Finding GEMS

AstraZeneca, PTC Therapeutics announce strategic oncology drug discovery collaboration

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SOUTH PLAINFIELD, N.J.—AstraZeneca PLC and PTC TherapeuticsInc. have forged an exclusive research collaboration and license agreement forthe application of PTC's proprietary Gene Expression Modulation bySmall-molecules (GEMS) technology for the discovery and development ofpotential new therapies for cancer and other diseases for which there is agreat unmet medical need.
PTC is focused on the discovery, development andcommercialization of orally administered, small-molecule drugs that targetpost-transcriptional control processes. Compounds identified using the GEMSscreening technology modulate gene expression by targeting thepost-transcriptional control processes that act through the UTRs of mRNAmolecules. The collaboration is geared toward discovering new remedies forcancer and other diseases.
Stuart W. Peltz, president and CEO of PTC, says his companyis pleased to be collaborating with AstraZeneca because the company "is widelyrecognized for its high scientific standards and track record in developingnovel therapies in areas of high unmet medical need."
Peltz explains that the initial focus of the collaboration willbe in oncology with an option to pursue a total of eight targets acrossdifferent therapeutic areas.
Cancer kills more than 7 million people globally every year.The World Health Organization estimates the incidence of cancer will continueto rise to an estimated 11 million deaths by 2030, highlighting the criticalneed to discover novel and targeted therapies that will offer benefits topatients.
According to Peltz, the collaboration between AstraZenecaand PTC is a step forward in drug discovery for novel therapies, consideringthat classical drug discovery approaches have proven ineffective for more than90 percent of all disease targets.
The collaboration also will give PTC the opportunity toshowcase GEMS, a novel technology that enables the discovery of drugs that canincrease or decrease the expression of a specific protein by targetingregulatory mechanisms.
"GEMS identifies compounds that can increase or decrease theexpression of a specific protein by targeting the untranslated areas of messengerRNA," Peltz says. "These regions contain important regulatory mechanisms thatwhen modulated can increase or decrease protein expression as necessary by thedisease being treated." 
Peltz added that GEMS is an alternative approach to drugdiscovery and among its advantages is the fact that GEMS delivers smallmolecules that can be taken orally.
PTC has successfully employed the GEMS technology in drugdiscovery programs in oncology, infectious diseases, cardiovascular diseasesand neuromuscular disorders. The most advanced compound identified through thistechnology is PTC299, a small-molecule inhibitor of VEGF expression currentlyin multiple oncology clinical trials.Under the terms of the agreement, AstraZeneca will make anundisclosed upfront cash payment for the initiation of the first target in thecollaboration plus committed research funding to PTC.
PTC will also qualify for significant future milestonepayments depending on the achievement of research, development, regulatory andcommercial milestones.
AstraZeneca retains the global commercialization rights andPTC has an option to participate in the development of select productcandidates emerging from the collaboration. AstraZeneca will pay PTC tieredroyalties on worldwide net sales.
Dr. Susan Galbraith, vice president and head of the OncologyInnovative Medicines Research Unit at AstraZeneca, says the collaboration withPTC Therapeutics poses an excellent opportunity for the pharma.
"PTC's RNA biology expertise and the uniqueness of the GEMStechnology are complementary to our internal efforts," she says. "We believethat the GEMS technology will enable AstraZeneca to address important diseasemechanisms that were intractable with our existing approaches."
PTC has discovery-stage programs in multiple fields,including antibacterial, oncology and musculoskeletal disorders, and ongoingcollaborations with a number of nonprofit and commercial organizations,including Celgene, Gilead, the Muscular Dystrophy Association, Pfizer, Rocheand the Spinal Muscular Atrophy Association and Parent Project MuscularDystrophy.
In February, the firm announced it had identified a chemicalseries of molecules that penetrates the blood-brain barrier in animal modelsand reduces levels of BMI1, a protein linked to drug-resistant cancers. Theachievement represented a milestone in the firm's collaboration with theWellcome Trust, triggering a $2.2 million payment. Initiated in June 2010, the$5.4 million collaboration with the Wellcome Trust aims to develop BMI1-targetingdrugs for the treatment of chemotherapy-resistant cancers.

AstraZeneca launches predictive science center in Russia

LONDON—On June 16, AstraZeneca also announced that it willestablish a Predictive Science Centre in St. Petersburg, Russia, over thecoming year. The move strengthens AstraZeneca's investments in Russia andsupports the Russian government's strategy to modernize and develop thecountry's pharmaceutical sector, according to the U.K.-based pharma.
The new AstraZeneca Predictive Science Centre, the company'sfirst in Russia, will leverage local scientific talent and focus on developingbioinformatics, data analysis methods, software and systems to better predictthe safety and efficacy of potential new medicines. Approximately 30 peoplewill work at the center through collaborations with local companies andorganizations as part of a related agreement with the St. Petersburggovernment.
Earlier this year, the company began construction of a new$150 million manufacturing facility in the Kaluga region to supply Russia withinnovative AstraZeneca medicines that are locally manufactured. In addition,AstraZeneca has established several partnerships with Russian developmentinstitutes, including the Skolkovo Innovation Centre and Russia VentureCompany, to share its global R&D expertise through research collaborations,grant programs and clinical trials.
According to AstraZeneca, its investment will contribute$1.2 billion to the Russian economy over the next five years.

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