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.

How we decide the overall verdict: How to read this site.

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.

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.

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.

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.

This is the most favourable result reported. See all key studies for the full picture.

Sources

  1. ATH-1105 experimental therapy found safe in healthy adultsALS News Today · 22 May 2025 · News
  2. 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.

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

  1. 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.

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 update14 Aug 2026 (3 months ago)24 / 40
Company updates in the last 12 months318 / 35
Promises kept0 of 2 on time0 / 25
Signal score42 / 100 · Quiet

Announced milestones

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

  1. Investor updateFrom the companyLeonaBio

    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.

  2. ConferenceFrom the companyLeonaBio

    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.

  3. Investor updateFrom the companyLeonaBio

    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

  1. ConferenceFrom the companyAthira Pharma

    Phase 1 results presented at ALS Nexus

    Athira presented the healthy-volunteer safety and drug-level results at the ALS Nexus meeting.

  2. NewsALS News Today

    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.

  3. Investor updateFrom the companyAthira Pharma

    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

  1. Press releaseFrom the companyAthira Pharma

    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.

  2. NewsReuters

    Reuters reports the Alzheimer's trial failure

    Independent coverage of fosgonimeton's failed Alzheimer's trial.