AstraZeneca, PTC Therapeutics announce strategic oncology drug discovery collaboration

AstraZeneca and PTC Therapeutics Inc announced that they have entered into an exclusive research collaboration and license agreement. This is for the application of PTC’s proprietary GEMS technology (Gene Expression Modulation by Small-molecules) for the discovery and development of potential new therapies for cancer and other diseases for which there is a great unmet medical need.

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 20301, highlighting the critical need to discover novel and targeted therapies that will offer benefits to patients.

According to the companies, this collaboration 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. GEMS is a novel technology that enables the discovery of drugs that can increase or decrease the expression of a specific protein by targeting regulatory mechanisms. It is an alternative approach to drug discovery and among its advantages is the fact that GEMS delivers small molecules that can be taken orally.

Under the terms of the agreement, AstraZeneca will make an undisclosed up-front cash payment for the initiation of the first target in the collaboration plus committed research funding to PTC. Initial efforts will be focused on oncology with an opportunity to pursue up to eight targets across different therapeutic areas.

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.

“We are delighted to enter into this collaboration with PTC Therapeutics. PTC’s RNA biology expertise and the uniqueness of the GEMS technology are complementary to our internal efforts,” said Dr Susan Galbraith, Vice President and Head of the Oncology Innovative Medicines Research Unit at AstraZeneca. "We believe that the GEMS technology will enable AstraZeneca to address important disease mechanisms that were intractable with our existing approaches.”

“We are honored to enter into this partnership with AstraZeneca,” said Stuart Peltz, PhD, President and CEO of PTC Therapeutics. “AstraZeneca is widely recognized for its high scientific standards and track record in developing novel therapies for the world’s most serious illnesses.”