– ATH434 is a Disease Modifying Drug Candidate Targeting Alpha-Synuclein and Iron in Parkinsonian Disorders –
– Topline Data Expected in Early 2025 –
MELBOURNE, Australia and SAN FRANCISCO, Dec. 04, 2024 (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 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. With the achievement of this milestone, topline results are expected to be reported in late January or early February 2025.
“We are very excited to announce that the last participant in our Phase 2 study has completed all clinical evaluations, the final milestone that starts the clock to reporting topline data in this rare neurodegenerative disease,” said, David Stamler, M.D., Chief Executive Officer of Alterity. “The completion of our ATH434-201 trial represents a major accomplishment for Alterity, and I would like to recognize the trial participants for their involvement in the study. I would also like to thank our clinical sites and our study team for their hard work and dedication in conducting the trial. Throughout the course of the trial, we have had tremendous interest from our clinical sites, doctors and patients around the globe as we seek a treatment that could potentially slow the progression of this devastating disease. With the last patient visit behind us, we can now focus our attention on cleaning and locking the database and reporting topline data early next year.”
About ATH434-201 Phase 2 Clinical Trial
The ATH434-201 Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will evaluate the effect of ATH434 treatment on neuroimaging and protein biomarkers to demonstrate target engagement and clinical endpoints to demonstrate efficacy, in addition to assessments of safety and pharmacokinetics. Selected biomarkers, such as brain iron and aggregating α-synuclein, are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors were also employed to evaluate motor activities that are important to patients with MSA. The study enrolled 77 adults who were randomly assigned to receive one of two dose levels of ATH434 or placebo. Participants received treatment for 12 months which will provide an opportunity to detect changes in efficacy endpoints to optimize design of a definitive Phase 3 study. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.
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. 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). ATH434 successfully completed Phase 1 studies demonstrating the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. ATH434 is currently being studied in two clinical trials: Study ATH434-201 is a randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA and Study ATH434-202 is an open-label Phase 2 Biomarker trial in patients with more advanced MSA. ATH434 has been granted Orphan drug designation for the treatment of MSA by the U.S. FDA and the European Commission.
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
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company’s lead asset, ATH434, has the potential to treat various Parkinsonian disorders and is currently being evaluated in two Phase 2 clinical trials in Multiple System Atrophy. Alterity also 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 web site at www.alteritytherapeutics.com.
Authorisation & Additional information This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
This press release contains "forward-looking statements" within the meaning of section 27A of the Securities Act of 1933 and section21EoftheSecuritiesExchangeActof1934.TheCompanyhastriedtoidentifysuchforward-lookingstatementsbyuse 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 facts.Suchstatementsinvolverisksanduncertainties,including,butnotlimitedto,thoserisksanduncertaintiesrelatingtothe 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.
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