Qualifying a human Liver-Chip for predictive toxicology: Performance assessment and economic implications

Human organ-on-a-chip (Organ-Chip) technology has the potential to disrupt preclinical drug dis-covery and improve success in drug development pipelines as it can recapitulate organ-level patho-physiology and clinical responses. The Innovation and Quality (IQ) consortium formed by multiple pharmaceutical and biotechnology companies, however, systematic and quantitative evaluation of the predictive value of Organ-Chips has not yet been reported. Here, 780 Liver-Chips were analyzed to determine their ability to predict drug-induced liver injury (DILI) caused by small molecules identified as benchmarks by the IQ consortium. The Liver-Chip met the qualification guidelines across a blinded set of 27 known hepatotoxic and non-toxic drugs with a sensitivity of 80% and a specificity of 100%. With this performance, a computational economic value analysis suggests that the Liver-Chip could generate $3 billion annually for the pharmaceutical industry due to increased R&D productivity.

Clinical Trials on COVID-19: What is Being Researched in the United States?

The emergence of Coronavirus Disease 2019 (COVID-19) in late 2019 has brought great challenges to public health worldwide and, to date, there is no specific approved therapeutic protocol. Therefore, this chapter will analyze types of intervention for use in patients with COVID-19 developed by American researchers from records made on the Clinical Trials platform. For the search strategy, keywords “COVID-19” in the “Condition or Disease” section and “United States” in the “Country” section were used. No filters were applied. Data were descriptively analyzed. In total, 1,182 studies were obtained, of which 496 met the eligibility criteria. Sample size ranged from 1 to 10,000 participants. Most studies involved the age group of 18–64 years (48.6%). As for design, randomized type (80.5%), parallel (75.6%), open designs (38.7%) and those with therapeutic purpose (88.3%) were more frequent. Most clinical trials used the two-arm trial (67.3%), researched drugs (64.8%), used placebo (55.2%) and were sponsored by pharmaceutical/biotechnology companies (35.4%). Clinical trials developed by American researchers on COVID-19 involve adult and elderly participants, with predominance of randomized, parallel and open design, for therapeutic purposes and mostly evaluated immunosuppressants or combinations of antivirals/immunosuppressants. The drugs and biological products Remdesivir, Baricitinib in combination with Remdesivir, Bamlanivimab and Etesevimab, REGEN-COV and COVID-19 convalescent plasma were also used, authorized for emergency use.

Trends of industry-leading biotechnology stocks during COVID-19

The 2019-nCoV coronavirus has significantly impacted the macroeconomic outlook for countries across the world. The biotechnology sector experienced a relatively positive stock price outlook, which corroborates the trends exhibited by biotechnology stocks in previous pandemics such as SARS (2003) and AH1N1 (2009). During the COVID-19 pandemic, vaccine roll-out rates have been more efficient than in any other pandemic, as companies today are more experienced in combating the time constraint to create vaccines. Gilead Sciences Inc. saw its shares rise 18% after developing the first FDA-approved COVID-19 vaccine, 'remdesivir'. In contrast, AstraZeneca's shares fell 2.28% in 2021 after its vaccine underwent investigations into blood-clotting side effects and subsequent suspension from several countries. Companies developing mRNA vaccines like Moderna, BioNTech, and Pfizer witnessed surges in share prices ranging from 10% to 20%. Many newer biotech companies such as Genexie, Sanovi, OnoSec, and Vaxart have also developed vaccines for COVID-19. Share prices are more volatile for these less established companies. This paper observes how developing new technologies, staging clinical trials, obtaining FDA approvals, gaining publicity, and several other complex factors have profound impacts on the stock prices of these biotechnology companies.

Riding the Biotechnology Wave: A Mixed-Methods Analysis of Malaysia's Emerging Biotechnology Industry

<p>Building a sustainable bioeconomy requires strategic alliances, intellectual property,funding and talent. The research focus of this empirical study was to assess Malaysian biotechnology companies regarding their opinions on priorities and capabilities necessary to establish a thriving bioeconomy. The research questions that form the basis of this paper explore the extent to which initial factor endowments affect the trajectory of biotechnology industry development and how Malaysia should prioritise, mobilise and coordinate resources to build a bioeconomy. A mixed methods approach using qualitative interviews and case studies, as well as a quantitative survey, indicated that respondents advocated a resource-based-view in terms of resource allocation and agglomeration towards building Malaysia's bioecnomy. That is, there was strong support to leverage Malaysia's existing capabilities in agriculture and biofuels to derive value-added products towards gaining leadership positions in these respective biotechnology sectors globally. Access to funding and talent emerged as the highest priority capabilities necessary for commercialising discoveries, conducting research and development and accelerating innovation. Respondents perceived the government as having a 'very important' role in building and accelerating the Malaysian biotechnology industry. The gap between required capabilities and strategic priorities provides a framework within which the government may play a central role in coordinate, accelerating and resourcing Malaysia's nascent bioeconomy.</p>

Riding the Biotechnology Wave: A Mixed-Methods Analysis of Malaysia's Emerging Biotechnology Industry

