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COMPANIES TO WATCH Column 14 LIFESCIENCELEADER.COM JUNE 2014 W A Y N E K O B E R S T E I N Executive Editor Finances Research Partnership Funding Other Partners Vital Statistics DAVID AVIEZER President and CEO PROTALIX BIOTHERAPEUTICS B y W. Koberstein Fundação Oswaldo Cruz, Ministry of Health, Brazil Uplyso supply & technology transfer agreement Teva R&D; with ProCellEx platform Raised $100M in underwritten public offerings 260 Employees Headquarters Carmiel, Israel Latest Updates February 2014: Reported top-line Phase 1 clinical trial results for oral glucocerebrosidase in Gaucher patients. 2013: Continued world- wide approvals of Elelyso / Uplyso. August 2013: Added three new plant-cell recom- binant proteins to the development pipeline. SNAPSHOT Protalix has a unique plant-cell platform for therapeutic protein production and an approved product, Elelyso/Uplyso (taliglucerase alfa), for Gaucher disease. In the company's pipeline is a chemically modified version of the recombinant alpha-Galactosidase-A protein for Fabry disease (Phase 1/2), an oral glucocerebrosidase (GCD) enzyme replacement therapy for Gaucher disease (Phase 1), an oral anti-TNF (tumor necrosis fac- tor) fusion protein for autoimmune/inflammato- ry conditions (preclinical), and a human deoxyri- bonuclease I (DNase I) for cystic fibrosis (preclin- ical), and other enzyme replacement therapies in early research. WHAT'S AT STAKE Be careful when someone claims to be number one; it depends on how you define the field. Just after filing my recent story on Medicago (March 2014), I encountered another plant-technology company, Protalix, that made the fine but sig- nificant distinction that I might have other- wise failed to mention: Medicago and others in the plant-based production sector grow whole plants, such as tobacco, in large gardens or greenhouses; Protalix produces proteins in plant cells, grown like other cell media in traditional bioreactors. But it is not just its platform that sets Protalix apart — it's also the company's approved plant-cell-produced product and sev- eral other candidates in its pipeline. "In the early days of therapeutic recombinant Using plant cells instead of mammalian cells to produce protein drugs, plus a partnered product already on the market, could possibly put this company in the lead. PROTALIX BIOTHERAPEUTICS proteins, manufacturing used bacteria for the simpler proteins, then evolved into using mam- malian cells such as Chinese hamster ovary [CHO] cells," explains David Aviezer, presi- dent and CEO. "But we offer a new way to transition from using mammalian cells to using plant cells as the cell source for special therapeutic proteins." Aviezer cites a "substantially lower" cost of goods as one advantage of the Protalix platform. "As an example, it is like comparing the cost of having a pet dog or cat to having a flower pot in your living room," he says. Safety is another factor. "Humans are not affected by any plant viruses so what we have is a built-in biological firewall, preventing any kind of transmission of mammalian-associated infectious viruses or prions." Why is it significant that Protalix uses plant cells rather than whole plants? Use of bioreac- tors versus plant-growing operations keeps the technology in familiar territory for engineers, manufacturing personnel, and perhaps above all, regulators. Equipment, guidelines, procedures, and processes remain essentially the same as they have been for decades in the biotech indus- try. With typically dry humor, Aviezer says, "We grow carrot cells. And, as we like to say, the only carrots we have in our facility are the carrots in chicken soup. But we have a very well-regulated and controlled system for producing our ther- apeutic proteins that really can comply with all the regulatory necessities needed for high- quality drug production." Oral delivery is a somewhat serendipitous out- come of the plant-cell product. Protalix takes advantage of the cellulose base in its oral-deliv- ered drugs as a natural way of keeping them pro- tected from digestion until they reach the small intestine, where the physical jostling opens up pores in the cellulose, releasing the active drug where it can be absorbed intact by the gut. Aviezer refers to the anti-TNF drug as "basically a plant-cell expressed Enbrel [etanercept]" that will go into human studies this year. Although Aviezer says there is room for other plant-based technologies in drug manufactur- ing, he clearly believes easy adoption, low cost, safety, effective delivery, and protein refinements will give his company's platform the competitive edge. After 10 years in the business, having a healthy product on the market and arguably taking a technology lead, Protalix gives its CEO good reason for careful optimism. l Pf zer $98.3M for Elelyso development and commercialization 0 6 1 4 _ C o l u m n s _ C T W . i n d d 1 0614_Columns_CTW.indd 1 5 / 2 1 / 2 0 1 4 1 1 : 4 9 : 4 7 A M 5/21/2014 11:49:47 AM