AstraZeneca and PTC Therapeutics, Inc. (PTC) today announced
that they have entered into an exclusive research collaboration and license
agreement for the application of PTC’s proprietary GEMST 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 2030(1), highlighting the critical
need to discover novel and targeted therapies that will offer benefits to
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.
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, Ph.D.,
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.”