Where ALS drugs are, in plain language

Clene is a small company based in Salt Lake City, Utah, and CNM-Au8 is its only drug, also studied in multiple sclerosis. Money is tight: it had $9.9 million in cash at the end of September 2026, which it says lasts only until the middle of the first quarter of 2027. More money from warrants depends on the FDA accepting its application for review.

Company website ↗

The gold crystals are meant to speed up chemical reactions that cells use to produce energy, helping stressed motor neurons and their support cells survive and work better, and reducing harmful oxidative stress. This mechanism comes mostly from lab studies and is not proven in people.

Scientific name: Catalytic gold nanocrystals (NAD+/NADH, mitochondrial energy)

Clene submitted a New Drug Application in October 2026 asking for accelerated approval, a pathway where the FDA can approve a drug based on a marker (here NfL) that is "reasonably likely" to predict benefit, with a later trial needed to confirm it. The FDA first decides whether to accept the file for review, then whether to approve. A confirmatory Phase 3 trial, RESTORE-ALS, has not started.

FDA accelerated approval application submitted Oct 2026

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In the HEALEY ALS Platform Trial, CNM-Au8 did not slow decline on the ALS functional score compared with placebo, and the earlier RESCUE-ALS trial also missed its main goal. In HEALEY, NfL, a blood marker of nerve damage, rose about 8% on placebo and fell about 2% on CNM-Au8 over 24 weeks, an exploratory finding. Company analyses, many done after the fact or using comparison groups from outside the trial, link NfL drops to longer survival. It is not yet proven that lowering NfL with this drug helps people live longer or function better.

All key studies

• ~

  Company NfL and survival analyses (HEALEY, RESCUE-ALS, expanded access)Mixed

  2025 · Post-hoc analysis · Company-run

  Clene's later analyses link NfL lowering to longer survival, partly using comparison groups from outside the trials. They underpin the FDA application but cannot by themselves show that the drug helps people live longer.

• ~

  HEALEY ALS Platform Trial, Regimen CMixed

  2022 · Placebo-controlled · Phase 2/3 · 161 people · Independent

  Missed its main goal (function and survival combined) and all three secondary goals over 24 weeks. The NfL nerve-damage marker was lower than on placebo, and there were fewer deaths on the 30 mg dose, both exploratory findings.

• ~

  RESCUE-ALSMixed

  2021 · Placebo-controlled · Phase 2 · 45 people · Company and academic

  Missed its main goal (a nerve-signal measure called MUNIX) and its breathing goal over 36 weeks. In later follow-up fewer people first given CNM-Au8 had died (7 vs 15), an exploratory finding in a small trial.

Results from different trials can't be compared directly. How to read effects

HEALEY and the expanded access programmes included adults with sporadic or familial ALS. Clene's application covers ALS generally rather than one genetic form; the exact proposed label has not been published.

How it's taken: Oral liquid, 30 mg daily

After an application is submitted, the FDA first decides whether it is complete enough to review. Clene has not given a date for this decision. Separately, it plans to start the RESTORE-ALS Phase 3 trial in the second quarter of 2027.

ClinicalTrials.gov lists one CNM-Au8 expanded access programme (NCT05281484) as available at participating US sites; ask your ALS clinic whether it takes part. The NIH-funded programme (NCT06408727) is listed as temporarily not available. No placebo-controlled trial is enrolling; RESTORE-ALS is planned for 2027.

An expanded access (compassionate use) programme exists. Ask your neurologist.

Trials and programmes

• CNM-Au8 expanded access (EAP01/02/06)NCT05281484 · available · USA
• NIH-funded expanded access (EAP04)NCT06408727 · unknown · USA

Signal measures how often, and how reliably, Clene Nanomedicine (Clene Inc.) 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.

Announced milestones

• Delivered late Submit accelerated approval NDA in mid-2025 (promised for July 2025, delivered 19 Oct 2026)
• Dropped Begin enrolling the Phase 3 RESTORE-ALS trial before the NDA (mid-2025) (promised for July 2025)
• Delivered late Submit accelerated approval NDA by end of 2025 (promised for Q4 2025, delivered 19 Oct 2026)
• Delivered late Submit NDA in Q3 2026 (promised for Q3 2026, delivered 19 Oct 2026)
• Dropped Start confirmatory Phase 3 RESTORE-ALS in Q1 2027 (promised for Q1 2027)
• Not due yet Randomise first RESTORE-ALS participants in Q2 2027 (promised for Q2 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

AB Science is a small Paris-listed company. Masitinib is its lead drug and has been tested in many diseases; in 2026 the company dropped several of those programmes to focus on ALS and one cancer drug. It has relied on repeated small share sales and debt deferrals for funding, and it changed chief executive in July 2026. In September 2026 inspectors from three European regulators carried out a "for cause" inspection of how it runs clinical trials.

Company website ↗

Masitinib blocks signalling enzymes (kinases) in immune cells called mast cells and microglia. In ALS these cells may become overactive and keep up inflammation that harms motor neurons. The idea is that calming them slows nerve damage. In 2026 the company reported a blood marker linked to inflamed microglia that it wants to test in the Phase 3 trial, but has not named it.

Scientific name: Tyrosine kinase inhibitor (mast cells, microglia; c-Kit, LYN, FYN)

The confirmatory Phase 3 trial AB23005 (about 408 people, 48 weeks) was cleared by the FDA and several European countries in 2025, but no patient has been enrolled. In April 2026 the company paused all European trials after regulators raised questions about its resources, and EU regulators listed AB23005 as suspended. After a regulatory inspection in September 2026, the company said it now aims to restart the trial in the fourth quarter of 2027, subject to health authority approval.

Phase 3 authorised but never started; suspended, restart aimed Q4 2027

Regulators' decisions for ALS

• Canada Health CanadaRefused · 19 Feb 2024Notice of deficiency-withdrawal on the conditional approval request. AB Science later dropped its reconsideration request.
• EU EMARefused · 19 Dec 2024Refused twice: Alsitek in 2018 and again in 2024 after re-examination, because study data were unreliable and no benefit was shown overall.

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In the earlier AB10015 trial, people progressing at a "normal" rate (losing under 1.1 points a month) lost 9.2 ALSFRS-R points on high-dose masitinib plus riluzole versus 12.6 on placebo plus riluzole over 48 weeks. This group was defined by a mid-trial change to the plan, and there was no clear benefit in the whole group treated or on survival. Serious side effects were more common on masitinib (31% vs 18%). European regulators found problems with how the study was run and did not consider the data reliable. Later company analyses of narrower subgroups are post hoc and partly unreviewed preprints.

All key studies

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  AB10015 re-analysis (patients before any complete loss of function)Mixed

  2025 · Post-hoc analysis · Phase 2/3 · Company-run

  A company analysis of a narrower group chosen after the trial reported longer survival. It is a preprint, not peer reviewed, and Health Canada considered this group post hoc.

• …

  AB23005 (confirmatory Phase 3)Pending

  2025 · Placebo-controlled · Phase 3 · Company-run

  About 408 people planned over 48 weeks. Authorised in 2025 but no patient enrolled; suspended in Europe, with a restart aimed for late 2027.

• ~

  AB10015Mixed

  2019 · Placebo-controlled · Phase 2/3 · 394 people · Company-run

  Met its main goal in "normal progressors" (3.4 points less decline at 48 weeks), a group set by a mid-trial amendment; no clear benefit in all patients. Regulators found study-conduct problems.

Results from different trials can't be compared directly. How to read effects

The Phase 3 trial is designed for people with ALS whose decline is under 1.1 ALSFRS-R points a month and who still score at least 1 on every item of the ALSFRS-R (no function completely lost). The company says this is about three-quarters of people with ALS. US participants may also take edaravone.

How it's taken: Pill, daily (with riluzole)

AB Science said on 29 September 2026 that it must first finish a new quality management system and have it audited by the end of 2026, then pass a new inspection, before resuming the ALS trial in the fourth quarter of 2027. The final inspection report is expected before the end of 2026.

No masitinib ALS trial is enrolling. The EU trial record (2024-511244-12-01) lists the study as suspended in the countries that had approved it. We did not find an active expanded access programme for ALS.

Trials and programmes

• AB23005 (Phase 3)NCT07174492 · suspended · Planned in EU, USA, Argentina, Serbia

Signal measures how often, and how reliably, AB Science 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.

