ATH434 / Alterity Therap - LARVOL DELTA

A Phase 1, 2-Part, Adaptive Design, Drug-Drug Interaction Study to Assess the Effect of Acid Reducing Agents on the Pharmacokinetics of ATH434 in Healthy Subjects (ANZCTR) - P1 | N=12 | Completed | Sponsor: Alterity Therapeutics Limited

New P1 trial • CNS Disorders • Movement Disorders • Multiple System Atrophy

Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France (clinicaltrials.gov) - P2 | N=5 | Not yet recruiting | Sponsor: Alterity Therapeutics | Initiation date: Jul 2026 ➔ Oct 2026

Trial initiation date • CNS Disorders • Movement Disorders • Multiple System Atrophy

Results from a Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in MSA using CSF NfL as a Covariate (MDS Congress 2026) - No abstract available

Clinical • CNS Disorders • CSF NfL

Alterity Therapeutics has secured a new US Composition of Matter patent covering its lead drug candidate ATH434, extending intellectual property (IP) protection for the potential neurodegenerative disease treatment to at least 2045. (The Australian) - "It also supports future development opportunities for ATH434 in Parkinson’s disease and other neurodegenerative disorders where iron dysregulation and protein aggregation are implicated."

Patent • Multiple System Atrophy • Parkinson's Disease

Alterity Therapeutics Announces Granting of New U.S. Composition of Matter Patent for ATH434 (GlobeNewswire) - "ATH434 is an oral agent designed to treat the underlying pathology of neurodegenerative diseases such as Multiple System Atrophy (MSA), Parkinson’s disease and related disorders. The newly granted patent represents a significant intellectual property milestone for Alterity and strengthens ATH434’s long-term commercial potential as the Company prepares to initiate Phase 3 trial activities in MSA by year-end 2026....The patent extends protection for ATH434 to at least 2045..."

New P3 trial • Patent • Multiple System Atrophy • Parkinson's Disease

Appendix 4C – Q4 FY26 Quarterly Cash Flow Report & Corporate Update (Alterity Therapeutics Press Release) - "FDA agreed that a single pivotal Phase 3 trial plus confirmatory evidence could support an approval of ATH434 for the treatment of MSA; Pivotal Phase 3 trial activities on track to initiate by year-end 2026...The study is expected to enroll approximately 200 patients who will be randomized 1:1 to ATH434 50 mg or matching placebo treatment twice daily for 12 months. Alterity plans to offer an open-label extension to participants who complete the Phase 3 trial, both to continue their treatment and to enhance the safety database for ATH434."

New P3 trial • CNS Disorders • Multiple System Atrophy

Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France (clinicaltrials.gov) - P2 | N=5 | Not yet recruiting | Sponsor: Alterity Therapeutics

New P2 trial • CNS Disorders • Movement Disorders • Multiple System Atrophy

Alterity Therapeutics (NASDAQ: ATHE) receives A$3.98m R&D tax refund (StockTitan.net) - "The incentive provides a refundable tax offset of up to 43.5% for eligible R&D activities. Alterity states that this cash refund will provide important funding to continue developing its clinical programs, including its lead candidate ATH434 for Multiple System Atrophy, where it has completed Phase 2 trials and is preparing a Phase 3 pivotal study."

Financing • Multiple System Atrophy

The FDA has confirmed End-of-Phase 2 outcomes for ATH434 in Multiple System Atrophy and agreed that one pivotal Phase 3 trial plus confirmatory evidence could support approval. (StockTitan.net) - "The planned Phase 3 will enroll about 200 patients on ATH434 50 mg or placebo for 12 months, using the 11‑item UMSARS Part I functional scale as the primary endpoint, with several clinically meaningful secondary measures. The FDA also indicated the anticipated safety database size is reasonable."

FDA event • New P3 trial • Multiple System Atrophy

Alterity Therapeutics…announced the successful outcome of its End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), achieving alignment on the key elements of the Company's registrational Phase 3 program for ATH434 in Multiple System Atrophy (The Manila Times) - "...FDA agreed with the proposed Phase 3 trial design, including the study population, treatment duration and primary endpoint ‒ the 11-item UMSARS Part I1 rating scale. The FDA also concurred with Alterity’s proposed Phase 3 dose regimen of ATH434 50 mg twice daily, which in Phase 2 achieved clinically and statistically significant efficacy on the 11-item UMSARS I, with 48% slowing of disease progression compared to placebo...we finalize the protocol and prepare to initiate trial activities by year-end 2026."

