The ASX healthcare industry has been offering enormous opportunities, well availed by astute investors. Many ASX Healthcare Companies, while being defensive, have huge addressable markets thus providing a low risk opportunity for good potential gains.
To name a few Healthcare Shares, Pro Medicus Limited (ASX: PME), Telix Pharmaceuticals Limited (ASX: TLX) and EZZ Life Science Holdings Limited (ASX: EZZ) not only performed well but turned multibaggers.
Neuren Pharmaceuticals Limited (ASX: NEU)
Neuren Pharmaceuticals Limited (ASX: NEU), is one of the Best Long Term Healthcare Stocks. It recently reported the key findings from its Phase 2 clinical trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS). Both medical professionals and caregivers noted a significant improvement from the initial stage as a result of the treatment, across all 4 efficacy measures specifically tailored to evaluate the fundamental characteristics of PTHS.
Neuren’s collaborator, Acadia Pharmaceuticals (NASDAQ: ACAD), launched DAYBUE (trofinetide) in the United States in April 2023 as the first and only approved therapy for Rett syndrome. Sales escalated rapidly, reaching US$177 million for 2023, leading to royalties of $27 million for Neuren.
At the recent American Academy of Neurology conference, Acadia presented extended efficacy and tolerability findings from the Daffodil study, concentrating on Rett syndrome patients aged 2-4 years. Data was gathered for up to 78 weeks of treatment with DAYBUE (the study was concluded prematurely when DAYBUE was approved in March 2023, so 9 out of the 12 participants had data at week 78, while all 12 had data at week 52). Efficacy data demonstrated sustained enhancements, which progressed with the duration of therapy. The average Clinical Global Impression of Improvement (CGI-I) score improved to 2.2 at 78 weeks.
The company continues to make significant commercial advancements with its DAYBUE (trofinetide) therapy, progressing towards launching the therapy on a global scale after its successful debut in the U.S. This launch delivered significant positive outcomes, supporting substantial revenue and earnings generation, and facilitating a notable financial breakthrough for the company and its stakeholders. Notably, DAYBUE remains the world’s first and only approved therapy for Rett syndrome. The company’s collaborative efforts with its partner, Acadia, to expand the therapy outside of the U.S. now stand to deliver a significant further breakthrough. Given the positive response in the U.S. market and the substantial clinical need that the therapy addresses, there is greater confidence in the potential for significant sales growth on a global scale.
Along with DAYBUE, the company also maintains a strong focus on advancing its extensive clinical pipeline, with clinical development in five additional neurological disorders progressing, all of which have received Orphan Drug Designation (ODD). This designation supports a relatively easier regulatory pathway, bolstered by the fact that there are no approved therapies for these disorders. Notably, after positive results in the Phase 2 trials, the company is now progressing with the End of Phase 2 meeting with the FDA for Phelan-McDermid syndrome in the ongoing third quarter of 2024. Additionally, the company plans to share top-line results for Angelman syndrome during the same period. Significant regulatory advancements are also expected globally for DAYBUE, with approval for New Drug Submission in Canada anticipated by the end of 2024. Furthermore, the company is actively engaging with regulatory agencies in Japan and Europe, highlighting its commitment to expanding the therapy's availability and capitalizing on its potential to address significant clinical needs in these markets.
Source: Company's Report
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