Alterity Therapeutics: Appendix 4C – Q1 FY26 Quarterly Cash Flow Report

Rhea-AI Summary

Alterity Therapeutics (NASDAQ: ATHE) released its Q1 FY26 Appendix 4C and program update for the quarter ended 30 September 2025. Key items: cash A$54.56M and operating cash outflows of A$5.34M for the quarter; A$20M gross raised via a strategic placement.

Clinical highlights include a strengthened efficacy signal for ATH434 75 mg (−2.8 UMSARS I points; 35% relative effect at 52 weeks), positive open‑label ATH434‑202 results with brain volume preservation, biomarker target engagement, and peer‑reviewed neuroimaging publication. An independent commercial assessment estimates a USD $2.4B peak sales opportunity in MSA. FDA Type C meetings and an End‑of‑Phase 2 meeting are planned, with the EOP2 expected mid‑2026.

Positive

• ATH434 75 mg showed −2.8 UMSARS I points at 52 weeks (35% relative effect)
• Preservation of brain volume observed in ATH434‑202 advanced MSA cohort
• Cash balance of A$54.56M as of 30 Sep 2025
• Raised A$20M gross proceeds in strategic placement
• Independent commercial assessment: USD $2.4B potential peak sales in MSA

Negative

• Operating cash outflow of A$5.34M in Q1 FY26
• Regulatory path requires sequential FDA Type C meetings and CMC/nonclinical data before Phase 3
• End‑of‑Phase 2 meeting not expected until mid‑2026, delaying Phase 3 start

Insights

Clinical and Commercial Analyst

Positive Phase 2 efficacy signals, regulatory engagement set path to Phase 3; cash runway extended by recent placement.

At a basic level, the company reports strengthened clinical effects for ATH434 at the 75 mg dose with a −2.8 point change at 52 weeks and a reported relative treatment effect of 35%, concordant open‑label data in advanced MSA, biomarker engagement, and a published imaging marker supporting disease measurement. These findings, combined with Fast Track status and planned staged meetings with the FDA culminating in an End‑of‑Phase‑2 meeting in mid‑year 2026, create a clear regulatory path to discuss Phase 3 design and required nonclinical/CMC data.

Key dependencies and risks include agreement with the FDA on nonclinical and CMC packages before a productive End‑of‑Phase‑2 meeting, and confirmation that the observed effect and safety profile replicate in larger studies. Financially, the company reported cash of A$54.56M as of 30 September 2025, operating outflows of A$5.34M for the quarter, and raised A$20M gross via placement; these items bear directly on near‑term ability to fund regulatory interactions and trial start‑up.

Watch for three concrete milestones: (1) completion of the FDA Type C meeting sequence and the End‑of‑Phase‑2 meeting in mid‑year 2026, (2) any detailed Phase 3 protocol elements agreed with FDA, and (3) replication of efficacy/safety in further datasets or interim analyses. The company’s independent commercial assessment citing a USD $2.4 billion peak sales opportunity frames upside if approval occurs, but commercial potential depends on confirmatory Phase 3 results and regulatory acceptance of endpoints.

Highlights

• New analysis of ATH434-201 double blind trial strengthens efficacy signal at high dose level
• Positive data from ATH434-202 open-label trial demonstrates similar treatment effect in advanced MSA as observed in earlier stage patients in double-blind study
• Independent commercial assessment indicates USD $2.4 billion dollar potential worldwide peak sales opportunity in MSA for ATH434
• Cash balance on 30 September 2025 of A$54.56M
  
  

MELBOURNE, Australia and SAN FRANCISCO, Oct. 31, 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 released its Appendix 4C Quarterly Cash Flow Report and update on company activities for the quarter ending 30 September 2025 (Q1 FY26).

“As we near the end of the calendar year, I am very proud of all we have accomplished in 2025, led by our compelling clinical results in Multiple System Atrophy (MSA), and I am excited about the prospect of delivering ATH434 to the MSA community,” said David Stamler, M.D., Chief Executive Officer of Alterity.

