NDX-1017 Phase 1 Trial Complete, Results Will Be Presented at CTAD Conference, Athira Says

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by Patricia Inacio, PhD |

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Athira Pharma has completed the Phase 1 trial testing NDX-1017, its lead therapeutic candidate for Alzheimer’s disease, and will present the results at the 12th Annual Clinical Trials on Alzheimer’s Disease (CTAD) Conference in December.

Preliminary results showed the therapy, which has the potential to be regenerative and to slow or stop disease progression, to be safe and well-tolerated.

Hans J. Moebius, MD, PhD, Athira’s chief medical officer, will present the trial’s final results during the CTAD Conference, which will take place in San Diego Dec. 4-7.

“NDX-1017 represents a new approach to the treatment of neurodegenerative diseases like Alzheimer’s. Completing our first clinical trial is a momentous event, and we look forward to the presentation of data from this trial later this year,” Moebius said in a press release.

The investigational medication is a small therapeutic molecule designed to increase the activity of the hepatocyte growth factor (HGF, also known as scatter factor), and its receptor protein MET. Known for its role in liver regeneration, several studies have reported that HGF-MET also can regulate various brain functions, including axonal growth — the long projections of nerve cells that allow them to communicate — neuronal survival, and transmission of information between cells (synapses).

The therapy has the potential to be regenerative, and was designed to slow or stop disease progression or even reverse it. In preclinical studies, NDX-1017 has shown the ability to regenerate nerve cells and improve cognitive function.

The Phase 1 clinical trial (NCT03298672) enrolled 88 healthy young (ages 18 to 45 years) and elderly (60 to 85 years) individuals, as well as elderly participants with amnestic mild cognitive impairment (MCI) or Alzheimer’s disease.

The study was divided in two parts. In Part A, healthy participants, both young and elderly, were randomly selected to receive single-ascending intravenous (into the vein) doses of NDX-1017 or a placebo for three days, to identify the maximum tolerated dose. In Part B, healthy elderly individuals and people with MCI and Alzheimer’s disease were randomly assigned to receive multiple ascending doses of the compound for 10 days, again with a goal of identifying the maximum tolerated dose.

The trial’s aim was to determine the safety, tolerability, and pharmacokinetics — essentially how the body affects and processes a medicine — of single and multiple ascending doses of NDX-1017.

Preliminary data has shown that NDX-1017 was safe and well-tolerated at multiple dose levels. Moreover, NDX-1017 was capable of crossing the blood-brain barrier and activating its targets across a range of doses. The blood-brain barrier is a highly selective membrane that shields the central nervous system (CNS) from the general blood circulation. Its function is crucial, because it protects the CNS from viruses, bacteria, and fungi, and other damaging agents. However, it may prevent the action of medicines that target the brain.

Increasing doses of NDX-1017 were linked with increases in electroencephalogram (EEG) signals, which correlate with improvements in learning and memory.

“We are encouraged by the results and by having achieved all of our primary goals from the Phase 1 study in these first cohorts,” Leen Kawas, PhD, CEO of Athira, said when releasing the preliminary data in July. “We see that NDX-1017 successfully crosses the blood brain barrier and clearly impacts the brain as noted through EEG as a biomarker.”

Athira, previously known as M3 Biotechnology, is now planning to test NDX-1017 in larger, Phase 2 trials.

“We are actively preparing for larger, later stage clinical studies to further investigate the potential of NDX-1017 as a therapeutic candidate for treatment of neurodegenerative diseases,” Moebius said.