“A Hit for Every Target” and Changing the Model for Pharma Innovation

For years, observers have criticized the pharmaceutical industry’s inefficiency in turning more of its discoveries into new drugs. To improve success, more companies are moving beyond high-throughput screening to “high content” screening and 3-D methods, bringing the spirit of high-throughput methods to biopharmaceuticals, and areas such as cell therapies. They are also rounding out target-based screening with more of the empirical aspects of old-fashioned phenotypic screening.

At the same time, “open” partnerships with academic research labs and even with competitors, are pushing back the traditional boundaries of collaborative research.  The goal of these new deals, which, typically do not involve funding or financial arrangements, is sharing the risk and increasing the chances of success.

In late November, AstraZeneca and Sanofi launched a precedent-setting partnership by agreeing to exchange 210,000 new compounds from their proprietary libraries, expanding each partner’s ability to find more diamonds in the rough.  The companies will share chemical structures and synthetic procedures, and sufficient quantities of compounds to allow them to carry out high-throughput screening for years. “The venture will accelerate our ability to identify unique starting points that could become new medicines for patients,” said Mene Pangalos, executive vice president of innovative medicines and early development at AstraZeneca.  Sanofi’s president of global R&D, Elias Zerhouni also commented, “We recognize that collaboration is the foundation of every medical breakthrough. We are happy to partner with other companies if it will speed the discovery of new life-saving or life-enhancing therapies for patients.”

This openness represents a major change in mindset from the past, and a new approach to innovation that is slowly becoming part of pharma company cultures. However, a new survey by PA Consulting Group suggests that some companies are still struggling to develop a cohesive strategy and implementation for open innovation. 

Collaborative partnerships can also extend to technology providers, at a time when pharma’s ability to develop trends and act on new leads has improved, and the assay has become far more powerful than it might have been in the past. As an article by Steve Rees, AstraZeneca’s VP of screening sciences and sample measurements, and his colleagues in the November 20, 2015 issue of Nature Reviews Drug Discovery suggests, scientists can now start to talk about a world where there might be “a hit for every target.”  

In an environment where all leading pharmaceutical companies have focused R&D efforts in “hubs” in research-heavy regions of the world, whether Cambridge, U.K. or Cambridge, Mass., AstraZeneca recently launched new partnerships, focused on compound screening and management, that aim to improve the efficiency of its small-molecule-based research efforts. Its efforts will focus on work underway at its AstraZeneca MRC UK Center for Lead Discovery at the Cambridge Biomedical Campus.

AstraZeneca’s Rees says his company is creating “cutting-edge capabilities for compound management and screening to deliver new ways of working, help reduce costs and transform our ability to identify new w molecules that could become medicines of the future."

The company plans to screen roughly 2 million chemical structures per target, using advanced robotics and screening, and exploiting potential advantages of ultrasound.  Its new technology partners include HighRes Biosolutions, a modular lab automation company that is developing new approaches to high-throughput genotyping, siRNA screening, next-generation sequencing, sample prep, and other functions based on its MicroDock system, which allows different robots or equipment to be connected much faster than possible with traditional systems.  The partnership’s goal is to develop robots that will interact more directly with researchers, to enhance flexibility and mobility.

AstraZeneca has also launched partnerships with Labcyte and Brooks Automation, which aim to improve liquid sample storage and handling. With Labcyte, AstraZeneca is working on an acoustic sampling method that would eliminate the need for pipetting and reduce compound volumes by a factor of ten.  The partners believe that the approach will vastly improve the quality of screening data generated from biological assays.

The drug manufacturer is also working with Genedata, and has signed a five-year contract that would make it one of the leading users of the company’s data-analysis system for high throughput screening.  This agreement would also allow AstraZeneca's early-stage collaborative research partners to gain access to the platform. The overall impact would significantly reduce the cost of data analysis, the partners say.  Genedata also offers other software tools designed to improve the impact of translational research. 

Other equipment vendors offering new technologies include Perkin Elmer with its high content analysis systems such as Phenix, as well as its sequencing tools and “sequencer-on-chip” products developed by companies such as ThermoFisher Scientific, which recently expanded its line of Ion Torrent sequencers.  

For some pharmaceutical manufacturers today, the question is not whether open collaboration strategies are underway, but how well they are being implemented and managed. 

The PA Consultancy recently issued results of a study in which their analysts interviewed 30 R&D directors from leading pharma and healthcare companies to get their perspectives on open innovation efforts in the industry. 

At this point, for many companies, results suggest that the desire and commitment to open innovation may be there, but questions of risk and reward remain.  Some companies have created dedicated in-house teams focused on open innovation.

However, a number of respondents noted a lack of company-wide strategy, and an ad hoc approach to open innovation partnerships, which as caused efforts to stall.  IT and cultural issues also remain an obstacle for some companies, the research suggests. 

“Gaining access to a range of insights, ideas, capabilities and partners for different sectors is seen as essential….but also a complex, time-consumng task regarding significant resources,” writes Paul Barrett, author of the report.

For now, for many companies, open innovation is a work in progress, but the factors driving this trend are not going away.  All indicators suggest that this risk-sharing model will continue to take root, and drive, discovery, research and development in the future.