Clinical protocol • New P3 trial • Multiple System Atrophy

A Study to Assess the Effect of Acid-Reducing Medicines on ATH434 in Healthy Volunteers (ANZCTR) - P1 | N=12 | Recruiting | Sponsor: Alterity Therapeutics Limited | Not yet recruiting ➔ Recruiting | Initiation date: Mar 2026

Enrollment open • Trial initiation date • CNS Disorders • Movement Disorders • Multiple System Atrophy

Results from a Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in MSA using CSF NfL as a Covariate (The Manila Times) - "ATH434 slowed functional decline in MSA: Significant effect at 50 mg BID (−4.0 points, p=0.035; ~48% slowing) and a consistent directional trend at 75 mg BID. Combined active arms significantly slowed UMSARS2 I progression vs placebo (p=0.047); CSF NfL is a meaningful prognostic covariate: Higher baseline CSF NfL predicted greater UMSARS-I worsening (β=0.90, p=0.033). Adjusting for it strengthened detection of treatment effects and supports CSF NfL for stratification in future trials."

P2 data • Multiple System Atrophy

ATH434 Clinical Update and Phase 3 Planning (The Manila Times) - "The biomarker data presented by Dr. Stamler included QSM MRI data from the ATH434-201, demonstrating that ATH434 decouples iron accumulation from clinical progression thus supporting its role as an iron chaperone. New data on the impact of ATH434 on swallowing were also presented, demonstrating that both doses of ATH434 reduce the progression of this key MSA symptom. Swallowing impairment was assessed with the 15-item Swallowing Disturbance Questionnaire (SDQ), a validated patient reported outcome. Over 52 weeks treatment, placebo patients worsened by a mean adjusted increase score of 8.5 points as compared to an increase of 1.2 and 5.0 points for the 50 mg and 75 mg groups, respectively, with the mean adjusted difference at 50 mg achieving statistical significance (p=0.003)."

Biomarker • New P3 trial • P2 data • Multiple System Atrophy

Alterity Therapeutics Announces Publication Demonstrating the Utility of Quantitative MRI as a Biomarker for Multiple System Atrophy (Yahoo Finance) - "The study analyzed high-resolution structural and QSM MRI data from 10 MSA patients followed prospectively for 12 months, and cross-sectional data from 28 MSA patients, 43 PD patients, and 23 age-matched healthy controls...MSA patients showed significantly higher iron content in the lentiform nucleus — comprising the globus pallidus and putamen — versus both healthy controls and PD (all p<0.05), with the most pronounced effect in the globus pallidus...Iron content in the globus pallidus distinguished MSA from PD with moderate-to-good accuracy (AUC = 0.76–0.79), with comparable performance in the early-stage subgroup...Alterity remains on track to hold its End-of-Phase 2 meeting with the FDA in mid-2026, the next key step toward initiation of a pivotal Phase 3 trial in MSA."

New P3 trial • Real-world • Multiple System Atrophy • Parkinson's Disease

Open-Label Extension Study to Provide Access to ATH434 in Patients with Multiple System Atrophy (clinicaltrialsregister.eu) - P1/2 | N=7 | Not yet recruiting | Sponsor: Alterity Therapeutics Limited

New P1/2 trial • CNS Disorders • Movement Disorders • Multiple System Atrophy

Alterity Therapeutics to Deliver Presentations at Multiple Medical Conferences in May 2026 (Alterity Therapeutics Press Release)

New P3 trial • P2 data • Multiple System Atrophy

Alterity Therapeutics Presents New Analysis of ATH434 Phase 2 Trial Data in Late Breaking Science Session of the American Academy of Neurology (The Manila Times) - "Presentation reinforces ATH434's clinical profile ahead of Phase 3 engagement with regulators...Consistent with prior data, ATH434 slowed disease progression on the MuSyCA assessment with a treatment effect of −1.9 (75 mg dose) to −4.0 points (50 mg dose, p=0.034, relative treatment effect 41%) at Week 52. In contrast, when utilizing a MMRM3 statistical analysis, ATH434 slowed disease progression on the modified UMSARS I versus placebo by -3.1 points at 75 mg (relative treatment effect of 35%) and -4.7 points at 50 mg (relative treatment effect of 53%, p=0.029).​"

