Throughout the year we have published the views of company executives, government regulators, industry analysts and scientists on a variety of topics and, in our popular annual feature, we include a selection of these that paints a picture of the significant events that shaped 2019. It was a year that marked impressive scientific milestones while the drug pricing debate and Brexit drama kept industry on its toes.
“The nature of science has changed in recent years and become more transdisciplinary. It doesn’t follow the traditional, rigid discipline that it has in the past. We need different types of facilities and spaces. That will be an increasing issue going forward for NIH. Almost three-quarters of buildings are more than 20 years old, and they are not designed and constructed to support where science is going.”
Kenneth Kizer, who chaired the Committee on Assessing the Capital Needs of the NIH. A two-year study concluded that the NIH’s Bethesda, Md., campus needs a $1.3 billion upgrade to its buildings and facilities
"You can extend longevity by suppressing neural excitation and, conversely, if you make neurons hyperexcitable, that shortens lifespan... one has to be cautious, at this point, about extrapolating our findings about neural activity to higher-order brain function."
Bruce Yankner, professor of genetics at Harvard Medical School and co-director of the Paul F. Glenn Center for the Biology of Aging, and principal investigator in a study that found "a correlation between down-regulation of excitation and extended longevity."
"A large fraction of our genome used to be a virus – our genome houses ancestral infections that we've had, and that have come along for the evolutionary ride."
Nicholas Haining, Dana-Farber Cancer Institute, commenting on work with colleagues published in Nature that showed in mice, blocking ADAR (an enzyme that makes edits to RNA after it is transcribed) could sensitize tumors to checkpoint blockade even when the animals had an additional mutation that prevented antigens from being presented in a way that enabled killer T cells to recognize them. The loss of the RNA editing enzyme ADAR sensitized tumor cells to immunotherapy by increasing their inflamed state, converting them from "cold" to "hot" tumors
"NASH is a complex disease with multiple biological pathways that influence its progression. Combination therapeutic approaches, which target these pathways are likely to be needed to effectively treat patients living with NASH, particularly those with advanced fibrosis who have the greatest unmet need."
John McHutchison, chief scientific officer, head of R&D, Gilead Sciences Inc., on new data from the company's clinical research program in nonalcoholic steatohepatitis presented at The International Liver Congress in Vienna
"The field of cancer therapy has focused on treating patients after they have contracted cancer. If we could vaccinate patients and never allow the cancer to develop, the impact would be immense for patients and our health care system. Over the last decade we have gained significant understanding about the immune system to contemplate developing cancer vaccines like this."
Vincent Tuohy, Cleveland Clinic, who has been developing a method to vaccinate women against contracting breast cancer, focused specifically on triple-negative breast cancer
"We believe modulators of lipid signaling have the potential to become the next generation of cancer immunotherapies."
Masaaki Sawa, chief scientific officer and head of R&D at Carna Biosciences Inc., which signed a $470 million R&D collaboration to develop and commercialize small-molecule compounds in immuno-oncology with Gilead Sciences Inc.
"The human-on-a-chip model is pushing the field forward and transforming drug development. Pharmaceutical companies and medicinal chemists can now do the preclinical testing of compounds without the use of animal studies. The preclinical space has relied upon animal models for looking at systemic or organ-organ communication issues since its inception. We hope to provide a lot of that information before they go into animal models to make the process more efficient and reduce the number of animal models required."
James Hickman, chief scientist, Hesperos Inc.
"CAR T cells, given our current state of knowledge, are very unlikely to impact solid tumors."
Steven Rosenberg, chief of surgery at the National Cancer Institute, speaking at the annual meeting of the American Association of Immunologists
“Magnetic nanoparticles are currently under evaluation for a range of biomedical applications, but nano/micro-robots are mostly at the research level or in the early stages of clinical use or trials. Nonetheless, I am reasonably confident that within the next five years or so, such devices will be in clinical trials or usage.”
