Landmark 180 · Trial in people
Fosgonimeton (SHAPE)
Stopped early at 28 of a planned 75 people, who had dementia linked to Parkinson's. It missed its main target. Too small to close the idea.
In short
An injected drug meant to boost a growth-factor pathway (HGF) was stopped at 28 of a planned 75 people with Parkinson's disease dementia or dementia with Lewy bodies, and missed its main target.
- What it is
- Trial in people
- Outcome
- Did not work
- When
- 2022–2023
- Size
- 28 people · Phase 2
- Approach
- Replace or repair what's lost
- Research lane
- Growth factors
- Trial registry
- NCT04831281
- Last checked
- 19 Oct 2026
The research record
This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".
Who
Athira Pharma (later renamed LeonaBio). NCT04831281; started 20 January 2022, primary completion 19 April 2023.
What happened
Hepatocyte growth factor (HGF) and its receptor MET help nerve cells survive and make connections. Fosgonimeton (ATH-1017) is a once-daily injection under the skin that raises HGF activity.
- Design. Phase 2, double-blind, placebo-controlled, 26 weeks. About 75 people planned; enrolment was ended in October 2022 at 28, citing study-design limits and a shift to the company's Alzheimer's programme. Registry reason: "Enrollment ended early due to study design limitations."
- Participants. The registry's results show 28 started (9 placebo, 9 on 40 mg, 10 on 70 mg); 19 finished (7, 7 and 5). Seven US centres.
- Main measure. A combined z-score of change in brain-signal timing (P300 latency) and thinking tests (ADAS-Cog13) at week 26. Registry results (posted March 2025) give group means of −0.22 (placebo, n=9), 0.33 (40 mg, n=7) and −0.04 (70 mg, n=9), with no between-group test posted. The registry notes the study stopped before the planned size of about 75.
- Company framing (Dec 2023). Athira reported the primary endpoint was not met, and an exploratory ADAS-Cog13 difference of −7.2 points at 40 mg (p=0.032, modified intent-to-treat population, group sizes unclear). This was not the primary result.
Why it moved us toward a cure
It adds a small negative data point for a growth-factor route in Lewy-body diseases, away from the gene-therapy delivery problem of landmark 179. It is mainly a dementia trial in Parkinson's-related diseases, so it is weak evidence about slowing motor Parkinson's.
Outcome
Dead end for this drug in this trial. Too small to close the HGF idea.
Sources
- ClinicalTrials.gov NCT04831281
- Athira release, Dec 2023 (BioSpace) · Patsnap Synapse news
- Not yet verified: a peer-reviewed publication; the direction of the z-score scale; the group sizes behind the company's p-value; the parent link (no clear upstream landmark; linking 179 or 147 would be a stretch).
How it connects
Built on
None recorded.
Led to
None recorded.