Latest news with #DavidStamler
The Australian
a day ago
- Health
- The Australian
Health Kick Podcast: Alterity Therapeutics
Stockhead's health and biotech expert Tim Boreham is back in the studio for another instalment of the Health Kick Podcast. In this episode, Tim speaks with Alterity Therapeutics (ASX:ATH) CEO Dr David Stamler, who sheds light on a little-known but devastating neurological condition, multiple system atrophy (MSA). MSA may not have the public awareness of Parkinson's or ALS, but it's every bit as serious, and Alterity is aiming to change that. With no approved treatments currently available, MSA represents a major unmet clinical need. Alterity's lead candidate, ATH434, is designed to address a unique biological mechanism involving iron imbalance in the brain. Tune in to hear how ATH434 is showing promise in slowing disease progression, the science behind iron maldistribution in neurodegenerative disease, and how Dr Stamler's decades of FDA drug development experience is helping drive this program forward. This podcast was developed in collaboration with Alterity Therapeutics, a Stockhead advertiser at the time of publishing. The interviews and discussions in this podcast are opinions only and not financial or investment advice. Listeners should obtain independent advice based on their own circumstances before making any financial decisions.
Business Upturn
28-07-2025
- Business
- Business Upturn
Alterity Therapeutics Reports Positive Topline Data from Open-Label Phase 2 Clinical Trial of ATH434 in Multiple System Atrophy
– ATH434 Demonstrated Clinical Benefit on the Unified MSA Rating Scale and Global Measures of Neurological Symptoms – – Neuroimaging Biomarkers Showed Target Engagement and Slowed Brain Atrophy – – ATH434 was Well-Tolerated with Favorable Safety Profile – – Data are Consistent with Phase 2 Double-Blind Trial and Support Advancement of ATH434 in MSA – MELBOURNE, Australia and SAN FRANCISCO, July 28, 2025 (GLOBE NEWSWIRE) — Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ('Alterity' or 'the Company'), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced positive topline data from the ATH434-202 open-label Phase 2 clinical trial in individuals with multiple system atrophy (MSA). The ATH434-202 trial evaluated a patient population with more advanced disease than was studied in Alterity's double-blind Phase 2 trial ATH434-201. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by redistributing iron in the central nervous system. The topline data showed that ATH434 conferred a clinical benefit on areas of impairment in MSA and stabilized key biomarkers that underpin the pathology of the disease. The results from the ATH434-202 trial indicate that ATH434 demonstrated a clinical benefit on the Modified Unified MSA Rating Scale Part I (UMSARS I)1, as well as stabilization of overall neurological symptoms. Over the 12-month treatment period, disease progression as assessed with UMSARS I was reduced by approximately half as compared to historical controls2. In addition, 30% of participants reported stable neurological symptoms over the course of the study. These outcomes are potentially promising as stabilization of MSA symptoms is unexpected in this patient population. On the important symptom of orthostatic hypotension3, ATH434 on average stabilized low blood pressure symptoms in study participants. Importantly, the aggregate data indicate that ATH434 has similar clinical efficacy in this advanced MSA population as was observed in the earlier stage patients in Study ATH434-201. Biomarker endpoints were used to evaluate potential drug effect and target engagement. Neuroimaging outcomes indicate that ATH434 slowed brain atrophy in MSA affected areas, as measured by the MSA Atrophy Index (MSA-AI)4, when compared to placebo-treated participants in Study 201. Moreover, the effects on brain volume were comparable to those observed in participants in the 75 mg dose group in Study 201. In addition, ATH434 led to lower iron accumulation in the putamen and globus pallidus as compared to placebo treated patients in Study 201, providing further evidence of target engagement. 'I am very encouraged by the positive results from the ATH434-202 trial, as they reinforce the robust efficacy we observed in our double-blind study,' said David Stamler, M.D., Chief Executive Officer of Alterity. 'The data from our Phase 2 studies are consistent and strongly support advancing our ATH434 program in MSA. With the favorable clinical and biomarker outcomes we have seen, we continue to believe that ATH434 has the potential to slow the progression of this devastating disease. We are committed to bringing this new therapy to patients as soon as possible.' Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center and principal investigator for the ATH434-202 Phase 2 study, commented, 'These results are very helpful in establishing the clinical response to therapy. The consistent changes in UMSARS, along with quantitative measures in imaging, support the findings we noted in Study 201. Currently, there are no disease modifying medications for the treatment of MSA, and these data encourage the continued development of ATH434 to treat this disease. We are indebted to the study participants and their families who contributed to this study.' ATH434-202 Topline Data Summary The ATH434-202 Phase 2 clinical trial was an open label study in advanced MSA. Ten (10) participants were enrolled in the trial and were diagnosed with MSA using a multimodal approach (clinical, neuroimaging, fluid biomarkers). Participants were treated with oral ATH434 75 mg twice daily for 12 months. The study assessed the safety and efficacy of ATH434 treatment on clinical and biomarker endpoints. The pre-specified key clinical endpoints included the modified UMSARS I, the clinical global impression of change, and the patient global impression of change. Enrolled participants were more advanced than those in the double blind ATH434-201 Phase 2 trial based on baseline variables including duration of motor symptoms, UMSARS I score, frequency of severe orthostatic hypotension, and plasma NFL levels. Based on the observed clinical and neuroimaging data, ATH434 improved overall neurological symptoms and slowed disease progression compared to historical data. Clinical Endpoints at 12 months Modified Unifie d MSA Rating Scale Part I (UMSARS I) The mean (SD) UMSARS scores increased from baseline to 12 months by 3.5 (4.7) points. These study data compare favorably to historical data in a similar MSA population, where an increase (worsening) of 6.5 (6.0) points over 12 months was observed 2 . . 43% (3/7) of participants who completed the study had stable UMSARS scores. Global Impression of Change 30% (3/10) of participants stabilized or improved on the Clinical Global Impression of Change (CGIC) 5 scale, which asks the investigator to evaluate overall neurological symptoms as compared to immediately before starting therapy. scale, which asks the investigator to evaluate overall neurological symptoms as compared to immediately before starting therapy. 30% (3/10) of participants also stabilized or improved on the Patient Global Impression of Change (PGIC)6 scale, which asks the patient to evaluate their overall neurological symptoms as compared to immediately before starting therapy. Orthostatic Hypotension Symptom Assessment (OHSA) The OHSA is a patient reported outcome that evaluates 6 symptoms of low blood pressure associated with moving from a sitting to a standing position. On average, symptoms were stable over the 12-month treatment period. Biomarker Endpoints at 12 months Brain volume The key biomarker endpoint was defined as the change in brain volume from baseline to 12-months, as measured by the MSA Atrophy Index (MSA-AI), which compares brain volume change in MSA affected regions to age matched controls using a composite z-score: Brain volume decreased on average, with a mean (SD) z-score of -0.44 (0.138), consistent with that seen in the 75 mg treatment group of Study 201. ATH434 slowed atrophy in MSA affected brain regions compared favorably to placebo-treated subjects in Study 201. Brain iron A secondary biomarker endpoint was change in iron content in MSA affected areas (substantia nigra, putamen, globus pallidus) from baseline to 12-months, as measured by MRI (quantitative susceptibility mapping): Reduced iron accumulation in the globus pallidus and putamen, as compared to placebo in Study 201, provide evidence of target engagement and support the clinical efficacy of ATH434. The pattern of iron accumulation in the substantia nigra over 12 months was comparable to that observed in the 75 mg dose group in Study 201. Neurofilament Light Chain (NfL) is a marker of axonal injury in neurons and is elevated in individuals with MSA. On average, plasma and CSF NFL levels were stable over the 12-month treatment period. Safety Results The safety population (n=10) includes all enrolled participants who received at least one dose of study drug. No serious adverse events (SAEs) related to ATH434 were reported. ATH434 was well-tolerated and most adverse events were mild to moderate in severity. There were 3 discontinuations from the study. Two participants discontinued due to progression of MSA and one discontinued for an AE that was not related to treatment. There was no evidence of adverse effects on hemoglobin or iron parameters Additional information on the open label Phase 2 trial can be found at NCT05864365 . About ATH434 Alterity's lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. As an iron chaperone, it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2 open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA, and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA. About Multiple System Atrophy Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the support cells of the central nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure7. Definitions and References 1 Unified MSA Rating Scale, Part I (historical review) assess activities of daily living. Domains assessed include speech, swallowing, handwriting, cutting food/handling utensils, dressing, hygiene, walking, falling, orthostatic symptoms, urinary function, sexual function and bowel function. 2 Wenning et al. The natural history of multiple system atrophy: a prospective European cohort study. Lancet Neurol 2013; 12: 264–74. 3 Orthostatic hypotension is a form of low blood pressure that might cause dizziness, lightheadedness or fainting when rising from sitting or lying down. Source: Mayo Clinic. 4 Trujillo et al. The MSA Atrophy Index (MSA-AI): An Imaging Marker for Diagnosis and Clinical Progression in Multiple System Atrophy . Annals of Clinical and Translational Neurology 2025. 5 Clinical Global Impression of Change: a clinician assessment to evaluate overall neurological symptoms as compared to immediately before starting therapy. 6 Patient Global Impression of Change: a patient assessment to evaluate their overall neurological symptoms as compared to immediately before starting therapy. 7 Multiple System Atrophy | National Institute of Neurological Disorders and Stroke ( . About Alterity Therapeutics Limited Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company's website at . Authorisation & Additional information This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited. Investor and Media Contacts: Australia Millie MacdonaldHead of Investor Relations and Business Development [email protected] +61 468 304 742
