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[e-drug] Press release: recruitment enabled of 2-5 year olds into Phase IIb trial for malaria cure

E-DRUG: Press release: recruitment enabled of 2-5 year olds into Phase IIb 
trial for malaria cure

Dear colleagues,

We have some exciting news to share with you: an Independent Safety Monitoring 
Board (ISMB) has recommended that recruitment of patients aged 2-5 years can 
begin for the ongoing Phase IIb safety and efficacy trial of a single dose of 
the antimalarial combination therapy OZ439/piperaquine phosphate.

This combination has the potential to be the long sought single dose cure for 
malaria. MMV and partners are therefore delighted that the medicine can now be 
offered, within the study setting, to patients from among the population that 
suffer the most from this deadly disease.

Please find the press release and notes for editors copied below and attached.

If you have any questions, don't hesitate to ask.

Best wishes,


Independent Safety Monitoring Board assessment enables recruitment of 2-5 year 
olds into Phase IIb trial for next-generation single-dose cure for malaria

* OZ439 (artefenomel) in combination with the established antimalarial 
piperaquine phosphate (PQP), is currently in a Phase IIb trial as the first 
potential single-dose cure for malaria.
* An assessment of safety data has led an Independent Safety Monitoring Board 
to conclude that OZ439/PQP is well-tolerated in adult patients as well as 
patients from 5 to 15 years. As a result, patients aged 2-5 years (among the 
primary target population for this medicine) may now also receive this 
treatment as part of the trial.
* MMV was able to achieve this milestone in collaboration with 10 trial sites 
from Burkina Faso, Gabon, Uganda and Vietnam. In addition, the following 
countries are also preparing to begin involvement, pending regulatory approval: 
Benin, Colombia, Democratic Republic of Congo and Mozambique.

Geneva, Switzerland, 20 January 2015. An Independent Safety Monitoring Board 
(ISMB) has recommended that recruitment of patients aged 2-5 years can begin 
for the ongoing Phase IIb safety and efficacy trial of a single dose of the 
antimalarial combination therapy OZ439 (artefenomel, a novel aromatic 
trioxolane)/piperaquine phosphate (PQP). The ISMB based this decision on 
assessment of safety data from over 80 patients, deeming it well-tolerated in 
patients above 5 years.

The main goal of the trial is to identify the optimal dose of OZ439/PQP. The 
ISMB's decision will now enable the correct dosage to be validated in the 
paediatric population, an important step given that the overwhelming burden of 
malaria is borne by children under 5 years of age.

If the programme is successful and the combination approved, it would add to 
the arsenal of malaria medicines. A single-dose treatment would help to improve 
patient compliance compared to current 3-day therapies.

The Phase IIb trial of OZ439/PQP began in July 2014 and will continue over the 
course of 2015 in eight countries: Benin, Burkina Faso, Colombia, DRC, Gabon, 
Mozambique, Uganda and Vietnam. Designed to deliver safety and efficacy data in 
young children as quickly as possible, the trial adopted a staggered approach, 
treating adults first, then, as sufficient safety data was obtained, extending 
to 5-15 year olds.

As of today (January 20th), 95 of the 495 patients required for the trial have 
been treated with the combination. Following the ISMB's recommendation, the 
treatment can now also be offered to 2-5 year olds (among the primary target 
population for this medicine). If the ISMB then deems it to be well-tolerated 
in this younger and more vulnerable group, the treatment can then be offered to 
infants aged 6 months to 2 years, the final age group in this trial, (also 
among the primary target population for this medicine).

"The safety data for this innovative combination antimalarial has been assessed 
in more than 60 adults and 20 children aged over 5 years," said Prof Dr Michael 
Ramharter, Coordinating Principal Investigator of the trial from the Medical 
University of Vienna and Albert Schweitzer Hospital, Lambaréné, Gabon where the 
first patient in this trial was enrolled. "The ISMB's recommendation after 
review of this data means that we can now enrol and treat children of 5 years 
and under in the efficacy study. This is a significant milestone, as we move 
closer to developing a potential single-dose cure that can not only be used by 
adults but also by young children, the population hardest hit by malaria."

"A single-dose cure would improve patient compliance considerably and would be 
a huge step forward for malaria treatment and its ultimate eradication," said 
Professor Dr Peter Kremsner, Director of the Institute for Tropical Medicine at 
the University of Tübingen, Germany and Scientific Director of the Albert 
Schweitzer Hospital in Gabon. "That's why testing a combination of the 
promising new compound, OZ439, with a known partner drug in a single-dose cure 
is so important. Medicines for Malaria Venture is dedicated to the eradication 
of this terrible disease and it's a privilege to work with them on this study."

