New antimalarial compound treats and protects in a single dose

Controlled infection models reveal exciting data on DSM265 earlier than previously possible

29 Mar 2017

Two articles published today in The Lancet Infectious Diseases demonstrate DSM265’s tolerability and efficacy for treatment and protection against P. falciparum malaria in a single dose. The phase Ia/Ib data, which support the continued development of DSM265, were gathered ahead of traditional drug development timelines thanks to the use of the innovative Controlled Human Malaria Infection (CHMI) model.

The CHMI model allows the activity of molecules to be tested in healthy volunteers inoculated with a low dose of malaria parasites in a tightly controlled clinical trial environment. In this way, the efficacy of compounds can be tested in humans earlier in clinical development than had been possible previously (in this case 6 months after the compound was administered to humans for the first time).

The first publication looks at DSM265’s safety and tolerability as well as its potential for treatment. It presents the findings of the first integrated phase Ia and phase Ib trial in antimalarial drug development. This was made possible thanks to the pioneering work in partnership with Prof. James McCarthy and his team at QIMR Berghofer Medical Research Institute, Queensland, Australia, to develop and utilise the "blood stage" CHMI model. The second paper demonstrates the prophylactic potential of a single dose of DSM265, thanks to the "sporozoite"1 CHMI model, in partnership with Prof. Peter Kremsner and his team at the University Tübingen, Germany.  

DSM265 is a novel antimalarial that inhibits an enzyme vital to the malaria parasite’s survival. The data from the integrated phase Ia and Ib trial provided a predicted efficacious dose for the phase IIa proof-of-concept clinical trial, which was recently successfully completed in Peru. These data are soon to be published. 

“This is a first in antimalarial drug development,” said Dr David Reddy, CEO of MMV. “It’s the first time we have been able to gather data on both the tolerability and efficacy of a compound in parallel, in early development. Thanks to MMV’s collaborations with QIMR Berghofer and University Tübingen, we have shown not only that DSM265 holds much promise as a future antimalarial for treatment and protection, but also that we can accelerate development times with innovative methods. It’s an exciting time to be involved in antimalarial drug research and development.”

DSM265 originates from a collaboration with Prof. Margaret Philips at the University of Texas Southwestern, Prof. Pradip Rathod, University of Washington, and Prof. Sue Charman, Monash University. Today, the compound is being developed by MMV in partnership with Takeda, with support from the Japanese Global Health Innovation and Technology Fund.


1. Sporozoites are the form of the malaria parasite that is transmitted from mosquito to man, when the mosquito takes a blood meal. In humans, the sporozoites enter liver cells where they develop into the next stage of the malaria parasite life cycle (the liver stage or exo-erythrocytic stage).