Arch2POCM
A new paradigm for drug development
The Arch2POCM is a Public Private Partnership (PPP) composed of an archipelago of participating academicians, pharmaceutical companies, regulatory scientists, public funders and patient advocacy groups.
Arch2POCM’s mission is to help invigorate and accelerate the pharmaceutical industry’s development of new and effective medicines for completely novel targets – those that are judged as slightly too risky to be pursued in industry, but that have significant scientific potential (i.e., disease areas of cancer, immunology, autism, schizophrenia). Arch2POCM will de-risk novel targets by developing and using pairs of test compounds (two different chemotypes) to determine if modulation of the selected target has an effect on human biology that might confer therapeutic benefit. Arch2POCM scientists and clinicians will take these compounds all the way through Phase IIB clinical trials to determine “Proof of Clinical Mechanism” (POCM): demonstration in a Ph II setting that the mechanism of the selected disease target can be safely and usefully modulated.
To generate a common stream of knowledge about the roles that Arch2POCM’s selected targets play in human biology, Arch2POCM will file no patents and will place all of its pre-clinical and clinical data into the public domain. The open sharing of this data is intended to alert the pharmaceutical industry regarding those targets that should be the focus of future development (positive POCMs) and those targets/indications that should be dis-continued (negative POCMs).
The Arch2POCM PPP will use its resources to test a limited set of hypotheses: To expand the impact of its funding, Arch2POCM (1) will make its pre-clinical and clinical test compounds and biomarker data available to any member of the scientific community that agrees to publish all their results and to do so without filing patents, and (2) will allow a wider set of qualified academic and industry clinicians to reference Arch2POCM’s IND databases in order to conduct experimental medicine studies with the test compounds in indications other than those studied by the PPP. By distributing its test compounds, Arch2POCM intends to seed crowd-sourced studies that (1) will increase our fundamental understanding of human disease biology, (2) will provide the pharmaceutical industry with “parallel shots on goal” to align Arch2POCM targets and test compounds to the most promising un-met medical need and (3) will help to reduce the industry’s current 80-90% Phase 2 attrition rate for novel target validation.
Based upon current Phase 2 attrition rates for novel targets, Arch2POCM anticipates that the majority of its POCM determinations will be negative: Arch2POCM will publish all its negative POCM data immediately in order to reduce the number of ongoing redundant proprietary studies (in pharma, biotech and academia) on an invalidated target and thereby:
- minimize unnecessary patient exposure;
- provide significant economic savings for the pharmaceutical industry;
- In the rare instance in which an Arch2POCM molecule achieves positive POCM, Arch2POCM will ensure that the compound has the ability to reach the market by arranging for exclusive access to the proprietary IND database for the molecule.
Championed by Drs. Stephen Friend of Sage Bionetworks and Aled Edwards and Chas Bountra of the Structural Genomics Consortium, Arch2POCM will share the risk by pooling public and private resources from government funders, pharmaceutical and biotechnology companies and private foundations and philanthropists.
Arch2POCM is bringing together a broad cross-section of the best in drug discovery and development science. Top pharma leaders from Takeda, Pfizer, J&J, GSK and Roche are helping define Arch2POCM operations and governance. Leaders from UCSF, the University of Toronto and Oxford University have already stated that their scientists are free to participate in Arch2POCM’s patent-free drug development model and a number of oncology and neuroscience experts are involved in scientific planning discussions. Top patient advocate leaders from Genetic Alliance and the Love Avon Army of Women are engaging Arch2POCM pharma and academic leaders to ensure that the scientific programs are optimally aligned with patient health and safety. And because the objective of the Arch2POCM PPP is to probe and elucidate disease biology as opposed to develop new proprietary products, an exciting opportunity exists for regulatory leaders and scientists to become active partners in the PPP. Specifically, it is anticipated that the FDA will play a key role and provide information to set up Arch2POCM in vitro screening assays for toxicities and biomarker development to improve patient selection. FDAs therapeutic experts in Oncology, Immunology and Schizophrenia/Autism will also have the opportunity to sit on the Arch2POCM scientific review boards
Arch2POCM is in the process of identifying its anchor pharmaceutical and government funding partners and expects to launch operations in 2012 with an initial focus on cancer and two areas in neuroscience, autism and schizophrenia. Recognizing that “…the world is becoming too fast, too complex, and too networked for any one company to have all the answers inside,” Arch2POCM’s open access PPP may represent a sustainable drug development model that can link fundamental discovery to development and accelerate the creation of innovative state-of-the-art medicines.
For more information please contact Dr. Thea Norman.