Fresh horizons in drug discovery

Horizon, H3 Biomedicine announce target validation collaboration

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CAMBRIDGE, U.K.—In a new approach intended to speed up drugdiscovery and hopefully ensure later clinical success, Horizon Discovery Ltd.and H3 Biomedicine Inc. have joined forces in a collaboration to identify andvalidate a panel of novel cancer drug targets.
Per the terms of the agreement, H3 Biomedicine will hold anexclusive option to acquire ownership of all program assets and intellectualproperty relating to each of the target programs, excluding Horizon backgroundintellectual property. Horizon in turn will retain commercial rights for anytarget program that H3 Biomedicine does not option, and will receive an initialupfront investment as well as milestone payments if certain preclinical andclinical goals are met. No additional financial details were disclosed.
"Horizon and H3 Biomedicine share a common belief thatsystematically validating mutant genes with clear roles in cancer evolution mayhelp yield rapidly translatable targets and drugs with greater clinicalsignificance," Darrin M. Disley, CEO of Horizon, said in a press release.
Horizon will work with H3 Biomedicine to performtarget-validation studies to identify cancer genome targets that are ideal formodulation. H3 will be responsible for early target discovery throughpreclinical research, and Horizon will make use of its drug discovery tools tosupport the discovery biology. The collaboration aims to make possible rapiddecisions on whether or not novel targets should be advanced to development. Ifany clinical trials result, they will be conducted by Eisai Inc., a partner ofH3 Biomedicine.
"The successful development of new personalized medicinesdepends upon well-validated and characterized targets and clearly definedpatient populations," Dr. Markus Warmuth, president and CEO of H3, said in apress release. "High attrition rates in clinical trials have been impactingindustry for a while. We believe that clinical success starts at targetselection and validation. We have elected to work with Horizon because theirtools will enable H3 Biomedicine to progress toward identifying the bestpossible targets for new drugs with the power to have meaningful therapeuticrelevance. That may help us to increase clinical success rates and expedite thedelivery of new therapies to the patients who need them."
The collaboration combines very specific and tailored skillsfrom the two companies. Horizon brings with it its proprietary GENESIS genomeediting technology, with which it can alter any endogenous gene sequence incells lines and has created over 400 X-MAN genetically defined andpatient-relevant human cell lines. The cells lines enable accurate modeling ofdisease-causing mutations in cancer patients. H3 Biomedicine specializes in thediscovery and development of cancer therapeutics, with experience in cancergenomics and chemical synthesis.
Both companies are hopeful that by starting with morefocused 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 targetdiscovery in certain ways in the past, but not being terribly fruitful. So H3has been set up and is driven by people that really want to break out of themold by doing drug discovery in a different way," says Dr. ChristopherTorrance, chief scientific officer of Horizon.
"The principles and the ethicsof doing that are very similar to why Horizon started, so we felt that therewas a lack of good patient-relevant disease models out there and that was onefactor in why drug discovery is so expensive."
He adds that H3 Biomedicine believes that spending the timeat the outset "to do the biological validation properly" and choose the righttarget can lead to a faster drug discovery process. There is a limited proof ofconcept, Torrance notes, but enough of one that "when you choose the righttarget, get a good drug to it and you get it in the right patient, you followthat 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 thattarget validation is very important … we believe that success in the clinicstarts with very solid target validation, and obviously the strategy that we'repursuing here will lead to insights and a level of confidence early on thattranslates into clinical success, but yes, we have to prove ourselves right,"says Warmuth.


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