Alterity Therapeutics Phase 2 Data Monitoring Committee Recommends Continuing Clinical Trial as Planned After Second Review
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Insights
The progression of the ATH434-201 Phase 2 clinical trial by Alterity Therapeutics is a pivotal moment for stakeholders, including patients, healthcare providers and investors. The trial is significant as it addresses multiple system atrophy (MSA), a rare and currently untreatable neurodegenerative disease. The involvement of an independent Data Monitoring Committee (DMC) provides an additional layer of scrutiny and validation, potentially enhancing the credibility of the study's outcomes. The continuation of the trial without concerns from the DMC suggests a positive safety profile and satisfactory trial conduct up to this point, which is encouraging for the future prospects of ATH434.
From a scientific standpoint, the double-blind, placebo-controlled design is the gold standard for clinical trials, ensuring that the results are reliable and free from bias. If ATH434 proves to be effective, it could become the first approved treatment for MSA, which would be a breakthrough in the field of neurology and could significantly improve the quality of life for patients suffering from this debilitating disease. The long-term implications for Alterity Therapeutics could include market exclusivity, potential partnerships and a strong position in the neurodegenerative treatment market.
The announcement that the ATH434-201 trial is on track and that top-line data is expected in January 2025 has direct implications for Alterity Therapeutics' financial outlook and stock performance. Investors will likely monitor this trial closely due to the high unmet medical need and the potential for ATH434 to capture a significant market share in the absence of approved treatments for MSA.
Given the rarity of the disease and the orphan drug designation that treatments like ATH434 may receive, there is potential for premium pricing and market exclusivity incentives, which could lead to substantial revenue streams for Alterity. However, investors should also consider the inherent risks of clinical trials, including the possibility of negative outcomes in efficacy or safety that could adversely affect the company's valuation. The timeline until January 2025 also introduces a holding period risk, where capital is tied up without any guarantee of return on investment.
It is also essential to consider the impact of trial costs on the company's financials. Clinical development is expensive and the company's ability to finance the trial to completion without diluting shareholder value is a critical factor that investors should evaluate.
Understanding the competitive landscape within the neurodegenerative disease market is crucial for assessing the potential impact of ATH434. Currently, there are limited options for MSA, which means a successful trial could position Alterity Therapeutics as a leader in this niche market. The rarity of MSA also implies that the patient population is small, which can affect the scalability of the drug's commercialization. However, orphan drugs often benefit from regulatory incentives and can command high prices due to the lack of alternatives.
Market dynamics, such as the entry of new competitors, advancements in alternative therapies and changes in healthcare policies, will also influence ATH434's market potential. The anticipation of trial results could lead to volatility in Alterity's stock as the market reacts to each incremental update. Market researchers will be keen to analyze the uptake of ATH434 post-approval, the effectiveness of Alterity's commercial strategy and the overall impact on the neurodegenerative treatment landscape.
- ATH434-201 Trial on Track to Complete in November 2024 –
- Top-Line Data Expected in January 2025 -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, Feb. 06, 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 an independent Data Monitoring Committee (DMC) has completed its second review of trial data and recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
The DMC conducted its second prespecified review of unblinded clinical data from study participants. The DMC expressed no concerns about safety and recommended that the study continue without modification. The plan for the DMC to review initial safety data has been cleared with the U.S. Food and Drug Administration.
“With accumulated data from our Phase 2 study, we are pleased to report that the Data Monitoring Committee has determined that there are no safety concerns and that the study can continue as planned,” said David Stamler, M.D., Chief Executive Officer of Alterity. “Participants in the trial are being dosed for 12 months; therefore, this recommendation is an important milestone as participants able to safely tolerate ATH434 as their time on study increases. We remain on track to complete the study in November 2024 and report top-line data in January 2025.”
The ATH434-201 Phase 2 clinical trial is evaluating 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 have also been 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 will receive 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
1Multiple System Atrophy | National Institute of Neurological Disorders and Stroke (nih.gov)
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. Alterity also has a broad drug discovery platform generating patentable chemical compounds to intercede in disease processes. 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
Hannah Howlett
we-aualteritytherapeutics@we-worldwide.com
+61 450 648 064
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
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