Alterity Therapeutics Issues Shareholder Letter Highlighting Pipeline Advances and Key Upcoming Milestones
Rhea-AI Summary
Alterity Therapeutics (NASDAQ: ATHE) has issued a shareholder letter highlighting significant progress in 2024 and upcoming milestones for 2025. The company completed a 12-month, double-blind Phase 2 clinical trial of ATH434 in early-stage Multiple System Atrophy (MSA), with topline data expected in late January or early February.
Preliminary results from their open-label biomarker study in advanced MSA patients showed stable or improved clinical measures after 6-months of ATH434 treatment. The study demonstrated stable iron levels and brain volumes in clinical responders, along with reduced neuronal injury compared to untreated patients.
The company also presented preclinical data showing ATH434's potential in a primate model of Parkinson's disease. Their bioMUSE Natural History study, in collaboration with Vanderbilt University Medical Center, led to developing a novel imaging biomarker for assessing brain volume in MSA-affected regions.
Positive
- Completion of Phase 2 clinical trial for ATH434 in MSA with imminent topline data
- Positive preliminary results from open-label biomarker study showing clinical improvements
- Development of novel imaging biomarker for MSA through bioMUSE study
- Promising preclinical data for ATH434 in Parkinson's disease model
Negative
- None.
Insights
The shareholder letter outlines significant clinical developments for Alterity Therapeutics' lead candidate ATH434 in Multiple System Atrophy (MSA). Two key milestones stand out: completion of a 12-month Phase 2 clinical trial with topline data expected in early 2025 and preliminary results from an open-label biomarker study showing stable or improved clinical measures in advanced MSA patients.
The biomarker study results are particularly noteworthy, demonstrating stable iron levels and brain volumes in clinical responders, along with reduced neuronal injury compared to untreated patients. This provides important validation of ATH434's mechanism of action and potential efficacy. For a rare neurodegenerative disease like MSA with no current disease-modifying treatments, these early signals of biological activity could be significant for both patients and investors.
The expansion into Parkinson's disease through preclinical primate studies suggests broader therapeutic potential beyond MSA, significantly expanding the drug's commercial opportunity. The novel imaging biomarker development through the bioMUSE study also strengthens the company's clinical development toolkit, potentially enabling more precise measurement of treatment effects in future trials.
From a market perspective, Alterity's strategic focus on MSA represents a calculated entry into an underserved market. MSA is an orphan indication with no approved disease-modifying treatments, potentially allowing for expedited regulatory pathways and premium pricing if ATH434 proves successful. The dual Phase 2 readouts expected in 2025 represent major catalysts that could significantly impact the company's
The expansion into Parkinson's disease is strategically sound, as it represents a much larger market opportunity while leveraging the same mechanism of action. The collaboration with Vanderbilt University Medical Center adds credibility and demonstrates the company's ability to forge valuable academic partnerships. However, investors should note that as a small-cap biotech, Alterity will likely need additional funding to advance their programs through later-stage clinical development, particularly if they pursue both MSA and Parkinson's indications.
MELBOURNE, Australia and SAN FRANCISCO, Jan. 09, 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 issued a letter to shareholders.
Dear Valued Shareholders:
As we again begin a new year, I am filled with optimism for 2025 and what lies ahead for Alterity Therapeutics.
I want to express my sincere gratitude for your unwavering support and belief in our mission to develop disease-modifying therapies for those living with neurodegenerative diseases. Your investment in Alterity allows us to pursue groundbreaking research and bring hope to patients and families affected by these devastating conditions.
2024 was a year of significant progress for Alterity. Most prominently, we completed our 12-month, double-blind Phase 2 clinical trial of ATH434 in early-stage Multiple System Atrophy (MSA). This milestone leads us to a topline data readout expected in late January or early February. Last July, we were pleased to report encouraging preliminary results from our open-label biomarker study in individuals with more advanced MSA.
The preliminary results from the open-label study showed that individuals receiving 6-months treatment with ATH434 had stable or improved clinical measures and that this clinical benefit was supported by biomarker data: stable iron levels and brain volumes in clinical responders and reduced neuronal injury compared to untreated patients from our MSA natural history study. Taken together, these data provide strong support for the potential of ATH434 to slow the progression of this very aggressive disease.
In addition to our advancements in clinical studies, we also continue to generate compelling data in MSA and other neurological diseases. Last year, we presented promising preclinical data demonstrating the potential of ATH434 in a primate model of Parkinson's disease. Our bioMUSE Natural History study, in collaboration with Professor Daniel Claassen’s neuroimaging group at Vanderbilt University Medical Center, yielded valuable insights into MSA progression and led to the development of a novel imaging biomarker for assessing brain volume in regions affected by MSA.
This year promises to be pivotal for Alterity with topline data expected from both of our Phase 2 clinical trials in MSA. Our team remains steadfast in their dedication to advancing our research and development efforts and bringing innovative therapies to patients with neurodegenerative diseases.
Thank you for your continued interest and support and we look forward to keeping you updated on our progress.
David Stamler, M.D., Chief Executive Officer of Alterity.
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 information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
Investor and Media Contacts:
Australia
Ana Luiza Harrop
we-aualteritytherapeutics@we-worldwide.com
+61 452 510 255
U.S.
Remy Bernarda
remy.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.
FAQ
When will Alterity (ATHE) release Phase 2 topline data for ATH434 in MSA?
What were the results of ATHE's open-label biomarker study for ATH434?
What new developments did ATHE achieve in their bioMUSE Natural History study?
What additional therapeutic potential has ATHE shown for ATH434?