Hongsoo Choi, a professor in the Department of Robotics Engineering at the Daegu Gyeongbuk Institute of Science and Technology and lead researcher in a study, published in Science Robotics, demonstrating that magnetically manipulated micro-robots facilitated the rapid and precise targeting of transplanted stem cells in vivo
“We made a semiconductor chip that mimics the behavior of ion channels of hippocampal and respiratory neurons. The chip itself is scaled to a few tens of microns in size. Our work is paradigm changing because it provides a robust method to reproduce the electrical properties of real neurons in minute detail.”
Alain Nogaret, professor of physics at Bath University, who led the research
Precision medicine at scale
Francis Collins, director of the U.S. NIH, said in a public forum that the agency is "really bullish" about precision medicine. However, while precision medicine requires mounds of data, which soon may be available, Collins said the NIH All of Us research program has drawn the interest of more than 300,000 willing participants to date, adding that the target enrollment of 1 million should be accomplished before the end of 2022.
“A decade from now, we’re going to have recruited a million people. You’ll have new findings on things like drug-gene interactions. Hopefully, we will have started a conversation on how we think of prescribing. Both by discovery and by virtue of making pharmacogenetics more commonplace, we can make more use of genetics to guide prescribing.”
Joshua Denny, a professor of biomedical informatics and medicine at Vanderbilt University Medical Center and the principal investigator for the All of Us Data and Research Center
"Drug prices are often increased substantially over time in the U.S., and questions are frequently raised regarding whether these price increases are justified. By identifying drugs with substantial price increases despite no new evidence of added benefit, we hope to make an important first step in providing policymakers with information they can use to advance the public debate on drug price increases."
David Rind, chief medical officer, The Institute for Clinical and Economic Review, which posted a draft protocol that will analyze significant prescription drug increases and determine whether or not new clinical evidence exists that could be used to support those increases
"Every year innovative drugmakers provide tens of billions of dollars in rebates to help expand affordable access to prescription medicines. But far too often these rebates are not passed along to patients and are instead used to pad the profits of middlemen."
Jim Greenwood, BIO president and CEO, commenting on BIO's support of a proposed rule that would alter safe harbor policies under the federal anti-kickback statute
"Across the industry, net prices for branded medicines have increased well below the rate of medical inflation in recent years."
Jennifer Taubert, executive vice president and worldwide chairman at Janssen Pharmaceuticals, a Johnson & Johnson company, testifying before the U.S. Senate Finance Committee's second hearing on prescription drug prices
One and done therapies
The past 48 hours "have been pretty emotional and overwhelming," Bluebird Bio Inc. CEO Nick Leschly said, and he wasn't talking about navigating the street crowd at the 37th Annual J.P. Morgan Healthcare conference in January. Between meetings in San Francisco, Leschly had fielded dozens of calls from biopharma CEOs, payer representatives, key opinion leaders, patient advocacy organizations, government officials around the world – even employees and board members – reacting to his presentation where he made the case for capped, at-risk, term-limited, installment-plan payments for potentially curative gene therapy and other one-time treatments. The response across stakeholders was uniformly heartening.
"This is bigger than Bluebird," he said. "We're hoping just to be part of that dialogue."
"Novartis and Bluebird Bio have each introduced outcomes-based pricing over up to a five-year timeframe for their respective products, exemplifying the movement toward alternative payment models. Legislation is currently pending that would enable such payment-over-time/outcomes models to be used in Medicaid," the Alliance for Regenerative Medicine commented on the issue.
Trying to make a living developing antibiotics
"[It sends out the message that] there is no viable route to commercial success for new antibiotics, however valuable they may be to society. Governments must send an immediate signal to companies and investors that the future is not as bleak as the present."
Jeremy Farrar, director of the U.K. research charity Wellcome Trust, commenting on the bankruptcy of Achaogen Inc. nine months after the U.S. launch of its new antibiotic, Zemdri (plazomicin)
“If you look around, the antibiotic space is not flavor of the month, which is interesting given all the press coverage of a looming AMR [antimicrobial resistance] apocalypse. The stock prices of most listed antibiotics companies in the U.S. have been hammered.”
