Quote:
slcimmuno Member Level Sunday, 05/14/17 04:58:21 PM
Re: Drakon 642 post# 181961
Post # of 181963

Glad to be back, and hope others who've been quiet start to join in

Re Big Pharma and what they want and when... great story on Novartis and their "Science First Approach."
Sound familiar?

Oldie but goodie 2013. Worth a read IMO. Reinforces why CTIX is entering that very attractive partnering stage... drug candidates with true platform potential approaching P 2 readouts

//

(Think B with HDPs, host defense... applicable to so many anti-inflammatory antibacterial indications)

(Think K with p53... applicable to half of all cancers)

(Think P... likely applicable to at least PsA, RA, and possible other autoimmune indications)

Novartis R& looking for the 'Sweet Spot'
http://www.pharmafile.com/news/180687/novarti...sweet-spot

Speaking at the R&D event in November, Mark Fishman encapsulated his approach: “Our aim is to discover drugs that really will change the practice of medicine. Our approach is to focus on fundamental mechanisms, and then to go into the clinic in a way that permits us to test that mechanism"

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"We feel there is a great overlap of mechanisms between diseases, so that if we allow our network to work together, we will see – as not uncommonly happens – a drug that goes from autoimmune disease or oncology, or vice versa. So our approach is to have a fundamental science platform that can be applicable across the whole world.”

This platform is in large part build around uncovering the role of signalling pathways in human biology and disease. A relatively small number of pathways play fundamental roles in the body, and defects in these pathways are often the underlying causes of disease.

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Proof of Concept trials

Another major part of the Novartis strategy is greater use of proof-of concept trials, in place of the traditional phases I to III progression. The new approach is particularly useful in oncology, where far earlier targeting of those patients likely to benefit most has reaped enormous rewards, cutting costs and producing proof of a drug’s efficacy (or otherwise) far earlier.

Novartis is now shifting towards a model of selecting patients most likely to benefit from the drug for the Phase I trial, and then following this up with a proof of concept trial which serves as the pivotal trial submitted to regulators. (see illustration).

Naturally this approach requires agreement from regulators about the validity and robustness of data from these trial designs, but the FDA in particular is now open to accelerated trial programmes. These trials often produce less mature data than traditional Phase III programmes, but this is partly offset by the science behind the design i.e. the targeting of patients based on their genetic profiles.

Proof of concept trials can’t reduce the failure rate of compounds in development, but they do shift any potential failure rate to earlier stages of the drug development process, saving huge amounts of time and money.