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New blindness treatment envisioned by MUSC spinoff; MitoChem raises $2M

5:53 pm by | 0 Comments

A Medical University of South Carolina (MUSC) spinoff researching a treatment for degenerative retinal conditions such as macular degeneration is closing in on a drug candidate that could slow vision loss or even restore some lost vision.

MitoChem has received a $2 million financial commitment from the nonprofit Foundation Fighting Blindness to select the best candidate from three compounds. Charleston, South Carolina-based MitoChem had previously screened a library of 50,000 compounds to narrow its choices to the three compounds with vision-saving properties. MitoChem will use the $2 million grant to conduct the necessary preclinical studies to take the best compound into human testing, Stephen Rose, the foundation’s chief research officer, told MedCity News.

Degenerative retinal conditions such as macular degeneration and retinitis pigmentosa affect more than 10 million Americans, according to Foundation Fighting Blindness. MitoChem is addressing this medical need by researching ways to protect mitochondrial function in cells. Mitochondria are elements in cells that produce energy.

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“They are the power plants of the cell,” Rose said. “If something goes wrong and you lose power, what happens? Stuff doesn’t run.”

Patients who have eye disorders such as retinitis pigmentosa and macular degeneration experience diminished mitochondria function, which contributes to vision loss. MitoChem’s approach would not cure the primary defect in these eye disorders. But a drug that improves mitochondria function could help keep cells going, Rose said. There are no U.S. Food and Drug Administration-approved drugs that address mitochondrial function. In animal studies, the three compounds that MitoChem selected showed good results preserving and even restoring vision. MitoChem aims to develop a new eye drug candidate in an eye drop formulation.

MitoChem was founded by Dr. Barb Rohrer, an MUSC professor and expert in retinal degeneration, and Dr. Craig Beeson, an MUSC medicinal chemist. Foundation Fighting Blindness funded Rohrer’s early mitochondria research. The foundation provides research, clinical trial and regulatory support to companies who are studying new eye treatments. Rose said that the foundation’s financial support is intended to fill the gaps left by early stage investors who have cut back plans for new investments.

“We look at things very early,” Rose said. “Our job is to be that angel investor to get them through the valley of death.”

If a company advances into clinical trials, the Foundation Fighting Blindness’ clinical trials support organization, National Neurovision Research Institute (NNRI), works with the company in phase 1 and possibly phase 2 clinical trials. NNRI may look to syndicate with additional investors to financially support further clinical work, with NNRI due to receive royalties from any commercialized treatment. Rose said the ultimate goal is get a company’s technology acquired or licensed by a larger company that has greater resources for late-stage testing as well as commercialization. That acquirer would buy out NNRI’s royalties. The foundation has committed about $8 million in funding to several companies. Milestones are attached to all of the financial commitments, which are grants and not equity investments.

[Photo from Flickr user orangeacid]

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By Frank Vinluan

Frank Vinluan is the North Carolina Bureau Chief for MedCity News.
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