Finding GEMS
August 2011
by David Hutton  |  Email the author


SOUTH PLAINFIELD, N.J.—AstraZeneca PLC and PTC Therapeutics Inc. have forged an exclusive research collaboration and license agreement for the application of PTC's proprietary Gene Expression Modulation by Small-molecules (GEMS) technology for the discovery and development of potential new therapies for cancer and other diseases for which there is a great unmet medical need.
PTC is focused on the discovery, development and commercialization of orally administered, small-molecule drugs that target post-transcriptional control processes. Compounds identified using the GEMS screening technology modulate gene expression by targeting the post-transcriptional control processes that act through the UTRs of mRNA molecules. The collaboration is geared toward discovering new remedies for cancer and other diseases.
Stuart W. Peltz, president and CEO of PTC, says his company is pleased to be collaborating with AstraZeneca because the company "is widely recognized for its high scientific standards and track record in developing novel therapies in areas of high unmet medical need."
Peltz explains that the initial focus of the collaboration will be in oncology with an option to pursue a total of eight targets across different therapeutic areas.
Cancer kills more than 7 million people globally every year. The World Health Organization estimates the incidence of cancer will continue to rise to an estimated 11 million deaths by 2030, highlighting the critical need to discover novel and targeted therapies that will offer benefits to patients.
According to Peltz, the collaboration between AstraZeneca and PTC is a step forward in drug discovery for novel therapies, considering that classical drug discovery approaches have proven ineffective for more than 90 percent of all disease targets.
The collaboration also will give PTC the opportunity to showcase GEMS, a novel technology that enables the discovery of drugs that can increase or decrease the expression of a specific protein by targeting regulatory mechanisms.
"GEMS identifies compounds that can increase or decrease the expression of a specific protein by targeting the untranslated areas of messenger RNA," Peltz says. "These regions contain important regulatory mechanisms that when modulated can increase or decrease protein expression as necessary by the disease being treated. " 
Peltz added that GEMS is an alternative approach to drug discovery and among its advantages is the fact that GEMS delivers small molecules that can be taken orally.
PTC has successfully employed the GEMS technology in drug discovery programs in oncology, infectious diseases, cardiovascular diseases and neuromuscular disorders. The most advanced compound identified through this technology is PTC299, a small-molecule inhibitor of VEGF expression currently in multiple oncology clinical trials.
Under the terms of the agreement, AstraZeneca will make an undisclosed upfront cash payment for the initiation of the first target in the collaboration plus committed research funding to PTC.
PTC will also qualify for significant future milestone payments depending on the achievement of research, development, regulatory and commercial milestones. AstraZeneca retains the global commercialization rights and PTC has an option to participate in the development of select product candidates emerging from the collaboration. AstraZeneca will pay PTC tiered royalties on worldwide net sales.
Dr. Susan Galbraith, vice president and head of the Oncology Innovative Medicines Research Unit at AstraZeneca, says the collaboration with PTC Therapeutics poses an excellent opportunity for the pharma.
"PTC's RNA biology expertise and the uniqueness of the GEMS technology are complementary to our internal efforts," she says. "We believe that the GEMS technology will enable AstraZeneca to address important disease mechanisms that were intractable with our existing approaches."
PTC has discovery-stage programs in multiple fields, including antibacterial, oncology and musculoskeletal disorders, and ongoing collaborations with a number of nonprofit and commercial organizations, including Celgene, Gilead, the Muscular Dystrophy Association, Pfizer, Roche and the Spinal Muscular Atrophy Association and Parent Project Muscular Dystrophy.
In February, the firm announced it had identified a chemical series of molecules that penetrates the blood-brain barrier in animal models and reduces levels of BMI1, a protein linked to drug-resistant cancers. The achievement represented a milestone in the firm's collaboration with the Wellcome Trust, triggering a $2.2 million payment. Initiated in June 2010, the $5.4 million collaboration with the Wellcome Trust aims to develop BMI1-targeting drugs for the treatment of chemotherapy-resistant cancers.

AstraZeneca launches predictive science center in Russia

LONDON—On June 16, AstraZeneca also announced that it will establish a Predictive Science Centre in St. Petersburg, Russia, over the coming year. The move strengthens AstraZeneca's investments in Russia and supports the Russian government's strategy to modernize and develop the country's pharmaceutical sector, according to the U.K.-based pharma.
The new AstraZeneca Predictive Science Centre, the company's first in Russia, will leverage local scientific talent and focus on developing bioinformatics, data analysis methods, software and systems to better predict the safety and efficacy of potential new medicines. Approximately 30 people will work at the center through collaborations with local companies and organizations as part of a related agreement with the St. Petersburg government.
Earlier this year, the company began construction of a new $150 million manufacturing facility in the Kaluga region to supply Russia with innovative AstraZeneca medicines that are locally manufactured. In addition, AstraZeneca has established several partnerships with Russian development institutes, including the Skolkovo Innovation Centre and Russia Venture Company, 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.
Code: E081118

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