This section highlights FDA-related milestones and regulatory updates for drugs developed by Alterity Therapeutics (ATHE).
Over the past two years, Alterity Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ATH434, ATH434-201, and ATH434-202. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
ATH434 FDA Regulatory Timeline and Events
ATH434 is a drug developed by Alterity Therapeutics for the following indication: To inhibit the aggregation of pathological proteins implicated in neurodegeneration.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ATH434
- Announced Date:
- May 11, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced the publication of a peer-reviewed study in NeuroImage, a leading journal in human brain imaging, demonstrating that quantitative susceptibility mapping (QSM) MRI can detect disease-specific iron accumulation in the brains of patients with Multiple System Atrophy (MSA), distinguish MSA from Parkinson's disease, and track clinical disease severity — including in early-stage disease.
AI Summary
Alterity Therapeutics announced a peer-reviewed study published in NeuroImage showing that quantitative susceptibility mapping (QSM) MRI can detect disease-specific iron build-up in the brains of people with Multiple System Atrophy (MSA). The study found QSM can distinguish MSA from Parkinson’s disease and track clinical disease severity, including in early stages.
Researchers analyzed high-resolution structural and QSM MRI data from a small prospective group of 10 MSA patients followed for 12 months, and cross-sectional data from 28 MSA patients, 43 Parkinson’s patients, and 23 age-matched healthy controls. Results indicate that QSM measures of iron correlate with clinical measures of severity.
These findings support QSM as an objective imaging biomarker to enable earlier and more accurate diagnosis of MSA and to help assess iron-modulating treatments, including Alterity’s lead candidate ATH434, in clinical trials and care.
Read Announcement- Drug:
- ATH434
- Announced Date:
- April 27, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced it has received positive regulatory feedback following a Type C Meeting with the U.S. Food and Drug Administration (FDA) regarding its planned Phase 3 development program for ATH434 in Multiple System Atrophy (MSA).
AI Summary
Alterity Therapeutics said it received positive regulatory feedback after a Type C meeting with the U.S. Food and Drug Administration about its planned Phase 3 program for ATH434 in multiple system atrophy (MSA). The company reported that the FDA’s responses were constructive and supportive of the program’s goals. This feedback relates to how Alterity will design and conduct late‑stage testing of ATH434 to assess safety and effectiveness in people with MSA.
The company said the FDA guidance will help shape next steps for the Phase 3 program, including trial design and data expectations, and supports moving forward with development plans. Alterity framed the outcome as an important regulatory milestone that informs its strategy as it prepares to advance ATH434 toward pivotal studies in MSA.Read Announcement
- Drug:
- ATH434
- Announced Date:
- April 22, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced the presentation of new data analyses from the Phase 2 trial of ATH434, demonstrating clinical efficacy in patients with Multiple System Atrophy (MSA).
AI Summary
Alterity Therapeutics reported new analyses from its Phase 2 trial showing ATH434 slowed functional decline in patients with Multiple System Atrophy (MSA). At Week 52, ATH434 reduced deterioration versus placebo on MuSyCA, a newly described composite MSA scale, with benefits seen in both daily function and neurological exam measures. These effects align with earlier activity reported on the modified UMSARS Part I assessment, supporting a consistent clinical signal for ATH434 as a potential therapy for MSA.
David Stamler, Alterity’s CEO, said the results reinforce ATH434’s potential as a disease‑modifying treatment in this rapidly progressive disorder. The company presented the data ahead of planned Phase 3 engagement with regulators, and the presentation and poster are available on Alterity Therapeutics’ website for review.Read Announcement
- Drug:
- ATH434
- Announced Date:
- April 16, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center and Chief Medical Advisor for Alterity, will deliver an oral presentation during a Late Breaking Session at the American Academy of Neurology (AAN) Annual Meeting taking place April 18-22, 2026 in Chicago, IL, USA.
AI Summary
Alterity Therapeutics announced that Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center and Chief Medical Advisor for Alterity, will deliver an oral presentation during a Late Breaking Session at the American Academy of Neurology (AAN) Annual Meeting. The meeting is scheduled for April 18–22, 2026, in Chicago, Illinois. The company said the presentation will be part of the meeting’s Late Breaking Session, which highlights new and potentially practice-changing research.
This opportunity gives Alterity a high-profile platform to share recent clinical or scientific findings with neurologists, researchers, and the media. The announcement was authorized by Alterity’s Board. Further details on the presentation’s timing, content, and abstracts are expected from Alterity or AAN as the conference approaches, and interested parties can follow official channels for updates.
