PARIS--(BUSINESS WIRE)--Pharnext SAS today announced its collaboration with OrphanDev, a dedicated platform offering support in regulatory, methodology and logistics for rare diseases. OrphanDev’s main mission is to accelerate the development of orphan drugs. This collaboration is related to the International pivotal Phase 3 trial of Pharnext’s investigational Pleodrug, PXT-3003, for the treatment of CMT 1A.
PLEO-CMT aims at recruiting 300 patients in 27 centers both in Europe and the United States. For the European part of the trial (18 centers spread over Germany, Belgium, Spain, France, the UK and the Netherlands), OrphanDev will play a critical role in the dissemination and availability of practical and logistical information related to the trial, notably via its website: www.orphan-dev.org.
Pharnext and OrphanDev had already collaborated on 3 studies related to CMT 1A: PXT-3003 Phase2 study, a meta-analysis and a prospective study (biomarkers).
About PXT-3003
PXT-3003 is a novel oral fixed-low dose
combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
which has been developed via Pharnext Pleotherapy R&D platform.
About PLEO-CMT
PLEO-CMT is an International pivotal,
multi-center, randomized, double blind, placebo-controlled, three-arm
Phase 3 study which will enroll patients
aged 16 to 65 with mild to
moderate CMT1A. Over 15 months, Pharnext will compare in parallel groups
the efficacy and safety of two orally administered dosage variations of
PXT-3003 versus 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 PXT-3003.
Additional secondary outcome measures will be assessed including
functional and electrophysiological endpoints. A nine month follow-up
study is planned thereafter, where all patients who will have completed
the first 15 months and agreed, will receive a dose of the active
product (patients who received placebo will be treated randomly with
PXT-3003 dose 1 or 2).
About CMT 1A
Charcot-Marie-Tooth (CMT) disease encompasses a
heterogeneous group of inherited, progressive, chronic peripheral
neuropathies. CMT type 1A (CMT 1A), the most common type of CMT, is an
orphan disease affecting at least 125,000 people in Europe and the U.S.
The genetic mutation responsible for CMT 1A is a duplication of the PMP
22 gene coding for a peripheral myelin protein. Overexpression of this
gene causes degradation of the neuronal sheath (myelin) responsible for
nerve dysfunction, followed by loss of nerve conduction. As a result of
peripheral nerve degradation, patients suffer from progressive muscle
atrophy of legs and arms causing walking, running, balance problems and
abnormal hand functioning. CMT 1A patients end up in wheelchairs in at
least 5% of cases. They might also suffer from mild to moderate
sensitive disorders. First symptoms usually appear during adolescence
and will progressively evolve through patients’ life.
To date, no
curative or symptomatic medications have been approved and treatment
consists of supportive care such as orthotics, leg braces, physical and
occupational therapy or surgery.
About OrphanDev
OrphanDev is an academic platform at the
heart of rare diseases, close to research teams, clinicians, industry
professionals and patient organizations. It offers its scientific,
regulatory and methodological expertise in the development of drugs for
rare diseases. OrphanDev assists its partners, particularly for Orphan
Drug Designation applications, the recruitment of patients for clinical
trials, and European projects. OrphanDev is a public platform linked to
the University of Aix Marseille (France) and the Timone Institute of
Neurosciences based in Marseille. OrphanDev is a component of the F-CRIN
infrastructure (French Clinical Research Network) which had mission to
reinforce the visibility and the competitiveness of French clinical
research.
For further information, visit http://orphan-dev.org
About Pharnext
Pharnext is an advanced clinical stage
biopharmaceutical company developing novel therapeutics that
simultaneously target multiple key disease pathways for severe orphan
and common neurological disorders. The proprietary research and
development platform of Pharnext, based on network pharmacology, is
applicable to a broad spectrum of diseases and allows the rapid
development of “pleodrugs”, synergistic combinations of repositioned
drugs with established safety profiles. The company’s two lead pleodrugs
are PXT-3003 for the treatment of orphan disease Charcot Marie Tooth
type 1A (Phase 3 clinical trial initiated) and PXT-864 for Alzheimer’s
disease (Phase 2 clinical trial ongoing) and other neurologic
indications (including Parkinson’s disease and amyotrophic lateral
sclerosis).
For further information, visit www.pharnext.com
