Neuren Pharmaceuticals Ltd (ASX: NEU) shares are starting the week with an almighty bang.
In morning trade, the ASX 200 biotech stock is up a massive 18% to $10.83.
Why is this ASX 200 stock jumping?
Investors have been bidding the pharmaceutical company's shares higher this morning after it announced a key regulatory milestone in the United States.
According to the release, its upcoming Phase 3 trial for NNZ-2591, a potential treatment for Phelan-McDermid syndrome (PMS), has received the green light from the US FDA to proceed as planned.
Management notes that it is an important step and one that could pave the way for a world-first treatment in an area of overwhelming unmet medical need.
So, what exactly is Neuren working on? And why are investors paying attention?
Phelan-McDermid syndrome is an ultra-rare neurological disorder caused by mutations or deletions in the SHANK3 gene, with symptoms that include severe developmental delays, absent speech, autism-like behaviours, epilepsy, and significant impairment in quality of life — not only for patients but for families as well.
There are currently no approved treatments for PMS. That makes Neuren's progress all the more important — and potentially valuable.
The company's planned Phase 3 trial will build on strong Phase 2 results, where 16 out of 18 children showed improvement in key measures of receptive communication and overall symptom change. The release notes that these weren't just statistically significant — they were clinically meaningful.
In the upcoming placebo-controlled trial, the two co-primary endpoints will assess changes in receptive communication and a global clinical impression of change, using tools already validated in the earlier study. Both were previously agreed upon with the FDA, and the company is now preparing to launch the trial mid-year 2025.
Why this matters for investors
The FDA's support means Neuren now has regulatory alignment on the core elements of its Phase 3 design — a major hurdle cleared in the biotech world. And with no need for additional funding, thanks to the company's current financial position, there's a clear runway for execution.
The ASX 200 stock's CEO, Jon Pilcher, commented:
We are very pleased with the outcome of another constructive discussion with the FDA and are now excited to be able to move forward as planned with the first ever Phase 3 trial in children with Phelan-McDermid syndrome.
Despite today's bounce, Neuren's shares remain down by almost 50% since this time last year.
