Pharnext Pharmaceuticals, a clinical-stage biopharmaceutical company, partnered with HNF in 2012 to support the development of PXT3003.
PXT3003 is a novel combination drug of baclofen, naltrexone, and sorbitol designed to downregulate the overexpression of PMP22, the protein impacted in CMT1A. This leads to the improvement of neuronal signaling in dysfunctional peripheral nerves.
Results of their pivotal phase III are expected by Q4, 2023.
“GRIN data has been essential in clinical trial protocol design, site selection, and efficient patient recruitment for the pivotal Phase III clinical study of our lead asset PXT3003, the PREMIER trial, in development for the most common type of CMT, CMT1A. In addition, the robust data collected in GRIN, together with the ongoing CMT&Me digital survey, will enable a better understanding of the burden of disease, comorbidities, and critical patient demographics as we move forward to a potential commercialization of PXT3003 if approved by the FDA. Topline data of the PREMIER trial are expected in the last quarter of 2023.” Xavier Paoli M.Sc. Pharnext, Chief Operating Officer
Learn more about HNF’s history with Pharnext.
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Pharnext unveils the latest progress of the PREMIER Phase III clinical trial for CMT1A
The PREMIER trial, initiated in March 2021, is an international, randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, where the primary objective is to evaluate the efficacy and safety of PXT3003 versus placebo in mild-to-moderate CMT1A...
CMT&ME App Study Presented at PNS Meeting 2022
The CMT&Me real-world digital lifestyle study self-reports data from patients with all types of CMT and is collected on a regular basis in both US and Europe.
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
New Study Measures Progression of CMT1A Nerve Impairment
New Study Measures Progression of CMT1A Nerve Impairment
Interim Analysis Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)
CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003
The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A.
Young Entrepreneur with CMT1A Launches Company
Dakota launched her own business, Blue Bear in August 2020 with a full line of greeting cards, stickers and enamel pins.
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
The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003.
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