Read Announcement- Drug:
- ATH434
- Announced Date:
- April 15, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that it will host a virtual key opinion leader (KOL) event featuring Roy Freeman, MD (Harvard Medical School, Beth Israel Deaconess Medical Center) and Daniel Claassen, MD, MS (Vanderbilt University Medical Center), alongside David Stamler, MD, (CEO, Alterity Therapeutics), to discuss the significant unmet need and current treatment landscape in Multiple System Atrophy (MSA), a rare, rapidly progressive neurodegenerative disease with no approved treatment.
AI Summary
Alterity Therapeutics will host a virtual key opinion leader event featuring Roy Freeman, MD (Harvard Medical School, Beth Israel Deaconess Medical Center), Daniel Claassen, MD, MS (Vanderbilt University Medical Center), and CEO David Stamler, MD. They will discuss the significant unmet need and current treatment landscape in Multiple System Atrophy (MSA), a rare, rapidly progressive neurodegenerative disease with no approved treatment.
The program will cover ATH434 — Alterity’s lead candidate and a potential first‑in‑class, disease‑modifying therapy for MSA — along with a disease overview and review of current therapeutic options. Speakers will review Phase 2 data, share new insights and analyses, and outline a high‑level Phase 3 planning approach. The event will also include KOL biographies and information about Alterity Therapeutics Limited.
The announcement was authorized by the Board of Alterity Therapeutics Limited. Investor contacts: Elyse Shapiro [email protected], Remy Bernarda [email protected], +1 (415) 203‑6386. Media contact: Casey McDonald, Tiberend Strategic Advisors, [email protected], +1 (646) 577‑8520. Forward‑looking statements will apply.
Read Announcement- Drug:
- ATH434
- Announced Date:
- March 30, 2026
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced it has received positive regulatory feedback following a Type C Meeting with the U.S. Food and Drug Administration (FDA) regarding its planned Phase 3 development program for ATH434 in Multiple System Atrophy (MSA).
AI Summary
Alterity Therapeutics announced it received positive regulatory feedback from the U.S. Food and Drug Administration following a Type C Meeting about its planned Phase 3 development program for ATH434 in Multiple System Atrophy (MSA). The meeting was part of a multidisciplinary strategy to seek alignment with the FDA on the company’s readiness to initiate a pivotal Phase 3 trial. Alterity said the discussion focused on key program elements needed to move forward.
Alterity reported it received written feedback supporting its plans related to clinical pharmacology and non-clinical development. The clinical pharmacology input addressed dose selection, exposure, and study approaches, while non-clinical feedback covered toxicology and supporting animal studies that back safety and mechanism. Alterity characterized the FDA’s feedback as constructive and said it helps clarify requirements as the company prepares to begin a Phase 3 pivotal trial in MSA.
Read Announcement- Drug:
- ATH434
- Announced Date:
- October 9, 2025
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that data from the ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) was featured at the 2025 International Congress of Parkinson's Disease and Movement Disorders (MDS) that took place in Honolulu, HI, USA.
AI Summary
Alterity Therapeutics announced that data from the ATH434-201 Phase 2 trial in Multiple System Atrophy (MSA) were featured at the 2025 International Congress of Parkinson’s Disease and Movement Disorders in Honolulu, HI. The randomized, double-blind study compared ATH434 at 50 mg and 75 mg twice daily with placebo over one year.
The data showed ATH434 slowed disease progression and stabilized orthostatic hypotension, a severe drop in blood pressure upon standing. The 50 mg dose reduced symptom severity by 48%; the 75 mg dose reduced symptoms by 30%, which increased to 35% after adjusting for blood pressure differences.
Neuroimaging and biomarker analyses refined MSA diagnosis and tracked progression across MSA-P and MSA-C subtypes. ATH434 reduced iron buildup in the globus pallidus, putamen, and substantia nigra and showed trends in preserving brain volume. These results boost confidence in ATH434’s potential as a disease-modifying therapy.
Read Announcement- Drug:
- ATH434
- Announced Date:
- July 28, 2025
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced positive topline data from the ATH434-202 open-label Phase 2 clinical trial in individuals with multiple system atrophy (MSA).
AI Summary
Alterity Therapeutics reported positive topline results from its ATH434-202 open-label Phase 2 trial in people with more advanced multiple system atrophy (MSA). Over 12 months, ATH434 treatment reduced disease progression on the Modified Unified MSA Rating Scale Part I by about half compared to historical data. Thirty percent of participants also showed stable or improved overall neurological symptoms, including orthostatic hypotension.
