Target product profile 1: A new treatment for stages 1 and 2 disease

DNDi aims to develop an oral or intramuscular treatment for sleeping sickness (human African trypanosomiasis), maximum one week in duration, that is effective against T.b. gambiense and rhodesiense parasites in all patients in both stages of the disease.

Ideal Acceptable
Target populationEffective against stages 1 and 2

Effective in melarsoprol refractory patients

All patients, including pregnant and lactating women
Effective against stages 1 and 2
Target speciesEfficacy against both T.b. gambiense and T.b. rhodesienseEfficacy against T.b. gambiense only
EfficacyClinical efficacy > 95% at 18-month follow-upClinical efficacy > 95% at 18-month follow-up
Safety/tolerability<0.1% drug related mortality

No monitoring for adverse events (AEs)
<1% possibly related mortality

Weekly simple lab testing (field testing) for AEs
FormulationAdult and paediatric formulations
Treatment regimen<7 days oral once daily or
<7 days intramuscular injection once daily
10 days oral or
<10 days intramuscular injection
StabilityStability in climatic zone 4 for >3 yearsStability in climatic zone 4 for > 12 months
Cost< EUR 30 / course (drug cost only)< EUR 100 / course
< EUR 200 / course OK if very good on other criteria

Target product profile 2: A treatment to be developed only if a newly developed second-stage treatment fails to show efficacy and is already in advanced clinical trials

DNDi aims to develop a three-day, oral or intramuscular treatment for sleeping sickness (human African trypanosomiasis) that is highly effective against T.b. gambiense and rhodesiense parasites in all stage-1 patients at a very low cost and with an impeccable safety profile.

Ideal Acceptable
Target populationEffective against stage 1

Suitable for pregnant and breastfeeding women, and for children
Effective against stage 1

Suitable for pregnant and breastfeeding women, and for children
Target speciesBroad spectrum (T.b. gambiense and rhodesiense)Efficacy against T.b. gambiense only
EfficacyClinical efficacy >95% at 18-month follow-upClinical efficacy no worse than pentamidine
Safety/tolerability0% drug related mortality
No monitoring for AEs*
0% drug-related mortality
No monitoring for AEs
FormulationAdult and paediatric formulationsAdult and paediatric formulations
Treatment regimen<3 days treatment
single dose oral or intramuscular injection (single dose in animal models, long t1/2)
<7 days treatment
2-3 daily doses oral or intramuscular injection
StabilityStability in climatic zone 4 for > 4 yearsStability in climatic zone 4 for >2 years
Cost<EUR 10 / course <EUR 30 / course

*   No specific monitoring needed