ATH434-201 Phase 2 baseline data confirm approach to target biomarkers for slowing disease progression
Presented promising nonclinical data on ATH434 in a primate model of Parkinson’s disease
Raised approximately A$5.25M to strengthen the balance sheet
Received an A$3.9M Research and Development Tax Incentive Refund
Cash balance on 31 March 2024 of A$18.3M
MELBOURNE, Australia and SAN FRANCISCO, April 30, 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 released its Appendix 4C Quarterly Cash Flow Report and update on company activities for the quarter ending 31 March 2024 (Q3 FY24).
“We are excited about the advancement of all of our programs during the third quarter of our fiscal year as we progress towards clinical data readouts from our Phase 2 trials,” said, David Stamler, M.D., Chief Executive Officer of Alterity. “Collectively, the data we presented from our clinical trials in Multiple System Atrophy (MSA) and the primate model of Parkinson’s disease continue to validate our approach of using biomarkers to monitor disease progression. Overall, these data improve our ability to predict clinical outcomes and increase our confidence level in our ongoing Phase 2 clinical trials in MSA. We remain on track to report preliminary data from our ATH434-202 study in the second quarter of this year, and topline results in ATH434-201 by January 2025.”
Alterity’s cash position on 31 March 2024 was A$18.3M with operating cash outflows for the quarter of A$2.6M. The company strengthened its balance sheet during the quarter by raising approximately A$5.25M.
In accordance with ASX Listing Rule 4.7C, payments made to related parties and their associates included in item 6.1 of the Appendix 4C incorporates directors’ fees, consulting fees, remuneration and superannuation at commercial rates.
On 6 February 2024, Alterity announced that an independent Data Monitoring Committee (DMC) completed its second prespecified review of unblinded clinical trial data from the ATH434-201 Phase 2 study. The DMC expressed no concerns about safety and recommended that the study continue as planned without modification. This recommendation is an important milestone as participants are able to safely tolerate ATH434 as their time on study increases.
In April 2024, a poster was presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, entitled, “A Phase 2 Study of ATH434, a Novel Inhibitor of α-Synuclein Aggregation, for the Treatment of Multiple System Atrophy”. The poster described the baseline characteristics for the 65 evaluable participants from the ATH434-201 with a focus on baseline fluid biomarkers, neuroimaging and clinical data. The participants met the strict criteria designed to confirm that participants were diagnosed with early-stage MSA and had a mean of two years of motor symptoms. The data showed increased iron in areas of pathology and elevated plasma Neurofilament Light Chain (NfL) levels at baseline that correlated significantly with disease severity.
The trial remains on track to complete in November 2024. The data from the trial will then be analyzed and the Company expects to report topline results by January 2025.
The ATH434-202 trial continues to enroll participants with more advanced MSA than in the 201 trial. A key aim of the 202 study is to assess the efficacy of ATH434 treatment on neuroimaging and protein biomarkers to evaluate target engagement, in addition to clinical measures, safety, and pharmacokinetics. While the 202 trial is also treating participants for 12-months, it has an open label design that will allow Alterity to perform interim analyses of biomarker and clinical data while the study is ongoing, providing a potential early indication of efficacy. The Company expects to report preliminary six-month data from the initial patients enrolled in the ATH434-202 trial in the second quarter of 2024.
ATH434fortheTreatmentofParkinson’sDisease
A poster was also presented at AAN entitled, “Effects of ATH434, a Clinical-Phase Small Molecule with Moderate Affinity for Iron, in Hemiparkinsonian Macaques”. The presentation showed that ATH434 can reduce Parkinsonism in a higher order animal, the monkey, with symptoms that closely parallel human disease. Importantly, the improvements in motor skills and general functioning that parallel human parkinsonism were associated with reductions in abnormal iron in affected brain regions. These favorable parkinsonian outcomes observed in each of the ATH434-treated monkeys were also associated with increased levels of striatal synaptophysin, a protein marker that reflects functional connections between neurons, suggesting functional recovery of nerve endings in this critical motor pathway. Taken together, the findings in this study increase the Company’s confidence in their approach in the ongoing Phase 2 program in MSA.
bioMUSENaturalHistoryStudy
The bioMUSE study continues to generate important data related to the understanding of MSA and its presentation as the disease progresses. The advancement of MSA is profoundly aggressive, highlighting the critical need for biomarkers to delineate its progression over time. Also at AAN, a poster was presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, entitled, “Neurofilament Light Chain and Clinical Progression in Early Multiple System Atrophy”. The poster described results from bioMUSE in which changes in clinical severity of 15 patients across a span of 12 months were compared with plasma biomarkers with a goal of establishing meaningful correlations. Importantly, the observational data suggest the fluid biomarker NfL may be a marker of disease modification in studies of MSA as it holds promise for measuring the extent of disease, tracking its progression, and forecasting the onset of clinical manifestations associated with MSA.
AboutAlterityTherapeuticsLimited
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&Additionalinformation This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
Thispressreleasecontains"forward-lookingstatements"withinthemeaningofsection27Aof theSecuritiesActof1933andsection21EoftheSecuritiesExchangeActof1934.TheCompany 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’sfilingswiththeSEC,includingitsmostrecentAnnualReportonForm20-Faswellas reports on Form 6-K, including, but not limited to the following: statements relating to the Company'sdrugdevelopmentprogram,including,butnotlimitedtotheinitiation,progressand 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 limitedto,ATH434,thatcouldslowor prevent productscoming to market, the uncertainty of obtaining patent protection for the Company's intellectual propertyortradesecrets,theuncertaintyofsuccessfullyenforcingtheCompany’spatentrights 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 speaksonly asofthe dateonwhich it is made. Weundertake no obligationtopubliclyupdateanyforward-lookingstatement,whetherwrittenororal,thatmay be made from time to time, whether as a result of new information, future developments or otherwise.