Announced milestones

• Dropped Start the confirmatory Phase 3 trial AB23005 (reported by analyst on company site) (promised for Q1 2026)
• Not due yet Finish new quality management system and its external audit (promised for Q4 2026)
• Not due yet Resume the Phase 3 ALS trial (promised for Q4 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

BrainStorm is a small company based in New York and Israel whose main product is NurOwn. Its shares moved from Nasdaq to the OTCQB market in July 2025, and in 2026 it has raised only small amounts of money (around US$1 million at a time). A former FDA associate commissioner, Peter Pitts, became executive chairman in July 2026.

Company website ↗

Doctors collect bone marrow from the person with ALS, and the lab turns its mesenchymal stem cells into cells that release high levels of growth factors that support nerves. These cells are then injected into the spinal fluid by lumbar puncture. The aim is to protect motor neurons and calm inflammation, not to replace lost neurons.

Scientific name: Neurotrophic factors (e.g. GDNF, VEGF) from modified MSCs

The first Phase 3 trial (189 people) missed its main goal, and in September 2023 FDA advisers voted 17 to 1 that the evidence did not show it works; BrainStorm then withdrew its approval application. The FDA agreed the design of a new Phase 3b trial in April 2024 and cleared it to start in May 2025. In May 2026 BrainStorm said starting depends on securing financing, and it reported only about US$0.2 million in cash at the end of June 2026.

New Phase 3b (ENDURANCE) cleared by FDA but not yet enrolling

Regulators' decisions for ALS

• USA FDAWithdrawn · 18 Oct 2023FDA first declined to review the application; BrainStorm forced a review. Advisers voted 17 to 1 that it was not shown to work, and BrainStorm then withdrew the application.

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In the Phase 3 trial, 32.6% of people on NurOwn met the "responder" definition versus 27.7% on placebo, which was not a significant difference (p=0.45). In a pre-planned group with less advanced ALS (ALSFRS-R score of 35 or more), the ALSFRS-R score fell about 2 points less with NurOwn, a borderline result (p=0.050) that is not considered proof. The earlier Phase 2 trial (48 people) also found no difference in decline overall. FDA reviewers concluded the data did not show substantial evidence that it works. The new ENDURANCE trial is meant to test the earlier-stage group properly.

All key studies

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  ENDURANCE (Phase 3b)Pending

  2025 · Placebo-controlled · Phase 3b · 200 people · Company-run

  Planned test in about 200 people with earlier-stage ALS over 24 weeks. Cleared by the FDA in 2025 but not yet enrolling for lack of funding.

• ~

  NurOwn Phase 3 (NCT03280056)Mixed

  2021 · Placebo-controlled · Phase 3 · 189 people · Company-run

  Missed its main goal: responders 32.6% on NurOwn vs 27.7% on placebo. A pre-planned group with less advanced ALS showed a borderline difference that FDA reviewers did not accept as proof.

• ~

  NurOwn Phase 2 (single dose)Mixed

  2019 · Placebo-controlled · Phase 2 · 48 people · Company-run

  Met its main goal of safety, but the rate of decline was similar on NurOwn and placebo. Fast-progressing patients looked better for the first weeks only.

Results from different trials can't be compared directly. How to read effects

ENDURANCE plans to enrol about 200 people whose symptoms began within the last 24 months and whose breathing (slow vital capacity) is still at least 65% of normal. Half will get NurOwn and half placebo for 24 weeks, followed by an open-label period where everyone can get it.

How it's taken: Spinal injection of own modified cells

BrainStorm says it is completing the operational and regulatory work to open ENDURANCE, and that enrolment depends on securing the necessary financing. It has not given a new start date; the registry still shows an old estimated start of June 2025.

No trial is enrolling yet. An earlier expanded access programme treated a small number of former trial participants; we found no programme open to new patients. BrainStorm's patient page lists trial information for anyone wanting to follow ENDURANCE.

Trials and programmes

• ENDURANCE (Phase 3b)NCT06973629 · not yet recruiting
• Phase 3 (completed)NCT03280056 · completed · USA

Signal measures how often, and how reliably, BrainStorm Cell Therapeutics 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.

Announced milestones

• Overdue Start the Phase 3b trial (dose first patients) in 2024 (promised for 2024)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Prilenia is a private biotech based in the Netherlands and Massachusetts, and pridopidine is its main drug, also being developed for Huntington's disease. The Spanish drug company Ferrer is its partner for Europe and some other markets and co-sponsors the ALS Phase 3 trial. Prilenia keeps the rights in North America and Asia.

Company website ↗

The sigma-1 receptor is a protein that sits where the cell's protein factory meets its power plants (mitochondria). Turning it on is thought to help nerve cells handle stress, keep their energy supply working and clear damaged material. Most of this evidence comes from lab and animal studies.

Scientific name: Sigma-1 receptor (S1R) agonist

PREVAiLS started in March 2026 and is planned across up to 60 ALS centres in 13 countries. In late October 2026 the companies said it was about 60% enrolled and expected to finish enrolling in the first quarter of 2027. Pridopidine has never been filed for approval in ALS anywhere. Its only marketing application, in Europe for Huntington's disease, received a negative EMA opinion in July 2025 and was withdrawn in November 2025.

Phase 3 PREVAiLS enrolling, ~60% of 500 people

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In the HEALEY ALS Platform Trial, pridopidine did not slow ALS compared with placebo in the full group of 162 people, on the main goal (function and survival combined) or on any key secondary goal, as published in JAMA in 2025. When researchers looked back at 34 people within 18 months of first symptoms, with definite or probable ALS and fast decline, those on pridopidine lost 7.5 ALSFRS-R points versus 12.7 on placebo, and some speech measures were better. Looking back at small subgroups can produce chance findings, which is why PREVAiLS is testing this group prospectively.

All key studies

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  PREVAiLSPending

  2026 · Placebo-controlled · Phase 3 · 500 people · Company-run

  48 weeks of pridopidine or placebo in people with definite or probable ALS within 18 months of first symptoms, the group that seemed to benefit in HEALEY. Results expected in early 2028.

• ~

  HEALEY ALS Platform Trial, Regimen DMixed

  2023 · Placebo-controlled · Phase 2/3 · 163 people · Independent

  Missed its main goal (function and survival combined) and all key secondary goals over 24 weeks. Some speech measures were better, and an after-the-fact look at 34 early, fast-progressing people suggested slower decline.

Results from different trials can't be compared directly. How to read effects

PREVAiLS only enrols adults with definite or probable ALS whose first symptoms began less than 18 months earlier, the group that seemed to benefit in HEALEY. People can stay on riluzole, edaravone or Nuedexta if their dose has been stable for at least four weeks.

How it's taken: Pill, 45 mg twice daily

Prilenia and Ferrer said on 28 October 2026 that they expect to finish recruiting about 500 people in the first quarter of 2027 and report the main results in the first quarter of 2028.

PREVAiLS is recruiting. Participants have a 3 in 5 chance of getting pridopidine for the first 48 weeks, and everyone gets it for a further 48 weeks afterwards. An NIH-funded expanded access programme run by the Healey Center gave pridopidine to 200 people who could not join trials; it completed enrolment and ClinicalTrials.gov now lists it as no longer available.

Trials and programmes

• PREVAiLSNCT07322003 · recruiting · USA, Canada, EU, UK, Israel
• Pridopidine expanded access (EAP 2)NCT06069934 · completed · USA

Signal measures how often, and how reliably, Prilenia Therapeutics (with Ferrer) 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.

Announced milestones

• Delivered on time Start enrolling the Phase 3 ALS trial (promised for Q1 2026, delivered 30 Mar 2026)
• Not due yet Complete PREVAiLS enrolment of ~500 participants (promised for Q1 2027)
• Not due yet PREVAiLS topline results (promised for Q1 2028)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Ionis, a US company that specialises in antisense (RNA-targeted) medicines, discovered and developed ulefnersen and ran the FUSION trial. In November 2024 Otsuka, a large Japanese drugmaker, took exclusive worldwide rights; Otsuka now leads regulatory filings, manufacturing and sales, while Ionis receives milestones and royalties.

Company website ↗

In FUS-ALS, a changed FUS gene makes a harmful form of the FUS protein that builds up in motor neurons. Ulefnersen sticks to the FUS gene's messenger RNA so the cell makes less FUS protein, including the harmful form. It is injected into the spinal fluid so it reaches the brain and spinal cord directly.

Scientific name: Antisense oligonucleotide against FUS pre-mRNA

FUSION, a placebo-controlled Phase 1-3 trial in 89 people, reported positive topline results on 22 September 2026. The companies say they will review the results with the FDA and other regulators to look for a faster (expedited) route to approval. No approval application had been announced as of 5 November 2026.

