Alterity Therapeutics Presents Phase 2 Data – ATH434 Shows Clinically Meaningful Efficacy - Multiple System Atrophy (MSA) - A neurodegenerative disease.
- Noel Ong
- Sep 30
- 5 min read
Announcement
Alterity Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) has presented important data from its ATH434-201 Phase 2 clinical trial at the 150th Annual Meeting of the American Neurological Association (ANA) in Baltimore, USA. The trial focused on Multiple System Atrophy (MSA), a rare and rapidly progressive neurodegenerative disease. These results mark a significant milestone for Alterity as it moves closer to providing the first disease-modifying therapy for MSA.
Key Highlights
Clinically Meaningful Efficacy: ATH434 slowed disease progression at both 50 mg and 75 mg doses (Figure 1). Patients receiving ATH434 experienced statistically significant improvements on the modified Unified MSA Rating Scale (UMSARS Part I), which measures activities of daily living.
Figure 1: Chart of UMSARS Part I score improvement vs placebo (source: ATH)
Target Engagement: Biomarker analysis confirmed ATH434’s mechanism of action, showing reduced iron accumulation in brain regions affected by MSA and trends toward preserving brain volume (Figure 2).
Figure 2: MRI images or chart showing iron reduction (source: ATH)
Improved Patient Function: Wearable sensor data demonstrated that patients on ATH434 had increased daily activity in outpatient settings, suggesting that benefits translated to real-world function.
Favourable Safety Profile: ATH434 was well-tolerated, with adverse event rates similar to those of the placebo. No serious adverse events were attributed to the study drug.
Broad Therapeutic Potential: As an iron chaperone, ATH434 is not only a candidate for MSA but also shows promise for Parkinson’s disease and other Parkinsonian disorders.
About the ATH434-201 Trial
The Phase 2 study enrolled 77 adult participants with early-stage MSA, randomized to receive ATH434 (50 mg or 75 mg twice daily) or placebo for 12 months. The endpoints assessed included efficacy (UMSARS Part I, motor performance, global impression scales), safety, pharmacokinetics, and biomarker effects. The trial’s design also incorporated wearable sensors to provide continuous monitoring of motor activity outside the clinic—a feature that adds depth to the clinical data.
About Multiple System Atrophy (MSA)
MSA is a rare Parkinsonian disorder affecting at least 15,000 people in the U.S. It is characterized by autonomic dysfunction, impaired movement, and progressive disability. Currently, no approved therapy slows disease progression, making disease-modifying approaches like ATH434 critical for patient outcomes.
Samso Concluding Comments
Alterity’s Phase 2 data continues to build a strong case for ATH434 as a first-in-class disease-modifying therapy for MSA. The statistically significant improvement on UMSARS Part I is particularly noteworthy, as it reflects real-world impact on patients’ ability to function day-to-day. For a condition as debilitating and fast-progressing as MSA, this is exactly the type of signal that clinicians and investors look for when assessing a therapy’s potential.
The confirmation of target engagement – iron reduction in affected brain regions and trends towards brain volume preservation is a validation that ATH434 is working where it should. In a space where many neurodegenerative programs fail due to a lack of biomarker correlation, this does appear to add an important layer of confidence for the next stage of development.
From an investor’s point of view, the fact that ATH434 was well tolerated and demonstrated comparable safety to placebo helps de-risk the program further. With Fast Track and Orphan Drug Designations already in hand, Alterity is positioned to move efficiently towards pivotal trials and potentially accelerated regulatory pathways. These factors could be key value drivers as the company progresses to its next inflection point.
As always, the real challenge will be execution—completing a successful Phase 3 trial, securing funding, and translating clinical promise into a registrational outcome. But with these results, Alterity has a compelling narrative: meaningful clinical benefit, mechanistic validation, and a clear plan forward. For investors following the neurodegeneration space, ATH434 and Alterity Therapeutics deserve to be firmly on the radar.
The Samso Way – Seek the Research
Facts first, context always. Dig into the clinical data, understand the endpoints, and assess how they align with market potential before making any investment decisions.
Our mission is simple: cut through the noise and spotlight what matters—genuine stories, grounded insights, and real opportunity.
Our content is well-researched and is only created if the team sees a merit in discussing the company or concept. Investors can explore our three core platforms:
There may be numerous paths to success in investing, but the common thread among successful individuals is that they remain committed to making informed decisions. Equip yourself with the right knowledge and tools, and you will be well on your way to achieving your financial goals.
Most importantly, investors need to be absolutely diligent in understanding their own risk-reward tolerance and capabilities. Never bite off more than you can chew. As they say, Rome wasn’t built in a day, and the Great Wall stood because it took centuries to complete.
The Samso Philosophy:
Stay curious. Stay sharp. And remember—digging deeper always uncovers the real value.
In Life, there is no such thing as a Free Lunch.
Happy Investing, and the only four-letter word you need to know is DYOR.
To support our independent nature of our work, please head over to our Support Page and give us a helping hand in any of the ways listed. This is a new initiate for the Samso Platform, and it was always the concept of Samso when we started this journey in 2018.
Disclaimer
The information or opinions provided herein do not constitute investment advice, an offer or solicitation to subscribe for, purchase or sell the investment product(s) mentioned herein. It does not take into consideration, nor have any regard to your specific investment objectives, financial situation, risk profile, tax position and particular, or unique needs and constraints.
Share to Grow: Your Bonus
Samso has just released an eBook: How to Add Value to your Share Portfolio
A lesson on geological models sought by mining companies that gives insight and an understanding of which portfolios are better - and potentially more lucrative – investments. Click here to download this eBook.
If you find this article informative and useful, please help me share the information. I try and write about topics that are interesting and have the potential to be of investment value. It is not easy to find stories that fit those parameters. If you or your organisation see the benefit of what Samso is trying to achieve and have a need to share your journey, please contact me at noel.ong@samso.com.au.
Samso is a trusted platform that equips dedicated investors with up-to-date industry knowledge and insights from top CEOs and thought leaders. By staying informed on business advancements and market trends, investors can enhance their financial decisions through a combination of expert guidance and their own research.
Comments