Neuroimaging biomarkers indicated that ATH434 slowed brain atrophy in MSA-affected regions and reduced iron accumulation in the putamen and globus pallidus, confirming target engagement. These effects matched those seen in an earlier double-blind Phase 2 trial at the same dose.
ATH434 was well tolerated, with no serious treatment-related adverse events and mostly mild to moderate side effects. These consistent clinical and biomarker findings support advancing ATH434 as a potential disease-modifying therapy for MSA.
Read Announcement- Drug:
- ATH434
- Announced Date:
- May 12, 2025
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that several oral and poster presentations related to Alterity's clinical programs in Multiple System Atrophy (MSA) were featured at the 2025 International MSA Congress.
AI Summary
Alterity Therapeutics recently showcased several oral and poster presentations at the 2025 International MSA Congress, emphasizing their promising clinical programs for Multiple System Atrophy (MSA). At the conference, the company shared key data from its ATH434 Phase 2 trial that demonstrated meaningful improvements in patients’ daily activities and symptom severity. Presentations highlighted the drug’s robust efficacy, as seen by reduced disease severity and target engagement in neuroimaging studies. Additionally, the Congress featured data on the MSA Atrophy Index (MSAai), an innovative imaging biomarker developed in collaboration with Vanderbilt University Medical Center. This tool not only enhances the diagnosis of MSA but also aids in monitoring disease progression. The presentations provided valuable insights into how increased levels of cutaneous α-synuclein are linked with more severe orthostatic symptoms, underscoring the potential of ATH434 to benefit MSA patients. Enthusiasm for the clinical progress was evident among leading clinicians, scientists, and community members.
Read Announcement- Drug:
- ATH434
- Announced Date:
- May 5, 2025
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATH434 for the treatment of Multiple System Atrophy (MSA).
AI Summary
Alterity Therapeutics announced that the FDA has granted Fast Track designation for ATH434 for the treatment of Multiple System Atrophy (MSA). This designation is meant to speed up the development and review process for drugs that address serious conditions with limited treatment options. ATH434 is an oral drug candidate that has shown promising results in a Phase 2 clinical trial, where it demonstrated significant improvements in patient function. The treatment works by restoring normal iron balance in the brain and reducing harmful protein build-up, which may slow the progression of MSA. With the Fast Track status, Alterity Therapeutics will have more frequent and earlier interactions with the FDA, potentially expediting the review and approval process. The company is hopeful that this designation will help bring a much-needed innovative treatment option to patients battling MSA more quickly.
Read Announcement- Drug:
- ATH434
- Announced Date:
- April 10, 2025
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that new presentations related to its Multiple System Atrophy (MSA) program were delivered at the American Academy of Neurology (AAN) 2025 Annual Meeting, one of the premier global neurology meetings.
AI Summary
Alterity Therapeutics announced new presentations on its Multiple System Atrophy (MSA) program at the American Academy of Neurology (AAN) 2025 Annual Meeting, one of the world’s leading neurology conferences. The company showcased findings from its ATH434-201 Phase 2 clinical trial in an oral presentation during a Scientific Platform Session on Movement Disorders.
The new data revealed clinically meaningful improvements across several assessments, including daily living activities and overall disease severity. In addition, neuroimaging analyses confirmed target engagement, as ATH434 was shown to reduce iron accumulation in brain regions affected by MSA. These results underline ATH434’s potential as a disease-modifying treatment for MSA—a condition currently lacking effective therapies. The presentations further highlighted innovative use of wearable sensors to monitor patient activity, supporting the trial’s findings and reinforcing the value of digital outcomes in tracking disease progression.
Read Announcement- Drug:
- ATH434
- Announced Date:
- October 11, 2024
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced promising new data related to ATH434 were presented at the Society for Neuroscience 2024 in Chicago, USA.
AI Summary
Alterity Therapeutics has presented promising new data on its lead candidate ATH434 at the Society for Neuroscience 2024 in Chicago. The research highlighted ATH434’s potent antioxidant and mitochondrial-protectant effects, showing that the drug can reduce lipid damage in two different models of neuronal injury. These effects suggest that ATH434 may help protect against oxidative stress, a major contributor to neurodegeneration. The study also demonstrated that ATH434 enhances cellular energy production by shifting mitochondrial pathways to reduce oxidative stress. According to the company’s CEO, these findings strengthen the potential of ATH434 as a disease-modifying treatment for neurodegenerative diseases, including Parkinson’s disease and related disorders. The novel approach of addressing both direct oxidative injury and the redistribution of excess labile iron enhances ATH434’s promise in slowing the progression of neurodegenerative conditions.