Phase 3 positive (company topline only); FDA talks under way

One positive trial. One controlled trial met its main goal, but no second trial has confirmed it yet.

The main goal combined three things: time to death or permanent breathing support, time to needing rescue treatment, and change in the ALSFRS-R function score over about 72 weeks. In the 73 people in the main analysis, ulefnersen did significantly better than placebo (p=0.0005), and blood neurofilament (a nerve-damage marker) also improved. These are company topline results; the companies have not yet said how many points of ALSFRS-R were saved, and the full data have not been presented or peer reviewed. Earlier, in a 12-person open-label case series, most people still declined, though two did unusually well.

All key studies

• ✓

  FUSIONMet its main goal

  2026 · Placebo-controlled · Phase 1-3 · 89 people · Company-run

  Met its main goal (function, rescue treatment and survival combined) at 72 weeks in the 73 people in the main analysis (p=0.0005), and NfL improved. Topline press release only; ALSFRS-R point differences and full data not yet published.

• ~

  Jacifusen expanded access case seriesMixed

  2025 · Open-label · 12 people · Company and academic

  Investigator-led, no placebo group. The nerve-damage marker NfL fell sharply, but most people kept losing function and two died. One young woman regained walking and breathing, and one person without symptoms stayed symptom-free.

Results from different trials can't be compared directly. How to read effects

FUS-ALS is rare (about 0.6% of people with ALS) but often starts young and progresses fast. You need a genetic test showing a FUS mutation to be eligible. The drug is not being studied in other forms of ALS.

How it's taken: Spinal injection (intrathecal)

Otsuka and Ionis said they will move quickly to review FUSION with the FDA and other regulators, and will present detailed results at a medical meeting and submit them to a journal "in the coming months". They have not given a date for an approval application.

FUSION has finished enrolling. In September 2026 Otsuka opened a global early access programme for people with a confirmed FUS-ALS genetic diagnosis who cannot join a trial. Requests must come from the treating doctor, not from patients or families, and each case still needs regulatory, ethics and treatment-centre approvals, so timing varies.

An expanded access (compassionate use) programme exists. Ask your neurologist.

Trials and programmes

• FUSIONNCT04768972 · active not recruiting · USA, Canada, Europe, Brazil, Japan, South Korea, Taiwan

Signal measures how often, and how reliably, Ionis Pharmaceuticals (licensed to Otsuka) 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.

Announced milestones

• Delivered on time Report Phase 3 FUSION results in 2026 (promised for 2026, delivered 22 Sept 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Coya is a small Nasdaq-listed company in Houston. Its partner Dr. Reddy's Laboratories supplies the CTLA4-Ig part of the combination, pays milestone fees and invested in Coya in January 2026. Coya says its cash should last into the second half of 2027, beyond the expected ALSTARS results.

Company website ↗

In ALS, regulatory T cells (Tregs), which normally keep inflammation in check, weaken as the disease progresses. COYA 302 pairs a low dose of interleukin-2, which strengthens Tregs, with CTLA4-Ig (a copy of the arthritis drug abatacept), which calms activated immune cells. The idea is that the two together reduce harmful inflammation around motor nerves.

Scientific name: Regulatory T cells (via IL-2) and CTLA-4/CD80/86

ALSTARS is a randomised, double-blind, placebo-controlled trial at about 25 sites in the US and Canada. Dosing began in December 2025 and enrolment of 120 people was completed in October 2026. The FDA granted Fast Track status in May 2026. Coya first called ALSTARS a Phase 2 trial; by October 2026 it was calling it Phase 2/3. Placebo patients switch to the drug after 24 weeks.

Phase 2/3 ALSTARS fully enrolled (120 people); results expected early Q2 2027

Early signal only. Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

In a Houston Methodist pilot study, 4 people with ALS received low-dose IL-2 and CTLA4-Ig for 48 weeks. Before treatment they lost about 1.1 ALSFRS-R points a month; over the first 24 weeks their average score was stable, and over 48 weeks they lost about 0.13 points a month, with one person improving and three declining by about 0.47 points a month. Treg function improved and some markers of inflammation fell, though the nerve-damage marker NfL did not change on average. With only 4 people and no placebo group this is a signal at most; ALSTARS is the first real test.

All key studies

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  ALSTARSPending

  2026 · Placebo-controlled · Phase 2/3 · 120 people · Company-run

  24 weeks of COYA 302 or placebo in adults with ALS in the US and Canada. Fully enrolled in October 2026; topline results expected in early Q2 2027.

• ~

  Houston Methodist pilot (low-dose IL-2 + CTLA4-Ig)Mixed

  2024 · Open-label · Phase 1 · 4 people · Company and academic

  Safe in all 4 people, its main goal. Function was stable for 24 weeks, but over 48 weeks three of four declined, though more slowly than before treatment. No placebo group.

Results from different trials can't be compared directly. How to read effects

ALSTARS enrolled adults with ALS at sites in the US and Canada. In 2026 Coya loosened the rules to allow people on blood-pressure medicines and people with controlled autoimmune diseases. Both medicines are given as injections under the skin.

How it's taken: Injection under the skin

When enrolment finished in October 2026, Coya said it expects topline results in early Q2 2027. Earlier in 2026 it had guided to Q1 2027. People who finish the blinded part can move into an open-label extension starting November 2026.

ALSTARS is no longer enrolling new people. Coya plans an open-label extension for people already in the trial, starting November 2026. We found no expanded access programme.

Trials and programmes

• ALSTARS (Phase 2/3)NCT07161999 · active not recruiting · USA, Canada

Signal measures how often, and how reliably, Coya Therapeutics 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.

Announced milestones

• Delivered on time Submit IND to FDA for ALS Phase 2 trial (promised for June 2024, delivered 14 Jun 2024)
• Delivered on time Submit extra nonclinical data FDA asked for (IND resubmission) (promised for Q2 2025, delivered 30 Jun 2025)
• Delivered late Publish longitudinal ALS biomarker (NfL, oxidative stress) data (promised for Q2 2025, delivered 9 Apr 2026)
• Delivered on time Complete ALSTARS enrolment (promised for Second half of 2026, delivered 5 Oct 2026)
• Dropped ALSTARS topline results in Q1 2027 (promised for Q1 2027)
• Not due yet ALSTARS topline results in early Q2 2027 (promised for Q2 2027)
• Not due yet Start ALSTARS open-label extension (promised for November 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

MediciNova is a small company based in La Jolla, California, listed in both the US and Tokyo. Ibudilast (MN-166) is its lead drug and is also being studied in multiple sclerosis and other conditions. It reported $25.4 million in cash in mid-2026.

Company website ↗

Ibudilast blocks an enzyme called PDE4 and an inflammatory signal called MIF. In ALS, immune cells in the nervous system (glia) become overactive and may damage motor neurons; ibudilast is meant to damp this down and reduce oxidative stress. Lab studies also suggest it helps clear clumps of the TDP-43 and SOD1 proteins. A small 2021 open-label study at high dose did not show changes in a brain inflammation scan or in neurofilament levels.

Scientific name: PDE4 and macrophage migration inhibitory factor (MIF) inhibitor

COMBAT-ALS randomised 234 people in the US and Canada to ibudilast or placebo for 12 months. The last blinded visit happened in September 2026, and the company is now locking and analysing the data. People still in the study are taking ibudilast in an open-label extension that ends around March 2027.

Phase 2b/3 COMBAT-ALS blinded phase done; results due by end of 2026

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

COMBAT-ALS has not reported results. Its main measure, CAFS, combines change in daily function with survival. An earlier 6-month trial in 51 people found no overall difference from placebo; the company has highlighted a later look-back analysis in which more people on ibudilast stayed stable, but such responder analyses are weak evidence. A separate open-label study at a higher dose found no change in a brain inflammation scan or in blood neurofilament, and about a third of people stopped early because of side effects.

All key studies

• …

  COMBAT-ALSPending

  2026 · Placebo-controlled · Phase 2b/3 · 234 people · Company-run

  12 months of ibudilast or placebo in people within 18 months of first weakness. Main goal combines function and survival (CAFS). Topline results expected by the end of 2026.

• ✗

  Mass General open-label biomarker studyNo benefit

  2021 · Open-label · Phase 1b · 35 people · Company and academic

  High-dose ibudilast did not change a brain inflammation scan or blood neurofilament, its two main goals, over 36 weeks. About a third of people stopped early because of side effects, mostly nausea and other stomach problems.

• ~

  MN-166-ALS-1201 (early-stage group)Mixed

  2018 · Placebo-controlled · Phase 1b/2a · 51 people · Company and academic

  Its main goal, safety, was met. Over 6 months there was no overall difference from placebo in function, strength or quality of life; only after-the-fact responder and subgroup analyses favoured ibudilast.

