Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose the first participant in the Phase 3 PREMIER trial before the end of the month.
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.
Moving Forward With PXT3003
PREMIER Trial
350 participants with mild-to-moderate CMT1A from 50 sites throughout the world are expected to enroll in this Phase 3 study. This trial is extremely important, as the FDA has stated that it is required for an upcoming New Drug Application (NDA).
An NDA is not the only way in which the FDA is involved in the PREMIER trial; Pharnext has applied for a Special Protocol Assessment (SPA) regarding this trial. The regulatory agency has been working with Pharnext on this assessment as well.
CLN-PXT3003-03
This ongoing Phase III extension study enrolled 187 of the original 323 participants of the first third phase study, PLEO-CMT. This open-label study has been gathering data from patients for over two years, as 130 patients are still receiving high doses of PXT3003. Researchers have been utilizing the data from this trial to evaluate the safety, tolerability, and long-term efficacy of the novel medicine. Data from this trial is expected to be released in the second quarter of this year.
Looking Forward
Hopefully, further research and development of this drug are successful, as CMT patients are in need of viable treatment options. As current data is positive, researchers are excited by upcoming trials and possible FDA approval.
