PharmaAI

The drug discovery process typically
begins with identifying a specific
molecular target associated with a disease
or condition. This could be a protein,
enzyme, receptor, or other biomolecule
involved in the disease pathway.
Validation of the target involves
confirming its relevance to the disease and
its potential as a therapeutic target

Once the target is validated, the next step
is to identify or generate “hits,” which are
compounds that have the potential to
interact with the target and modulate its
activity. Hits can be identified through
various methods, including high-
throughput screening of chemical libraries,
virtual screening using computational
methods, or fragment-based screening.

Selected hits are further optimized to
improve their potency, selectivity,
pharmacokinetic properties, and safety
profile. This process involves medicinal
chemistry techniques to modify the
chemical structure of the hits while
maintaining or enhancing their biological
activity. Iterative cycles of synthesis,
testing, and structure-activity relationship
(SAR) analysis are conducted to identify
lead compounds with improved drug-like
properties.

Lead compounds with the most promising
pharmacological profiles are subjected to
further optimization to enhance their
efficacy, safety, and drug-like properties.
This involves fine-tuning the chemical
structure of the lead compounds and
evaluating their pharmacokinetic and
toxicological properties through in vitro
and in vivo studies. The goal is to identify
candidate compounds suitable for
preclinical testing.

Candidate compounds undergo preclinical
testing to assess their safety,
pharmacokinetics, pharmacodynamics, and
toxicology in animal models. These studies
provide crucial data for evaluating the
compound’s potential for human use and
determining the optimal dose range for
clinical trials.

Phase I: Conducted in a small number of healthy volunteers to evaluate the
compound’s safety, pharmacokinetics, and
initial tolerability.
Phase II: Involves testing the compound
in a larger group of patients to assess its
efficacy and further evaluate safety.
Phase III: Conducted in a larger patient
population to confirm efficacy, monitor
adverse effects, and gather additional
safety data. Successful completion of
Phase III trials may lead to regulatory
approval for marketing