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Our proprietary drug discovery platform combines target-based and chemistry-based technologies to enable efficient discovery of new compounds that may serve as potential treatments for significant unmet medical needs. We have established drug discovery and technical expertise in the areas of molecular biology, ultra-high throughput screening, molecular and behavioral pharmacology, and combinatorial, medicinal and analytical chemistry. In addition, we collaborate with world-renowned scientists, clinicians and academic institutions.

All of our drug candidates that are currently in clinical trials, preclinical testing and earlier stages of discovery were discovered using our proprietary drug discovery platform. We believe that the breadth of our discovery and development programs and the rapid pace at which we have discovered drug candidates provide strong validation of our technology platform.

Drug Discovery Approach

Our drug discovery approach is designed to introduce chemistry at an early stage in the discovery process and to enable selection of the most attractive key drug targets using established clinical experience. We have developed three proprietary target-based platforms to screen G-protein coupled receptors, nuclear receptors, and tyrosine kinase linked receptors, some of the most relevant and feasible targets for small molecule discovery.

We use our proprietary assay platforms in two ways. First, we profile our collection of reference drugs, which consists primarily of currently and formerly marketed central nervous system drugs, over the range of targets in our assays to identify targets that we believe are responsible for various side effects of these drugs.

Our discoveries of pimavanserin and ACP-104 resulted from the successful application of this approach. We discovered that the only property that predicted atypical antipsychotic clinical activity was inverse agonism at the 5-HT_2A receptor. This important finding led us to the discovery of selective 5-HT_2A inverse agonists that we are developing as treatments for a variety of central nervous system disorders. In the case of ACP-104, we found that, of all of the clinical compounds within our reference library, only ACP-104 was a robust m1 muscarinic agonist, thus suggesting the cognitive benefits of ACP-104.

Second, we broadly screen large numbers of targets to identify the most attractive target-specific chemistries that we can then use as starting points for our drug programs.

Using this approach, we discovered that one of our target-specific chemistries demonstrated activity in preclinical models of neuropathic pain, providing the starting point for our collaborative neuropathic pain development program. Similarly, one of our selective muscarinic agonists was active in a glaucoma model without showing classical side effects, providing the starting point for our collaborative glaucoma development program.

Target-Based Discovery Technologies

The human genome project has provided information about the genetic structure of essentially all of the potential drug targets in the human genome. This knowledge, when combined with our proprietary technologies, allows for the efficient testing of the effects of chemical compounds on a wide range of potential drug targets. Within the human genome there are families of genes that include the most frequent targets of drugs. We focus our drug discovery efforts on those families of targets that are most likely to be affected by small molecule drugs.

R-SAT Functional Assay Technology – Our proprietary receptor selection and amplification technology, which we refer to as R-SAT®, is a valuable component of our drug discovery platform. R-SAT is a cell-based assay system where genes are transferred to cultured cells. The functional activities of the gene products, or potential drug targets, are then evaluated through signal transduction pathways that lead to cellular growth. The growth signals are reported using marker gene technologies. Thus, effects of drugs on potential drug targets can be efficiently detected as changes in color or fluorescence. R-SAT enables the efficient screening of large compound libraries for identification of new chemistries at given targets, as well as detailed pharmacological testing of compounds at a wide range of targets.

Proprietary Receptor Assay Platforms – Our scientists have cloned the genes for the majority of the targets in the G-protein coupled receptor and nuclear receptor gene families. These represent the largest families of genes targeted by known drugs. Our R-SAT assay system has enabled the building of functional assays for most of these genes yielding robust assay platforms, which we refer to as GPCR-SAT and NR-SAT.

Chemistry-Based Discovery Technologies

Our drug discovery approach aims to identify small molecules that can serve as chemical starting points, or leads, for optimization efforts providing novel, potent and selective drug candidates for targets that are most likely to be affected by small molecule drugs. To enable our screening operation to identify high quality leads, we have assembled a large proprietary chemical library of diverse compounds. Our reference drug library provides us with the opportunity to validate targets and is another key component of our drug discovery platform. Our reference drug library includes a wide range of the known central nervous system active drugs, and our diverse compound library consists of roughly 800,000 small organic molecules. We have also developed proprietary synthetic methods for library construction and lead optimization.

Drug Discovery Opportunities

Our proprietary drug discovery platform has generated a wide range of novel chemistries that we believe will continue to provide us with starting points for additional drug programs. Using these target-specific chemistries, we have established a portfolio of proprietary drug discovery assets and projects in four key therapeutic areas, including neuropsychiatry, neuropathic pain/inflammation, endocrinology, and metabolic syndrome. Our discovery projects aim to answer specific scientific questions using relatively-limited synthetic chemistry and biological efforts. When all key criteria have been fulfilled, these earlier-stage discovery projects may be advanced into preclinical programs.



