Neuren Pharmaceuticals Ltd (ASX: NEU) shares have returned from their trading halt with an almighty bang.
In morning trade, the ASX 200 healthcare stock is up 32% to a record high of $22.70.
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Why is this ASX 200 healthcare stock rocketing?
The catalyst for this rise has been the top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS).
According to the release, a significant improvement was observed by both clinicians and caregivers from treatment, across multiple efficacy measures. Pleasingly, improvements were consistently seen across many of the core PMS characteristics.
The company notes that PMS has severe quality of life impacts for those living with the syndrome, as well as parents and siblings. At present, there are no approved treatments for PMS despite its severely debilitating impact.
The ASX 200 healthcare stock's CEO, Jon Pilcher, commented:
The strength and consistency of these results has exceeded our expectations and gives us high confidence as we strive to accelerate the development of a potential first therapy to address the overwhelming unmet medical need of PMS. We are very grateful to all the people at the trial sites in the United States and in the PMS community who enabled this groundbreaking trial to be completed successfully.
An investigator in the study, Elizabeth Berry-Kravis, MD, PhD, from Rush University Medical Center, adds:
Although the Phase 2 trial was an open label study, I am very encouraged that both clinicians and caregivers observed pervasive improvements across multiple, clinically important features of PMS including communication, cognition, learning, socialisation and behaviour. Improvements typically considered clinically meaningful were achieved in the clinician rated CGI-I as well as the caregiver rated CIC. I look forward to seeing this program advance to the next stage of development for the PMS community.
Based on these results, investors appear optimistic that this could be another treatment that Neuren generates significant royalties from in the near future.