"This trial is a key milestone in the development of OZ439," said Dr David 
Reddy, MMV's CEO. "Children below the age of 5 are the most vulnerable to 
malaria and the ISMB's confirmation that OZ439 is sufficiently well-tolerated 
to be offered to this young population in a study setting is a vital step 
towards developing a new cure appropriate for young children. This medicine 
could be a game changer for malaria case-management, as it also has the 
potential to be the long sought-after single-dose cure. The fact that almost 
100 patients required in the trial have been dosed with this new combination is 
real credit to our partners. I applaud each of the 10 trial sites - it is their 
commitment and collaboration that has enabled us to achieve this milestone."

Notes for editors

About OZ439 (artefenomel)
The aromatic trioxolane, OZ439 is the first drug that MMV, together with its 
academic partners,[1] has independently brought forward from laboratory to 
clinic. OZ439 is a fully synthetic alternative to artemisinin derivatives. 
Phase IIa trials demonstrated that OZ439 has excellent activity against both P. 
falciparum and P. vivax, which together cause the majority of malaria cases 
worldwide. Because OZ439 has a longer half-life than artemisinin-derived 
compounds it stays in the blood for longer providing an ideal foundation for a 
single-dose malaria cure, and is being investigated in combination with both 
novel and existing antimalarials, such as piperaquine.

About a single dose cure for malaria
A single-dose cure would be a major treatment advance compared to currently 
available 3-day therapies, as it would enable healthcare workers to guarantee 
patients receive a complete curative dose of treatment. This is important, as 
we know that patients often do not complete the full course of treatment once 
they see their symptoms improve and unfortunately, this proves a perfect 
breeding ground for drug resistance.

About Medicines for Malaria Venture (MMV)
MMV is recognized as the leading product development partnership (PDP) in the 
field of antimalarial drug research and development. Its mission is to reduce 
the burden of malaria in disease-endemic countries by discovering, developing 
and facilitating delivery of new, effective and affordable antimalarial drugs.

Since its foundation in 1999, MMV has developed and brought to registration 
four new medicines with its partners: Pyramax®, (pyronaridine-artesunate) 
co-developed with Shin Poong; Eurartesim® (dihydroartemisinin-piperaquine) with 
Sigma-Tau; Guilin's artesunate injection for the treatment of severe malaria, 
Artesun®; and Coartem® Dispersible (artemether-lumefantrine), a child-friendly 
formulation developed with Novartis. Since 2009, over 250 million courses of 
Coartem Dispersible treatment have been supplied to 50 malaria-endemic 
countries; and since prequalification in 2010, an estimated 25 million vials of 
artesunate injection have been delivered, saving an additional 165,000 young 

Managing the largest portfolio of antimalarial R&D projects ever assembled, of 
over 65 projects, MMV has seven new drugs in clinical development addressing 
unmet medical needs in malaria, including medicines for children, pregnant 
women and relapsing malaria, and drugs that could support the 
elimination/eradication agenda. MMV's success in research and access & product 
management comes from its extensive partnership network of over 300 
pharmaceutical, academic and endemic-country partners in 50 countries.

MMV's vision is a world in which innovative medicines will cure and protect the 
vulnerable and under-served populations at risk of malaria, and ultimately help 
to eradicate this terrible disease.
For more information, please visit http://www.mmv.org

For more information:

Dr. Ghyslain Mombo-Ngoma
Albert Schweitzer Hospital, Medical Research Unit, Epidemiology & Control of 
Infectious Disease
Lambaréné,[2] Gabon and
Eberhard-Karls-University of Tübingen, Department of Tropical Medicine
Tel.: +24 1 0709 2785
Email: ghyslain.mombongoma@gmail.com

Assoc. Prof. Dr. Michael Ramharter
Department of Medicine I, Div. of Infectious Diseases and Tropical Medicine
Medical University of Vienna1 and
CERMEL (Centre de Recherches Médicales de Lambaréné)
Albert Schweitzer Hospital2
Lambaréné, GABON
Tel.: +43 1 40400 44400
Email: michael.ramharter@meduniwien.ac.at

[1]The collaborative research activities between the CERMEL (Centre de 
Recherches Médicales de Lambaréné), MMV, and the Medical University of Vienna 
are co-funded by the Austrian Ministry of Science, Research, and Economy.
2 The Albert Schweitzer Hospital, Gabon, is part of the German Center for 
Infection Research (DZIF) and receives funding for a clinical research group 
from the Federal Ministry of Education and Research (BMBF), Germany, via DZIF.

Prof. Dr. Peter G. Kremsner
Centre de Recherches Medicales de Lambaréné, Gabon and Universitätsklinikum 
Tübingen, Instititut für Tropenmedizin Germany
Tel.: +49 7071 2987179
Email: peter.kremsner@uni-tuebingen.de

Jaya Banerji
Director Communications and Advocacy, MMV
Tel: +41 22 555 0327
Mob: +41 79 707 7181
Email: banerjij@mmv.org

Elizabeth Poll | Medicines for Malaria Venture
Editor and Publications Officer
T: +41 22 555 03 17 | M: +41 79 907 59 92
Email: polle@mmv.org

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