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Agreements

Date: 2011-06-29

Type of information: R&D agreement

Compound: PTC's proprietary GEMS™ technology (Gene Expression Modulation by Small-molecules)

Company: AstraZeneca (UK) PTC Therapeutics (USA - NJ)

Therapeutic area: Cancer - Oncology

Type agreement: research - R&D - licensing

Action mechanism: 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.

Disease: cancer and other diseases for which there is a great unmet medical need

Details: AstraZeneca and PTC Therapeutics have entered into an exclusive research collaboration and license agreement 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. 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% of all disease targets. 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.

Financial terms: Financial details were not disclosed.

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