Proof-of-Concept: record productivity at Novartis R&D

Guided by a proven research strategy, Novartis scientists delivered more hope for patients with a record twelve positive Proof-of-Concept (PoC) compounds in 2009.

The Novartis Institutes for BioMedical Research (NIBR), the global Novartis drug discovery organization, exceeded goals by declaring their largest number of PoC compounds last year.

“2009 was a banner year for NIBR,” reports Mark Fishman, NIBR President. “We delivered more medicines to the clinic and to clinical trials than ever before in our history.”

At Novartis, a PoC compound is a potential medicine that has successfully demonstrated a benefit to patients in a small clinical trial. These focused trials, called PoC studies, are an efficient and well-designed bridge between the laboratory and the clinic.

“PoC is where science meets medicine,” says Tim Wright, Global Head of NIBR Translational Sciences. “A positive PoC study can translate an exciting discovery in the lab to an innovative therapy for patients.”

PoC and pathways: transforming drug discovery
“The premise of PoC studies is that we go into a narrow set of patients to understand if a medicine works,” explains Mark Fishman. “Once we are convinced that a medicine does work and that patients are responding, we expand into broader populations and look at related dis­eases.”

The Novartis R&D philosophy is that diseases are caused by an imbalance in molecular pathways – the complex communication highways in a cell. NIBR explores pathways to find a key node, or protein associated with disease, that can be targeted by medicines to change the pathway and treat disease.

Once a PoC is declared positive, Novartis initiates parallel studies in other indications because illnesses can share a common underlying pathway. NIBR has already discovered single medicines, such as the compound ACZ885, with the potential to treat multiple diseases. This “one pathway – many diseases” view is transforming the process of drug discovery.

Changing the practice of medicine
The ultimate goal of NIBR is to discover therapies with the potential to change the practice of medicine. This means changing the way physicians can help patients who do not respond to current therapy, who suffer unacceptable side effects from their medicines, or worse, have no treatment option or hope for their disease.

“Our success in 2009 is evidence once again that our pathways approach to drug discovery is working,” confirms Fishman.

“Our current class of PoC compounds open some therapeutic arenas in oncology, neuroscience, and autoimmunity not previously available.” says Fishman. “That is changing the practice of medicine.”