Pharnext Shares Top-Line Results from Pivotal Phase 3 Studies of PXT3003; More to Come

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Pharnext recently provided an update from its ongoing Phase 3 clinical trials evaluating PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) in adults, and also announced that some top-line results are expected by October 2018.

PXT3003 is a fixed-dose combination of baclofen, naltrexone and sorbitol with orphan drug status in the United States and Europe.

PXT3003’s clinical program is comprised of Phase 3 PLEO-CMT trial (NCT02579759) in Europe and the United States, and the Phase 3 follow-up study, PLEO-CMT-FU (NCT03023540).

The PLEO-CMT trial is studying the safety and effectiveness of two doses of PXT3003 compared to placebo in 323 patients with mild-to-moderate CMT1A.

The PLEO-CMT-FU trial is treating all patients who were given PXT3003 for the first 15 months in the first trial with an additional nine months of PXT3003; the goal is to assess the long-term safety and tolerability of the potential therapy.

“We are thrilled to bring this Phase 3 clinical trial to completion and we now expect to disclose top-line results by October of this year. Our Pleodrug PXT3003 has already shown initial signals of efficacy in our Phase 2 trial in CMT1A,” Daniel Cohen, MD, PhD, co-founder and CEO of Pharnext, said in a press release.

The drug was developed using Pharnext’s new approach to developing new drug combinations, based on big genomic data and artificial intelligence (AI).

Known as Pleotherapy, the drug discovery approach seeks synergic combinations of drugs, called Pleodrugs, which offer several advantages, such as increased effectiveness and safety.

“We are hopeful we can bring this much-needed therapy to patients suffering from this debilitating condition, as they currently have limited therapeutic options, most of which are palliative in nature,” Cohen added.

Earlier this month, Pharnext announced that PXT3003 was granted priority review by the China Food and Drug Administration (CFDA), based on preliminary results from the same clinical program.

Charcot-Marie-Tooth disease type 1 is the most common type of CMT, accounting for about two-thirds of all recorded cases of the disease. CMT1 is caused by genetic mutations that damage the myelin sheath,  a protective layer that ensures the correct transmission nerve signals from the brain to the muscles. The loss of myelin results in a slow transmission of these nerve signals, resulting in decreased sensitivity.

Specifically, in the subtype CMT1A, there is an extra copy of the PMP22 gene, which encodes the peripheral myelin protein 22. This protein is a key component of the myelin sheath; however, its duplication disrupts its structure and function. CMT1A is the most common subtype of CMT1, accounting for about 60 percent of all CMT1 cases.