Glyn Edwards, CEO, Summit Therapeutics plc, which intends to cancel trading on the Alternative Investment Market (AIM) in London, but it will still retain its Nasdaq listing
"Traditional antibiotics kill bacteria by interfering with primary metabolism. Because of that, there is high pressure to generate resistance. We are not targeting essential genes and our products don't kill bacteria, so we don't expect to create the same pressure for them to develop resistance. We are pitching ourselves as a company that is developing non-traditional solutions for antimicrobial resistance. Our contribution is pursuing first-in-class molecules that inhibit bacterial virulence."
Graham Dixon, CEO, Neem Biotech Ltd.
"The rapidly rising rates of antibacterial resistance in pathogens associated with uncomplicated urinary tract infections are making successful treatment more challenging for physicians while patients are experiencing more significant clinical consequences. The elderly and those with diabetes mellitus are especially at risk for treatment failure and need better diagnostic and therapeutic options for this disease."
Michael Dunne, chief scientific officer of Iterum Therapeutics plc
"Bacterial infections are a growing threat around the globe and are driving the need for innovative therapeutics with new mechanisms of action. GNA NOW gives us the opportunity to join forces with leading institutions of both the public and the private sector across Europe to develop new gram-negative antibacterial agents as quickly and as efficiently as possible."
Cord Dohrmann, chief scientific officer, Evotec SE, which announced a new initiative for the development of novel antibacterial agents - Gram-Negative Antibacterials, or GNA NOW
“MRSA and antimicrobial resistance must immediately become a top global political priority.”
Jeanine Thomas, founder of MRSA Survivors Network and World MRSA Day
The never-ending Brexit saga
"Now is the time for policymakers in the U.K. and the EU to put politics aside and put measures in place to prevent patients being harmed by the consequences of Brexit."
Nathalie Moll, director general of the European Federation of Pharmaceutical Industries and Associations
"There is no escaping that these cutbacks have hampered, at least in the short term, the ability of this agency to keep abreast of scientific and regulatory development and to support research and development of medicines in Europe."
Guido Rasi, executive director, lists a catalogue of Brexit-related damages in his introduction to EMA's operations in the agency's 2018 annual review
"If Mr. Johnson really believed in the U.K. as a science superpower, he would not be in favor of Brexit. The prime minister's announcement may be a positive step, but the brute fact remains that Brexit is absolutely dreadful news for U.K. science. We know that EU scientists have already left, talented young people have decided not to come here, and U.K. scientists have been excluded from EU projects because of Brexit."
John Krebs, professor at Oxford University, commenting on government plans to preserve the status of U.K. research by setting up a new fast track visa system to attract top scientists post-Brexit
Growth of biotech in Asia
The new Shanghai Stock Exchange's Science and Technology Innovation board, also known as the STAR board, officially launched in late July and marked China's boldest move yet to attract biopharma and technology companies to its often-turbulent financial markets.
"The goal of the Chinese government is to use the STAR exchange as an avenue for new, disruptive companies to raise funds, and ultimately accelerate the pace of tech development in China. Indirectly, the exchange is also a way for the government to divert local funds away from overheated asset segments, like the property market."
Kenny Liew, ICT analyst at Fitch Solutions
"Over the past 10 years in Asia, the number of life sciences and health care companies listed on stock exchanges has steadily increased by about 8 percent. Shanghai just launched a Nasdaq-like exchange. It's expected to allow pre-revenue biotech companies to list. As of last week, about 150 companies have applied to raise funds on the stock market through IPOs."
Katherine Andersen, head of life science and health care relationship banking at Silicon Valley Bank, speaking at the 2019 BIO Asia-Taiwan Conference and Exhibition
Editor’s note: See part 1 for the industry’s take on markets, M&A and gene therapy.