Neuren Pharma (ASX:NEU) has popped 20% as the FDA greenlights its upcoming Phase 3 trial in children with Phelan-McDermid syndrome (PMS) – the first ever to be conducted.

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A rare disorder, PMS ultimately leads to developmental disabilities in children with the condition that acts on chromosomes. The condition can be inherited genetically, but it can also form sporadically on its own.

The FDA approval for Neuren’s Phase 3 trial comes on the back of a meeting it held with regulators in the U.S. establishing primary endpoints for the study.

In other words: Deciding what milestones the company would put in place to determine whether or not its drug NNZ-2591 is effective in treating PMS. (For the study to satisfy FDA’s internal scientific standards, a general methodology needs to be determined.)

That methodology is fairly stock standard: The double-blind study will give some patients NNZ-2591, while others will receive a placebo.

Caregivers and clinicians will also give feedback data with one key area of focus being communication – as in, whether or not children who take NNZ-2591 during these trials do improve their ability to follow verbal instructions and then interpret other information compared to the placebo group.

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While the FDA needs to give another final approval before the trial kicks off around halfway through 2025, this penultimate step has made its investors happy.

And that may be a boost Neuren shareholders needed to hear – Neuren’s one-year returns are down -48% YoY.

NEU last traded at $11.03/sh.

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