PharmacologyAdvanced~18 min

Dose–Response & IC50 Lab

Potency is not efficacy, and the curve proves it

Fit sigmoidal dose–response curves, compare compounds head to head, add experimental noise, and compute a therapeutic index against a toxicity curve.

The takeaway

A compound can be ten times more potent and still be the worse drug. Potency moves the curve left; efficacy sets how high it goes — and only one of those decides whether the patient gets better.

Hill equationIC50 / EC50Potency vs efficacyCooperativityCurve fittingTherapeutic index
Read the theory: Immuno-Oncology & Checkpoint Blockade

R = Bottom + (Top − Bottom) / (1 + ([D]/IC50)^h) · signal falls with dose

Log-dose response curve

% activity remaining vs log₁₀[drug]

Plotted on a log x-axis the hyperbola straightens into a sigmoid, and the IC50 lands exactly at the inflection. Sliding a curve LEFT is potency. Raising or lowering its PLATEAU is efficacy. They are independent — and confusing them is one of the most expensive mistakes in pharmacology.

Live verdict · potency vs efficacy

CompoundIC50 (potency)Max effect (efficacy)Hill slopeFold vs best
BNX-10112.0 nM96%1.006.0×
BNX-207400 nM99%1.90200.0×
BNX-3302.0 nM48%0.70

Potency ≠ efficacy. BNX-330 is the most potent (IC50 2.0 nM) — it works at the lowest dose — but it tops out at only 48% effect. BNX-207 needs 200.0× more drug, yet reaches 99% — it is the more efficacious molecule. If the biology demands near-complete target suppression, the potent compound is the wrong drug at any dose. Potency is a dosing convenience; efficacy is whether the drug can do the job at all.

Experimental noise & curve fitting

Turn this on to scatter simulated replicate wells around the selected compound's curve, then watch a four-parameter fit try to recover the parameters you set.

Therapeutic index · efficacy vs toxicity

Every drug has two dose–response curves: the one you want and the one you do not. Their separation on the x-axis is the safety margin. TI = TD50 / ED50.

30.0 nM
1.40
240 nM
1.80

A steep toxicity curve is dangerous: the gap between 'fine' and 'harmed' collapses.

Therapeutic index

8.0

Moderate window

Certain safety factor

0.02

TD01 / ED99 — overlap!

Usable band

799 nM18.7 nM

ED99 up to TD01

TI of 8.0 — a workable but unforgiving margin. Common for oncology cytotoxics, where the tumour and the bone marrow are dosed by the same needle.The certain safety factor has fallen below 1: the dose needed to treat 99% of patients already poisons 1% of them. The curves overlap — no dose is simultaneously fully effective and clean.

What IC50 really means

IC50 is not a property of the molecule. It is a property of the molecule in your assay: change the substrate concentration, the enzyme concentration, the incubation time or the cell line, and the number moves. A competitive inhibitor assayed at high [S] will look weak; the same compound at low [S] will look potent. This is why IC50 values from different papers are almost never directly comparable, and why the Cheng–Prusoff correction exists to translate an IC50 back into the assay-independent Ki.

Potency vs efficacy vs affinity

  • Affinity (Kd) — how tightly the drug sticks to the target. Thermodynamics. Measured by binding, not function.
  • Potency (IC50/EC50) — how much drug the system needs to hit half-effect. Depends on affinity, but also on receptor reserve, cell permeability and assay design.
  • Efficacy (Top/Emax) — what the drug can achieve once it is bound. A full agonist and a partial agonist can share identical affinity and wildly different ceilings.

A drug with picomolar affinity and 20% efficacy is a beautifully bound failure.

Why this is THE core readout

Every screening cascade in the industry funnels into this curve. A hit is a molecule with a real sigmoid; a lead is one whose sigmoid keeps moving left across a chemical series; a candidate is one whose efficacy curve is far enough from its toxicity curve to survive a human being. Structure–activity relationship campaigns are, quite literally, thousands of these plots stacked on top of each other.

And a shallow Hill slope (h < 1) or a stubbornly high floor is often the earliest hint that a compound is hitting something other than the intended target.