A touching and instructive testimony on CMT disease and its effects realized by CMT UK, one of the ECMTF founding members.
A touching and instructive testimony on CMT disease and its effects realized by CMT UK, one of the ECMTF founding members.
Vivi in Italia? Ti è stata diagnosticata la #CMT? Unisciti a noi nell’aiutare la ricerca a migliorare il trattamento della malattia partecipando al nostro studio usando il tuo smartphone o tablet.
Per il sistema operativo iOS, clicca qui https://itunes.apple.com/us/app/cmt-me/id1417129091… oppure,
per Android, clicca qui https://play.google.com/store/apps/details…
#salutedigitale #rara #evidenzamondoreale #genomica #molecola #CMT #CMTandMe Pharnext ACMT Rete per la Charcot Marie Tooth ONLUS European CMT Federation
Leben Sie in Deutschland? Wurde bei Ihnen die Erkrankung #CMT diagnostiziert? Dann unterstützen Sie durch Ihre Teilnahme an unserer Studie mittels der #BYOD Technologie unsere Forschung zur Verbesserung der Behandlung dieser Erkrankung.
Für iOS-Geräte klicken Sie hier https://itunes.apple.com/us/app/cmt-me/id1417129091…
oder für Android-Geräte hier https://play.google.com/store/apps/details…
#DigitaleGesundheit #selten #echteNachweise #Genomik #Molekül #CMT #CMTandMe Pharnext European CMT Federation
Is now available, in Germany and in Italy, the free app CMT&Me thanks to which it will be possible to further knowledge of CMT and improve care. Each CMT patient can install the app on his smartphone and fill out questionnaires, record symptoms in a diary, access the knowledge section and much more. Do not miss the opportunity to help research and, at the same time, to better manage your CMT.
Da oggi è disponibile in Italia e in Germania l’applicazione gratuita CMT&Me grazie alla quale sarà possibile approfondire la conoscenza sulla CMT e migliorarne la cura. Ogni malato CMT può caricarla sul proprio smartphone e compilare questionari, memorizzare i propri sintomi su un diario, accedere ad una pagina di informazioni e altro ancora. Non perdete l’occasione di aiutare la ricerca e, al tempo stesso, di gestire al meglio la vostra CMT.
Nantes, France – May 22th, 2018.
InFlectis BioScience SAS, a drug discovery company committed to the development of innovative therapeutics harnessing the Integrated Stress Response for the treatment of a broad range of diseases, today announced that the French National Agency for Medicines and Health Products Safety (ANSM) approved the Company’s Clinical Trial Application (CTA) to begin a Phase 1 study of IFB-088 (study P-188). First results from the study are expected in the first half of 2019. This study will provide
the safety data necessary for Phase 2 studies in Charcot-Marie-Tooth (CMT) patients that are expected to begin end of 2019.
A Clinical Trial Application (CTA) was submitted to ANSM in March 2018 for a phase 1 clinical trial to investigate the safety, tolerability and pharmacokinetics of IFB-088 when administered in single and multiple doses in healthy volunteers. It was approved on May 18th, 2018. This Phase 1 trial of IFB-088 follows a standard single ascending dose and multiple ascending dose design with the enrolment of 72 healthy volunteers.
Following the successful completion of the Phase 1 study, InFlectis BioScience will transition the IFB-088 program into a Phase 2 clinical trial to test the drug treatment’s efficacy in treating patients with Charcot-Marie-Tooth disease. Based on preclinical evidences and proofs of concept in CMT1A and CMT1B animal models, the European Commission and the Food and Drug Administration (FDA) have both already granted orphan drug designation (ODD) to IFB-088 in CMT.
Philippe Guédat, Président and CEO of InFlectis BioScience SAS said: “We are very pleased to have received the authorization to initiate a human clinical trial with IFB-088. Entering the clinic represents a significant step for IFB-088 and one step closer to reaching CMT patients who are suffering from this rare and debilitating neuropathy. In addition to CMT, the drug might also be assessed in the future in other degenerative pathologies for which InFlectis has already obtained preclinical efficacy in animal models”.
