Pharnext's process can be applied to any chronic disease, provided enough genomics information is available (which is now the case for most diseases).
Pharnext runs all its own activities from research through to clinical proof of concept (the end of Phase II clinical trials) via both in-house research and clinical departments and outsourced activities. The following R&D process is used to identify and develop PleodrugsTM:
- Once a disease has been selected:
- The corresponding network is reconstructed in silico. Generally 50 or so off-patent, "other disease" drugs are deduced using the Nexus process.
- These drugs are tested one by one in cell-based disease models and then animal disease models.
- Combinations of positive compounds are then tested.
- The most efficient, synergistic combination is selected as the lead, which enters into pharmaceutical development (including simplified regulatory preclinical studies).
- The PleodrugTM candidate enters Phase I-IIa clinical trials or may even go straight into a Phase II clinical trial.
- After Phase II at the latest, the product will be licensed to a partner who will proceed with further development and marketing.
In parallel, biomarker candidates are identified using network knowledge, and screened with a view to developing a companion test for use in the Phase II trial.
The overall R&D process is several years shorter than the standard process. most of this timesaving comes during the preclinical phase because known, approved drugs are being used. The first-in-man trial would occur generally 2-3 years after initiation of the R&D program (instead of the usual 6-8 years) and thus speeds up the overall development phase. By way of example, Pharnext started a Phase II clinical trial just three years after initiation of its program on Charcot-Marie-Tooth type 1A disease.
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