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Summit, Phylonix Score New Funding to Screen Zebrafish for DMD, Eye Disease

An influx of funding was announced this week by two players in the zebrafish screening market. Summit announced that it received £220,000 ($448,000) from Parent Project UK to support Summit’s preclinical Duchenne muscular dystrophy program as well as an early-stage research project called the ZF Partnership that is seeking to identify additional DMD therapies using zebrafish models of the disease.
Separately, Phylonix said that it received a three-year, $1,092,031 Phase II Small Business Innovation Research Grant from the National Eye Institute to develop zebrafish models for ophthalmic diseases such as diabetic retinopathy and age-related macular degeneration.
Reaching the Summit
Summit, formerly called VASTox, has zebrafish models of DMD, but lacks the budget to develop the model further and scale up its screening efforts, CEO Steven Lee told CBA News this week.
Lee said that the current round of PPUK funding “is only the first half of the project” and that the company is “working closely with PPUK to raise even more funds.”
The agreement is open ended, Lee said, because as hits emerge from the screens that Summit is now doing, it will need more financing for lead optimization programs, medicinal chemistry, and clinical trials. 
Lee said that the company has already established tissue culture labs, medicinal chemistry facilities, and other infrastructure through investment financing, so the PPUK funding will go directly into the DMD program.
The company will use the PPUK funds to hire two or three scientists to run the screens and to purchase any additional compound libraries that may be necessary, Lee said.
Summit said that PPUK raised the funding for the project from private sources including Charley’s Fund in the US and the Gavriel Meir Trust in the UK. PPUK will seek to raise additional funds to further support Summit’s DMD program in the future, said Lee. He was unable to specify a definite timeline, however, beyond saying that the partnership between Summit and PPUK is ongoing.
Lee said that he hopes that Summit finds new hits within the next six to nine months, and then in about 12 months or so, the company will require more funding to start medicinal chemistry programs around those hits.
Proactive Parents
PPUK was founded as a charity about five or six years ago by parents of children with DMD, Lee said, noting that these parents had become “a little disenfranchised” by the Muscular Dystrophy Campaign in the UK, because the MDC was satisfied by funding just “the odd bit of research here and there” at universities.
The PPUK parents were adamant about finding a cure, said Lee, so when they heard of Summit’s DMD research program and its lead candidate SMT C1100, there was a good alignment of both group’s goals.
PPUK did not respond to requests for comment before the CBA News deadline.
Eye to Eye
Phylonix, meanwhile, will use its SBIR funds to develop drugs that promote ocular neovascularization, said Ed O’Lear, vice president of finance and business development at Phylonix. He said that the company does not have plans to hire additional people or expand its facilities specifically for this project.
Phylonix will screen compound libraries that it has purchased and compare the results of the hits identified in zebrafish to those identified using a mouse corneal pocket assay, O’Lear said.
According to the company, zebrafish is an effective model for eye disease because the basic structure between human and zebrafish eyes is “indistinguishable.”
Phylonix is modeling several eye diseases in zebrafish including diabetic retinopathy and age-related macular degeneration. Both conditions involve vascular abnormalities, proliferation, and leakage of new blood vessels.
According to the company, mammalian models for ocular neovascularization “require lengthy, tedious surgical manipulation and do not always result in improved vision.” The company is betting that its zebrafish platform will prove to be “an alternative rapid, less invasive animal model for studying the process of ocular neovascularization and assessing drug effects.”
More Fish in the Sea       
The two awards underscore a rising trend in the use of zebrafish screening in the industry — a pattern that some observers see as destined to continue.

“I suspect it will be used more by academics, and I think that the pharmaceutical industry will use zebrafish more as well.”

“I suspect it will be used more by academics, and I think that the pharmaceutical industry will use zebrafish more as well,” William Trevarro, director of the zebrafish facility at the University of Oregon and a self-described “zebrafish evangelist,” told CBA News.
Trevarro said that researchers from a drug company recently spoke at his lab about studies they were doing to demonstrate that they could use zebrafish screening as an initial screen for compounds prior to testing in more expensive models, such as mice and humans. “I would expect that there are a lot of other things that those guys could use zebrafish to do,” he said.
In addition, Trevarro said, while zebrafish were traditionally used primarily in the field of developmental genetics, they are becoming more prevalent in other fields, such as toxicology.
Peter Eimon, director of research at Zygogen, said that one of the most attractive features of the zebrafish model is the fact that it is an intact living organism on which one can conduct high-throughput screens. He said that some companies are currently doing zebrafish screens on the order of greater than 1,000 compounds.
Phylonix cites a number of advantages of zebrafish over other animal models, including their small size, low maintenance costs, and their ability to breed quickly in large numbers. The company estimates that a typical zebrafish assay costs less than $100.
In another sign that the market for zebrafish screening is opening up, new companies are entering the field. One new player, Marksman Cellject, a Toronto-based startup formed to commercialize automated zebrafish embryo microinjection technology, published a paper in PLoS One last month describing its platform.
The company is now seeking seed funding so it can deliver prototypes of its instrument to drug-screening labs by the end of the year, CBA News sister publication Biotech Transfer Week reported two weeks ago.
The company is commercializing the work of Yu Sun, a professor of mechanical and industrial engineering at the University of Toronto, who developed a fully automated platform for injecting biological materials into zebrafish embryos.
As reported in the PLoS One paper, the microrobotic system can inject foreign material such as DNA, RNA, sperm, protein, or drug compounds into zebrafish embryos at a speed of 15 embryos per minute, with a survival rate of 98 percent and a success rate of 99 percent — a far greater efficiency than most available automated techniques, and much faster than manual microinjection.
Trevarro said that Sun’s work is “technically pretty cool” and that the automated method would likely be useful for a large operation such as a pharmaceutical or biotech company.
Eimon pointed out that the 99 percent success rate of the system is in the range of what a skilled technician would be able to achieve manually.
“Their throughput also looked pretty good to me,” Eimon said, acknowledging that the rate of 15 embryos per minute is not quite as high as a technician, but pointing out that a technician would not be able to go straight through for a couple of hours.
Trevarro and Eimon both said that the use of an automated system would free up technicians for other tasks, such as collecting and preparing the eggs to do the next injection.
Biotech Transfer Week reported that the initial cost of one of Marksman Cellject’s instruments for zebrafish embryo injection would likely be around $200,000.

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