Positive results of regulatory toxicology studies pave the way for first in human clinical trials

Basel, Switzerland – PIQUR Therapeutics AG, focusing on the discovery and the development of innovative anti-cancer drugs, announces today that it has successfully completed preclinical profiling and the GLP toxicology studies with its most advanced compound PQR309. The preclinical studies confirm that the compound is a highly selective brain penetrant dual PI3K/mTOR inhibitor, which displays potent anti-proliferative in vitro and in vivo antitumor efficacy. The standard GLP 4-week toxicology studies, which have been successfully completed, will allow PIQUR to start its clinical trials with PQR309 in selected cancer patients.

Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR commented: “This is excellent news for PIQUR. Based on these results no special safety monitoring appears to be necessary during Phase I clinical studies planned to start in Q1/2014. Our compound has a benefit to risk ratio that is anticipated to be superior to other PI3K inhibitor front-runners. This makes PQR309 an excellent candidate to treat cancer patients.” The protocol for Phase I study with PQR309 is being submitted to regulatory authorities and ethics committees for approval to start in January 2014. Phase I will run in four centers in Switzerland, one center in Spain and one center in England. The study focus is on safety and the identification of the maximum tolerated dose.

To turn cancer into a manageable disease

The aim of PIQUR’s activities is to turn cancer into a manageable disease. Given the relatively small number of effective cancer treatments available today, there is an unmet medical need for new therapeutic approaches. One in every two men and one in every three women, living in the developed world, will develop cancer at some stage in their lives. PIQUR targets the PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin) pathways.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries as well as cellular technology platform and their excellent products with novel, dual-acting ‘strong PI3K plus finely-tuned weaker mTOR’ inhibitors that fully address the given challenges, meeting therapeutic, tolerance and galenic needs.


PIQUR is a Swiss pharmaceutical company incorporated in August 2011 as a spin-off of the University of Basel focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. While mTOR is a clinically validated drug target, PI3K inhibition achieved proof of concept status in clinical phase III. PIQUR has a secured patent scope protecting many chemical compounds. www.piqur.com

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Contact PIQUR Therapeutics AG
Vladimir Cmiljanovic, PhD
Chief Executive Officer
Mattenstrasse 22
4002 Basel
Tel +41 61 695 31 40