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Early pipeline

AP-2

Bottom line

Very early: a first safety test in healthy volunteers that began in 2026. It helped nerve cells in lab and animal tests, but most drugs at this stage never become approved treatments.

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

AP-2 is an experimental drug taken by mouth, discovered at Spain's national research council (CSIC). It blocks an enzyme called CK1δ to help the TDP-43 protein go back to its normal place in nerve cells. Its first human trial, in healthy volunteers in Madrid, began in 2026. A small trial in people with ALS is planned for 2027.

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

CompanyMolefy PharmaExplanation and sources

Molefy Pharma is a small Madrid company founded in 2024 as a spin-off of CSIC, Spain's national research council, and majority-owned by the ARQUIMEA group. AP-2 is its most advanced drug. In December 2025 the ALS Association gave it about $1 million through a Hoffman award to support early clinical testing.

Sources

  1. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
  2. AEMPS authorizes the initiation of the clinical trial of AP-2, Molefy Pharma's therapeutic candidate for ALSMolefy Pharma · Company press release
  3. ALS Association commits $2 million to support early-stage clinical trials of two emerging ALS therapiesThe ALS Association · 4 Dec 2025 · Patient organisation
How it worksDrug taken by mouth, meant to put TDP-43 back where it belongsCK1δ (casein kinase 1 delta) inhibitorExplanation and sources

In ALS the protein TDP-43 gets extra chemical tags (phosphorylation), leaves the cell nucleus and builds up where it should not be, so nerve cells stop making some proteins they need. AP-2 blocks CK1δ, one of the enzymes that adds these tags, to help TDP-43 return to the nucleus and work normally.

Sources

  1. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
  2. AEMPS authorizes the start of the clinical trial of AP-2, a promising drug for ALS discovered at the CIB Margarita SalasCIB Margarita Salas (CSIC) · 18 Mar 2026 · News
StagePhase 1Phase 1a in healthy volunteers, about 72 people · Dosing: healthy volunteersExplanation and sources

Spain's medicines agency authorised the first human trial in March 2026. It gives rising doses to healthy men at Hospital Universitario de La Princesa in Madrid and started in spring 2026. In September 2026 Molefy said results so far looked favourable for safety and that the trial should finish in early 2027. The European Medicines Agency gave AP-2 orphan drug status in October 2025; that is not an approval.

Sources

  1. First administration of AP-2 to humans (EU CT 2025-524480-21-00)EU Clinical Trials Information System · Trial registry
  2. AEMPS authorizes the initiation of the clinical trial of AP-2, Molefy Pharma's therapeutic candidate for ALSMolefy Pharma · Company press release
  3. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
Best reported effectNo ALS data yetSafety and drug levels in healthy volunteersExplanation and sources

No one with ALS has taken AP-2. Molefy reports that in cells from patients and in animal models it reduced the harmful tags on TDP-43, moved it back to the nucleus and helped motor neurons survive. These are lab and animal 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. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
  2. AEMPS authorizes the initiation of the clinical trial of AP-2, Molefy Pharma's therapeutic candidate for ALSMolefy Pharma · Company press release
Who it's forAimed at ALS with TDP-43 problems; trial is in healthy menAll ALS · By mouthExplanation and sources

TDP-43 problems are found in almost all people with ALS, so Molefy is aiming at ALS broadly, and later possibly other diseases such as a form of frontotemporal dementia. The current trial only takes healthy volunteers.

Sources

  1. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
  2. First administration of AP-2 to humans (EU CT 2025-524480-21-00)EU Clinical Trials Information System · Trial registry
What's nextEnd of healthy-volunteer trial, then a small trial in people with ALSExpected 2027Explanation and sources

Molefy says the healthy-volunteer trial should finish in early 2027, followed by a Phase 1b study in a small group of people with ALS. CSIC has said recruitment for that study could start in January 2027; the company itself says "from 2027".

Sources

  1. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release
  2. AEMPS authorizes the initiation of the clinical trial of AP-2, Molefy Pharma's therapeutic candidate for ALSMolefy Pharma · Company press release
  3. AEMPS authorizes the start of the clinical trial of AP-2, a promising drug for ALS discovered at the CIB Margarita SalasCIB Margarita Salas (CSIC) · 18 Mar 2026 · News
Can I join?Healthy volunteers only; people with ALS can't join yetExplanation and sources

The only trial takes healthy volunteers at one hospital in Madrid. People with ALS cannot join until the planned Phase 1b study opens. We found no expanded access programme.

Sources

  1. First administration of AP-2 to humans (EU CT 2025-524480-21-00)EU Clinical Trials Information System · Trial registry
  2. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release

Signal: Steady

Signal measures how often, and how reliably, Molefy 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 update16 Sept 2026 (50 days ago)32 / 40
Company updates in the last 12 months16 / 35
Promises keptNot enough history yet (neutral score)12.5 / 25
Signal score51 / 100 · Steady

Announced milestones

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

Timeline

3 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.

2026

  1. Press releaseFrom the companyMolefy Pharma

    Molefy says early AP-2 safety data look favourable

    Presenting its strategy, Molefy said the healthy-volunteer trial is under way with favourable data so far, should finish in early 2027, and will be followed by a small Phase 1b in ALS.

  2. NewsCIB Margarita Salas (CSIC)

    Spain authorises first human trial of AP-2

    CSIC reported that the Spanish medicines agency had authorised a Phase 1 trial in about 70 healthy volunteers in Madrid, with a study in people with ALS possibly from January 2027.

2025

  1. NewsThe ALS Association

    ALS Association funds early testing of AP-2

    The ALS Association announced Hoffman awards for two early-stage ALS drugs, including about $1 million for Molefy Pharma's AP-2.

All sources for this page

  1. First administration of AP-2 to humans (EU CT 2025-524480-21-00)EU Clinical Trials Information System · Trial registry
  2. ALS Association commits $2 million to support early-stage clinical trials of two emerging ALS therapiesThe ALS Association · 4 Dec 2025 · Patient organisation
  3. AEMPS authorizes the initiation of the clinical trial of AP-2, Molefy Pharma's therapeutic candidate for ALSMolefy Pharma · Company press release
  4. AEMPS authorizes the start of the clinical trial of AP-2, a promising drug for ALS discovered at the CIB Margarita SalasCIB Margarita Salas (CSIC) · 18 Mar 2026 · News
  5. Molefy Pharma presents its strategy to develop new therapies for neurodegenerative diseases such as ALSMolefy Pharma · 16 Sept 2026 · Company press release