Results from different trials can't be compared directly. How to read effects

COMBAT-ALS enrolled people with sporadic or familial ALS whose first weakness began within the last 18 months and whose breathing capacity was at least 70% of normal. Riluzole and edaravone were allowed.

How it's taken: Pill, up to 50 mg twice daily

MediciNova has said several times, most recently in its 12 October 2026 shareholder update, that it expects to report topline results from the blinded part of COMBAT-ALS by the end of 2026.

COMBAT-ALS closed recruitment in September 2025. SEANOBI-ALS, an NIH-funded expanded access programme led by Mayo Clinic for people who cannot join trials, reached its 200-person target in mid-2026 and is no longer enrolling. MediciNova says it supports individual expanded access for people finishing COMBAT-ALS who want to continue.

Trials and programmes

• COMBAT-ALS (Phase 2b/3)NCT04057898 · active not recruiting · USA, Canada
• SEANOBI-ALS expanded accessNCT06743776 · no longer available · USA

Signal measures how often, and how reliably, MediciNova 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.

Announced milestones

• Not due yet COMBAT-ALS topline data by end of 2026 (promised for Q4 2026)
• Not due yet SEANOBI-ALS final patient follow-ups (promised for First half of 2027)
• Not due yet COMBAT-ALS last patient last visit for whole study (incl. extension) (promised for March 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Neurizon is a small Melbourne-based company listed on the Australian stock exchange; it was formerly called PharmAust. NUZ-001 is its lead drug, and it licenses monepantel from the animal-health company Elanco. It reports A$11.4 million cash at the end of September 2026 and says it is funded through the HEALEY results.

Company website ↗

Monepantel is thought to dampen mTOR, a cell "growth switch", which can turn up autophagy, the cell's recycling system for clearing clumped proteins such as TDP-43 that build up in ALS. This idea comes from lab studies; whether it works this way in people with ALS is not proven.

Scientific name: mTOR pathway (4EBP1 phosphorylation); autophagy

NUZ-001 is "Regimen I" of the HEALEY ALS Platform Trial in the US, where 250 people were randomly given the drug or placebo (3 to 1) for 36 weeks, followed by 36 weeks where everyone gets the drug. The FDA paused the programme from January to October 2025 to ask for more animal data, and in January 2026 it declined a Fast Track request. In October 2026 the FDA said a positive HEALEY result could on its own support an approval application.

In the HEALEY Phase 2/3 platform trial, fully enrolled; results mid-2027

Early signal only. Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

In the Phase 1 MEND study, 12 people took monepantel without a placebo group. Their ALSFRS-R score fell about 0.74 points a month, which the company says is 39% slower than matched people from an old trial database (58% slower in the 6 people on the higher dose). Comparisons with historical patients are often misleading, so this is only an early hint. The placebo-controlled HEALEY result will be the first real test.

All key studies

• …

  HEALEY ALS Platform Trial, Regimen IPending

  2026 · Placebo-controlled · Phase 2/3 · 250 people · Independent

  36 weeks of NUZ-001 or placebo (3 to 1), then everyone gets the drug. Fully enrolled in July 2026; topline results expected in late Q2 2027.

• ~

  MEND and its open-label extensionMixed

  2024 · Open-label · Phase 1 · 12 people · Company-run

  Main goal was safety and dosing, which was met. Decline looked slower than in matched historical patients, but there was no placebo group and the higher-dose result was not statistically significant (p=0.11). Ten people continued in a 12-month extension.

Results from different trials can't be compared directly. How to read effects

HEALEY enrols adults with sporadic or familial ALS at US sites, and Regimen I uses 10 mg per kg of body weight daily, the dose picked from the Phase 1 study. The drug is not limited to any genetic form of ALS.

How it's taken: Oral, 10 mg/kg once daily

Neurizon expects the 36-week placebo-controlled part to end in early Q2 2027 and to report the main results in late Q2 2027. Separately, an NIH-funded expanded access programme for up to 200 people who cannot join trials is due to start enrolling in Q1 2027.

Regimen I stopped enrolling in July 2026 after reaching 250 participants, so new patients cannot join it. An NIH-funded expanded access programme, run by the Healey & AMG Center at about 20 US sites plus a virtual site, is expected to start enrolling in Q1 2027 for people who do not qualify for trials.

Trials and programmes

• HEALEY ALS Platform Trial, Regimen I (NUZ-001)NCT04297683 · active not recruiting · USA (78 sites)

Signal measures how often, and how reliably, Neurizon Therapeutics (formerly PharmAust) 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.

Announced milestones

• Delivered late Start pivotal Phase 2/3 study in Q3 2024 (promised for Q3 2024, delivered 26 Feb 2026)
• Delivered late Enter the HEALEY ALS Platform Trial this quarter (Q4 2025) (promised for Q4 2025, delivered 26 Feb 2026)
• Delivered on time Complete HEALEY Regimen I enrolment in H2 2026 (promised for Second half of 2026, delivered 17 Jul 2026)
• Not due yet Start enrolling NIH-funded expanded access programme by Q1 2027 (promised for Q1 2027)
• Not due yet Report HEALEY Regimen I topline results in late Q2 2027 (promised for Q2 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Rapa Therapeutics is a small private biotech in Rockville, Maryland that develops reprogrammed T-cell therapies. Its ALS work is run with the Healey & AMG Center for ALS at Massachusetts General Hospital, which also leads the NIH-funded expanded access programme. Rapa itself publishes very little news, so most updates come from MGH.

Company website ↗

Inflammation in the brain and spinal cord is thought to speed up ALS. RAPA-501 is made by collecting a person's T cells and treating them in the lab so they become regulatory cells that dampen inflammation. They are then infused back into the same person.

Scientific name: Hybrid regulatory T cells (Treg/Th2)

The registry lists the trial as Phase 2/3, but the current part is a single-group study in which everyone gets RAPA-501 and the main goals are safety and whether treatment is feasible. It started in January 2025 and expects its main results around July 2027. It is not a large placebo-controlled trial.

Open-label Phase 2/3 cohort recruiting at MGH

Early signal only. Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

In the expanded access programme for advanced ALS, 26 of 34 treated people (76.5%) were alive at a median follow-up of 8.9 months. MGH reported this was better than expected from computer-modelled "virtual" control patients (p=0.01). Such predictions cannot account for every way the treated people differed from typical patients, and there was no placebo group. The earlier Phase 1 study in 9 people was about safety. None of this is published in a peer-reviewed paper yet.

All key studies

• ~

  RAPA-501 expanded access, advanced ALS (NCT06169176)Mixed

  2026 · Open-label · 34 people · Company and academic

  NIH-funded programme at MGH for people with breathing below 50%. Survival looked better than computer-predicted "virtual" controls (p=0.01), but there was no placebo group and no paper yet.

• …

  RAPA-501 Phase 2/3, standard-risk ALS (NCT04220190)Pending

  2025 · Open-label · Phase 2/3 · 41 people · Company-run

  Single-group study of about 41 people in which everyone gets RAPA-501; main goals are safety and feasibility. Main results expected around July 2027.

• ~

  RAPA-501 Phase 1 (NCT04220190)Mixed

  2024 · Open-label · Phase 1 · 9 people · Company-run

  Everyone got RAPA-501. It was described as safe with signs of calming the immune system and trends toward slower breathing decline, but it was too small and uncontrolled to judge benefit.

Results from different trials can't be compared directly. How to read effects

The trial is for "standard-risk" ALS: symptoms for 24 months or less, breathing (slow vital capacity) at least 70% and ALSFRS-R between 34 and 45. The expanded access programme was for people with advanced ALS whose breathing was below 50%, who could not join trials.

How it's taken: IV infusion of own reprogrammed T cells

ClinicalTrials.gov lists July 2027 as the estimated primary completion date; the company has not publicly promised a date. MGH also said it plans to analyse the access programme data and seek funding for a second access programme.

The Phase 2/3 trial is enrolling at one site, Massachusetts General Hospital. The expanded access programme for advanced ALS has finished enrolling and is listed as temporarily not available. Ask your clinic or the MGH Healey Center about eligibility.

Trials and programmes

• RAPA-501 Phase 2/3 (standard-risk ALS)NCT04220190 · recruiting · Massachusetts General Hospital, Boston, USA
• Expanded access programme for advanced ALSNCT06169176 · active not recruiting · Massachusetts General Hospital, Boston, USA

Signal measures how often, and how reliably, Rapa Therapeutics 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.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

AL-S Pharma is a small private Swiss biotech near Zurich that develops only AP-101. It is co-owned by Neurimmune, which discovered the antibody, and the investor TVM Capital Life Science. It appointed a new chief executive, Harry Welten, in October 2026.

