CTx1000 ALS Trial: First Patient Dosed in Phase 1b Study Targeting TDP-43 Protein
SYDNEY, March 23, 2026 – Celosia Therapeutics, an Australian biotechnology firm, today initiated dosing in its Phase 1b KOANEWA clinical trial, evaluating CTx1000, a genetic medicine designed to target the TDP-43 protein in patients with amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). The first patient received the investigational treatment at the Neurology Department of Macquarie University Hospital in Sydney.
The trial marks a significant step in the development of CTx1000, which aims to address a key pathological mechanism in ALS – the accumulation of toxic forms of the TDP-43 protein. According to Celosia Therapeutics, the therapy is designed to selectively bind and clear these toxic protein aggregates.
“ALS is a progressive and ultimately fatal neurodegenerative disease with limited therapeutic options,” said Dr. Kathryn Sunn, Chief Executive Officer at Celosia Therapeutics. “The initiation of dosing in the KOANEWA study marks an important milestone for Celosia and, most importantly, for the ALS community. Treating the first patient begins the clinical evaluation of this genetic medicine and represents a meaningful step toward a potential disease-modifying therapy for people living with ALS.”
The KOANEWA trial is an open-label, first-in-human Phase 1b study designed to assess the safety and tolerability of a single administration of CTx1000. Researchers will also monitor biomarkers and clinical measures as secondary exploratory endpoints, according to information provided by Celosia.
The development of CTx1000 stems from research led by Professor Lars Ittner and Professor Yazi Ke at Macquarie University. Their 2024 publication in the journal Neuron detailed the identification of a unique binder to TDP-43, a protein directly linked to ALS pathology. Celosia Therapeutics was formed as a spin-out company from Macquarie University to advance the research.
“This study is a major milestone for our research program, advancing a novel disease-modifying therapeutic strategy into the clinic that, for the first time, directly targets a fundamental disease mechanism in ALS – the pathological accumulation of TDP-43,” said Professor Ittner, who also serves as Chief Medical Officer at Celosia and Director of the Macquarie University Dementia Research Centre. “Evaluating CTx1000 in people living with ALS will enable us to assess the safety of this approach while also exploring its potential to therapeutically address one of the key drivers of disease.”
Preclinical studies, conducted in multiple ALS mouse models, demonstrated that CTx1000 halted disease progression, even at advanced stages, and in some instances, partially reversed disease manifestations. The therapy’s potential extends beyond ALS, with researchers suggesting it may also offer opportunities for treating frontotemporal dementia (FTD) and other forms of dementia, including Alzheimer’s disease.
Celosia Therapeutics secured $16.75 million in funding to advance the development of CTx1000, according to a Macquarie University press release. The company anticipates that clinical testing will continue, with further results expected to be reported as the KOANEWA trial progresses.