<p>Building a sustainable bioeconomy requires strategic alliances, intellectual property,funding and talent. The research focus of this empirical study was to assess Malaysian biotechnology companies regarding their opinions on priorities and capabilities necessary to establish a thriving bioeconomy. The research questions that form the basis of this paper explore the extent to which initial factor endowments affect the trajectory of biotechnology industry development and how Malaysia should prioritise, mobilise and coordinate resources to build a bioeconomy. A mixed methods approach using qualitative interviews and case studies, as well as a quantitative survey, indicated that respondents advocated a resource-based-view in terms of resource allocation and agglomeration towards building Malaysia's bioecnomy. That is, there was strong support to leverage Malaysia's existing capabilities in agriculture and biofuels to derive value-added products towards gaining leadership positions in these respective biotechnology sectors globally. Access to funding and talent emerged as the highest priority capabilities necessary for commercialising discoveries, conducting research and development and accelerating innovation. Respondents perceived the government as having a 'very important' role in building and accelerating the Malaysian biotechnology industry. The gap between required capabilities and strategic priorities provides a framework within which the government may play a central role in coordinate, accelerating and resourcing Malaysia's nascent bioeconomy.</p>

The Influence of Earnings Management and Board Characteristics on Company Efficiency

Earnings management is a means by which managers manipulate earnings to conceal the true performance of a company. The characteristics of the board of directors can also influence firm performance. This study applies data envelopment analysis (DEA) and the Tobin regression model to investigate the influence of earnings management and board characteristics on company efficiency. The data sample includes 396 Taiwanese electronics and biotechnology companies from 2009 to 2017. The results indicate that earnings management has an insignificant influence on company efficiency with mixed results on the interactions between earnings management and board characteristics. When companies practiced earnings management, director experiences, a higher proportion of female directors, and a higher number of board meetings increased company efficiency. In contrast, a higher number of independent directors and a higher attendance rate of the directors at the board meeting decreased company efficiency. The results of this study suggest that board diversity, more female directors, and meetings could still improve firm performance despite companies’ engagement in earnings management.

Rare disease patients’ needs: an up-to-date analysis and future directions

The interest to rare diseases has increased in the recent decades. Legislation seeks to facilitate patients’ access to innovative and effective treatment and to define incentives for pharmaceutical and biotechnology companies to develop new medicines for rare diseases.The current review presents the current knowledge and adopted solutions in the field of rare diseases and discusses the future issues and unmet needs that should be resolved for affected patients and their families. Along with the positive trends in the field of rare diseases, there are still issues related to diagnosis and inequal care for some patients groups that should be solved over the next decade. The innovative digital health methods, which have been improved continuously in the recent years, implementation of improved versions of patient-centered policy plans and programs and investment in advanced therapies could move forward the rare diseases to new horizons giving them the opportunity to overcome the main barriers and challenges in the whole journey of the patients – from diagnosis through treatment to follow-up.

The Code Breaker: Jennifer Doudna, gene editing, and the future of the human race

In this book review and accompanying commentary and Addendum, we focus on 5 principal topics/major themes that are of interest for our readership, with a focus on framing the translation of transformative technology into a platform business model in biopharma. We focus on: 1) the behavioral and personal side of the story of the academic scientist, in this case the principal “code breaker” – Jennifer Doudna; 2) the innovation/technology transfer models, including team building appropriate for successfully translating technology from the academic laboratory into the private sector; 3) the IP considerations needed for broad commercialization and dissemination of pivotal, platform inventions in biopharma; and, 4) framing the issues surrounding the ethical discussion related to use in patients associated with a transformative, gene based technology like CRISPR. We also include an Addendum that covers, 5) Some pertinent, concluding comments on the importance of high–performance, diverse teams for founding, building, and growing successful biotechnology companies.

THE PROFITABILITY DETERMINANTS OF THE GLOBAL PHARMACEUTICAL & BIOTECHNOLOGY COMPANIES DURING THE COVID-19 PANDEMIC

In today's chaotic, complex, unpredictable, and extremely dynamic business environment, it is more than ever essential to operate in a way that will ensure sustainable operations and demand for companies’ goods or services. Employing contemporary corporate governance, advanced information technology, and adequate financial management are significant foundations for sustaining high profitability and adequate financial stability as crucial objectives that ensure adding value for companies' owners. Covid-19 is an excellent stress test for every entity, as for micro-companies, so for the largest multinational corporations. Nowadays, in the era of the world Covid-19 pandemic, companies challenge to maintain business operations and going concern assumption. However, in every crisis or adverse situation, some companies take enormous benefits of it. Pharmaceutical and biotechnology companies can be characterized as the major players during this last, still actual crisis. Their role can be observed from two angles, first as a provider of necessary drugs for curing Covid-19 patients and from the other side, as a developer of effective and efficient vaccine which will ‘save the world’ and brought us back to the ‘old normal’. This paper seeks to investigate financial determinants that are the most significant for the profitability of the pharmaceutical and biotechnology companies. The question is how those companies coped with the Covid-19 crisis, and what is the impact on their profitability in 2020 compared to previous operating years? Another question is how is the development of the vaccine financed and are R&D expenses significantly increased in 2020? Therefore, the objective of the paper is to investigate the impact of the Covid-19 crisis on the profitability of global pharmaceutical and biotechnology companies. The research covers 52 entities on the global level for the period from 2010 to 2020. Data will be analysed by applying adequate panel data analysis and moderator regression analysis. Keywords: profitability ratios, pharmaceutical and biotechnology companies, Covid-19

Big pharma’s search for a COVID-19 vaccine: Take It To The Limit!

The 2020 COVID-19 pandemic undermines the world economy, the affordability and accessibility of our health systems. It puts an increasing strain on global production, supply and value chains. The research and development of a set of COVID-19 vaccines is an unprecedented triumph of rapid innovation. The sense of urgency has prompted academic research institutions, big pharmaceuticals and dedicated biotechnology companies to develop vaccines targeting the SARS-CoV-2 coronavirus disease. Simultaneously, it unlocks new value for business, patients and society. Consequently, new business models have come to the forefront.