Company website ↗

SOD1 is a normal protein, but when it folds the wrong way it becomes toxic to motor neurons and can spread from cell to cell. AP-101 is designed to bind and remove only the misfolded form, leaving normal, protective SOD1 alone rather than lowering all SOD1. The idea is that this could help both SOD1-ALS and the sporadic ALS cases where misfolded SOD1 is also found.

Scientific name: Misfolded SOD1

The Phase 2 trial (AP-101-02) is complete. In October 2026 the company described a Phase 3 trial, AP101-03, of about 300 people given AP-101 or placebo for 12 months, run with the NEALS network. It now expects to start that trial in 2027, later than the "end of 2026" target it gave in March 2026.

Phase 2 done; Phase 3 planned to start in 2027

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

The trial's main goal was safety, which it met. During the 24-week placebo-controlled period, the ALS functional score (ALSFRS-R) did not differ from placebo in the main sporadic ALS group. In exploratory analyses, people who started AP-101 straight away did better on a combined death-or-ventilation measure than people who switched from placebo to AP-101 after 24 weeks (sporadic ALS p=0.013; SOD1-ALS p=0.036). Slower ALSFRS-R decline was seen only in SOD1 carriers, where the groups were not evenly matched at the start, and in people with high misfolded SOD1. These are company-reported results, not yet in a peer-reviewed journal.

All key studies

• ~

  AP-101-02 (Phase 2)Mixed

  2025 · Placebo-controlled · Phase 2 · 73 people · Company-run

  Met its main goal of safety. Function did not differ from placebo in the sporadic ALS group over 24 weeks; exploratory survival, subgroup and biomarker results looked favourable but are not yet published in a journal.

Results from different trials can't be compared directly. How to read effects

Phase 2 included 52 people with sporadic ALS and 21 with SOD1-ALS. The company says the strongest signals were in SOD1 carriers and in people with high levels of misfolded SOD1, which it hopes to measure to pick patients in future.

How it's taken: IV infusion every 3 weeks

The company says the Phase 3 trial will test 2,500 mg of AP-101 by IV every three weeks against placebo for 12 months. It gave 2027 as the start year; the exact sites and month have not been announced.

The Phase 2 trial has finished and no trial is currently enrolling. We found no announced expanded access programme. Watch for site lists when the Phase 3 trial opens.

Trials and programmes

• AP-101-02 (Phase 2)NCT05039099 · completed · Europe and North America

Signal measures how often, and how reliably, AL-S 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.

Announced milestones

• Delivered on time Present full Phase 2 results at the International Symposium on ALS/MND (promised for December 2025, delivered 5 Dec 2025)
• Dropped Start a confirmatory Phase 3 trial by the end of 2026 (promised for Q4 2026)
• Not due yet Start the Phase 3 trial in 2027 (promised for 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

The trials are run by doctors, not a company: Professor Haruhisa Inoue's team at Kyoto University's Center for iPS Cell Research and Application (CiRA), with Japanese government research funding. Pfizer Japan, which sells bosutinib for leukaemia, supplied the drug and advice for Phase 2. For Phase 3, Japanese trade press reports that generic maker Towa Pharmaceutical will supply the study drug.

Company website ↗

In nerve cells made from ALS patients' stem cells, two enzymes called Src and c-Abl are overactive. This blocks autophagy, the cell's system for clearing out damaged proteins, so misfolded proteins build up and the cells die. Bosutinib blocks these enzymes, and in lab and mouse tests it helped nerve cells survive.

Scientific name: Src/c-Abl kinases

A small Phase 1 study ran from 2019 to 2021. The Phase 2 trial started in 2022 at 7 Japanese hospitals and is listed as completed in July 2024. Preliminary results were announced in June 2024, but we could not find a full peer-reviewed report. In October 2026 Japanese trade press reported that an investigator-initiated Phase 3 trial should begin in Japan in 2027.

Open-label Phase 2 done; doctor-led Phase 3 expected in Japan in 2027

Early signal only. Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

All 26 participants took bosutinib (13 at 200 mg, 13 at 300 mg) for 24 weeks; there was no placebo group. Their ALS functional score (ALSFRS-R) fell significantly less than in the placebo group of an earlier edaravone trial, which met both main goals the researchers had set in advance. But the lead researcher said they could not show a clear advantage over the edaravone-treated group of that trial, results varied a lot between people, and one of two secondary goals was missed. Comparisons with other trials' patients are much weaker evidence than a placebo group, and the full Phase 2 results have not been published in a journal.

All key studies

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  iDReAM Phase 2Mixed

  2024 · Open-label · Phase 2 · 26 people · Company and academic

  Met its pre-set goals against the placebo group of an older edaravone trial, but missed one of two secondary goals and did not clearly beat that trial's edaravone group. Full results not yet in a journal.

• ~

  iDReAM Phase 1Mixed

  2022 · Open-label · Phase 1 · 13 people · Independent

  Safe up to 300 mg a day; all 3 people at 400 mg had dose-limiting side effects. 5 of 9 people who finished 12 weeks were stable, with no comparison group.

Results from different trials can't be compared directly. How to read effects

Phase 2 included people whose ALS began within the previous 2 years and who lost 1 to 4 points on the ALSFRS-R during a 12-week observation period. The researchers saw hints that people with lower neurofilament light (NfL, a blood marker of nerve damage) did better. Side effects included diarrhoea and liver problems, which sometimes needed dose changes or extra treatment.

How it's taken: Pill, once daily

Japanese pharmaceutical trade press reported on 1 October 2026 that the doctor-led Phase 3 is expected to start in Japan in 2027, with Towa Pharmaceutical supplying the drug. No trial has been registered yet, and design details have not been published.

No bosutinib trial for ALS is enrolling now. Bosutinib is approved for leukaemia, but it is not approved for ALS and has real side effects, so it should not be used for ALS outside a trial.

Trials and programmes

• iDReAM Phase 1 and Phase 2NCT04744532 · completed · Japan

Signal measures how often, and how reliably, Kyoto University (CiRA) 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.

Announced milestones

• Not due yet Start investigator-initiated Phase 3 in Japan (promised for 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Corcept is a profitable company in Redwood City, California, that sells cortisol-blocking medicines for Cushing's syndrome (Korlym) and ovarian cancer (Lifyorli). It reported $340.5 million in revenue for the third quarter of 2026 and $662.3 million in cash and investments, so money is not the main constraint. ALS is one of several programmes; most of its effort goes to hormone disorders and cancer.

Company website ↗

Cortisol is a stress hormone. Dazucorilant attaches to the glucocorticoid receptor, the docking site cortisol uses, and blocks or tunes down its signal. The idea is that too much cortisol signalling may add to nerve damage and inflammation in ALS. How this would protect motor neurons is not yet proven.

Scientific name: Selective glucocorticoid receptor modulator (cortisol modulator)

DAZALS, a placebo-controlled Phase 2 trial of 249 people in Europe, the US and Canada, reported in December 2024 that it missed its main goal. Its open-label extension continues, and a second part of the study is testing whether starting at a low dose and slowly increasing it reduces stomach pain. Corcept has moved the planned Phase 3 start three times: by mid-2026, then later in 2026, then early 2027; in November 2026 it said only "next year". The FDA has granted Fast Track and orphan drug status.

Phase 2 done (missed main goal); Phase 3 planned for 2027

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

Over 24 weeks, the drug did not slow the loss of daily function (ALSFRS-R), the main goal: people on either dose lost about 7 to 7.5 points, and people on placebo 6.8. On survival, nobody on 300 mg died in those 24 weeks versus 5 of 82 on placebo. The headline 84% (one year) and 87% (two years) lower risk of death compare the 83 people on 300 mg with only the 18 placebo patients who never switched to the drug, an exploratory comparison that can be biased. Counting everyone as first assigned, deaths by April 2026 were 22 of 83 on 300 mg, 27 of 83 on 150 mg and 32 of 82 on placebo.

All key studies

• ~

  DAZALS 1- and 2-year survival analysesMixed

  2026 · Post-hoc analysis · Phase 2 · 249 people · Company-run

  The 84-87% lower risk of death compares 83 people on 300 mg with only 18 placebo patients who never switched to the drug. Counting everyone as first assigned, deaths were 22 of 83 on 300 mg and 32 of 82 on placebo.

