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.
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 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- Drug:
- ATH434
- Announced Date:
- April 29, 2024
- Indication:
- Multiple System Atrophy (MSA)
Announcement
Alterity Therapeutics announced that important new data on its lead drug candidate ATH434 was presented at the World Orphan Drug Congress USA 2024 in Boston, MA.
AI Summary
Alterity Therapeutics announced that new data on its lead drug candidate ATH434 was presented at the World Orphan Drug Congress USA 2024 in Boston, MA. The presentation featured a poster titled "Biophysical Characteristics of ATH434, a Unique Iron-Targeting Drug for Treating Friedreich’s Ataxia," presented by Ashley Pall from Wayne State University. The study showed that ATH434 functions as an iron chaperone, meaning it binds and redistributes the harmful ferrous iron responsible for oxidative stress in neurodegenerative diseases, such as Friedreich’s Ataxia.
This new evidence highlights that ATH434 differs from traditional iron chelators, which only remove excess iron from the body. By selectively targeting the toxic form of iron, ATH434 may help slow the progression of diseases associated with iron mismanagement, offering a unique approach to treatment. The findings provide valuable insights into the drug’s mechanism and support its potential as a disease-modifying treatment.
Read Announcement- Drug:
- ATH434
- Announced Date:
- April 10, 2024
- Indication:
- Multiple System Atrophy (MSA)
Announcement
Alterity Therapeutics announced that new data on ATH434 will be presented at the World Orphan Drug Congress USA 2024 taking place April 23-25, 2024 in Boston, MA.
AI Summary
Alterity Therapeutics announced that new data on ATH434 will be presented at the World Orphan Drug Congress USA 2024. The event is scheduled for April 23-25, 2024, in Boston, MA. The data, led by Ashley Pall from the Department of Pharmaceutical Sciences at Wayne State University, focuses on the biophysical characteristics of ATH434. This drug is designed to target iron in the brain and may help reduce the build-up of pathological proteins linked to neurodegenerative diseases. ATH434 is an oral treatment that has shown promise in preclinical studies by restoring normal iron balance and protecting neuronal functions. Alterity Therapeutics is dedicated to developing treatments that modify the disease process, and this presentation underscores their commitment to advancing potential therapies for conditions like Friedreich’s Ataxia and Parkinsonian disorders.
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:
- 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 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- 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:
- May 8, 2024
- Estimated Event Date Range:
- January 1, 2025 - January 31, 2025
- Target Action Date:
- January 1, 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 Top-Line Data Expected in January 2025 -
AI Summary
Alterity Therapeutics recently announced that its top-line data is expected to be available in January 2025. This update has drawn significant attention from industry experts and investors who are keen to see how the data will impact future regulatory decisions. The upcoming data release is seen as a critical milestone in the company’s development of its innovative treatments. Although the announcement provided limited details on the trial parameters or the therapy’s specific goals, it has set the stage for a period of close observation and anticipation within the medical and investment communities. Analysts believe that the data could offer valuable insights into the treatment’s effectiveness and safety profile, ultimately influencing the pathway towards full FDA approval. Stakeholders are advised to keep an eye on further communications from Alterity Therapeutics as the planned release date approaches.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- May 8, 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 ATH434-201 Trial on Track to Complete in November 2024
AI Summary
Alterity Therapeutics recently announced that its ATH434-201 trial is on track to complete in November 2024. This trial is a key step in their development efforts and is expected to provide clear and important data that could guide future treatment options.
The company is focused on ensuring that the study meets all milestones while continuing to adhere to regulatory requirements. Completing this trial is an important achievement that shows progress in their research and development efforts.
With the trial scheduled for completion next November, healthcare professionals and investors are watching closely. The anticipated success of ATH434-201 could play a significant role in advancing new therapeutic options and offer hope for improved treatments in the future.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- May 8, 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 an independent Data Monitoring Committee (DMC) has completed its third review of trial data and recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201 clinical trial is a randomized, double-blind, placebo-controlled 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.
AI Summary
Alterity Therapeutics announced that an independent Data Monitoring Committee (DMC) has completed its third review of trial data from the ATH434-201 Phase 2 study. The DMC recommended that the study continues as planned, supporting the idea that it is safe for participants to proceed. This clinical trial is carefully designed as a randomized, double-blind, placebo-controlled investigation. It focuses on evaluating the safety and potential effectiveness of ATH434 in patients who have early-stage multiple system atrophy (MSA). MSA is a rare neurodegenerative disease that currently has no approved treatments to slow or stop its progression. The positive recommendation from the DMC boosts hope that ATH434 may turn into one of the first treatment options for this challenging disease, addressing an urgent unmet medical need in the field of neurodegeneration by ensuring robust and safe trial practices.
Read Announcement- Drug:
- ATH434-201
- Announced Date:
- April 17, 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 three posters were presented at the American Academy of Neurology (AAN) 2024 Annual Meeting taking place April 13-18, 2024, in Denver, Colorado, USA. Featured presentations described the Company's work in Parkinson's disease and Multiple System Atrophy (MSA), including initial biomarker data and baseline characteristics from the ATH434-201 Phase 2 clinical trial.
AI Summary
Alterity Therapeutics announced that three posters were presented at the American Academy of Neurology (AAN) 2024 Annual Meeting in Denver, Colorado from April 13–18, 2024. The presentations focused on the company’s research work in Parkinson’s disease and Multiple System Atrophy (MSA), emphasizing the potential of its lead candidate, ATH434.
One key poster highlighted a primate study where ATH434 treatment improved motor performance and general functioning by reducing abnormal iron levels in the brain. Another presentation detailed initial biomarker data and baseline characteristics from the ATH434-201 Phase 2 clinical trial in early-stage MSA patients. The study showed increased brain iron and higher plasma Neurofilament Light Chain levels, which correlated with disease severity. These findings support further investigation of ATH434 as a promising disease-modifying therapy for neurodegenerative disorders.
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:
- 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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