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Dose and schedule optimisation trials specific to antibody-drug conjugates

Antibody-drug conjugates carry potent chemotherapy into tumours but also cause lung, eye and nerve damage that depends on total exposure. Trials comparing lower doses, longer intervals and payload caps could keep the benefit while cutting these harms.

Randomised dose and schedule comparisons for ADCs (e.g., lower dose per cycle, extended intervals, capped cumulative payload dose, response-adapted de-escalation) with PK of conjugate and free payload, tolerability estimands (ILD, ocular toxicity, neuropathy) and efficacy. The 5.4 versus 6.4 mg/kg trastuzumab deruxtecan comparison and dose modifications for several approved ADCs show dose was not optimised at approval.

Hypothesis
Optimised ADC dosing will reduce grade 2+ exposure-driven toxicities by a third and discontinuation rates substantially, with non-inferior efficacy, for most ADCs tested.
Rationale
ADC toxicity is largely payload-exposure-driven and cumulative, while efficacy depends on target saturation that occurs at lower doses; the therapeutic window is being wasted by MTD-based dosing.
What would test it
Two randomised phase 2/3 dose-comparison trials of approved ADCs with high discontinuation rates, with tolerability co-primary and efficacy non-inferiority.
Maturity
early clinical
Who has to act
industry
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks

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