• ~

  DAZALSMixed

  2024 · Placebo-controlled · Phase 2 · 249 people · Company-run

  Missed its main goal: over 24 weeks, function declined about as much on either dose (about 7 to 7.5 points) as on placebo (6.8). Survival, a secondary goal, was better on 300 mg (0 vs 5 deaths), but many people on the drug stopped early because of stomach pain.

Results from different trials can't be compared directly. How to read effects

DAZALS enrolled adults with ALS at 35 sites in 10 countries in Europe and North America. The dose being taken forward is 300 mg, taken as capsules once a day. Stomach pain was the main side effect: BioSpace reports that about 58% of people on 300 mg stopped the drug because of side effects, mostly stomach-related.

How it's taken: Pill, once daily

In July 2026 Corcept said the Phase 3 trial would start early in 2027; in its November 2026 update it said only that the trial is planned to begin next year. The goal is to confirm the survival benefit. Design details, including sites, have not been published yet.

The DAZALS registry entry is listed as active but not recruiting, so new people cannot join. The Phase 3 trial is not yet registered or enrolling. We found no expanded access programme.

Trials and programmes

• DAZALS (Phase 2, with dose-titration part)NCT05407324 · active not recruiting · Europe, USA, Canada

Signal measures how often, and how reliably, Corcept Therapeutics 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.

Announced milestones

• Delivered on time One-year overall survival results from DAZALS (promised for Q2 2025, delivered 5 Jun 2025)
• Dropped Start Phase 3 trial by mid-2026 (promised for First half of 2026)
• Dropped Start Phase 3 trial later in 2026 (promised for 2026)
• Not due yet Start Phase 3 trial early in 2027 (promised for First half of 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Woolsey is a small private company in New York. It raised extra Series B funding in February 2025 to advance its ALS programme. Its website's newsroom has not posted anything since that announcement, and the study results came out as a journal article in June 2026 rather than a company press release.

Company website ↗

Fasudil blocks Rho kinase (ROCK), an enzyme that becomes overactive in ALS and is thought to damage the connections between nerves and muscles, increase inflammation and stop nerve fibres regrowing. In the oral study, a marker of ROCK being blocked (pAKT) rose in tiny particles from nerve cells in the blood and spinal fluid, suggesting the drug reaches its target.

Scientific name: Rho kinase (ROCK)

The REAL study tested two doses (180 mg and 300 mg a day) in two groups of 31 people for 24 weeks in the US and Australia. Results were published in June 2026. The registry still lists the study as active, not recruiting, with long-term follow-up to 2029. The authors say the 180 mg dose should now be tested against placebo, but as of October 2026 no such trial was registered.

Open-label Phase 2a done; placebo-controlled trial not yet announced

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In the 180 mg group, blood neurofilament light (NfL, a marker of nerve damage that normally creeps up) fell by 15.4% over 24 weeks; at 300 mg it did not change. Function, breathing and muscle strength results were not statistically significant. Everyone got the drug, so the study cannot show it slows ALS; a company comparison with patients from an older trial also found no significant difference in function. An earlier placebo-controlled trial of fasudil given into a vein for 20 days (ROCK-ALS, 118 people) found it safe but showed no effect on function or survival.

All key studies

• ~

  REAL study (oral fasudil, 180 and 300 mg)Mixed

  2026 · Open-label · Phase 2a · 62 people · Company-run

  Met its main goal of safety. Blood NfL fell 15.4% at 180 mg but not at 300 mg; function, breathing and strength changes were not statistically significant.

• ~

  ROCK-ALS (intravenous fasudil)Mixed

  2024 · Placebo-controlled · Phase 2 · 118 people · Independent

  Met its main goals of safety and tolerability. Fasudil by drip for 20 days did not change ALSFRS-R decline or survival over 180 days; one nerve-muscle measure (MUNIX) looked better.

• ~

  REAL 180 mg vs matched patients from an older trialMixed

  2024 · Post-hoc analysis · 31 people · Company-run

  Compared with 31 matched patients from the ceftriaxone ALS trial, function declined 28% more slowly but this was not significant (p=0.12); breathing decline was 42% slower (p=0.05).

Results from different trials can't be compared directly. How to read effects

The oral study enrolled adults with ALS at sites in the United States and Australia. More people stopped early at the higher dose (71% finished) than the lower dose (81%). Side effects were mostly mild.

How it's taken: Pill, daily

The published study recommends a double-blind, placebo-controlled trial of the 180 mg dose. Woolsey has not announced plans, timing or funding for it, and an October 2026 registry search by the Compass project found no such trial.

The oral fasudil study is no longer enrolling and no expanded access programme has been announced. Fasudil is not approved in the US or Europe.

Trials and programmes

• REAL study (oral fasudil, Phase 2a)NCT05218668 · active not recruiting · USA, Australia

Signal measures how often, and how reliably, Woolsey Pharmaceuticals 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.

Announced milestones

• Delivered late Report results from the 300 mg cohort (promised for First half of 2025, delivered 29 Jun 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Tiziana is a small Nasdaq-listed biotech that issues its news from Boston and whose pipeline is built around foralumab, tested in multiple sclerosis, Alzheimer's disease, multiple system atrophy and ALS. In January 2026 it raised $8.8 million. The ALS trial is co-funded by the ALS Association through a $1 million Hoffman award, with extra support from two ALS foundations.

Company website ↗

Foralumab attaches to CD3, a marker on immune T cells. Given in small doses through the nose, it is meant to boost regulatory T cells (Tregs), immune cells that calm inflammation, which then travel to the brain and spinal cord. Inflammation from immune cells called microglia is thought to speed up ALS. In multiple sclerosis studies the spray reduced signs of microglia activity on brain scans.

Scientific name: CD3 on T cells (anti-CD3 monoclonal antibody)

Tiziana filed its US trial application for ALS in March 2025. The trial was accepted into the Healey ALS MyMatch programme at Mass General Brigham in November 2025, and the first three participants were dosed in September 2026. The trial was first described as a 20-person study and has grown to 44 people.

Phase 2a BANYAN trial began dosing in September 2026

Results pending. Trials are running, but there are no efficacy results yet.

BANYAN is the first clinical study of foralumab in ALS, so there is no evidence yet that it helps in ALS. Its main goal is safety. It will also look at immune cells, the nerve-damage marker NfL, and brain inflammation on PET scans, not at whether people decline more slowly.

All key studies

• …

  BANYAN (Phase 2a, NCT07688239)Pending

  2026 · Placebo-controlled · Phase 2a · 44 people · Company and academic

  44 people with early ALS get nasal foralumab or placebo (3 to 1) for 12 weeks, then all get foralumab. Main goal is safety; first participants dosed in September 2026.

Results from different trials can't be compared directly. How to read effects

BANYAN is enrolling adults aged 18 to 75 whose first symptoms began within the last 24 months, whose breathing capacity is at least 65% of normal, and whose blood NfL level is at least 40 pg/mL. Three in four people get foralumab and one in four placebo for 12 weeks, then everyone gets foralumab for 12 more weeks. The spray is used three times a week, two weeks on and one week off, mostly at home.

How it's taken: Nasal spray, 3 times a week

The trial registry estimates the main data will be collected by July 2027 and the study will finish in November 2027. Tiziana itself has not given a date for results.

BANYAN is run through Healey ALS MyMatch, which screens people at Mass General in Boston, Northwestern in Chicago, the University of Minnesota and Nova Southeastern in Florida. The registry lists it as enrolling by invitation, meaning people join through these centres' screening rather than by signing up directly. We found no expanded access for ALS.

Trials and programmes

• BANYAN (Phase 2a)NCT07688239 · enrolling by invitation · Boston MA, Chicago IL, Minneapolis MN, Davie FL

Signal measures how often, and how reliably, Tiziana Life Sciences 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.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

IFB-088 was developed by InFlectis BioScience, a small biotech in Nantes, France, which a court placed in judicial liquidation in September 2025. Its former chief executive and finance chief set up a new company in November 2025, renamed Enemsa Pharma in early 2026, which now presents icerguastat as its lead drug and is seeking pharmaceutical partners for ALS. We could not find a public statement explaining how the drug rights moved to Enemsa.

Company website ↗

When nerve cells are stressed by misfolded proteins, they briefly slow down protein production to recover. IFB-088 stretches out this recovery period, giving the cell more time to cope. The company says it also dampens a type of glutamate receptor (NR2B-containing NMDA receptors) that can overexcite nerve cells, and helps cells recycle damaged mitochondria. In mice and cell studies it reduced TDP-43 clumps, a hallmark of most ALS.

