Alpha Blue Ocean, Pharnext Strike Deal on PXT3003 Study

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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PXT3003 for CMT1A

A new financial partnership between Pharnext and Alpha Blue Ocean will help support the Phase 3 clinical trial PREMIER, which is testing Pharnext’s investigational therapy PXT3003 for Charcot-Marie-Tooth disease type 1A (CMT1A).

CMT1A is the most common subtype of CMT type 1, which is the most common type of CMT. CMT1A is caused by a genetic mutation that leads to excessive production of the PMP22 protein. Excess levels of this protein lead to a loss of myelin, the fatty substance that surrounds nerve fibers and enhances nerve cell communication.

PXT3003 is a combination of three approved treatments — the muscle relaxant baclofen, naltrexone (used to treat opiate and alcohol addiction), and the laxative sorbitol — that seeks to reduce PMP22 levels and increase myelin production.

The PREMIER clinical trial (NCT04762758), which recently enrolled its first participant, intends to enroll about 350 participants with mild-to-moderate CMT1A, ranging in age from 16 to 65 years. The Pharnext-sponsored study is actively recruiting participants at sites in the U.S., with locations in Canada, Israel, and several countries in Europe yet to open.

Participants in PREMIER will be assigned randomly to receive either a high dose of PXT3003 or a placebo, given as an oral solution two times per day for 15 months.

The study’s primary endpoint, or main measure of effectiveness, is the extent of physical disability as measured with the Overall Neuropathy Limitation Scale (ONLS). Total ONLS scores can range from zero (no disability) to 12, or maximum disability. Changes in participants’ walking ability and muscle strength also will be assessed, along with participant-reported measurements of disease severity, and safety and tolerability outcomes.

Pharnext previously announced plans to seek approval of PXT3003 in the U.S. and the European Union, if results from PREMIER are positive and other laboratory data also support the investigational medication.

In a prior Phase 3 trial, PLEO-CMT (NCT02579759), participants given the same high dose of PXT3003 that is being used in PREMIER experienced reductions in their ONLS scores compared with those on a placebo, as well as improved walking ability. However, the previous trial had missing data due to a manufacturing problem and a temporary treatment interruption, leading the U.S. Food and Drug Administration to request an additional Phase 3 trial in 2019.

The new financing agreement between Pharnext and Alpha Blue Ocean is for up to 81 million Euros (about $98.4 million).

“We are pleased to announce this financing that extends our cash position to continue and complete our pivotal Phase III study in CMT1A,” David Horn Solomon, managing director at Pharnext, said in a press release.

“We are excited to embark on this journey with Pharnext to provide the funding necessary to complete their pivotal Phase III study,” added Pierre Vannineuse, founder and CEO of Alpha Blue Ocean.