Read Announcement- Drug:
- ATH434
- Announced Date:
- October 2, 2024
- Indication:
- To inhibit the aggregation of pathological proteins implicated in neurodegeneration
Announcement
Alterity Therapeutics announced that multiple oral and poster presentations were presented at the International Congress of Parkinson's Disease and Movement Disorders® (MDS).
AI Summary
Alterity Therapeutics recently showcased multiple oral and poster presentations at the International Congress of Parkinson’s Disease and Movement Disorders (MDS). The presentations highlighted key findings from the ATH434-202 Phase 2 open-label trial, which focused on treating Multiple System Atrophy (MSA). During this trial, about 30% of participants, known as clinical responders, experienced stabilizing or improved clinical outcomes, including stable brain imaging results and biomarker levels such as brain iron and neurofilament light chain (NfL).
Additional data were presented on the ATH434-201 Phase 2 randomized, double-blind trial for early-stage MSA and on preclinical studies in Parkinson’s disease. Overall, the results enhanced the profile of ATH434 as a promising, well-tolerated candidate that may slow neurodegeneration. Alterity expects further data updates, including topline results for the ATH434-201 study in January 2025, reinforcing its approach to disease modification.
Read Announcement
ATH434-201 FDA Regulatory Timeline and Events
ATH434-201 is a drug developed by Alterity Therapeutics for the following indication: Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ATH434-201
- Announced Date:
- November 10, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that analyses on the baseline characteristics related to orthostatic hypotension (OH) from the ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) were featured in an oral presentation at the American Autonomic Society (AAS) 36th International Symposium on the Autonomic Nervous System that took place in Clearwater Beach, Florida, USA.
AI Summary
Alterity Therapeutics reported that analyses of baseline characteristics related to orthostatic hypotension (OH) from its ATH434-201 randomized, double‑blind Phase 2 trial in Multiple System Atrophy (MSA) were presented orally at the American Autonomic Society 36th International Symposium in Clearwater Beach, Florida. The work, led by Daniel Claassen, M.D., with Amy E. Brown, M.D., as presenter, examined how OH — a low blood pressure on standing that predicts rapid disease progression — affected trial outcomes and interpretation.
Key findings showed severe OH (a sustained systolic drop >30 mmHg after three minutes standing) was much higher in the 75 mg group (29.2%) than in the 50 mg (4%) or placebo (4.5%) arms. Adjusting for orthostatic blood pressure change strengthened the 75 mg efficacy signal on UMSARS Part I from −2.4 to −2.8 points, shifting the relative treatment effect from 30% to 35%. Patient‑reported OH scores worsened ~6 points on placebo over 52 weeks but remained stable in both ATH434 dose groups. The analysis suggests future studies should control for OH when designing Phase 3 protocols.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- October 2, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that data from the ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) will be featured at the 2025 International Congress of Parkinson's Disease and Movement Disorders (MDS) taking place October 5-9, 2025 in Honolulu, HI, USA.
AI Summary
Alterity Therapeutics announced that data from its ATH434-201 randomized, double-blind Phase 2 trial in Multiple System Atrophy (MSA) will be featured at the 2025 International Congress of Parkinson’s Disease and Movement Disorders (MDS) in Honolulu, October 5–9, 2025.
The main oral platform session, “ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy,” will be presented by CEO David Stamler on October 8. Two poster presentations will take place on October 5:
• Margaret Bradbury, Ph.D., will discuss “Relationship Between Alpha-Synuclein Aggregation Profiles, Imaging Biomarkers, and Disease Severity.”
• Paula Trujillo, Ph.D., will present “Differences Between Clinical and Imaging Phenotypes.”
In the ATH434-201 study, 77 adults received ATH434 or placebo for 12 months, showing significant improvement in daily living activities and motor function, as tracked by clinical scales and wearable sensors. Biomarker changes were positive and safety was good. These sessions will highlight ATH434’s potential to modify disease progression in MSA.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- September 15, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that data from the ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) was presented at the 150th Annual Meeting of the American Neurological Association (ANA), held in Baltimore, MD.