Scientific name: PPP1R15A/PP1c (integrated stress response); NR2B-NMDA receptors

The Phase 2 trial in France and Italy finished in January 2025. A larger Phase 2b trial has been discussed since mid-2025, but needs a partner to pay for it, and none has been announced. The original company was liquidated in September 2025. IFB-088 has orphan drug designation for ALS from the FDA and the European Medicines Agency.

Small Phase 2 done; no further trial planned or funded

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

The trial's main goal was safety, and it was too small to prove benefit. In February 2025 the company announced a statistically significant slowing among the 40 people who followed the protocol (0.95 vs 1.42 ALSFRS-R points lost per month). The later full poster shows this was judged against a loose threshold (p=0.209), that the difference was not significant across all patients, and that breathing results did not differ. A post-hoc adjustment for nerve-damage levels (NfL), because the drug group started out sicker, gave p=0.11.

All key studies

• ~

  P288ALS TRIAL (bulbar-onset ALS)Mixed

  2025 · Placebo-controlled · Phase 2 · 51 people · Company-run

  The main goal was safety, and the drug was judged reasonably safe, though 5 of 34 people on it died versus 2 on placebo. Slowing of decline was not significant in all patients and only approached the trial's loose threshold in a smaller per-protocol group.

Results from different trials can't be compared directly. How to read effects

The trial enrolled 51 people whose ALS started with speech or swallowing problems, at 9 sites in France and Italy, and they kept taking riluzole. Two people received the drug for every one on placebo. Stomach side effects were more common on the drug (35.3% vs 11.8%), and serious side effects were similar (23.5% in each group). Seven people died (5 on the drug, 2 on placebo), and two sudden deaths on the drug were judged possibly related.

How it's taken: Pill, twice daily

Enemsa says it is seeking pharmaceutical partners to take icerguastat forward in ALS. It has not announced a Phase 2b trial, a start date, or a partner. Its publications page lists the Phase 2 paper as accepted in the journal Brain, but we could not confirm a publication date.

The only ALS trial finished in January 2025, and no new trial is registered. We found no expanded access programme, so the drug is not available to patients at present.

Trials and programmes

• IFB-088 Phase 2 in bulbar-onset ALSNCT05508074 · completed · France, Italy

Signal measures how often, and how reliably, Enemsa Pharma (formerly InFlectis BioScience) 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.

Announced milestones

• Delivered late First results from the Phase 2 trial (promised for Q4 2024, delivered 6 Feb 2025)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

MIROCALS was an academic trial sponsored by the University Hospital of Nîmes (France) and run by a European consortium, with funding from the EU and the UK MND Association. The Paris company ILTOO Pharma holds exclusive rights to the trial data and makes the IL-2 product (ILT-101), so any approval application would likely come from ILTOO.

Company website ↗

In ALS, immune cells in the nervous system can become overactive and add to nerve damage. Regulatory T cells normally keep this in check, and they tend to be weaker in faster progressing ALS. Small doses of IL-2 selectively increase regulatory T cells without strongly activating other immune cells.

Scientific name: Regulatory T cells (Tregs) via IL-2

MIROCALS was a Phase 2b placebo-controlled trial and reported in The Lancet in May 2025. UK ALS experts said a Phase 3 trial (or a funded early access programme) would be needed before the drug could be recommended. As of November 2026 we found no announced confirmatory trial, though the programme has not been formally stopped.

Phase 2b done; main goal missed; no follow-up trial announced

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

For everyone in the trial, 41 people on IL-2 (37%) and 49 on placebo (45%) had died by 21 months, a difference that was not statistically significant (hazard ratio 0.81, p=0.33), so the main goal was missed. In the 70% of people with lower levels of a nerve-damage marker (pNFH) in their spinal fluid, the risk of death was 48% lower with IL-2 (hazard ratio 0.52, p=0.016), and there was no benefit in people with high levels. This subgroup was planned in advance, but it still needs to be confirmed in a new trial.

All key studies

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  MIROCALSMixed

  2025 · Placebo-controlled · Phase 2b · 220 people · Independent

  Missed its main goal: survival at 21 months was not significantly better overall (37% vs 45% died). In the 70% of people with lower spinal-fluid pNFH, risk of death was 48% lower, a planned analysis that still needs confirming.

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  IMODALSMixed

  2020 · Placebo-controlled · Phase 2a · 36 people · Independent

  Met its main goal, which was biological: IL-2 raised regulatory T cells versus placebo over 3 months. It was not designed to show a clinical benefit, and the nerve-damage marker NfL did not differ between groups.

Results from different trials can't be compared directly. How to read effects

MIROCALS enrolled people early after diagnosis who were already taking riluzole. Treatment was a small injection under the skin on five days in a row, repeated every 28 days for 18 months. The apparent benefit was in people with lower pNFH, which requires a spinal fluid test.

How it's taken: Injection under skin, 5 days a month

In 2025 ILTOO said it was talking with European and US regulators about faster access, and UK experts called for a Phase 3 trial. As of November 2026 we could not find a public plan, date or funding for either. A UK patient campaign said in February 2026 it had seen no new activity.

MIROCALS has finished. ILTOO Pharma has run a managed access programme in some countries, including France, the UK and Austria, but in the UK it has been limited to former MIROCALS participants, and the drug is not available on the NHS. Ask your neurologist whether any access route exists where you live.

An expanded access (compassionate use) programme exists. Ask your neurologist.

Trials and programmes

• MIROCALSNCT03039673 · completed · France, UK, other European sites

Signal measures how often, and how reliably, MIROCALS consortium (CHU de Nîmes); rights held by ILTOO 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.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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NeuroSense is a small company based in Israel and Cambridge, Massachusetts, and PrimeC is its only drug. It is financially fragile: in mid-2026 it reported about $231,000 in cash, it is fighting to keep its Nasdaq listing, and it says the large Phase 3 trial needs outside funding that has taken longer than expected to find. A partnership with an unnamed big pharma company, announced as a term sheet in December 2024, has not been finalised.

Company website ↗

Ciprofloxacin is an antibiotic that in lab studies affects how cells process RNA (genetic messages), and celecoxib is an anti-inflammatory painkiller. Together they are meant to hit several things that go wrong in ALS at once: inflammation, oxidative stress, iron build-up and faulty RNA handling. The company also reports that PrimeC lowered TDP-43, the protein that clumps in nerve cells in almost all ALS, in blood-based measurements.

Scientific name: Ciprofloxacin + celecoxib (fixed-dose combination)

The Phase 2b PARADIGM trial is finished, and no Phase 3 trial has started. In November 2025 the FDA allowed the company to start a Phase 3 trial called PARAGON (about 300 people, 12 months), but it has not enrolled anyone because the money to run it has not been secured. The company is now redesigning it to be smaller and shorter, possibly with a head-to-head comparison against edaravone. Separately it plans to ask Health Canada for approval based on the Phase 2b data.

Phase 2b done; Phase 3 cleared by FDA but not started; Canada filing planned

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

In PARADIGM, 68 people took PrimeC or placebo for 6 months, then everyone took PrimeC. At 6 months, the only placebo-controlled comparison, the difference in the ALS functional score (ALSFRS-R) was 2.2 points and not statistically significant. At 18 months, people who started PrimeC first were 7.9 points better off than those who started on placebo, and a later company analysis reports a 65% lower risk of death. These come from the open-label period, compare small groups (45 vs 23 people), and the survival analysis has only been released by the company, so a larger trial is needed to confirm any benefit. The trial's main goals were safety and a blood marker (TDP-43).

All key studies

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  PARADIGM long-term follow-up (early vs delayed start)Mixed

  2026 · Post-hoc analysis · Phase 2b · 68 people · Company-run

  After everyone switched to PrimeC, people who started it first had 7.9 points better function at 18 months and, in a company analysis, a 65% lower risk of death. Small groups and the open-label switch make this hard to interpret.

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  PARADIGMMixed

  2023 · Placebo-controlled · Phase 2b · 68 people · Company-run

  Met its main goal of safety. At 6 months the function difference versus placebo (2.2 points) was not statistically significant. The company later reported that its TDP-43 blood-marker goal was met (press release only).

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  NST002 (earlier formulation)Mixed

  2022 · Open-label · Phase 2a · 15 people · Company and academic

  12 months of an earlier, three-times-daily form of PrimeC with no placebo group. It was safe and blood markers changed, but decline was not significantly slower than in historical patients.

Results from different trials can't be compared directly. How to read effects

PARADIGM enrolled adults with sporadic or familial ALS, who could keep taking riluzole or edaravone. The redesigned Phase 3 plan would preferentially enrol people earlier in their disease, and the new NfL analysis suggested larger effects in people with higher starting function.

