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 30 June 2024 (Q4 FY24).
“We have made great strides over the last two
months with the positive interim data readout from our ATH434-202
Phase 2 clinical trial and the important observations from our
bioMUSE Natural History Study that continue to guide development of
ATH434,” said David Stamler, M.D., Chief Executive Officer of
Alterity. “I am very encouraged by the results from our 202 study
in patients with advanced Multiple System Atrophy (MSA) where we
saw favorable clinical and biomarker outcomes in some patients
suggesting that ATH434 has the potential to modify the course of
this devastating condition. We were also very pleased to see that
the clinical responders had biomarker evidence of stable disease as
this provides an objective indication of potential efficacy.”
“Our bioMUSE study continues to provide valuable
information to inform our patient selection criteria and choose
endpoints for our Phase 2 clinical trials. This observational study
has allowed us to monitor the progression of MSA in earlier stage
patients and further characterize this devastating disease. Working
with our colleagues at Vanderbilt University, we have employed
novel MRI technology and machine learning to precisely analyze
brain iron content and brain volumes in these patients over time.
The results from the study have guided us to modify our endpoints
in the ATH434-202 study. The data from our 202 and bioMUSE studies
increases my overall confidence in the ATH434 development program,”
concluded Dr. Stamler.
Alterity’s cash position on 30 June 2024 was
A$12.6M with operating cash outflows for the quarter of A$5.6M.
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.
Operational Activities
ATH434–201: Randomized, Double-Blind Phase 2
Clinical Trial in Early-State MSA
On 8 May 2024, Alterity announced that an
independent Data Monitoring Committee (DMC) completed its third
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, important new data on ATH434 was
presented at the World Orphan Drug Congress in a poster
presentation, entitled, “Biophysical Characteristics of ATH434, a
Unique Iron-Targeting Drug for Treating Friedreich’s Ataxia.” The
study evaluated the ability of ATH434 to target the toxic form of
iron that drives the pathology of Friedreich’s Ataxia, a rare
neurodegenerative disease that affects young children to young
adults. The investigation provides important insights into the
mechanism of action of ATH434, namely that it selectively targets
the labile iron implicated in the pathology of important
neurodegenerative diseases. In this way, ATH434 behaves like a
chaperone to redistribute iron within the body.
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
strict selection criteria designed to confirm they had early-stage
MSA. Overall, the participants had a mean duration of motor
symptoms of two years. 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.
ATH434–202: Open-label, Biomarker Phase 2
Clinical Trial in More Advanced MSA
Subsequent to the quarter end, on 17 July 2024,
Alterity reported positive interim data from the ATH434-202 trial
in participants with advanced MSA. The interim analysis included
clinical and biomarker data on 7 participants treated with ATH434
for 6 months and neuroimaging data on 3 participants who were
treated for 12 months. After 6 months of treatment, 43% of
participants showed improvement on the UMSARS1, indicating reduced
disability on activities of daily living. Over the same period, 29%
of participants had stable or improved neurological symptoms
(clinical responders) as assessed by the global impression of
change by both the treating physician and the patient. Importantly,
the clinical responders on average had reduced accumulation of iron
on MRI in the substantia nigra, putamen and globus pallidus and
stable levels of NFL, a marker of axonal injury, when compared to
participants who declined.
bioMUSE Natural History Study
On 30 May 2024 Alterity hosted a webinar to
discuss data from the bioMUSE Natural History Study. The goal of
the observational bioMUSE study is to optimize patient selection
and choose endpoints for the Company’s Phase 2 clinical trials.
This study enrolled 21 individuals who were observed for 12 months
to characterize early-stage MSA in terms of various biomarkers. In
particular, the focus is on brain iron, brain volume, and the
pathology in glial support cells. Utilizing novel MRI technology,
Alterity’s partners at Vanderbilt University have optimized
specialized MRI methods, including machine learning (a form of
artificial intelligence), to establish standardized methods to
analyze brain iron and brain volumes with precision. Importantly,
they developed a new, novel imaging biomarker to assess brain
volume in MSA affected regions. The bioMUSE data showed a
statistically significant increase in iron over 12 months in the
substantia nigra, and statistically significant decreases in brain
volume observed in affected regions at 12 months.
Also at AAN, a poster was presented at the 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 used as a marker of disease severity 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.
ATH434 for the Treatment of Parkinson’s
Disease
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 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.
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’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 informationThis
announcement was authorized by David Stamler, CEO of Alterity
Therapeutics Limited.
Investor and Media Contacts:
AustraliaHannah
Howlettwe-aualteritytherapeutics@we-worldwide.com+61 450 648
064
U.S.Remy Bernardaremy.bernarda@iradvisory.com
+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.
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