AI Summary
Alterity Therapeutics announced that data from the ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) was presented at the 150th Annual Meeting of the American Neurological Association in Baltimore. Daniel Claassen, M.D., presented a poster titled “ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy,” showing that ATH434 at both 50 mg and 75 mg doses produced clinically meaningful effects. The drug reduced iron accumulation in MSA-affected brain regions, and analysis revealed that initial disease severity explained the different responses between dose groups.
The trial found ATH434 was well tolerated, with adverse event rates similar to placebo and no serious drug-related events. Patients treated with ATH434 showed reduced scores on the MSA activities of daily living scale, improvements in core symptoms, and maintained function in outpatient settings. These results underscore ATH434’s potential as a disease-modifying therapy for MSA and have generated optimism among neurologists and researchers.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- April 28, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that David Stamler, M.D., Chief Executive Officer presented the ATH434-201 Phase 2 clinical trial results at the annual MSA Research Symposium hosted by University College London, Institute of Neurology in partnership with the MSA Trust of the U.K.
AI Summary
Alterity Therapeutics announced that its CEO, David Stamler, M.D., presented the ATH434-201 Phase 2 clinical trial results at the annual MSA Research Symposium. The symposium was hosted by University College London’s Institute of Neurology in partnership with the MSA Trust of the U.K. At the event, Dr. Stamler shared promising data from the double-blind study, which involved patients with Multiple System Atrophy (MSA). The clinical trial evaluated the effect of ATH434 on disease symptoms by measuring improvements in activities of daily living and motor performance over a 52-week period. The results showed a significant treatment benefit at the 50 mg dose, with encouraging trends in reducing disease severity. This presentation highlighted the potential of ATH434 as a novel treatment for MSA and brought together top clinicians and researchers dedicated to finding better solutions for patients with this challenging disorder.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- April 3, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that an oral presentation and a poster presentation related to Alterity's clinical programs in Multiple System Atrophy (MSA) will be delivered at the American Academy of Neurology (AAN) 2025 Annual Meeting taking place April 5 - 9, 2025 in San Diego, CA.
AI Summary
Alterity Therapeutics announced that it will deliver two key presentations at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, CA, scheduled for April 5–9, 2025. The company’s oral presentation will present topline data from its ATH434-201 Phase 2 clinical trial, which assessed the efficacy, safety, and pharmacokinetics of ATH434 in treating Multiple System Atrophy (MSA). Additionally, a poster presentation will showcase data linking wearable sensor technology with clinical scores in individuals with early-stage MSA. These presentations are significant as they highlight promising clinical outcomes and innovative methods for monitoring patient performance outside the clinical setting. Alterity Therapeutics’ efforts at AAN aim to advance the understanding and treatment of neurodegenerative diseases, particularly MSA, and reinforce the potential of ATH434 as a disease-modifying therapy for these conditions.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- January 30, 2025
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced positive topline results from the ATH434-201 randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage multiple system atrophy (MSA).
AI Summary
Alterity Therapeutics announced positive topline results from its ATH434-201 Phase 2 clinical trial in patients with early-stage multiple system atrophy (MSA). In this randomized, double-blind, placebo-controlled study, the 50 mg dose of ATH434 achieved a 48% slowing of clinical progression on the UMSARS Part I scale compared with placebo, showing statistically significant improvement. The trial also demonstrated that ATH434 helped stabilize iron levels in key brain regions affected by MSA, as shown by MRI scans, supporting the potential for a disease-modifying effect. Furthermore, the treatment was well tolerated and exhibited a favorable safety profile. These findings provide important evidence for ATH434’s ability to slow the progression of MSA—a rare, rapidly progressing neurodegenerative disorder with no current treatments—and may help guide future discussions with the FDA.Read Announcement
- Drug:
- ATH434-201
- Announced Date:
- December 4, 2024
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that the last patient in the ATH434-201 Phase 2 trial, a randomized, double-blind, placebo-controlled investigation in early-stage multiple system atrophy (MSA), has completed the study.
