Imagine a protein in the brain acting like a volume knob that is turned up too high, causing memory and learning problems. This project created a chemical 'dimmer switch' called Leucettinib-21 to turn that protein back down to normal levels. By doing this, it helps the brain better store and recall information, similar to fixing a glitchy hard drive in a computer.
People with Down Syndrome face severe cognitive decline and a lack of medical treatments to improve learning and memory, leading to limited autonomy and early onset of Alzheimer's-like symptoms.
A small molecule drug candidate, Leucettinib-21 (LCTB-21), which inhibits the DYRK1A protein kinase to correct cognitive impairments.
If you are a drug developer dealing with the lack of treatments for cognitive decline in Down Syndrome — this project developed Leucettinib-21 that corrects memory and learning disorders in animal models. This provides a validated candidate for a market serving 150,000 children in Western countries.
If you are a biotech firm dealing with the high failure rate of Alzheimer's drugs — this project developed a DYRK1A inhibitor that has already passed preclinical regulatory toxicology studies. It offers a targeted mechanism to address neuropathology that starts at age 40 in people with Down Syndrome.
If you are an investor dealing with the search for high-impact orphan drug opportunities — this project developed a lead candidate moving toward phase 2a clinical trials. It targets a specific genetic overexpression to improve patient autonomy and societal integration.
Based on available project data, there is no information regarding the final price or cost of the drug; the project is currently focused on clinical trial funding.
Based on available project data, the drug is currently in the clinical trial phase (Phase 1/2a) and has not yet reached industrial manufacturing scale.
Perha Pharmaceuticals aims to outlicense Leucettinib-21 to a large pharmaceutical partner, such as Otsuka Pharmaceuticals or Intra-Cellular Therapies, following a successful phase 2a clinical trial.
The project has completed preclinical regulatory toxicology studies and has been entitled by French health authorities to assess safety in humans during a phase 1 trial.
The project period runs from 2022-06-01 to 2026-06-30, with the goal of reaching proof-of-concept via a phase 2a clinical trial.
The project is led by a single SME, Perha Pharmaceuticals SAS, based in France. With a 100% industry ratio and no university or research partners in the consortium, the project is streamlined for commercial development and rapid transition to clinical trials, though it relies on external funding (Series A) for the phase 2a stage.
Contact Perha Pharmaceuticals SAS regarding LCTB-21 licensing
Contact us to connect with Perha Pharmaceuticals for licensing opportunities.
