The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.
Blog Category
CMT Type – CMT1A
Total posts in Category: 22
Clinical Trial for CMT1A begins in Korea
Helixmith, specializing in gene therapy research for over 20 years, has kicked off its phase I and 2a clinical trial for using VM202 (Engensis) to treat CMT1A.
Press Release: Pharnext provides regulatory and clinical update on PXT3003 Phase III study for the treatment of Charcot-Marie-Tooth Type 1A
US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study
Good News for CMT1A Patients – PXT3003
Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Pharnext raises € 7.7 million in a private placement
Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
Hot Off the Press – Potential Treatment for CMT1A
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.
Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A
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.
Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext
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
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.
Categories
Advocacy (7)
Awareness (34)
Centers of Excellence (13)
CMT (2)
CMT en Español (1)
CMT Summit (53)
CMT Update (74)
CMT-Connect (29)
CMT-Connect Webinars (10)
Webinars – Accessible Travel (3)
Webinars – Adaptive Driving (2)
Webinars – Bracing (1)
Webinars – Breathing (1)
Webinars – Canine Companions (1)
Webinars – Cannabis (3)
Webinars – Dating and Relationships (3)
Webinars – Family Planning (1)
Webinars – FEATURED (6)
Webinars – Genetic Testing (1)
Webinars – Home Accessibility (2)
Webinars – Inclusive Employment (3)
Webinars – Inspire Community (1)
Webinars – Legal Rights and Benefits (3)
Webinars – Newly Diagnosed (2)
Webinars – Nutrition (1)
Webinars – Pain (5)
Webinars – Podcasts (1)
Webinars – PT and Exercise (9)
Webinars – Research and Clinical Trials (9)
Webinars – School and College (4)
Webinars – Surgery (2)
Webinars – Telemedicine (1)
Webinars- Emotional and Mental Health (5)
Community (4)
Emerging Technologies (1)
Faces of CMT (1)
Faces of CMT – Addie (2)
Faces of CMT – Bernadette (9)
Faces of CMT – CMT4A (7)
Faces of CMT – Grace (18)
Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (78)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (21)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (11)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (9)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (11)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (7)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (29)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (10)
Registry (16)
Research (95)
School Outreach Program and Team CMT Kids (1)
Special Events (2)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (61)
The CMT Genie (6)
TRIAD (28)
TRIAD – Clinical Trial Readiness (5)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
TRIAD – Sponsored Research (0)
Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (4)
Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (22)
CMT Type – CMT1B (2)
CMT Type – CMT1X (3)
CMT Type – CMT2A (9)
CMT Type – CMT2C (5)
CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
CMT Type – HNPP (3)
CMT Type – MTRFR-C12orf65 (6)
CMT Type – SORD Deficiency (9)
Upcoming Events (3)
Ways to Give (4)
Awareness (34)
Centers of Excellence (13)
CMT (2)
CMT en Español (1)
CMT Summit (53)
CMT Update (74)
CMT-Connect (29)
CMT-Connect Webinars (10)
Webinars – Accessible Travel (3)
Webinars – Adaptive Driving (2)
Webinars – Bracing (1)
Webinars – Breathing (1)
Webinars – Canine Companions (1)
Webinars – Cannabis (3)
Webinars – Dating and Relationships (3)
Webinars – Family Planning (1)
Webinars – FEATURED (6)
Webinars – Genetic Testing (1)
Webinars – Home Accessibility (2)
Webinars – Inclusive Employment (3)
Webinars – Inspire Community (1)
Webinars – Legal Rights and Benefits (3)
Webinars – Newly Diagnosed (2)
Webinars – Nutrition (1)
Webinars – Pain (5)
Webinars – Podcasts (1)
Webinars – PT and Exercise (9)
Webinars – Research and Clinical Trials (9)
Webinars – School and College (4)
Webinars – Surgery (2)
Webinars – Telemedicine (1)
Webinars- Emotional and Mental Health (5)
Community (4)
Emerging Technologies (1)
Faces of CMT (1)
Faces of CMT – Addie (2)
Faces of CMT – Bernadette (9)
Faces of CMT – CMT4A (7)
Faces of CMT – Grace (18)
Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (78)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (21)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (11)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (9)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (11)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (7)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (29)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (10)
Registry (16)
Research (95)
School Outreach Program and Team CMT Kids (1)
Special Events (2)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (61)
The CMT Genie (6)
TRIAD (28)
TRIAD – Clinical Trial Readiness (5)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
TRIAD – Sponsored Research (0)
Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (4)
Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (22)
CMT Type – CMT1B (2)
CMT Type – CMT1X (3)
CMT Type – CMT2A (9)
CMT Type – CMT2C (5)
CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
CMT Type – HNPP (3)
CMT Type – MTRFR-C12orf65 (6)
CMT Type – SORD Deficiency (9)
Upcoming Events (3)
Ways to Give (4)
Recent Comments