People with Down syndrome have a third copy of chromosome 21, which carries the gene for an enzyme called DYRK1A; the extra dose is linked to cognitive impairment. Perha Pharmaceuticals, founded in Roscoff, Brittany, in 2019, is developing Leucettinib-21, a DYRK1A inhibitor derived from a molecule found in a marine sponge, to improve memory and learning in people with Down syndrome and Alzheimer's disease.
The European Innovation Council granted €2,500,000 towards DOWN-AUTONOMY, an €11.36 million project running from June 2022 to June 2026, as part of blended finance of about €8 million according to the company's grant adviser. Its CORDIS report records completed preclinical toxicology and approval to start Phase 1. The Phase 1 trial, registered as NCT06206824, enrolled 152 participants and was completed on 25 June 2026; no results have been posted. The Phase 2a efficacy trial, the project's last milestone, has not begun.
Company file
- Legal entity
- Perha Pharmaceuticals SAS, SIREN 850 455 429, Roscoff
- Founded
- 18 April 2019
- Co-founder and CSO
- Laurent Meijer
- Drug
- Leucettinib-21, oral DYRK1A kinase inhibitor
- Investors
- Jérôme Lejeune Foundation, business angels, Bpifrance, crowdfunding via Capital Cell
- EIC project
- DOWN-AUTONOMY, 1 Jun 2022 to 30 Jun 2026
- EIC funding
- Blended finance, about €2.5M grant and €5.5M equity, per Catalyze
Where Perha Stands Today
The Phase 1 trial tested Leucettinib-21 in healthy volunteers and in adults with Down syndrome and with Alzheimer's disease, starting in January 2024. ClinicalTrials.gov lists it as completed in June 2026 without results. The French register shows share capital rising from €190,250 to €546,310 in August 2024 and €683,240 by January 2026, a series of capital increases whose investors and amounts have not been announced. The company has no insolvency proceedings.
The Technology: Turning Down an Overactive Enzyme
According to the CORDIS report, Leucettinib-21 showed target engagement in cells and animals and improved memory and learning in Ts65Dn mice, an animal model of Down syndrome, with three papers in the Journal of Medicinal Chemistry. A paper in PubMed Central reported that it reduced the effects of the extra DYRK1A dose in neural cells derived from people with trisomy 21. None of this shows benefit in people yet.
What the EIC Project Promised, and What CORDIS Shows
DOWN-AUTONOMY was to take Leucettinib-21 through toxicology, a Phase 1 safety trial and a Phase 2a trial of efficacy. The report, covering the period to November 2023, says the company "needs to raise series A funds to co-finance preparation and conduct of the phase 2a clinical trial". No Series A has been announced. The project's end date has now passed with Phase 1 complete and Phase 2a not started.
- Preclinical toxicologyDeliveredPhase 1 authorised.
- Phase 1 safety trialCompleted152 people; no results posted.
- Phase 2a efficacy trialNot startedSeries A needed.
What Has Happened Since the Award
Perha raised €3.1 million in autumn 2021, €1.5 million of it from the Jérôme Lejeune Foundation, and in March 2024 opened a crowdfunding campaign on Capital Cell, naming the EIC, the Lejeune Foundation, France 2030 and the national research agency among the trial's funders.
The Money
The net losses recorded by Pappers were €1.71 million in 2023, €2.04 million in 2024 and €2.49 million in 2025. Whether the EIC Fund has invested the equity part of the blended award has not been announced.
What cannot be checked
The Phase 1 safety results, whether the EIC Fund took its planned equity stake, who funded the 2024 to 2026 capital increases, and when Phase 2a will start are not public.
Why Europe Should Care
The EIC backed a small French company with a first-in-class approach and paid part of its first human trial. The public will learn whether that was a good bet only when the safety data are published and an efficacy trial is funded.
What the project promised
Leucettinib-21 through Phase 1 and a Phase 2a efficacy trial in Down syndrome.
What the record shows
Toxicology done, Phase 1 completed without posted results, Phase 2a unfunded at the project's end.
The Verdict
Halfway through the plan
DOWN-AUTONOMY got Leucettinib-21 into people and through a completed safety trial. The efficacy trial it also promised depends on a Series A that has not been announced, and the Phase 1 results are not yet public.
Perha Pharmaceuticals has not been asked for comment for this article. It is based entirely on company statements, French company data, clinical registries and EU project data, linked throughout.