AI Summary
Alterity Therapeutics announced that the final patient in its ATH434-201 Phase 2 trial has completed all clinical evaluations. This Phase 2 study is a randomized, double-blind, placebo-controlled investigation in patients with early-stage multiple system atrophy (MSA). The trial enrolled 77 participants who received one of two dose levels of ATH434 or placebo over 12 months. ATH434 is designed to address neurodegeneration by targeting the buildup of alpha-synuclein and abnormal iron levels in the brain. Completing the study marks a significant milestone, allowing the team to begin cleaning and locking the trial database. Topline results are expected to be reported in early 2025. Alterity thanked the trial participants, clinical sites, and the study team for their valuable contributions, emphasizing that this achievement could play a key role in moving toward a definitive Phase 3 trial for this rare, debilitating disease.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- December 4, 2024
- Indication:
- Investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
Announcement
Alterity Therapeutics announced that Topline Data Expected in Early 2025
AI Summary
Alterity Therapeutics announced the completion of the final patient visit in its ATH434-201 Phase 2 trial for early-stage multiple system atrophy (MSA). With this milestone reached, the company expects to report topline data in early 2025, likely between late January and early February. The trial is a randomized, double-blind, placebo-controlled study that evaluates ATH434’s effect on brain iron, alpha-synuclein levels, and motor functions in patients with MSA. ATH434 is designed to reduce the aggregation of pathological proteins and restore proper iron balance in the brain, potentially slowing the progression of neurodegenerative diseases like MSA and Parkinsonian disorders. This achievement marks an important step forward for Alterity Therapeutics as it continues to seek innovative treatments for debilitating neurodegenerative conditions.
Read Announcement
ATH434-202 FDA Regulatory Timeline and Events
ATH434-202 is a drug developed by Alterity Therapeutics for the following indication: In Multiple System Atrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ATH434-202
- Announced Date:
- July 28, 2025
- Indication:
- In Multiple System Atrophy
Announcement
Alterity Therapeutics announced positive topline data from the ATH434-202 open-label Phase 2 clinical trial in individuals with multiple system atrophy (MSA).
AI Summary
Alterity Therapeutics announced positive topline data from the ATH434-202 open-label Phase 2 trial in advanced multiple system atrophy (MSA). Ten participants received ATH434 75 mg twice daily for 12 months. The drug cut disease progression by about half on the Modified Unified MSA Rating Scale Part I compared to historical controls and stabilized overall neurological symptoms in 30% of patients. Orthostatic hypotension symptoms also remained steady over the year.
Neuroimaging biomarkers showed ATH434 slowed brain atrophy in MSA-affected regions, matching results from an earlier placebo-controlled study. MRI scans found reduced iron accumulation in the putamen and globus pallidus, confirming target engagement and supporting the observed clinical benefits.
ATH434 was well tolerated with no serious adverse events related to treatment and mostly mild to moderate side effects. These consistent clinical and biomarker findings support advancing ATH434 as a potential disease-modifying therapy for MSA.
Read Announcement- Drug:
- ATH434-202
- Announced Date:
- March 27, 2025
- Indication:
- In Multiple System Atrophy
Announcement
Alterity Therapeutics announced that the last patient in the ATH434-202 Phase 2 trial has completed the study.
AI Summary
Alterity Therapeutics announced that the last patient in the ATH434-202 Phase 2 trial for advanced multiple system atrophy (MSA) has completed the study. This open-label trial is designed to test the safety, effectiveness, and target engagement of ATH434, a drug candidate aimed at treating Parkinsonian disorders. The study involved 10 participants with advanced MSA who received a 75 mg dose of ATH434 over 12 months. The trial focuses on measuring changes in brain volume along with neuroimaging and protein biomarkers to determine how well the drug acts on the disease. Alterity expects to share the topline data from the study around mid-year 2025. The company is encouraged by the progress and newly gained insights, which will help shape the future development of the treatment for patients facing severe challenges with their illness.
Read Announcement- Drug:
- ATH434-202
- Announced Date:
- July 17, 2024
- Indication:
- In Multiple System Atrophy
Announcement
Alterity Therapeutics announced positive interim data from the ATH434-202 open-label Phase 2 clinical trial in patients with multiple system atrophy (MSA).
AI Summary
Alterity Therapeutics announced positive interim results from its open-label Phase 2 ATH434-202 trial in patients with multiple system atrophy (MSA). The study involved advanced MSA patients treated with ATH434, an oral agent designed to reduce α-synuclein pathology and restore normal iron levels in the brain. After six months of treatment, 43% of the participants showed improvement on the Unified MSA Rating Scale-Part I, which measures the impact of MSA on daily activities, while 29% had stable or improved overall neurological symptoms.
Additionally, objective biomarker assessments, including neuroimaging and fluid markers, supported these clinical findings by showing reduced brain iron accumulation and stable NFL levels in the clinical responders. ATH434 was well-tolerated, with no significant safety concerns reported. These promising interim results suggest that ATH434 could modify disease progression in MSA patients, increasing confidence in its potential as a disease-modifying treatment.
Read Announcement
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