Dr. Stamler continued, “During the recent quarter, we reported positive results from our trial in advanced MSA, published important neuroimaging findings from our natural history study, and completed our commercial assessment indicating a potential market opportunity of approximately USD$2.4 billion dollars. The totality of the data from our combined studies and interest from the medical and scientific community continues to give us great confidence in the potential of ATH434 as a first-in-class, disease-modifying therapy for MSA.”

“We are actively engaging with the U.S. Food and Drug Administration (FDA) on ATH434 to conduct a series of meetings to discuss emerging nonclinical and chemistry and manufacturing data required for Phase 3 conduct. Reaching agreement on these elements with the FDA is critical for ensuring a productive End-of-Phase 2 meeting to enable us to initiate a Phase 3 trial in MSA,” concluded Dr. Stamler.

Alterity’s cash position on 30 September 2025 was A$54.56M with operating cash outflows for the quarter of A$5.34M. In accordance with ASX Listing Rule 4.7C, payments of A$108k 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 Highlights

ATH434 Regulatory Update

Alterity has continued to generate the required data and analyses that are needed to engage regulatory authorities about the path forward for ATH434. With respect to the U.S. Food and Drug Administration (FDA), this process necessitates a proactive, staged approach.

The Fast Track Designation granted to ATH434 affords Alterity the opportunity to engage with the FDA in a series of Type C meetings related to the nonclinical and the chemistry, manufacturing, and controls (CMC) data necessary to support Phase 3. Following these meetings, an End-of-Phase 2 meeting will be held to align with the FDA on all elements of the Phase 3 program. Conducting these meetings in sequence allows Alterity to focus the End-of-Phase 2 meeting on the clinical development topics, including the Phase 3 protocol, and data requirements to commence the Phase 3 study. This series of meetings is expected to occur over the next several months with the End-of-Phase 2 meeting in mid-year 2026.

ATH434–201: Randomized, Double-Blind, Placebo Controlled Phase 2 Clinical Trial in MSA

Subsequent to the end of the quarter, Alterity presented a new analysis of the modified USMARS I¹ data from the ATH434-201 trial at the International Congress of Parkinson’s Disease and Movement Disorders meeting. The analysis, which incorporated baseline orthostatic blood pressure change as a covariate, led to a strengthened efficacy signal in the 75 mg dose group at 52 weeks of −2.8 points, for a relative treatment effect of 35%. The baseline differences in the rate of severe orthostatic hypotension (OH)² largely explains the different responses in 50 mg and 75 mg treatment groups. In addition, ATH434 demonstrated a beneficial effect on OH symptoms as assessed with the OH Symptom Assessment, a patient reported outcome. On this scale, placebo patients worsened on average by approximately 6 points over 52 weeks whereas the 50 mg and 75 mg groups were stable over the same period.

Multiple presentations were delivered on the positive results from the ATH434-201 trial:

• September 2025 – American Neurological Association (ANA), Title: “ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy”
• October 2025 - International Congress of Parkinson’s Disease and Movement Disorders (MDS), Title of Oral Platform Presentation: “ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy”
• October 2025 – MDS, Title: “Relationship Between Alpha-Synuclein Aggregation Profiles, Imaging Biomarkers, and Disease Severity in a Phase 2 Study of ATH434 in MSA”
• October 2025 – MDS, Title: “Differences Between Clinical and Imaging Phenotypes in Phase 2 Study of ATH434 in Multiple System Atrophy”
  
  

ATH434–202: Open-label, Biomarker Phase 2 Clinical Trial in Advanced MSA

In July 2025, Alterity announced positive data from the ATH434-202 open-label Phase 2 clinical trial in more advanced MSA than was studied in the double-blind Phase 2 trial. A key objective of the study was met as the 75 mg dose in this study demonstrating comparable efficacy to that observed in the ATH434-201 double-blind study, including the key efficacy endpoint of UMSARS I and preservation of brain volume. Importantly, biomarkers demonstrated target engagement and a safety profile that was comparable to prior studies. Overall, the Phase 2 studies confirmed ATH434’s favorable profile and provided further evidence that its mechanism of action has utility in addressing the underlying pathology of disease.

