of
2019. Long-term safety data from PLEO-CMT-FU would then be
submitted to regulatory authorities during their review of the
marketing authorization application. Pharnext expects PXT3003
market approval during the second half of 2019, as scheduled.
As previously communicated, the independent Data Safety
Monitoring Board (iDSMB) evaluated the safety data of all patients
on September 5th, 2017, as it had no safety concern for both
PXT3003 doses, it recommended study continuation. Of note, in
agreement with regulatory agencies, the dose 2 was included in the
PLEO-CMT study based on the dose response from the Phase 2 trial.
Only the dose 1 was evaluated in the Phase 2 trial and demonstrated
safety, tolerability and improvement beyond stabilization of CMT1A
patient disability.
Quote:
“We have found a satisfactory solution for this unexpected
stability event of the PXT3003 highest dose that was not previously
investigated in our Phase 2 trial.” said Daniel Cohen, M.D.,
Ph.D., Co-Founder and Chief Executive Officer of Pharnext. “All
our objectives and development milestones remain unchanged and we
look forward to bringing this innovative therapy to CMT1A
patients.”
About PXT3003PXT3003, developed using Pharnext’s R&D
platform PLEOTHERAPYTM, is a novel oral fixed-low dose combination
of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol with
Orphan Drug Designation in Europe and the United States.
About PharnextPharnext 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.
Pharnext is listed on Euronext Growth Stock Exchange in Paris
(ISIN code: FR00111911287).For more information, visit
www.pharnext.com
DISCLAIMERThis press release contains certain
forward-looking statements concerning Pharnext and its business.
Such forward-looking statements are based on assumptions that
Pharnext considers to be reasonable. However, there can be no
assurance that the estimates contained in such forward-looking
statements will be verified, which estimates are subject to
numerous risks including the risks set forth in the Company’s
document de base registered by the French Financial Markets
Authority (Autorité des marchés financiers) on June 2, 2016 under
number I.16-0050 (a copy of which is available on
www.pharnext.com). The occurrence of all or part of such risks
could cause actual results or achievements of Pharnext to be
materially different from such forward-looking statements.
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PharnextRené Goedkoop, MDChief Medical
Officermedical@pharnext.com+33 (0)1 41 09 22 30orInvestor
Relations (Europe)MC Services AGAnne
Henneckeanne.hennecke@mc-services.eu+49 211 529252 22orInvestor
Relations (U.S.)Stern Investor Relations, Inc.Matthew
Shinsekimatthew@sternir.com+1 212-362-1200orFinancial
Communication (France)ActifinStéphane Ruizsruiz@actifin.fr+33
(0)1 56 88 11 15orMedia Relations (Europe)ALIZE RPCaroline
CarmagnolMargaux Pronostpharnext@alizerp.com+33 (0)1 44 54 36
64orMedia Relations (U.S.)RooneyPartnersMarion
Janicmjanic@rooneyco.com+1 212.223.4017
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