Associated Press
12-05-2025
- Business
- Associated Press
Alterity Therapeutics Prominently Featured at the International MSA Congress
–ATH434 Phase 2 data demonstrated clinically meaningful efficacy on multiple clinical endpoints – –MSA Atrophy Index (MSAai) enhances MSA diagnosis and monitoring – –bioMUSE Study shows higher α- synuclein concentration is associated with greater burden of orthostatic symptoms – MELBOURNE, Australia and SAN FRANCISCO, May 12, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ('Alterity' or 'the Company'), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today 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. The Congress was presented by Mission MSA, a non-profit organization dedicated to improving the quality of life and building hope for people affected by MSA through support services, educational resources, research funding, and community engagement. 'The MSA Congress gave us an opportunity to share the results of our Phase 2 double-blind trial of ATH434 with prominent MSA clinicians and scientists as well as community members affected by MSA,' said David Stamler, M.D., Chief Executive Officer of Alterity. 'The robust efficacy of ATH434, as indicated by reduced disease severity on the MSA activities of daily living scale along with improvement in key symptoms of MSA and preserved activity in the outpatient setting, continue to generate enthusiasm. Our clinical progress is generating significant excitement, and we are focused on bringing this therapy to patients as quickly as possible.' 'In addition, our colleagues at Vanderbilt University Medical Center presented data from our bioMUSE Natural History Study that highlights the understanding of MSA we have brought to our development program. Because of the variability in MSA presentation and progression and its similarity to Parkinson's disease, it is critical to improve diagnostic and monitoring tools. Through development of a novel imaging biomarker known as the MSA Atrophy Index (MSAai), we have a new tool to measure and track brain volume in individuals with MSA. In a separate presentation, the bioMUSE study demonstrated that α-synuclein levels in the skin increased over the 12-month follow-up period, and that a higher concentration of α-synuclein in skin is associated with greater burden of orthostatic symptoms, a valuable finding that confers insight into disease progression.' Presentation Highlights: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy Presenter: David Stamler, M.D., Chief Executive Officer of Alterity The oral and poster presentation highlighted data from Alterity's ATH434-201 Phase 2 clinical trial. The clinical analysis included 71 patients who had at least one post-baseline assessment of the key clinical endpoint, the modified UMSARS1 I activities of daily living scale. On this endpoint, ATH434 demonstrated a clinically significant reduction in disease severity versus placebo, with a 48% relative treatment effect at the 50 mg dose (p=0.02)^ and a 30% relative treatment effect at the 75 mg dose (p=0.16) at 52 weeks. Additional efficacy assessments showed improvement consistent with the UMSARS I findings: the Clinical Global Impression of Severity Scale2 demonstrated improvement compared to placebo at both dose levels, with difference at 50 mg achieving nominal statistical significance (p=0.0088). On the Orthostatic Hypotension Symptom Assessment (a patient reported outcome), on average placebo patients worsened by approximately 6 points over 52 weeks whereas both ATH434 treatment groups improved over the same period (p=0.08 at 50 mg, p=0.14 at 75 mg). Baseline differences in disease severity likely explain different response in 50 mg and 75 mg treatment groups. Increased activity in the outpatient setting was observed at both dose levels as compared to placebo with wearable sensors, with clinically meaningful improvements in step count, bouts of walking, total walking time, and total standing time. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Regarding neuroimaging in 61 participants, ATH434 demonstrated target engagement by stabilizing or reducing iron accumulation at both dose levels compared to placebo in MSA affected brain regions. In addition, ATH434 demonstrated trends in reducing brain atrophy at both dose levels compared to placebo. Overall, the study results support continued advancement of ATH434 for the treatment of MSA. MSA Atrophy Index (MSAai): A Quantitative Imaging Marker for Diagnosis and Monitoring of Multiple System Atrophy Presenter: Amy Brown, M.D., M.S., Assistant Professor, Movement Disorders Division, Department of Neurology, Vanderbilt University Medical Center This oral presentation described the MSA Atrophy Index (MSAai) as a promising imaging biomarker that distinguishes MSA from related disorders, correlates with clinical presentation, and tracks disease progression. Its implementation has the potential to enhance MSA diagnosis and monitoring in both clinical trials and clinical practice. The MSAai imaging tool was developed by the team at Vanderbilt University Medical Center as part of the bioMUSE Natural History Study. The study evaluated the utility of volumetric measures and the MSAai in distinguishing early MSA from other movement-related disorders, correlating with clinical presentation, and tracking longitudinal changes in brain structure. The study results showed that fluid biomarkers classified bioMUSE participants into MSA (n=10), Lewy body disorders (n=5; PD or DLB), and α-synuclein-negative (n=2) groups. At baseline, MSA patients had significantly lower LN, brainstem, cerebellum, and MSAai values compared to HC, PD, DLB, and PAF (all p<0.001). The MSAai demonstrated strong group discrimination, correlated with UMSARS scores (ρ=-0.46, p=0.038), and predicted clinical progression (ρ=0.58, p=0.022). Longitudinally, reductions in LN, brainstem, and MSAai volumes were strongly associated with UMSARS progression. Cutaneous Phosphorylated Alpha-Synuclein Deposition Informs Autonomic Function in Individuals with Early-Stage Multiple System Atrophy Presenter: Leah Mann, PhD, Postdoctoral Research Fellow, Vanderbilt University Medical Center This poster presentation evaluated 17 participants from the bioMUSE Natural History Study who met criteria for clinically probable MSA. The study compared α-synuclein deposition in skin at baseline and 12 months later and examined associations between clinical measures and α-synuclein quantitation over the year. The analysis described the relationship between α-synuclein and autonomic function and revealed that cutaneous α-synuclein detection may serve as an effective diagnostic biomarker that can additionally track progression of MSA. Importantly, deposition of α-synuclein may confer insight into symptomatology, as higher α- synuclein concentration is associated with greater burden of orthostatic symptoms. In the analysis, 100% (17/17) of patients exhibited detection of phosphorylated α-synuclein by skin biopsy. Cutaneous α-synuclein deposition significantly increased ( p = 0.042) from baseline (mean = 6.59 ± 4.37) to 12-month follow-up (mean = 7.71 ± 4.22). Deposition at 12-month follow-up was positively correlated with Orthostatic Hypotension Questionnaire (OHQ) and Composite Autonomic Symptom Score-31 (COMPASS-31) (OHQ: coefficient = 0.579, p = 0.015; COMPASS-31: coefficient = 0.560, p = 0.024). Presentations will be available on the Alterity Therapeutics website here. About ATH434 Alterity's lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. As an iron chaperone, it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2 open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA, and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA. About ATH434-201 Phase 2 Clinical Trial The ATH434-201 Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of 12 months treatment with ATH434 in patients with MSA. The study evaluated the efficacy, safety and pharmacokinetics of ATH434 as well as the effect of ATH434 on neuroimaging and protein biomarkers. Wearable sensors were employed to evaluate motor activities outside of the clinic. The study enrolled 77 adults who were randomly assigned to receive ATH434 50 mg or 75 mg twice daily or matching placebo. The data showed that, compared to placebo, ATH434 produced clinically and statistically significant improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. Additional efficacy assessments demonstrated improvement consistent with the positive UMSARS Part I findings including trends in improved motor performance on the Parkinson's Plus rating scale, the Clinical Global Impression of Severity Scale, and the Orthostatic Hypotension Symptom Assessment (a patient reported outcome). Wearable sensor data indicated that ATH434 also led to increased activity in an outpatient setting. Biomarkers were used to evaluate potential drug effect and target engagement relative to placebo. Both dose levels reduced iron accumulation in MSA affected brain regions with trends in preservation of brain volume. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Additional information on the Phase 2 trial can be found by Identifier: NCT05109091. About bioMUSE Biomarkers of progression in Multiple System Atrophy (bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a parkinsonian disorder without approved therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity's randomized ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently enrolling in the Phase 2 clinical trial. About Multiple System Atrophy Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the support cells of the central nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure.1 1 Multiple System Atrophy | National Institute of Neurological Disorders and Stroke ( About Alterity Therapeutics Limited Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company's website at Authorisation & Additional information This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited. Investor and Media Contacts: Australia Millie Macdonald Head