Early pipeline
Brelgometon
Also known as ATH-1105
Bottom line
Very early: tested only in healthy volunteers, where it was safe. A trial in people with ALS has been delayed twice and has no date. A closely related drug failed in Alzheimer's.
Very early. This drug is in its first safety trial in people and has not been tested for whether it helps in ALS. Most drugs at this stage never reach approval. More about early drugs
Brelgometon (ATH-1105) is an experimental drug taken by mouth that is meant to boost a natural growth-factor system (HGF) that helps nerve cells survive. A Phase 1 study in 80 healthy volunteers, finished in 2024, found it safe and showed it reaches the brain and spinal cord. A trial in people with ALS has been promised twice but has not started, and the company has stopped giving a date.
Information checked 5 Nov 2026
Evidence: Results pending
Studies: 1 running
Trials are running, but there are no efficacy results yet.
ATH-1105 Phase 1 in healthy volunteers (NCT06432647)Pending
80 healthy adults got single doses, or 10 days of doses, of brelgometon or placebo. It was safe and reached the brain and spinal cord. It did not include anyone with ALS.
At a glance
CompanyLeonaBio (formerly Athira Pharma)Explanation and sources
LeonaBio, formerly called Athira Pharma, is a small Nasdaq-listed biotech in Bothell, Washington. Its closely related drug fosgonimeton failed a large Alzheimer's trial in 2024. In 2026 the company shifted its main focus to lasofoxifene, a breast cancer drug, and said it would update the ALS timeline later.
Sources
- Athira Pharma announces topline results from Phase 2/3 LIFT-AD clinical trial of fosgonimetonAthira Pharma · 3 Sept 2024 · Company press release
- LeonaBio reports second quarter 2026 financial results and business highlightsLeonaBio · 14 Aug 2026 · Company press release
How it worksDrug taken by mouth, meant to boost a nerve-protecting growth factorPositive modulator of the HGF/MET neurotrophic systemExplanation and sources
HGF (hepatocyte growth factor) is a natural signal that helps nerve cells survive and reduces inflammation. Brelgometon is designed to strengthen this signal. The company's earlier drug, fosgonimeton, works the same way and failed to help people with Alzheimer's disease in a Phase 2/3 trial in 2024.
Sources
- ATH-1105 experimental therapy found safe in healthy adultsALS News Today · 22 May 2025 · News
- Athira Pharma announces topline results from Phase 2/3 LIFT-AD clinical trial of fosgonimetonAthira Pharma · 3 Sept 2024 · Company press release
- Athira Pharma's Alzheimer's drug fails mid-to-late-stage trialReuters · 3 Sept 2024 · News
StagePhase 1Phase 1 done in 80 healthy volunteers; no ALS trial yet · Dosing: healthy volunteersExplanation and sources
The only human study gave single doses, or 10 days of daily doses, of brelgometon or placebo to 80 healthy adults in Dallas, and finished in November 2024. In 2025 the company said it would start a trial in people with ALS in late 2025; that did not happen. In May and June 2026 it said a Phase 2 trial would start in the second half of 2026, then in August 2026 withdrew that timing.
Sources
- A SAD and MAD study of the safety, tolerability and pharmacokinetics of ATH-1105 (NCT06432647)ClinicalTrials.gov · Trial registry
- Athira Pharma reports first quarter 2025 financial results and recent business highlightsAthira Pharma · 9 May 2025 · Company press release
- LeonaBio presents ATH-1105 at the 5th Annual ALS Drug Development SummitLeonaBio · 4 Jun 2026 · Company press release
- LeonaBio reports second quarter 2026 financial results and business highlightsLeonaBio · 14 Aug 2026 · Company press release
Best reported effectNo ALS data yetSafety and drug levels in healthy volunteersExplanation and sources
No one with ALS has taken brelgometon. In healthy volunteers it was safe and reached the brain and spinal cord. The company reports that in ALS mice it reduced nerve damage, inflammation and TDP-43 clumps and helped them live longer. These are mouse results; most drugs that work in mice do not work in people.
Sources
- ATH-1105 experimental therapy found safe in healthy adultsALS News Today · 22 May 2025 · News
- Athira Pharma presents ATH-1105 Phase 1 trial results at ALS NexusAthira Pharma · 14 Aug 2025 · Company press release
Who it's forAimed at ALS generally; not yet tested in people with ALSAll ALS · By mouthExplanation and sources
The company is developing brelgometon for ALS in general, not a genetic subgroup. In the healthy volunteer study it was given by mouth as a liquid, once a day.
