How Japan Is Creating New Opportunities in the Field of Regenerative Medicine - SPONSOR CONTENT FROM THE GOVERNMENT OF JAPAN
As biotech companies race toward the opportunities in Japan, no life sciences sector has received more attention, government support, and regulatory reform than the important field of regenerative medicine. Regeneratives are pharmaceutical cell therapy products that replace or restore cells and tissues lost to disease or aging. Some regenerative medicines stimulate our bodies to regrow or repair, so the field can include the use of stem cells for tissue engineering. Japan’s rapidly aging population creates a perfect test kitchen for such regeneratives, especially as the aging trend expands to Europe and North America. Meanwhile, regenerative medicine is at the forefront of scores of research partnerships between private biotech companies and Japanese universities. “The Japanese want to be the regenerative medicine center of the world,” said Colin Lee Novick, managing director of the CJ Partners biotech consulting firm in Tokyo. “To be able to do that, they need to entice companies to come to Japan, and they need to entice their own pharmaceutical companies to license in and obtain the best.” The “regen” boom in Japan is based on a series of reforms and new laws for pharmaceuticals, but especially the landmark Pharmaceuticals and Medical Devices Act (PMD Act) and the Act on the Safety of Regenerative Medicine. The result is the ability to offer conditional marketing approval for a regen so it can move more quickly to commercialization – an approval process widely seen as the fastest in the world. Regens also benefit from what is known as the Sakigake Package of policy changes, which speed up the introduction of innovative medical products. The package focuses on everything from basic research to clinical research/trials, approval reviews, safety measures, insurance coverage, improvement of infrastructure and the environment for corporate activities, and global expansion. It includes a designation system that promotes R&D in Japan aimed at early practical application for innovative pharmaceutical products, medical devices, and regenerative medicines. It also includes a scheme for rapid authorization of unapproved drugs, which speeds the practical application of unapproved/off-label use of drugs for serious and life-threatening diseases. But the benefits don’t stop there, Novick points out. In addition to the conditional approval and Sakigake benefits, Japanese regens arrive in one of the world’s largest markets for pharmaceuticals. The result since 2014 has been a flood of partnerships, research studies, and licensing deals. Gene therapy is also a part of this trend. Agilis Biotherapeutics, a U.S. company, is partnered with Gene Therapy Research Institution of Japan to develop and obtain approval for medicines to treat Parkinson’s disease and another nerve disease. “R&D requires extremely high skills, but manufacturing requires even higher skills. There are very few places that can provide high-quality manufacturing capabilities in the U.S. and also in the world. After a thorough valuation, they came to believe the Japanese Gene Therapy Research Institution had the best capabilities (talent and skills),” said President Katsuhito Asai of Agilis GTRI Japan, a joint venture between Agilis and the Japanese Gene Therapy Research Institution. Foreign companies that have decided to conduct R&D in Japan for innovative medical products are entitled to a research grant from METI to help with their capital investment. In addition, local governments provide their own grants. Agilis GTRI Japan is located in a public-private research park in Kanagawa Prefecture, which has adopted the National Strategic Special Zone concept to cut regulation and offer other benefits for innovation companies. Among the larger partnerships are some iconic Japanese companies. Nikon continues to move into health care by partnering with Lonza of Switzerland – the world’s leading developer of regenerative medicine cells – to build a cell and gene therapy manufacturing plant. Hitachi has partnered with Caladrius Biosciences of the U.S., and Asahi Glass Co. is working with Regeneus of Australia. That these classic Japanese companies known for products in other fields are moving into life sciences underscores the lure of both regens and Japan’s lucrative home markets. Other partnerships or licensing deals involve Athersys of the U.S. and Healios of Tokyo, to develop and produce the MultiStem therapy for stroke victims; Celixir of the U.K., working with Daiichi Sankyo on the Heartcel heart failure medicine; the Belgian firm TiGenix partnering with Takeda Pharmaceuticals on a Crohn’s disease treatment; and Kolon Life Science of South Korea collaborating on the Invossa arthritis drug with Mitsubishi Tanabe Pharmaceutical Co. The mutual benefits of such partnerships were demonstrated when Takeda recently announced it was buying TiGenix. Among the companies directly crediting the conditional approval process in Japan for its interest is Israel’s Pluristem, which is partnering with Sosei CVC to develop placenta-derived cells to speed healing in patients with critical limb ischemia, or CLI. “The development of our CLI program through the accelerated regulatory pathway could allow a more rapid entrance into the sizeable Japanese market, as has been our strategy,” said Zami Aberman, Pluristem’s chairman and CEO. In regeneratives and the broader field of pharmaceuticals, a plethora of non-Japanese companies have formed research collaborations with Japanese universities, which have in recent years moved strongly into commercialization and outside relationships. From the U.S., Merck and Pfizer are working with the University of Tokyo, and the U.K’s AstraZeneca conducts collaborative research with Osaka University and National Cancer Center Japan. Pluristem works with Fukushima Medical University. Pointing out the many deals, collaborations, and new regen opportunities, the consultant Novick advises non-Japanese companies to “definitely jump in. … It’s a nascent and rapid-growth market. Because the cement is still drying on the rules and regulations and guidelines, if you want to be one of the parties that is going to be able to set the tone in this market, you must enter now.” Find more at Japan.go.jp/abenomics.
