Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350 participants from 50 various sites throughout the world. They are hoping for a successful trial, as positive results may aid in the approval process for PXT3003 later on.
About Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth (CMT) disease, also known as hereditary motor and sensory neuropathy, is a neurological disorder that affects the peripheral nervous system. It occurs as the result of mutations in various genes (CMT1, CMT2, CMT3), all of which result in the death of peripheral nerves. Depending on which gene is affected, this condition can be inherited in an autosomal dominant or recessive pattern. There is also an X-linked version of the disease, which is passed down through mutations of the GJB1 gene. In this case, CMT1A is the result of an extra PMP22 gene, which is passed down in an autosomal dominant pattern.
Regardless of inheritance, affected individuals may experience foot deformities, difficulty with walking and fine motor skills, weakness in the hands and feet, and lower leg deformities. There is currently no cure for this disease, although physical and occupational therapy are used to maintain muscle strength and use. Pain medications may also be prescribed.
About the Study
This Phase 3 study, titled PREMIER, will evaluate PXT3003 as a treatment for CMT1A with the goal of compiling evidence to support FDA approval. Already, Pharnext is working with the regulatory agency on a special protocol assessment. If all goes well, the trial will begin by the end of the first quarter of this year, and it will lead to approval from both the FDA and European Medicines Agency (EMA).
For 15 months, participants will receive either a placebo or high doses of PXT3003. Researchers will focus on the primary endpoint, which is to evaluate changes in functional disability using the Overall Neuropathy Limitation Scale. Secondary endpoints are the strength of hand and feet muscles, walking speed, and severity on the CMT neuropathy score version 2.
About PXT3003
PXT3003 is a combination of three therapies: sorbitol, baclofen, and naltrexone. Each component targets a different receptor in the nervous system in order to modify pathways and ultimately decrease the expression of the PMP22 gene. This will lead to increased myelination, which will improve patients’ quality of life.
Looking Forward
Beyond the PREMIER trial, Pharnext is also conducting a preclinical trial with animal models on each separate component of PXT3003. This study will allow researchers and other medical professionals to better understand the treatment as a whole.
The PLEO-CMT-FU study, an open-label extension trial, will continue as well. 130 of the 187 original patients are participating, all of whom have taken PXT3003 for more than two years. Data from this trial is expected in June.
Hopefully these trials and further efforts lead to FDA approval for this treatment, as it stands to improve the lives of those living with CMT1A.
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