		Home » Technology Technology Our proprietary drug discovery platform combines target-based and chemistry-based technologies to enable efficient discovery of new compounds that may serve as potential treatments for significant unmet medical needs. We have established drug discovery and technical expertise in the areas of molecular biology, ultra-high throughput screening, molecular and behavioral pharmacology, and combinatorial, medicinal and analytical chemistry. In addition, we collaborate with world-renowned scientists, clinicians and academic institutions. All of our drug candidates that are currently in clinical trials, preclinical testing and earlier stages of discovery were discovered using our proprietary drug discovery platform. We believe that the breadth of our discovery and development programs and the rapid pace at which we have discovered drug candidates provide strong validation of our technology platform. Drug Discovery Approach Our drug discovery approach is designed to introduce chemistry at an early stage in the discovery process and to enable selection of the most attractive key drug targets using established clinical experience. We have developed three proprietary target-based platforms to screen G-protein coupled receptors, nuclear receptors, and tyrosine kinase linked receptors, some of the most relevant and feasible targets for small molecule discovery. We use our proprietary assay platforms in two ways. First, we profile our collection of reference drugs, which consists primarily of currently and formerly marketed central nervous system drugs, over the range of targets in our assays to identify targets that we believe are responsible for various side effects of these drugs. Our discoveries of pimavanserin and ACP-104 resulted from the successful application of this approach. We discovered that the only property that predicted atypical antipsychotic clinical activity was inverse agonism at the 5-HT_2A receptor. This important finding led us to the discovery of selective 5-HT_2A inverse agonists that we are developing as treatments for a variety of central nervous system disorders. In the case of ACP-104, we found that, of all of the clinical compounds within our reference library, only ACP-104 was a robust m1 muscarinic agonist, thus suggesting the cognitive benefits of ACP-104. Second, we broadly screen large numbers of targets to identify the most attractive target-specific chemistries that we can then use as starting points for our drug programs. Using this approach, we discovered that one of our target-specific chemistries demonstrated activity in preclinical models of neuropathic pain, providing the starting point for our collaborative neuropathic pain development program. Similarly, one of our selective muscarinic agonists was active in a glaucoma model without showing classical side effects, providing the starting point for our collaborative glaucoma development program. Target-Based Discovery Technologies The human genome project has provided information about the genetic structure of essentially all of the potential drug targets in the human genome. This knowledge, when combined with our proprietary technologies, allows for the efficient testing of the effects of chemical compounds on a wide range of potential drug targets. Within the human genome there are families of genes that include the most frequent targets of drugs. We focus our drug discovery efforts on those families of targets that are most likely to be affected by small molecule drugs. R-SAT Functional Assay Technology – Our proprietary receptor selection and amplification technology, which we refer to as R-SAT®, is a valuable component of our drug discovery platform. R-SAT is a cell-based assay system where genes are transferred to cultured cells. The functional activities of the gene products, or potential drug targets, are then evaluated through signal transduction pathways that lead to cellular growth. The growth signals are reported using marker gene technologies. Thus, effects of drugs on potential drug targets can be efficiently detected as changes in color or fluorescence. R-SAT enables the efficient screening of large compound libraries for identification of new chemistries at given targets, as well as detailed pharmacological testing of compounds at a wide range of targets. Proprietary Receptor Assay Platforms – Our scientists have cloned the genes for the majority of the targets in the G-protein coupled receptor and nuclear receptor gene families. These represent the largest families of genes targeted by known drugs. Our R-SAT assay system has enabled the building of functional assays for most of these genes yielding robust assay platforms, which we refer to as GPCR-SAT and NR-SAT. Chemistry-Based Discovery Technologies Our drug discovery approach aims to identify small molecules that can serve as chemical starting points, or leads, for optimization efforts providing novel, potent and selective drug candidates for targets that are most likely to be affected by small molecule drugs. To enable our screening operation to identify high quality leads, we have assembled a large proprietary chemical library of diverse compounds. Our reference drug library provides us with the opportunity to validate targets and is another key component of our drug discovery platform. Our reference drug library includes a wide range of the known central nervous system active drugs, and our diverse compound library consists of roughly 800,000 small organic molecules. We have also developed proprietary synthetic methods for library construction and lead optimization. Drug Discovery Opportunities Our proprietary drug discovery platform has generated a wide range of novel chemistries that we believe will continue to provide us with starting points for additional drug programs. Using these target-specific chemistries, we have established a portfolio of proprietary drug discovery assets and projects in four key therapeutic areas, including neuropsychiatry, neuropathic pain/inflammation, endocrinology, and metabolic syndrome. Our discovery projects aim to answer specific scientific questions using relatively-limited synthetic chemistry and biological efforts. When all key criteria have been fulfilled, these earlier-stage discovery projects may be advanced into preclinical programs. back to top