Therapy shows promise in preventing retinal degeneration
CLEVELAND — May 17, 2018 — OptiKira, LLC, a privately-held biotechnology startup developing drugs that inhibit the unfolded protein response (UPR) with applications in a variety of diseases, including retinitis pigmentosa, type I diabetes, idiopathic pulmonary fibrosis (IPF), amyotrophic lateral sclerosis (ALS), and immuno-oncology announced today that it achieved a significant proof-of-principle milestone.
OptiKira’s compounds are the most highly potent and selective known inhibitors of IRE1α, an enzyme responsible for pathogenic UPR and cell destruction. These agents significantly slowed the progression of retinitis pigmentosa, providing protection from degeneration of the retina when tested in a representative genetic model of the disease at Charles River Laboratories.
“Demonstrating the efficacy of our molecules in a retinitis pigmentosa rat model is a critical proof-of-principle milestone,” said Ted Torphy, CSO of BioMotiv and Chairman of the OptiKira board. “These compounds protect the eye’s photoreceptor in an aggressive model that is representative of the human disorder — a very encouraging result that could lead us toward a therapy to manage a disease for which there is currently no treatment.”
Retinitis pigmentosa is a rare, genetic disorder that causes the breakdown and loss of cells in the retina, resulting in progressive loss of vision. OptiKira’s research team discovered that, by targeting the UPR and regulating its activity to favor an adaptive rather than destructive pathway, cell death and degeneration is prevented not only in retinitis pigmentosa, but in a variety of other diseases.
Proof of concept has also been demonstrated in mouse models of diabetes; with ongoing proof of concept studies underway for ALS and IPF. OptiKira has received funding from the ALS Association and is currently conducting in vivo studies in ALS mouse models.
OptiKira was formed in 2015 by BioMotiv, LLC, the for-profit collaborator of The Harrington Project for Discovery & Development, the University of California, San Francisco, and the University of Washington. The company has received support from Arix Bioscience, the Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, which is supported in part by a grant from the Ohio Third Frontier, two National Institutes of Health Small Business Technology Transfer grants, and the ALS Association .
For additional information about OptiKira, go to: http://www.optikira.com.
BioMotiv is the mission-driven accelerator associated with The Harrington Project for Discovery & Development, a $340 million initiative for advancing medicine centered at University Hospitals in Cleveland. The focus is to accelerate breakthrough discoveries from research institutions into therapeutics for patients through an innovative model that efficiently aligns capital and collaborations. The company leverages an experienced team and advisory board to select, fund, and actively manage and advance a portfolio of drug development programs.
For more information, go to: www.biomotiv.com.
About The Harrington Project for Discovery & Development
The Harrington Project for Discovery & Development (The Harrington Project) is a $340 million national initiative built to bridge the translational valley of death. It includes the Harrington Discovery Institute at University Hospitals in Cleveland and BioMotiv, a for-profit, mission-aligned drug development company that accelerates early discoveries into medicines for benefit of society.
For more information about The Harrington Project and the Harrington Discovery Institute, visit: HarringtonDiscovery.org.
About Arix Bioscience Limited and Group
Arix Bioscience plc is a global healthcare and life science company supporting medical innovation. Headquartered in London and with an office in New York, Arix Bioscience sources, finances and builds world class healthcare and life science businesses addressing medical innovation at all stages of development. Operations are supported by privileged access to breakthrough academic science and strategic relationships with leading research accelerators and global pharmaceutical companies.
Arix Bioscience plc is listed on the Main Market of the London Stock Exchange.
For more information, go to: www.arixbioscience.com