of Investor Relations and Business Development [email protected] +61 468 304 742 Ana Luiza Harrop [email protected] +61 452 510 255 U.S. Remy Bernarda [email protected] +1 (415) 203-6386 Forward Looking Statements This press release contains 'forward-looking statements' within the meaning of section 27A of the Securities Act of 1933 and of such words as 'expects,' 'intends,' 'hopes,' 'anticipates,' 'believes,' 'could,' 'may,' 'evidences' and 'estimates,' and other similar expressions, but these words are not the exclusive means of identifying suchstatements. Importantfactorsthatcouldcauseactualresultstodiffermateriallyfromthoseindicatedbysuchforward-lookingstatements aredescribedinthesectionstitled'RiskFactors'intheCompany'sfilingswiththeSEC,includingitsmostrecentAnnualReport onForm20-FaswellasreportsonForm6-K,including,butnotlimitedtothefollowing:statementsrelatingtotheCompany's drug development program, including, but not limited to the initiation, progress and outcomes of clinical trials of the Company'sdrugdevelopmentprogram,including,butnotlimitedto,ATH434,andanyotherstatementsthatarenothistorical difficultiesordelaysinfinancing,development,testing,regulatoryapproval,productionandmarketingoftheCompany'sdrug components,including,butnotlimitedto,ATH434,theabilityoftheCompanytoprocureadditionalfuturesourcesoffinancing, unexpected adverse side effects or inadequate therapeutic efficacy of the Company's drug compounds, including, but not limitedto,ATH434,thatcouldslowor prevent productscomingtomarket,the uncertaintyof obtaining patent protectionfortheCompany's intellectualpropertyortradesecrets, the uncertainty of successfully enforcing the Company's patent rights and the uncertainty of the Company freedom to operate. Any forward-looking statement made by us in this press release is based only on information currently available to us and whetherwrittenororal,thatmaybemadefromtimetotime,whetherasaresultofnewinformation,futuredevelopmentsor otherwise.
Yahoo
12-05-2025
- Business
- Yahoo
Alterity Therapeutics Prominently Featured at the International MSA Congress
– ATH434 Phase 2 data demonstrated clinically meaningful efficacy on multiple clinical endpoints – – MSA Atrophy Index (MSAai) enhances MSA diagnosis and monitoring – – bioMUSE Study shows higher α- synuclein concentration is associated with greater burden of orthostatic symptoms – MELBOURNE, Australia and SAN FRANCISCO, May 12, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ('Alterity' or 'the Company'), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today 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. The Congress was presented by Mission MSA, a non-profit organization dedicated to improving the quality of life and building hope for people affected by MSA through support services, educational resources, research funding, and community engagement. 'The MSA Congress gave us an opportunity to share the results of our Phase 2 double-blind trial of ATH434 with prominent MSA clinicians and scientists as well as community members affected by MSA,' said David Stamler, M.D., Chief Executive Officer of Alterity. 'The robust efficacy of ATH434, as indicated by reduced disease severity on the MSA activities of daily living scale along with improvement in key symptoms of MSA and preserved activity in the outpatient setting, continue to generate enthusiasm. Our clinical progress is generating significant excitement, and we are focused on bringing this therapy to patients as quickly as possible.' 'In addition, our colleagues at Vanderbilt University Medical Center presented data from our bioMUSE Natural History Study that highlights the understanding of MSA we have brought to our development program. Because of the variability in MSA presentation and progression and its similarity to Parkinson's disease, it is critical to improve diagnostic and monitoring tools. Through development of a novel imaging biomarker known as the MSA Atrophy Index (MSAai), we have a new tool to measure and track brain volume in individuals with MSA. In a separate presentation, the bioMUSE study demonstrated that α-synuclein levels in the skin increased over the 12-month follow-up period, and that a higher concentration of α-synuclein in skin is associated with greater burden of orthostatic symptoms, a valuable finding that confers insight into disease progression.' Presentation Highlights: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy Presenter: David Stamler, M.D., Chief Executive Officer of Alterity The oral and poster presentation highlighted data from Alterity's ATH434-201 Phase 2 clinical trial. The clinical analysis included 71 patients who had at least one post-baseline assessment of the key clinical endpoint, the modified UMSARS1 I activities of daily living scale. On this endpoint, ATH434 demonstrated a clinically significant reduction in disease severity versus placebo, with a 48% relative treatment effect at the 50 mg dose (p=0.02)^ and a 30% relative treatment effect at the 75 mg dose (p=0.16) at 52 weeks. Additional efficacy assessments showed improvement consistent with the UMSARS I findings: the Clinical Global Impression of Severity Scale2 demonstrated improvement compared to placebo at both dose levels, with difference at 50 mg achieving nominal statistical significance (p=0.0088). On the Orthostatic Hypotension Symptom Assessment (a patient reported outcome), on average placebo patients worsened by approximately 6 points over 52 weeks whereas both ATH434 treatment groups improved over the same period (p=0.08 at 50 mg, p=0.14 at 75 mg). Baseline differences in disease severity likely explain different response in 50 mg and 75 mg treatment groups. Increased activity in the outpatient setting was observed at both dose levels as compared to placebo with wearable sensors, with clinically meaningful improvements in step count, bouts of walking, total walking time, and total standing time. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Regarding neuroimaging in 61 participants, ATH434 demonstrated target engagement by stabilizing or reducing iron accumulation at both dose levels compared to placebo in MSA affected brain regions. In addition, ATH434 demonstrated trends in reducing brain atrophy at both dose levels compared to placebo. Overall, the study results support continued advancement of ATH434 for the treatment of MSA. MSA Atrophy Index (MSAai): A Quantitative Imaging Marker for Diagnosis and Monitoring of Multiple System AtrophyPresenter: Amy Brown, M.D., M.S., Assistant Professor, Movement Disorders Division, Department of Neurology, Vanderbilt University Medical Center This oral presentation described the MSA Atrophy Index (MSAai) as a promising imaging biomarker that distinguishes MSA from related disorders, correlates with clinical presentation, and tracks disease progression. Its implementation has the potential to enhance MSA diagnosis and monitoring in both clinical trials and clinical practice. The MSAai imaging tool was developed by the team at Vanderbilt University Medical Center as part of the bioMUSE Natural History Study. The study evaluated the utility of volumetric measures and the MSAai in distinguishing early MSA from other movement-related disorders, correlating with clinical presentation, and tracking longitudinal changes in brain structure. The study results showed that fluid biomarkers classified bioMUSE participants into MSA (n=10), Lewy body disorders (n=5; PD or DLB), and α-synuclein-negative (n=2) groups. At baseline, MSA patients had significantly lower LN, brainstem, cerebellum, and MSAai values compared to HC, PD, DLB, and PAF (all p<0.001). The MSAai demonstrated strong group discrimination, correlated with UMSARS scores (ρ=-0.46, p=0.038), and predicted clinical progression (ρ=0.58, p=0.022). Longitudinally, reductions in LN, brainstem, and MSAai volumes were strongly associated with UMSARS progression. Cutaneous Phosphorylated Alpha-Synuclein Deposition Informs Autonomic Function in Individuals with Early-Stage Multiple System AtrophyPresenter: Leah Mann, PhD, Postdoctoral Research Fellow, Vanderbilt University Medical Center This poster presentation evaluated 17 participants from the bioMUSE Natural History Study who met criteria for clinically probable MSA. The study compared α-synuclein deposition in skin at baseline and 12 months later and examined associations between clinical measures and α-synuclein quantitation over the year. The analysis described the relationship between α-synuclein and autonomic function and revealed that cutaneous α-synuclein detection may serve as an effective diagnostic biomarker that can additionally track progression of MSA. Importantly, deposition of α-synuclein may confer insight into symptomatology, as higher α- synuclein concentration is associated with greater burden of orthostatic symptoms. In the analysis, 100% (17/17) of patients exhibited detection of phosphorylated α-synuclein by skin biopsy. Cutaneous α-synuclein deposition significantly increased (p = 0.042) from baseline (mean = 6.59 ± 4.37) to 12-month follow-up (mean = 7.71 ± 4.22). Deposition at 12-month follow-up was positively correlated with Orthostatic Hypotension Questionnaire (OHQ) and Composite Autonomic Symptom Score-31 (COMPASS-31) (OHQ: coefficient = 0.579, p = 0.015; COMPASS-31: coefficient = 0.560, p = 0.024). Presentations will be available on the Alterity Therapeutics website here. About ATH434 Alterity's lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. As an iron chaperone, it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2 open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA, and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA. About ATH434-201 Phase 2 Clinical Trial The ATH434-201 Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of 12 months treatment with ATH434 in patients with MSA. The study evaluated the efficacy, safety and pharmacokinetics of ATH434 as well as the effect of ATH434 on neuroimaging and protein biomarkers. Wearable sensors were employed to evaluate motor activities outside of the clinic. The study enrolled 77 adults who were randomly assigned to receive ATH434 50 mg or 75 mg twice daily or matching placebo. The data showed that, compared to placebo, ATH434 produced clinically and statistically significant improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. Additional efficacy assessments demonstrated improvement consistent with the positive UMSARS Part I findings including trends in improved motor performance on the Parkinson's Plus rating scale, the Clinical Global Impression of Severity Scale, and the Orthostatic Hypotension Symptom Assessment (a patient reported outcome). Wearable sensor data indicated that ATH434 also led to increased activity in an outpatient setting. Biomarkers were used to evaluate potential drug effect and target engagement relative to placebo. Both dose levels reduced iron accumulation in MSA affected brain regions with trends in preservation of brain volume. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Additional information on the Phase 2 trial can be found by Identifier: NCT05109091. About bioMUSE Biomarkers of progression in Multiple System Atrophy (bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a parkinsonian disorder without approved therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity's randomized ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently enrolling in the Phase 2 clinical trial. About Multiple System Atrophy Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the support cells of the central nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure.1 1Multiple System Atrophy | National Institute of Neurological Disorders and Stroke ( About Alterity Therapeutics Limited Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company's website at Authorisation & Additional informationThis announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited. Investor and Media Contacts: Australia Millie MacdonaldHead of Investor Relations and Business Developmentmmacdonald@ +61 468 304 742 Ana Luiza Harropwe-aualteritytherapeutics@ 452 510 255 +1 (415) 203-6386 Forward Looking Statements This press release contains "forward-looking statements" within the meaning of section 27A of the Securities Act of 1933 and section 21E of the Securities Exchange Act of 1934. The Company has tried to identify such forward-looking statements by use of such words as "expects," "intends," "hopes," "anticipates," "believes," "could," "may," "evidences" and "estimates," and other similar expressions, but these words are not the exclusive means of identifying such statements. Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are described in the sections titled 'Risk Factors' in the Company's filings with the SEC, including its most recent Annual Report on Form 20-F as well as reports on Form 6-K, including, but not limited to the following: statements relating to the Company's drug development program, including, but not limited to the initiation, progress and outcomes of clinical trials of the Company's drug development program, including, but not limited to, ATH434, and any other statements that are not historical facts. Such statements involve risks and uncertainties, including, but not limited to, those risks and uncertainties relating to the difficulties or delays in financing, development, testing, regulatory approval, production and marketing of the Company's drug components, including, but not limited to, ATH434, the ability of the Company to procure additional future sources of financing, unexpected adverse side effects or inadequate therapeutic efficacy of the Company's drug compounds, including, but not limited to, ATH434, that could slow or prevent products coming to market, the uncertainty of obtaining patent protection for the Company's intellectual property or trade secrets, the uncertainty of successfully enforcing the Company's patent rights and the uncertainty of the Company freedom to operate. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
07-05-2025
- Business
- Associated Press
Alterity Therapeutics Announces Multiple Oral and Poster Presentations to be Featured at the International MSA Congress
MELBOURNE, Australia and SAN FRANCISCO, May 07, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ('Alterity' or 'the Company'), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that multiple oral and poster presentations related to Alterity's clinical programs in Multiple System Atrophy (MSA) will be featured at the 2025 International MSA Congress taking place May 9 - 11, 2025 in Boston, MA, USA. The Congress is presented by Mission MSA, a non-profit organization dedicated to improving the quality of life and building hope for people affected by MSA through support services, educational resources, research funding, and community engagement. David Stamler, M.D., Chief Executive Officer of Alterity, commented, 'We are excited to play a prominent role at the MSA Congress which brings together a noteworthy group of physicians and scientists as well as patients and care partners, all of whom are focused on advancing MSA research and care. Mission MSA is one of the primary patient education and advocacy organizations for the disease in the U.S., and we are also proud to support the event as a sponsor. We look forward to presenting our double-blind Phase 2 data for ATH434 as well as our bioMUSE natural history study with our colleagues at Vanderbilt University Medical Center. Our clinical advancements in MSA continue to generate significant interest throughout the community, bolstered by the recent Fast Track Designation for ATH434 that was granted by the US FDA.' Type: Oral Presentation Session: Opening Plenary: Toward Disease Modifying Therapies of MSA Title: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy Presenter: David Stamler, M.D., Chief Executive Officer of Alterity Date/Time: Friday, May 9, 2025 at 9:10 AM EDT (USA) Type: Oral Presentation Session: Biomarkers in MSA: Imaging Title: MSA Atrophy Index (MSAai): A Quantitative Imaging Marker for Diagnosis and Monitoring of Multiple System Atrophy Presenter: Amy