Fresh horizons in drug discovery
June 2012
by Kelsey Kaustinen  |  Email the author


CAMBRIDGE, U.K.—In a new approach intended to speed up drug discovery and hopefully ensure later clinical success, Horizon Discovery Ltd. and H3 Biomedicine Inc. have joined forces in a collaboration to identify and validate a panel of novel cancer drug targets.  
Per the terms of the agreement, H3 Biomedicine will hold an exclusive option to acquire ownership of all program assets and intellectual property relating to each of the target programs, excluding Horizon background intellectual property. Horizon in turn will retain commercial rights for any target program that H3 Biomedicine does not option, and will receive an initial upfront investment as well as milestone payments if certain preclinical and clinical goals are met. No additional financial details were disclosed.  
"Horizon and H3 Biomedicine share a common belief that systematically validating mutant genes with clear roles in cancer evolution may help yield rapidly translatable targets and drugs with greater clinical significance," Darrin M. Disley, CEO of Horizon, said in a press release.  
Horizon will work with H3 Biomedicine to perform target-validation studies to identify cancer genome targets that are ideal for modulation. H3 will be responsible for early target discovery through preclinical research, and Horizon will make use of its drug discovery tools to support the discovery biology. The collaboration aims to make possible rapid decisions on whether or not novel targets should be advanced to development. If any clinical trials result, they will be conducted by Eisai Inc., a partner of H3 Biomedicine.  
"The successful development of new personalized medicines depends upon well-validated and characterized targets and clearly defined patient populations," Dr. Markus Warmuth, president and CEO of H3, said in a press release. "High attrition rates in clinical trials have been impacting industry for a while. We believe that clinical success starts at target selection and validation. We have elected to work with Horizon because their tools will enable H3 Biomedicine to progress toward identifying the best possible targets for new drugs with the power to have meaningful therapeutic relevance. That may help us to increase clinical success rates and expedite the delivery of new therapies to the patients who need them. "  
The collaboration combines very specific and tailored skills from the two companies. Horizon brings with it its proprietary GENESIS genome editing technology, with which it can alter any endogenous gene sequence in cells lines and has created over 400 X-MAN genetically defined and patient-relevant human cell lines. The cells lines enable accurate modeling of disease-causing mutations in cancer patients. H3 Biomedicine specializes in the discovery and development of cancer therapeutics, with experience in cancer genomics and chemical synthesis.  
Both companies are hopeful that by starting with more focused and in-depth target validation in the early phases of development, there can be a greater assurance of clinical success down the road.  
"The industry has been doing drug discovery and target discovery in certain ways in the past, but not being terribly fruitful. So H3 has been set up and is driven by people that really want to break out of the mold by doing drug discovery in a different way," says Dr. Christopher Torrance, chief scientific officer of Horizon.
"The principles and the ethics of doing that are very similar to why Horizon started, so we felt that there was a lack of good patient-relevant disease models out there and that was one factor in why drug discovery is so expensive."  
He adds that H3 Biomedicine believes that spending the time at the outset "to do the biological validation properly" and choose the right target can lead to a faster drug discovery process. There is a limited proof of concept, Torrance notes, but enough of one that "when you choose the right target, get a good drug to it and you get it in the right patient, you follow that paradigm," it could be possible to break out of the "15-year, $1 billion … metric in the industry."  
"We're not the only group that has come to realize that target validation is very important … we believe that success in the clinic starts with very solid target validation, and obviously the strategy that we're pursuing here will lead to insights and a level of confidence early on that translates into clinical success, but yes, we have to prove ourselves right, " says Warmuth.  

Code: E061219

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