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Donald Trump will nominate Scott Gottlieb, a conservative drug industry insider and former FDA and CMS official, to serve as FDA commissioner, industry sources close to the White House tell POLITICO.Gottlieb, a physician, is a far more traditional pick to lead the Food and Drug Administration than other candidates Trump had considered. One, Silicon Valley investor Jim O’Neill, had shocked public health advocates with his comments that the FDA should approve drugs without proof they worked. However, Gottlieb’s close ties with industry are likely to draw Senate scrutiny. A classic Washington insider who has occupied conservative turf in the health care debate for two decades, Gottlieb is a board member or adviser to various drug companies and plays a role in funding the industry through a venture capital firm, New Enterprise Associates.If confirmed, Gottlieb would play a major role in the Trump’s administration’s efforts to speed up the regulatory approval of drugs. Trump, who’s been critical of high drug prices, has often argued that shortening the FDA approval process and cutting regulations would bring prices down.The White House could announce Gottlieb's nomination as soon as Friday.The FDA, which oversees the review and approval of drugs, vaccines and medical devices, and the regulation of tobacco, food and cosmetics, has an annual budget of $5 billion, close to 15,000 employees, and plays a regulatory role in about a fifth of the economy. A slew of FDA user fee programs, in which industry essentially funds half the FDA budget, are up for reauthorization in the fall; it would fall upon Gottlieb, if approved, to shepherd the agreements — negotiated under his predecessor — through Congress.Gottlieb has broad experience in regulating health care and working for the industry. He received nearly $200,000 in payments in 2015 from eight pharmaceutical companies, according to federal database tracking drug industry payments. All were classified as “general” payments, meaning they were for things like travel and lodging, consulting or speaking fees. In 2014, he received more than $160,000 in general payments from companies.Gottlieb has been on GlaxoSmithKline’s research and development board since 2010, according to his résumé, and previously served on its oncology board. He advises Bristol Meyers Squibb on its cancer drugs and Cell Biotherapy, an oncology start-up. Gottlieb was a senior adviser to Vertex Pharmaceuticals, maker of expensive cystic fibrosis drugs, from 2009 through 2016. He holds seats on the boards of drug companies Daiichi Sankyo and Tolero Pharmaceuticals; medical lab company American Pathology Partners; MedAvante, a contract research organization, and Glytech, which makes an FDA-approved insulin dosing support system. He also has served on the board of insurance and medical diagnostic companies.Gottlieb was the FDA’s deputy commissioner for medical and scientific affairs from 2005 to 2007, and chief policy adviser to the CMS administrator in 2004, during implementation of Medicare Part D. Earlier, he was a senior adviser and director of medical policy development at the FDA, where he worked on issues like orphan drugs and combination products. Gottlieb has long been a resident fellow at the right-leaning American Enterprise Institute. He serves on the Federal Health IT Policy Committee, which makes recommendations to the Department of Health and Human Services Office for Healthcare Information Technology. He practices medicine at New York University where he is a clinical assistant professor. Gottlieb's free-market health policy positions are well known, thanks to regular op-eds in The Wall Street Journal and Forbes, and FDA-related testimony to Congress. This past fall, he gave testimony criticizing the FDA’s regulation of generic drugs, during a hearing on pharmaceutical industry competition occasioned by the rising prices of the EpiPen.
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