bioMUSE Natural History Study

In July 2025, the Company announced publication of a study in conjunction with researchers at Vanderbilt University Medical Center on an innovative neuroimaging measure developed in Alterity’s Biomarkers of Progression in Multiple System Atrophy (bioMUSE) Natural History Study. The publication, entitled “The MSA Atrophy Index (MSA-AI): An Imaging Marker for Diagnosis and Clinical Progression in Multiple System Atrophy”, was featured in the peer-reviewed journal Annals of Clinical and Translational Neurology. Development of the MSA Atrophy Index can enhance the understanding of MSA progression and provide support for using brain atrophy markers for diagnosis and evaluation of disease-modifying therapies.

Corporate Activities

Alterity continues to engage with the investment community with participation in the Bioshares Biotech Summit and Biotech Showcase events in Australia, as well as a panel presentation focused on neurodegenerative diseases at the Maxim Growth Summit Healthcare Day in the U.S.

During the period, Alterity completed an independent commercial assessment of ATH434 in MSA that resulted in a potential worldwide peak sales opportunity of USD$2.4 billion dollars, if approved. Physicians surveyed noted the importance of inhibiting α‐synuclein aggregation to address the underlying pathology of disease as addressed by the targeted mechanism of action of ATH434. The key driver of physician interest in ATH434 was the Phase 2 data that demonstrated a slowing of disease progression and stabilization of orthostatic hypotension, leading more than 70% of the neurologists surveyed being “extremely likely” or “very likely” to prescribe ATH434.

During the period, Alterity strengthened its balance sheet with a total of A$20M raised in gross proceeds from a strategic placement, led by high-quality healthcare-focused funds, to advance its programs.

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 is initially focused on developing disease modifying therapies in Parkinson’s disease and related disorders. Alterity has demonstrated clinically meaningful efficacy for its lead asset, ATH434, in a randomized, double-blind, placebo-controlled Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 recently reported positive data in its open label Phase 2 clinical trial in advanced MSA. In addition, Alterity 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 website at www.alteritytherapeutics.com.

References:

¹ Unified MSA Rating Scale, Part I (historical review) assess activities of daily living. Domains assessed include speech, swallowing, handwriting, cutting food/handling utensils, dressing, hygiene, walking, falling, orthostatic symptoms, urinary function, sexual function and bowel function.
² Orthostatic hypotension is a form of low blood pressure that might cause dizziness, lightheadedness or fainting when rising from sitting or lying down. Source: Mayo Clinic.

Authorisation & Additional information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.

Contacts:

Investors
Remy Bernarda
Investor Relations Advisory Solutions
ir@alteritytx.com
+1 (415) 203-6386

Media
Casey McDonald
Tiberend Strategic Advisors, Inc.
cmcdonald@tiberend.com
+1 (646) 577-8520

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

What cash position did Alterity (NASDAQ: ATHE) report on 30 September 2025?

Alterity reported a cash balance of A$54.56M on 30 September 2025.

How did ATH434 perform in the ATH434‑201 Phase 2 trial at the 75 mg dose?

A new analysis showed a −2.8 point change in UMSARS I at 52 weeks for 75 mg, a 35% relative treatment effect.

What were the key results from the ATH434‑202 open‑label study in advanced MSA?

ATH434‑202 reported comparable efficacy to ATH434‑201, preservation of brain volume, target engagement biomarkers, and a consistent safety profile.

What is the expected regulatory timeline for ATH434 Phase 3 discussions with the FDA?

Alterity expects a series of FDA Type C meetings on nonclinical and CMC data, followed by an End‑of‑Phase 2 meeting mid‑2026.

How large is the commercial opportunity estimated for ATH434 in MSA?

An independent commercial assessment estimated a potential worldwide peak sales opportunity of USD $2.4 billion if approved.

How much did Alterity raise in the recent strategic placement and what is the use?

Alterity raised A$20M in gross proceeds from a strategic placement to advance its ATH434 programs.