Sources
- A SAD and MAD study of the safety, tolerability and pharmacokinetics of ATH-1105 (NCT06432647)ClinicalTrials.gov · Trial registry
- LeonaBio presents ATH-1105 at the 5th Annual ALS Drug Development SummitLeonaBio · 4 Jun 2026 · Company press release
What's nextUpdate on timing of a Phase 2 trial in ALSExplanation and sources
In August 2026 LeonaBio said it would give an update on the ALS trial's timing once enrolment in its breast cancer trial, ELAINE-3, is complete, which it expects in late 2026. It has not given a new date for the ALS trial.
Sources
- LeonaBio reports second quarter 2026 financial results and business highlightsLeonaBio · 14 Aug 2026 · Company press release
Can I join?No trial open; an ALS trial is planned but has no dateExplanation and sources
The only trial, in healthy volunteers, has finished. There is no trial open to people with ALS and we found no expanded access programme.
- ATH-1105 Phase 1 (healthy volunteers) (NCT06432647) completed · Dallas TX
Sources
- A SAD and MAD study of the safety, tolerability and pharmacokinetics of ATH-1105 (NCT06432647)ClinicalTrials.gov · Trial registry
- LeonaBio reports second quarter 2026 financial results and business highlightsLeonaBio · 14 Aug 2026 · Company press release
Signal: Quiet
Signal measures how often, and how reliably, LeonaBio (formerly Athira Pharma) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.
| Last company update | 14 Aug 2026 (3 months ago) | 24 / 40 |
|---|---|---|
| Company updates in the last 12 months | 3 | 18 / 35 |
| Promises kept | 0 of 2 on time | 0 / 25 |
| Signal score | 42 / 100 · Quiet | |
Announced milestones
- Dropped Enable dosing of people with ALS (promised for Second half of 2025)
- Dropped Start a Phase 2 trial in ALS (promised for Second half of 2026)
70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.
Timeline
8 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.
2026
LeonaBio withdraws timing for the ALS trial
The company said it would update the ALS trial timeline after enrolment in its breast cancer trial finishes, expected in late 2026. No new ALS date was given.
Drug named brelgometon; Phase 2 design presented
At the ALS Drug Development Summit, LeonaBio announced the name brelgometon and described a planned placebo-controlled Phase 2 trial testing several doses.
LeonaBio sets Phase 2 ALS start for second half of 2026
In its first-quarter results, the renamed company said a Phase 2 trial in ALS was on track to start in the second half of 2026.
2025
Phase 1 results presented at ALS Nexus
Athira presented the healthy-volunteer safety and drug-level results at the ALS Nexus meeting.
ALS News Today reports healthy-volunteer results
Independent coverage of the Phase 1 results in 80 healthy adults, which found the drug safe and able to reach the brain and spinal cord.
Athira says an ALS trial could start in late 2025
With healthy-volunteer results in hand, Athira said it was on track to enable dosing in people with ALS in late 2025. That did not happen.
2024
Sister drug fosgonimeton fails in Alzheimer's
Athira's related HGF drug fosgonimeton missed its main goal in the Phase 2/3 LIFT-AD trial in Alzheimer's disease. Brelgometon works in a similar way.
Reuters reports the Alzheimer's trial failure
Independent coverage of fosgonimeton's failed Alzheimer's trial.
All sources for this page
- A SAD and MAD study of the safety, tolerability and pharmacokinetics of ATH-1105 (NCT06432647)ClinicalTrials.gov · Trial registry
- Athira Pharma announces topline results from Phase 2/3 LIFT-AD clinical trial of fosgonimetonAthira Pharma · 3 Sept 2024 · Company press release
- Athira Pharma's Alzheimer's drug fails mid-to-late-stage trialReuters · 3 Sept 2024 · News
- Athira Pharma reports first quarter 2025 financial results and recent business highlightsAthira Pharma · 9 May 2025 · Company press release
- ATH-1105 experimental therapy found safe in healthy adultsALS News Today · 22 May 2025 · News
- Athira Pharma presents ATH-1105 Phase 1 trial results at ALS NexusAthira Pharma · 14 Aug 2025 · Company press release
- Experimental ALS therapy shows promise in Phase 1 trial; patient study setALS News Today · News
- LeonaBio reports first quarter 2026 financial results and business highlightsLeonaBio · 7 May 2026 · Company press release
- LeonaBio presents ATH-1105 at the 5th Annual ALS Drug Development SummitLeonaBio · 4 Jun 2026 · Company press release
- LeonaBio reports second quarter 2026 financial results and business highlightsLeonaBio · 14 Aug 2026 · Company press release