ABOUT IFB-088 (also known as Sephin1)
IFB-088 is a first-in-class orally available small molecule drug candidate with a validated mechanism of action and a promising pharmacokinetic profile for targeting the central and peripheral nervous system. IFB-088 is a selective inhibitor of PPP1R15A (GADD34), a stress-induced PP1 phosphatase regulatory subunit involved in the unfolded protein response. PPP1R15A inhibition by IFB-088 regulates the protein translation rate in stressed cells to a level manageable by the available cellular proteins that assist in protein folding (so-called “chaperones”), thereby restoring proteostasis. IFB-088 is strikingly specific for stressed cells, avoiding persistent inhibition of protein synthesis in normal, non-stressed cells.
ABOUT INFLECTIS BIOSCIENCE (www.inflectisbioscience.com)
InFlectis BioScience is a clinical stage company committed to the development of innovative therapeutics harnessing the Integrated Stress Response (ISR) for the treatment of a broad range of diseases. The company plans to demonstrate the clinical effectiveness of its drug candidate IFB-088 for the treatment of Charcot-Marie-Tooth diseases type 1A (CMT-1A) and 1B (CMT-1B). The company is also developing IFB-088 for the treatment of rare eye diseases. Meanwhile, InFlectis BioScience develops new chemical series for the treatment of non-orphan diseases. Based in Nantes in Western France, InFlectis BioScience is part of the science park of the economic area of Nantes Atlantique.
Researchers have found that disease progression in Charcot-Marie-Tooth type 1A (CMT1A) patients reaches a critical point at age 50, after which clinical decline occurs faster. In response, patients should be encouraged to engage in a healthy and active lifestyle in order to preserve their function, a new study recommends.
The article “Motor performance deterioration accelerates after 50 years of age in Charcot-Marie-Tooth type 1a patients” was published in the European Journal of Neurology.
The aim of the study was to describe the clinical decline in CMT1A patients, by comparing their clinical impairment and age. CMT1A is the most common CMT subtype.
Researchers at the University of Naples “Federico II” in Naples, Italy, recruited and evaluated 70 CMT1A patients (26 men and 44 women from 20 to 81 years of age) and 70 sex- and age-matched healthy participants as controls.
All healthy participants had no neurological symptoms or signs or other disabilities. In the CMT1A patient group, 38 patients were from 14 different families.
Clinical impairment was measured through motor performance using three different tests: the 10-Meter Walk Test, the 6-Minute Walk Test, and the 9-Hole Peg Test of dominant and non-dominant side. All three tests determine the ability and capacity of the participant to achieve specific physical tasks.
In addition, muscle strength, disability and quality of life were evaluated in CMT1A patients.
The researchers analyzed the relationships between age and all clinical measures. In general, motor performance deteriorated with age in both CMT1A patients and healthy participants. However, motor performance deterioration was more accentuated in CMT1A patients.
The authors found that CMT1A patients and healthy participants had similar clinical measures until 50 years of age. However, from age 50 onwards, CMT1A patients had significantly greater clinical impairment when compared with healthy participants.
“Our results support this assumption demonstrating that after the 50th year of age the extent of deterioration of clinical impairment accelerates in CMT1A patients,” the authors wrote. “This is relevant for clinicians in the current management of CMT1A patients. A healthy lifestyle and physical activity should be encouraged in order to preserve the functional reserve.”
Paris, France, 6:00pm, September 18, 2017 (CEST) – Pharnext SA (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.
“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.”
PXT3003, 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.
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.
Pharnext is listed on Euronext Growth Stock Exchange in Paris (ISIN code: FR00111911287).
For more information, visit www.pharnext.com
This 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.
We are delighted to acknowledge the very kind support of French pharmacological company, Pharnext, for their donation which allowed us to have this website created.
Pharnext are currently developing a new drug to treat CMT Type 1a. The drug is in Phase 3 trials across Europe and the States currently, and the results of this trial will be available sometime in 2018.
You can read more about PXT3003 here – https://www.pharnext.com/en/products/pxt3003
The European CMT Federation is delighted to announce the launch of it’s new website.
At present, there is not too much content, but once the Federation is registered in Belgium, and we can begin our work properly, there will be more added to the site.
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