New P3 trial • P2 data • Multiple System Atrophy

ATH434 Demonstrates Disease-Modifying Signal in Multiple System Atrophy Using the MuSyCA Composite Scale (AAN 2026) - "Reference: Kaufmann H, Palma JA, Millar Vernetti P, et al for the MuSyCA Working Group. Multiple System Atrophy Combined Outcome Assessment (MuSyCA): Process, format, and validation plan (Clinical Autonomic Research, in Press)"

Late-breaking abstract • CNS Disorders • Movement Disorders • Multiple System Atrophy • Plasma NfL

Alterity Therapeutics Announces Late-Breaker Oral Presentation at the American Academy of Neurology Annual Meeting (GlobeNewswire) - "Type: Oral Presentation; Title: ATH434 Demonstrates Disease-Modifying Signal in Multiple System Atrophy Using the MuSyCA Composite Scale; Date/Time: Tuesday, April 21, 2026, 6:21 PM CT."

Clinical data • Late-breaking abstract • Multiple System Atrophy

Post-Trial Access to ATH434 for Patients With Multiple System Atrophy (clinicaltrials.gov) - P=N/A | N=0 | Available | Sponsor: Alterity Therapeutics

New trial • CNS Disorders • Movement Disorders • Multiple System Atrophy

Alterity Therapeutics…announced it has received positive regulatory feedback following a Type C Meeting with the U.S. Food and Drug Administration (FDA) regarding its planned Phase 3 development program for ATH434 in Multiple System Atrophy (MSA) (GlobeNewswire) - "The Type C Meeting is part of a multidisciplinary strategy to seek alignment with the FDA on readiness to initiate a Phase 3 pivotal trial in MSA. Alterity received written feedback supporting its plans related to the clinical pharmacology and non-clinical development elements of the program...'Today’s favorable outcome sets the stage for our future discussions, culminating in an End-of-Phase 2 meeting that remains on track for mid-year 2026'."

FDA event • Multiple System Atrophy

A Phase 1 Study to Evaluate a Tablet Formulation of PBT434 in Healthy Volunteers (ANZCTR) - P1 | N=42 | Completed | Sponsor: Alterity Therapeutics Limited | Recruiting ➔ Completed

Trial completion • CNS Disorders • Movement Disorders • Multiple System Atrophy • Parkinson's Disease

A Study to Assess the Effect of Acid-Reducing Medicines on ATH434 in Healthy Volunteers (ANZCTR) - P1 | N=12 | Not yet recruiting | Sponsor: Alterity Therapeutics Limited

New P1 trial • CNS Disorders • Movement Disorders • Multiple System Atrophy

Advancing Toward Phase 3 and Potential Future Commercialization (The Manila Times) - "Following the successful Phase 2 program, we are now focused on aligning with the FDA on key clinical and regulatory considerations for Phase 3 and expect to hold our End-of-Phase 2 meeting in mid-2026. The goal of this meeting is to finalise the Phase 3 design, including patient selection, efficacy and safety endpoints, and the statistical framework for analyzing the results."

Clinical protocol • New P3 trial • Multiple System Atrophy

Alterity Therapeutics Presents Promising Impact of ATH434 on Orthostatic Hypotension and Disease Progression in MSA at the 36th International Symposium on the Autonomic Nervous System (GlobeNewswire) - "'These data are very important as we design our Phase 3 protocol and prepare for our upcoming FDA interactions.'....In the trial, severe OH is defined as a sustained decrease in systolic blood pressure > 30mm Hg after three minutes of standing. Baseline data from the trial revealed that severe OH was substantially higher in the 75 mg dose group at 29.2% of participants, versus 4% in the 50 mg arm and 4.5% in the placebo arm. When orthostatic blood pressure change was used as a covariate in the analysis of the UMSARS I at 52 weeks, the efficacy signal in 75 mg dose group strengthened from -2.4 to -2.8 points, improving the relative treatment effect from 30% to 35%. This baseline difference in severe OH largely explains the different responses in the 50 mg and 75 mg treatment groups."

New P3 trial • P2 data • Hypotension • Multiple System Atrophy