An analysis of un-blinded interim data showed fosgonimeton provided clinically meaningful improvements in cognition and function in patients with mild-to-moderate Alzheimer’s disease.
Athira CEO Mark Litton/courtesy Athira Pharma
Shares of Athira Pharma climbed Monday morning as an analysis of un-blinded interim data showed its Alzheimer’s drug candidate, fosgonimeton, provided clinically meaningful improvements in cognition and function.
Bothell, Washington-based Athira shared data that showed an analysis of about 100 patients in the Phase II/III LIFT-AD study saw these improvements. The data informed Athira of the necessary sample size to power the clinical trial, the company reported.
The data support the advancement of the LIFT-AD study in patients with mild to moderate Alzheimer’s disease (AD), said Athira Chief Executive Officer Mark Litton on a call with investors Monday. Litton quipped that Oct. 17 was an appropriate day to make the announcement given fosgonimeton was previously known as ATH-1017.
He said the unblinded interim analysis of the LIFT-AD trial supports the “clinically meaningful activity” of fosgonimeton in patients who have not received background therapy. That mitigates program risk, he stated.
More than 35 million people globally have been diagnosed with AD. By the year 2050, it is estimated those numbers could triple to more than 100 million people. Approximately 81 percent of current Alzheimer’s cases are considered mild-to-moderate.
New Data Builds on Phase II Trial
The unblinded data builds on previous results from the Phase II ACT-AD study. That trial showed a favorable profile for fosgonimeton but also suggested positive efficacy in measures of cognition, function and neurodegeneration in those patients who did not have background therapy, Litton said.
In the ACT-AD study, Athira assessed fosgonimeton with standard-of-care acetylcholinesterase inhibitors. As BioSpace previously reported, the study missed its primary endpoint.
However, a post hoc analysis of subgroup data showed cognitive and functional improvements through fosgonimeton’s modulation of the HGF/MET system in a population of patients who had not received background therapy. The company amended LIFT-AD to investigate the efficacy of fosgonimeton as a monotherapy in this subgroup.
Hans Moebius, chief medical officer of Athira, said the ACT-AD study was an exploratory study conducted to inform the larger LIFT-AD study in the same population.
The unblinded interim data from the 100 patients in LIFT-AD corroborated the observations seen in ACT-AD. It also informed Athira the trial was well powered to determine the effect of fosgonimeton on clinically meaningful and commercially relevant endpoints, Litton said.
LIFT-AD trial is well-powered to achieve its primary endpoint of the Global Statistical Test, which measures cognition and function, according to Athira.
To achieve this, the company will enroll an additional number of patients, expected to be fewer than 150. When fully enrolled, the trial it is expected to have less than 300 patients who have not received background therapy. Full enrollment of the Phase II/III trial is expected in the middle of 2023, with topline results anticipated in early 2024.
The recommended sample size was based on the actual preliminary effects size observed, not on an estimate, Litton said.
Moebius said stringent evaluation criteria were applied in the analysis, which was based on validated and clinically meaningful cognitive and functional outcomes.
With few available treatments for Alzheimer’s today, Litton noted there is a significant opportunity for fosgonimeton to make a difference should it eventually be approved for commercialization.
“Our goal for this program has remained steadfast,” he said. “Our belief in the science has guided us to this point and our confidence has never wavered.”
As Athira presses forward with LIFT-AD, Litton said it is well-financed, with more than $282 million in cash and cash equivalents. This puts the company in a good position through its key readouts and beyond, he said.
