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Drug discovery collaboration could lead to a long-term clinical development effort

Melissa Fassbender

By Melissa Fassbender

09-Jan-2017
Last updated on 09-Jan-2017 at 18:25 GMT2017-01-09T18:25:13Z

(Image: iStock/ktsimage)
(Image: iStock/ktsimage)

Sharp Edge Labs will use its propriety biosensor platform to identify lead compounds for Sumitomo Dainippon Pharma – technology that the company says will help focus discussions on outcomes rather than cost.

As part of the newly announced drug discovery collaboration, Sumitomo Dainippon Pharma (SDP) will provide its proprietary compound library to Sharp Edge Labs for the company to use in its drug discovery platform.

The goal of the collaboration is to identify lead compounds and potential development candidates for neurodegenerative disorders.

The collaboration involves SDP providing a specially selected compound library for us to run in an assay for restoration of a trafficking defect occurring in a genetic subset of Parkinson’s patients,” Scott Sneddon, Sharp Edge Labs, president and CEO, told us.

We run the compounds, produce the data, and decide together what compounds are worth further characterization.” 

The biosensor platform

According to the company, Sharp Edge Labs’ biosensor platform allows large-scale screening for drugs that restore proper trafficking.

The Sharp Edge Biosensor Platform provides a means of directly observing normal and altered trafficking with the sensitivity and speed required to perform full high throughput screening in the search for chemical chaperones,” explained Sneddon.

Additionally, he said the same technology allows the company to probe details of the trafficking mechanism to better understand efficacy and selectivity. “Having found a small number of hits, the compounds can then by validated in lower-throughput assays involving cells from patients with the same genetic mutation,” he added.

Validation is the last step – and as Sneddon explained – provides “a way to move the program forward in the most biologically and clinically relevant way possible.

Depending on the potency and selectivity of the compounds discovered, this effort could lead to a long-term preclinical and clinical development effort,” he added.

Next generation sequencing

According to Sneddon, next generation sequencing (NGS) is now providing the datasets that tell researchers if certain diseases can be divided into genetic cohorts. 

For many of these cohorts the cellular defect will be a trafficking mutation involving the altered protein, and for these cohorts, a small-molecule chemical chaperone approach will have attractive advantages,” he said.

Additionally, he said the same genetic patient cohorts could provide stem cell-derived model cells for studying the effects of compounds on restoring the molecular defect and that having a genetically based cohort could allow for patient stratification and the possibility of orphan drug designation.

Finally, such targeted therapies will have associated companion diagnostics, which should make pricing and reimbursement discussions more focused on outcomes than on cost,” added Sneddon.

We think these technologies: NGS, patient derived stem cells, and a precision, patient-targeted approach will drive the future value proposition for the next generation drugs to the benefit of patients, regulators, payers, and the companies that undertake drug development.”

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