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BIOMARKET TRENDS Biobusiness Top Biotech & Pharma Companies As the global business environment continues to recover from the fnancial meltdown in 2008, biotech and pharma companies look like they are mending well. Indeed, as veteran bioindustry market guru G. Steven Burrill reported in July, there's been a strong demand for life sciences IPOs this year. Through the frst seven months of 2013, 29 life science companies raised a total of $5 billion on U.S. stock exchanges. This compares to 11 companies raising $771 million during the same period a year ago. Earlier this year, Burrill pointed out that Big Biotech frms (e.g., Amgen, Gilead Sciences, Regeneron Pharmaceuticals, and Biogen Idec) have outpaced Big Pharma (e.g., Johnson & Johnson, Pfzer, Roche, Novartis, and Sanof) in terms of growth of sales, income, and investment in R&D; during the past three years. "As the industry migrates away from an era of the one-size-fts all blockbuster, the bio- technology industry's strength at developing innovative therapies that meet unmet medical needs and target the molecular mechanisms of diseases gives it an upper hand in creating value," said Burrill, who serves as CEO of Burrill & Company, a global fnancial services frm. "But as pricing pressures become an increasingly challenging prospect for pharmaceutical and biotechnology companies alike, they will need to fnd new ways to build relationships Annual Top 25 Biotechnology Companies Rank Company 2013 Market Cap (in billions) 1,10 2012 Market Cap (in billions) 1,10 % Change 1. Novo Nordisk $85.335 $62.809 2. Amgen $78.695 $55.054 42.9% 3. Gilead Sciences $78.373 $19.751 296.8% 4. Celgene $52.557 $34.319 53.1% 5. Biogen Idec $47.157 $31.582 49.3% 4,9 4,9 35.9% 6. 2013 Market Cap (in billions) Teva Pharmaceutical Industries $33.768 $39.292 -14.1% 7. Annual Top 15 Pharmaceutical Firms Rank Firm with payers, providers, and patients to capture value outside of their products." But, as you can see from our two GEN listings—Top 25 Biotech Companies of 2013 and Top 15 Pharma Companies of 2013— the market caps of the leading frms in both sectors are healthy indeed. These numbers not only provide a reading on a company's current value but hint at, not guarantee, a frm's future performance. Merck KGaA $33.3644 $22.3194 49.5% 1,8 1,8 8. 2012 Market Cap (in billions) % Change CSL $27.440 $18.359 49.5% 9. Regeneron $20.2697 $11.8947 70.4% 10. Alexion $17.893 $17.152 4.3% 1. Johnson & Johnson $248.39313,14 $189.04613,14 31.4% 11. Shire $17.252 $17.205 0.3% 2. Pfizer $203.717 $168.109 21.2% 12. Vertex Pharmaceuticals $17.495 $8.151 114.6% 3. Novartis $173.4001,12 $133.9001,12 29.5% 13. UCB $11.6501,6 $7.4491,6 1,11 1,11 15.0% 14. BioMarin Pharmaceutical $9.097 $3.788 140.2% $98.3221,10 50.9% 15. Elan $7.0414,5 $8.8684,5 -20.6% 1,3 1,3 4. Roche $166.477 5. Sanofi $148.3811,10 $144.778 9 56.4% 6. Merck & Co. $141.922 $118.710 19.6% 16. Actelion $6.885 $4.548 51.4% 7. GlaxoSmithKline $112.5111,7,8 $104.8521,7,8 7.3% 17. Onyk Pharmaceuticals $6.580 $3.010 118.6% 8. Bayer $87.5271,6 $57.3651,6 52.6% 18. Dr. Reddy's Laboratories $5.0141,2 $5.0011,2 9. 10. 11. 2 2 0.3% Bristol-Myers Squibb $67.657 $57.007 18.7% 19. Seattle Genetics $4.390 $2.316 89.6% AbbVie $64.5842 N/A5 N/A5 20. Alkermes $4.011 $2.356 70.2% $46.312 39.7% 21. Ariad Pharmaceuticals $3.300 $2.701 22.2% Eli Lilly 4 $64.687 2 2 12. AstraZeneca $62.475 $56.680 10.2% 22. Cubist Pharmaceuticals $3.059 $2.668 14.7% 13. Takeda $40.0561,2,3 $29.0271,2,3 38.0% 23. United Therapeutics $2.966 $2.260 31.2% 1,2,3 1,2,3 14. Astellas $23.888 $16.050 48.8% 24. Ipsen Group $2.889 $2.230 29.6% 15. Daiichi Sankyo $12.8761,2,3 $10.7011,2,3 20.3% 25. Isis Pharmaceuticals $2.324 $0.803 189.2% *Notes available on GEN's website: www.GENengnews.com 1 1 *Notes available on GEN's website: www.GENengnews.com CORPORATE PROFILE Warp Drive Bio part of the company's efforts. With sequence assemblies in hand, Warp Drive is performing what it calls a "genomic search"—extracting biosynthetic pathways of interest. This step, Dr. Verdine said, "is sort of like looking through a blueprint for a very particular type of machine on a factory foor." Having identifed the DNA encoding the entire pathway of interest, the team clones it, using genomeediting techniques to make manipulations at the single-nucleotide level. From there, purifcation and target identifcation/mechanismof-action studies are performed. "Every single one of these operations is completely interdependent on the operation that came before it," Dr. Verdine said. "It's the convergence of all these unique areas of expertise and really getting them to work 14 | Continued from page 12 with each other that makes this such an extraordinary operation." In essence, Warp Drive is bringing natural products up to speed in the genomics era. And that's part of the story behind how the frm got its name. When the company was incorporated, Dr. Verdine chose its name as a placeholder. "I thought: We're basically taking this feld and putting it through a time warp…so I said 'Let's call it Warp Drive'—because we were providing that propulsion," he said. However, it was because of Sanof's global R&D; president Elias Zerhouni, M.D., that the name stuck. "Elias loved the name. He loved it so much he began using the term 'warp speed' at Sanof," Dr. Verdine explained. Eventually, he added, "people in Sanof started September 1, 2013 | GENengnews.com | Genetic Engineering & Biotechnology News calling Elias 'Warp Speed'—that became his nickname. I think he loved it because it really symbolized what he was trying to do in Sanof when he came in." What Dr. Verdine is trying to do with Warp Drive, he said, is not unlike what many frms are doing in the systematic search for biologics. "Acquiring sequence information, assembling sequence information, fguring out how to search it effectively, moving genes around [is] something that people have shown you could do, but had not been in any way industrialized," he said. "So we had to show that we could do all of that…and that, when we did the sequencing, we could prove that there were novel, exciting molecules hiding in plain sight in the genomes of these bacteria." Though fewer are submitted, when it comes to FDA approvals, natural products outperform their synthetic counterparts. But natural products are not without their problems. Without critical discoveries related to deconstructing complex biosynthetic pathways and advances in assembling highly repetitive genomes made largely in academic labs, Warp Drive scientists may not have even fathomed attempting what they have been able to accomplish thus far. "As pharma was abandoning it [natural products], academic groups had effectively reinvented the feld from the ground up," Dr. Verdine said. "And we capitalized on those fndings in the academic world. That set of discoveries—and also the access to ultrahigh-throughput DNA sequencing—made it possible to do what Warp is doing."