After several years of research, a first clinical trial on healthy volunteers and then on a few adults with Down syndrome (trisomy 21) or Alzheimer’s (phase 1) has been authorized to test the safety of Leucettinib-21, a drug developed by Perha Pharmaceuticals with the support of the Fondation Jérôme Lejeune. The trial will start early 2024. The drug is designed to correct cognitive deficits and improve quality of life and autonomy in people with trisomy 21 and Alzheimer’s patients.

For over 10 years, the Fondation Jérôme Lejeune has been providing financial support and backing along with other partners (the French National Research Agency or ANR, FUI, France 2030, Bpifrance, European Innovation Council) Dr. Laurent Meijer’s research work from the biotechnology company Perha Pharmaceuticals with a view to develop a drug candidate for people with Down syndrome.

Perha Pharmaceuticals‘ drug candidate, Leucettinib-21 was selected on the basis of multiple parameters: in vitro and in vivo safety, oral bioavailability, favorable pharmacological properties and strong intellectual property. It is expected to normalize the activity of the protein derived from the DYRK1A gene which is located on chromosome 21 in triplicate (instead of in duplicate) in trisomy 21. Excessive activity of this enzyme generates a number of disorders, including the cognitive deficit associated with trisomy 21. It is also implicated in the development of Alzheimer disease.

Leucettinib-21 is a molecule inspired by a natural substance produced by the marine sponge Leucetta microraphis. After selecting and optimizing the molecule, preclinical tests concluded that Leucettinib-21 was safe in animals with much higher doses than those to be administered to humans. Following the green light of the French National Drug Agency or ANSM

(Agence Nationale de Sécurité du Médicament) Leucettinib-21 is now entering Phase 1 clinical trial: a safety and pharmacology study involving 96 healthy volunteers, 12 adults with trisomy 21 and 12 patients with Alzheimer’s disease. The clinical trial is designed to check that the molecule is well tolerated. It will start early 2024.

According to Grégoire François-Dainville, Fondation Jérôme Lejeune’s Managing Director, « a new step has been taken in the search for a treatment for the cognitive disorders associated with trisomy 21 (Down syndrome) and Alzheimer disease. On behalf of all the people with trisomy 21 whom we support, and on behalf of the Fondation Lejeune, I would like to express my great gratitude to the Perha Pharmaceuticals teams. The Fondation Lejeune is proud to be associated with this remarkable milestone in the race for a drug candidate. All is not yet won, and it is only at the end of an engaging journey that we will know with certainty whether this drug candidate can be used safely and effectively. However, by dint of intuition, perseverance and confidence, hope is growing that we will be able to correct intellectual deficiency and enable greater autonomy for people with trisomy 21. »

 About the Fondation Jérôme Lejeune

The Fondation Jérôme Lejeune has been recognized as a charitable organization since 1996. It works on behalf of people with genetic intellectual disabilities (Trisomy 21, Williams-Beuren, X-fragile, Cri du Chat, Smith Magenis, Rett syndrome and Angelman syndrome, unexplained mental retardation, etc.). In the service of patients and their families, it pursues three missions: research, care and the defense of human life and dignity. 

For over 25 years, the Fondation Jérôme Lejeune has been involved in therapeutic research for trisomy 21 and other genetic intellectual disabilities.

 About Perha Pharmaceuticals

Perha Pharmaceuticals is a biotechnology company focused on the development of molecules capable of limiting the problematic action of certain proteins (kinases). Founded by Dr. Laurent Meijer in 2019 and based in Roscoff, the company aims to identify, optimize and characterize active ingredients from marine organisms.

Press contact

Véronique Bourgninaud – Clotide Micheneau 

+33 (0)6 47 95 66 60