Announcement PDF MND Extension Study Begins Statistical Review Indicates Survival Benefit Highlights: • First patient dosed in the 12-month Open-Label Extension study for patients with MND/ALS • All 12 patients from the Phase 1 MEND Study are expected to be enrolled, meeting the minimum requirement of being able to swallow • Statistical survival estimations based on comparisons to the PRO-ACT historical MND/ALS database, points to the probability that all 12 patients treated with monepantel being alive today being less than 1 in 1,000 14 February 2024 – Perth, Australia: PharmAust Limited (ASX: PAA & PAAOA) (“PharmAust” or “the Company”), a clinical-stage biotechnology company, is pleased to announce that the first patient has been dosed in the Open-Label Extension (OLE) study at Calvary Health Care Bethlehem, Melbourne. The OLE study will investigate the long-term safety, tolerability, and efficacy of monepantel (MPL) in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS) who previously completed the Phase 1 MEND Study. In December 2023, the Company announced the completion of the Phase 1 MEND study, involving two cohorts of six patients, each progressively receiving higher dose levels of MPL in a staggered design approach over time. All 12 patients have elected to continue treatment with MPL through a compassionate-use program and are now willing and able to participate in the 12-month OLE study. Patients will receive a daily dose of 10 mg/kg body weight of MPL for an additional 12 months during the OLE study, to further test if MPL will safely reduce diseaseassociated protein accumulation in motor neurons and provide therapeutic benefits for patients with MND/ALS. The OLE study involves two sites in Australia, Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers and Macquarie University, led by Professor Dominic Rowe. The study has been registered on the ClinicalTrials.gov (https://clinicaltrials.gov/study/NCT06177431). Calvary Health Care Bethlehem Principal Investigator Associate Professor Susan Mathers commented: “We are very grateful to all of our participants and their families. Everyone is keen to join the OLE Study, working with us to understand the longer-term effects and possible benefits of monepantel in MND/ALS.” Survival estimations / statistical review To assess the likelihood of a 100% survival rate among the 12 Phase 1 MEND patients without treatment, statistical consultant specialists and PharmAust partner Berry Consultants (Berry) utilises baseline survival rates derived from historical control data within the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database1. After adjusting for differing diagnosis durations, Berry’s analysis involved comparing patients in the PRO-ACT database with similar characteristics to those in PharmAust’s Phase 1 study. Berry’s conservative sensitivity analyses identified the one-year study survival rate estimate of 67.7% with a 95% Confidence Interval. Considering differential diagnosis durations, the probability estimates of all 12 Phase 1 MEND patients surviving today without treatment are less than 0.1% (less than 1 in 1,000). The PRO-ACT database is the largest publicly available repository of merged ALS clinical study data. Clinical study data were pooled from 16 completed Phase 2/3 ALS/MND clinical studies and one observational study. Over 8 million de-identified longitudinally collected data points from more than 8,600 persons with ALS were standardised across studies and merged to create the PRO-ACT database. This database includes demographics, family histories, and longitudinal clinical and laboratory data. PharmAust Limited ABN 35 094 006 023. Suite 116, 1 Kyle Way, Claremont, Western Australia Tel: 08 9202 6814 Fax: 08 9467 6111 www.pharmaust.com PharmAust Chief Executive Officer Dr Michael Thurn commented: “Initiation of the OLE study is a significant milestone for PharmAust and the 12 patients who began their treatment journey with monepantel in October 2022. It is remarkable and satisfying to know that all 12 patients are still alive and capable of participating in this important extension study. The survival statistics based on these 12 patients are extremely encouraging for the company and the wider patient population with MND/ALS. This provides an exciting backdrop ahead of the release of the top-line data from the recently completed Phase 1 MEND study, due by the end of February 2024.” The Board authorises this announcement. Enquiries: Dr Michael Thurn Chief Executive Officer investorenquiries@pharmaust.com Media: Matthew Wright NWR Communications matt@nwrcommunications.com.au 0451 896 420 P +61 (8) 9202 6814 F +61 (8) 9467 6111 www.pharmaust.com About PharmAust Limited: PharmAust Limited is listed on the Australian Securities Exchange (ASX Code: PAA). PAA is a clinical-stage biotechnology company developing therapeutics for human and animal health applications. The company is focused on repurposing monepantel (MPL) for human neurodegenerative diseases and treating cancer in dogs. MPL is a potent and safe inhibitor of the mTOR pathway. This pathway plays a central role in cell growth and proliferation of cancer cells and degenerating neurons. The mTOR pathway regulates the cellular “cleaning process”, where toxic protein is broken down into macromolecules to be reused. This autophagic process is disrupted in most neurodegenerative diseases, including motor neurone disease (MND/ALS). PAA’s lead MPL program is for the treatment of MND/ALS, a rare, incurable disease. The company is currently completing a Phase 1 study in patients with MND/ALS. Top-line results are expected to be announced in Q1 CY2024. PAA anticipates starting a Phase 2 study in H2 CY 2024 that could lead to accelerated approval with the US Food and Drug Administration in 2026. PAA is preparing to begin a pivotal field trial in dogs with B-Cell Lymphoma to enable product registration in the US in 2025. PAA has previously completed a Phase 1 oncology clinical study of monepantel in humans and pilot studies in canine cancer. 1 Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, Walker J, Katsovskiy I, Schoenfeld D, Cudkowicz M, Leitner M. The PRO-ACT database: design, initial analyses, and predictive features. Neurology. 2014 Nov 4;83(19):1719-25. doi: 10.1212/WNL.0000000000000951. Epub 2014 Oct 8. PMID: 25298304; PMCID: PMC4239834. 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