How it's taken: Pill, twice daily

NeuroSense said on 2 November 2026 that it will file in March 2027 instead of December 2026 so it can add a new neurofilament (NfL) analysis. If Health Canada accepts the file, a standard review takes about 300 days, so a decision would not come before 2028. The company also expects a decision on a grant to fund Phase 3 in Q1 2027.

No PrimeC trial is enrolling right now, and we found no ClinicalTrials.gov record for PARAGON yet. In October 2026 the company signed an agreement aiming to sell PrimeC to eligible patients through a licensed clinic in one US state, using a pathway that allows some investigational drugs before approval, with first sales targeted for the first half of 2027. Details such as the state and eligibility have not been published.

Trials and programmes

• PARADIGM (Phase 2b)NCT05357950 · completed · Canada, Israel, Italy
• PARAGON (Phase 3, planned)not yet recruiting · Mainly USA (planned)

Signal measures how often, and how reliably, NeuroSense Therapeutics 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.

Announced milestones

• Overdue Sign definitive partnership agreement with global pharma company (promised for Q1 2025)
• Overdue Start Phase 3 trial (promised for Second half of 2025)
• Delivered late Pre-NDS meeting with Health Canada (promised for April 2026, delivered 22 Jul 2026)
• Dropped File New Drug Submission in Canada by mid-2026 (promised for First half of 2026)
• Dropped File New Drug Submission in Canada in December 2026 (promised for December 2026)
• Not due yet File New Drug Submission in Canada in March 2027 (promised for March 2027)
• Not due yet Decision on non-dilutive funding for Phase 3 PARAGON (promised for Q1 2027)
• Not due yet First revenue from PrimeC supply via US clinic (promised for First half of 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Spinogenix is a private company in Los Angeles. Tazbentetol is its lead drug and it is also testing it in Alzheimer's disease and schizophrenia, so many of its press releases are about those diseases. It issues frequent news releases about the drug.

Company website ↗

In ALS, nerve cells lose their synapses, the contact points that pass signals to other nerve cells and on to muscles. Tazbentetol is designed to make nerve cells grow new excitatory (glutamatergic) synapses. This is a new approach, and it has not yet been proven to work in people.

Scientific name: Glutamatergic synapse formation

The Phase 2a study in Australia is complete. The FDA granted Fast Track status in May 2026. The Phase 2b/3 trial (about 430 people, 36 weeks of drug or placebo) was first registered in January 2026 with a start date of March 2026; in September 2026 the start date was moved to October 2026. The registry still showed it as not yet recruiting, with no sites listed, as of early November 2026.

Phase 2a done; Phase 2b/3 registered but not yet recruiting

Hints only. Controlled trials missed their main goal. Some measures or subgroups looked better, which needs confirming.

The Phase 2a study's main goal was safety. It gave 23 people drug or placebo (18 and 5) for only 28 days, too short to compare decline, then everyone took 300 mg a day for another 140 days. The company reports that 82% had a stable or better rate of decline, and that on average they declined 76% more slowly than matched patients in an old database of trial records (PRO-ACT). People who join a new trial often differ from past patients, so such comparisons can make a drug look better than it is. Results are from a company poster and press release, not a peer-reviewed journal.

All key studies

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  SPARK Phase 2b/3 (NCT07325591)Pending

  2026 · Placebo-controlled · Phase 2b/3 · 430 people · Company-run

  About 430 people to get tazbentetol or placebo for 36 weeks. Registered in January 2026 but not yet recruiting as of early November 2026.

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  SPG302 Phase 2a in ALS (NCT05882695)Mixed

  2025 · Placebo-controlled · Phase 2a · 23 people · Company-run

  Met its main goal of safety. The placebo period lasted only 28 days; slower decline was claimed only against historical PRO-ACT records during the open-label period, and is unpublished.

Results from different trials can't be compared directly. How to read effects

The Phase 2b/3 trial plans to enrol adults aged 18 to 80 with ALS who are on stable standard treatment, are within a set range on the TRICALS risk score, and do not use breathing support.

How it's taken: Pill, once daily

The registry lists an estimated start of 1 October 2026, but the trial was still marked "not yet recruiting" in early November 2026, after the start date had already been pushed back once from March 2026. Spinogenix has not issued a press release about the start.

Since May 2025 the FDA has allowed an expanded access programme in the US for up to 200 adults with ALS who cannot join a trial. Spinogenix says eligibility criteria apply; ask your ALS clinic or contact the company.

An expanded access (compassionate use) programme exists. Ask your neurologist.

Trials and programmes

• SPARK Phase 2b/3NCT07325591 · not yet recruiting
• Phase 1/2a studyNCT05882695 · completed · Australia

Signal measures how often, and how reliably, Spinogenix 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.

Announced milestones

• Delivered on time Present full Phase 2a data at the ALS/MND Symposium on 5 December 2025 (promised for December 2025, delivered 5 Dec 2025)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Eledon is a Nasdaq-listed biotech in Irvine, California. Its main programme is tegoprubart to prevent rejection after kidney transplants. ALS is listed in its pipeline as a completed Phase 2 biomarker study; a June 2026 company presentation said Eledon is seeking non-equity financing to take it to Phase 3.

Company website ↗

CD40 ligand is a protein on immune cells that switches on inflammatory responses. In ALS, inflammation in the nervous system is thought to add to motor neuron damage. Tegoprubart blocks CD40 ligand with the aim of turning down that inflammation.

Scientific name: CD40 ligand (CD154)

The 54-person Phase 2a trial finished in 2022 and was published in 2024. In its August 2026 quarterly report, Eledon said it cannot continue ALS development without additional funding dedicated to ALS. No Phase 3 trial has been scheduled.

Phase 2a done in 2022; ALS work on hold until funding found

Early signal only. Only small, open-label or historical-comparison studies so far. Promising at best; not proof.

This was a small, open-label trial without a placebo group, testing four dose levels over 12 weeks. It was safe and well tolerated, and 18 pro-inflammatory markers in the blood went down. The ALS functional score (ALSFRS-R) fell by 0.99 points a month across all 54 people, which the authors describe as similar to the natural course of ALS, and there was no dose-related pattern. The company's comparison with past trial patients only looked favourable after leaving some participants out. The study was not designed to show an effect on symptoms or survival.

All key studies

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  Tegoprubart Phase 2a in ALS (NCT04322149)Mixed

  2022 · Open-label · Phase 2a · 54 people · Company-run

  Met its main goals of safety and lowering inflammation markers over 12 weeks. Function declined about as fast as is usual in ALS (0.99 ALSFRS-R points a month), with no dose pattern.

• ✗

  Company comparison with PRO-ACT historical recordsNo benefit

  2022 · Post-hoc analysis · 54 people · Company-run

  Across all 54 people, decline was about 22% faster than in matched past placebo patients. Slower decline appeared only after the company removed 7 people and those without target engagement, an after-the-fact choice.

Results from different trials can't be compared directly. How to read effects

The Phase 2a trial enrolled 54 adults with ALS at sites in the US and Canada. Infusions were given every two weeks, six times in total.

How it's taken: IV infusion

Eledon has said since early 2025 that it wants to run a fully powered ALS Phase 3 trial but only with outside funding such as grants or partners. It has not announced any such funding or a start date.

The only ALS trial is finished, and no expanded access programme for ALS has been announced. Current tegoprubart trials are in kidney transplantation.

Trials and programmes

• Phase 2a open-label study in ALSNCT04322149 · completed · USA, Canada

Signal measures how often, and how reliably, Eledon Pharmaceuticals 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.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

See the timeline of news and updates

Nura Bio is a private biotech in South San Francisco and NB-4746 is its lead drug. It raised more than $140 million in its first funding rounds and a further $73.8 million in June 2026, led by The Column Group with Euclidean Capital, Samsara BioCapital and Sanofi Ventures. It is also testing a second SARM1 blocker, NB-9402, in healthy volunteers.

Company website ↗

SARM1 is an enzyme inside nerve cells that, once switched on by injury or stress, burns up the cell's energy molecule NAD and makes the long nerve fibre (axon) fall apart. Blocking it protects nerve fibres in lab and animal studies, and rare overactive versions of the SARM1 gene are found more often in people with ALS. But removing SARM1 entirely did not slow disease in the most widely used ALS mouse model (SOD1), so it is unclear how much this pathway drives ALS. Eli Lilly is also starting trials of its own SARM1 blocker in ALS.

Scientific name: SARM1 inhibitor (reversible, oral, brain-penetrant)