CATEGORIES: CMT Type - CMT1A | Research | TRIAD

Good News for CMT1A Patients – PXT3003

by | Jun 9, 2020 | 0 comments

ALLISON MOORE, FOUNDER/CEO, HNF

pharnextI’m excited by Pharnext’s recent announcement that three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A. 

The United Kingdom has granted Pharnext a Promising Innovative Medicine (PIM) designation as an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS) in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with unmet need.

Despite the climate of COVID-19, our industry partner, Pharnext SA, continues to plan for the additional pivotal Phase 3 PXT3003 trial for CMT1A in patients 16 years and older in the US and Europe.

“All existing data indicate that PXT3003 is a safe and well tolerated drug combination. We look forward to continuing our discussions with U.S. and European regulatory authorities to advance the clinical development of PXT3003 and initiate as quickly as possible an additional pivotal Phase 3 trial in the U.S. and Europe.” 

–  Daniel Cohen, M.D., Ph.D., Co-Founder and Chairman of the Scientific Advisory Board of Pharnext

You may be eligible to participate. By creating a profile in HNF’s Global Registry in Inherited Neuropathies (GRIN), you may be considered as a potential candidate for the Phase 3 study. 

JOIN GRIN TODAY! www.hnf-cure.org/registry/

You can also help advance the knowledge of CMT and the impact on Quality of Life, by joining the CMT&Me App. The objective of the app is to better understand the impact of the disease on patients’ daily lives: the burden of CMT, its natural history and treatment, and medical, social and pharmacoeconomic effects.

Read full press release from Pharnext www.hnf-cure.org/research/pharnext-pim

Learn more on this topic

Related Blog Posts

A New Mouse Model for Charcot-Marie-Tooth (CMT2)

by | May 27, 2015 | , , | 0 Comments

We were recently informed that The Jackson Laboratory (JAX, a nonprofit biomedical research institution headquartered in Bar Harbor, Maine) had taken delivery and will be distributing a newly generated CMT-related mouse model. The new model expresses mutant mitofusin 2, a mitochondrial membrane protein involved in mitochondrial fusion and regulation of vascular smooth muscle cell proliferation.

Hot Off the Press – Potential Treatment for CMT1A

by | Mar 9, 2015 | , , | 53 Comments

Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.

Support CMT Therapeutic Alliance

by | Mar 9, 2015 | , | 0 Comments

HNF has entered into a joint venture – the CMT Therapeutic Alliance – with a unique non profit organization (BioPontis Alliance for Rare Diseases) that brings professional drug discovery capabilities to translate our research results into potential treatments.

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A

by | Dec 19, 2014 | , , | 12 Comments

PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.

Sneak Peak of Fall Newsletter: CMT Research Study Survey

by | Sep 16, 2014 | , | 2 Comments

My name is Elizabeth Francisco and I am a graduate student from the Genetic Counseling program at the University of North Carolina Greensboro. I am inviting you to participate in a research study. The goal of my study is to learn more about the experiences of people with Charcot-Marie-Tooth (CMT) with genetic counseling and genetic testing. Adults with CMT and parents or legal guardians of someone of any age who has a diagnosis of CMT are eligible to participate

Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext

by | Aug 19, 2014 | , , | 1 Comment

HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.

Targeting PMP22 in CMT1A Patients with Gene Duplication

by | Aug 19, 2014 | , | 1 Comment

In previous collaborative work with a group of investigators including Dr Rolf Renne from the University of Florida, Dr. Alex Murashov from East Carolina University and Dr. Lynn Hudson from the NIH-NINDS, we validated a microRNA known as miR29a as a reagent that corrected the expression level of PMP22 in rodent Schwann cells.

Quality of Life and CMT Research

by | Jul 7, 2014 | | 4 Comments

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Join the conversation

Leave a Comment

0 Comments

Submit a Comment

Your email address will not be published. Required fields are marked *

Newsletter

Join for notifications on events, campaigns, & news