Brown, M.D., M.S., Assistant Professor, Movement Disorders Division, Department of Neurology, Vanderbilt University Medical Center Date/Time: Saturday, May 10, 2025 at 2:55 PM EDT Type: Poster Session Title: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy Presenter: David Stamler, M.D., Chief Executive Officer of Alterity Date/Time: Saturday, May 10, 2025 at 12:00 PM EDT Poster: #85 Type: Poster Session Title: Cutaneous Phosphorylated Alpha-Synuclein Deposition Informs Autonomic Function in Individuals with Early-Stage Multiple System Atrophy Presenter: Leah Mann, PhD, Postdoctoral Research Fellow, Vanderbilt University Medical Center Date/Time: Saturday, May 10, 2025 at 12:00 PM EDT Poster: #86 About ATH434 Alterity's lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. As an iron chaperone, it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2 open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA, and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA. About ATH434-201 Phase 2 Clinical Trial The ATH434-201 Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of 12 months treatment with ATH434 in patients with MSA. The study evaluated the efficacy, safety and pharmacokinetics of ATH434 as well as the effect of ATH434 on neuroimaging and protein biomarkers. Wearable sensors were employed to evaluate motor activities outside of the clinic. The study enrolled 77 adults who were randomly assigned to receive ATH434 50 mg or 75 mg twice daily or matching placebo. The data showed that, compared to placebo, ATH434 produced clinically and statistically significant improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. Additional efficacy assessments demonstrated improvement consistent with the positive UMSARS Part I findings including trends in improved motor performance on the Parkinson's Plus rating scale, the Clinical Global Impression of Severity Scale, and the Orthostatic Hypotension Symptom Assessment (a patient reported outcome). Wearable sensor data indicated that ATH434 also led to increased activity in an outpatient setting. Biomarkers were used to evaluate potential drug effect and target engagement relative to placebo. Both dose levels stabilized or reduced iron accumulation in MSA affected brain regions with trends in preservation of brain volume. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Additional information on the Phase 2 trial can be found by Identifier: NCT05109091 . About bioMUSE Biomarkers of progression in Multiple System Atrophy (bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a Parkinsonian disorder without approved therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity's randomized ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently enrolling in the Phase 2 clinical trial. About Multiple System Atrophy Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the support cells of the central nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure.1 1 Multiple System Atrophy | National Institute of Neurological Disorders and Stroke ( About Alterity Therapeutics Limited Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company's website at . Authorisation & Additional information This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited. Investor and Media Contacts: Australia Millie Macdonald Head of Investor Relations and Business Development [email protected] +61 468 304 742 Ana Luiza Harrop [email protected] +61 452 510 255 U.S. Remy Bernarda [email protected] +1 (415) 203-6386 Forward Looking Statements This press release contains 'forward-looking statements' within the meaning of section 27A of the Securities Act of 1933 and section 21E of the Securities Exchange Act of 1934. The Company has tried to identify such forward-looking statements by use of such words as 'expects,' 'intends,' 'hopes,' 'anticipates,' 'believes,' 'could,' 'may,' 'evidences' and 'estimates,' and other similar expressions, but these words are not the exclusive means of identifying such statements. Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are described in the sections titled 'Risk Factors' in the Company's filings with the SEC, including its most recent Annual Report on Form 20-F as well as reports on Form 6-K, including, but not limited to the following: statements relating to the Company's drug development program, including, but not limited to the initiation, progress and outcomes of clinical trials of the Company's drug development program, including, but not limited to, ATH434, and any other statements that are not historical facts. Such statements involve risks and uncertainties, including, but not limited to, those risks and uncertainties relating to the difficulties or delays in financing, development, testing, regulatory approval, production and marketing of the Company's drug components, including, but not limited to, ATH434, the ability of the Company to procure additional future sources of financing, unexpected adverse side effects or inadequate therapeutic efficacy of the Company's drug compounds, including, but not limited to, ATH434, that could slow or prevent products coming to market, the uncertainty of obtaining patent protection for the Company's intellectual property or trade secrets, the uncertainty of successfully enforcing the Company's patent rights and the uncertainty of the Company freedom to operate. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
