Articles in 2019

Oral delivery of peptide therapeutics could have benefits for treatment adherence, but it faces barriers related to the structural organization and physiological function of the gastrointestinal tract. This article highlights strategies to overcome these barriers and discusses experience with oral peptides that have reached clinical trials, including the recent landmark approval of an oral formulation of semaglutide for the treatment of type 2 diabetes.

Most rare diseases still lack approved treatments. This article analyses the main therapeutic modalities available to researchers interested in translating advances in the scientific understanding of rare diseases into therapies, highlights progress so far and discusses overarching issues in drug development for rare diseases.

Continued VC largesse and a maturing biotech industry in China helped boost 2019’s IPO totals.

Immunotherapies that activate costimulatory receptors on T cells have failed so far in the clinic, but the first phase III trial of an agonist antibody in cancer has just begun and second-generation candidates are advancing.

With Takeda’s US$62 billion acquisition of Shire in 2018, the Japanese firm scored a spot as a top 10 pharmaceutical firm. But well before the deal was even on the radar, President of R&D Andy Plump and CEO Christophe Weber were already working to transform the company. Once a small-molecule specialist, it is now embracing new modalities to be better positioned to follow the biology of disease. Once internally focused, it is now partnering whenever possible both to bring compelling new projects in and to let promising assets out. And Plump, now celebrating 5 years at the firm, is ready to showcase Takeda’s new look. He spoke with Asher Mullard about Takeda’s plans to leapfrog competitors in immuno-oncology, cellular therapy, rare diseases, gene therapy and more.

The complement pathway plays key roles in host defence, but its excessive activation or dysregulation can lead to a variety of disorders. This poster overviews strategies to therapeutically intervene in the pathway and the new generation of complement inhibitors that are now in the clinic.

Artificial intelligence (AI) tools are increasingly being applied in drug discovery. This article presents the views of a group of international experts on the ‘grand challenges’ in small-molecule drug discovery with AI, including obtaining appropriate data sets, generating new hypotheses, optimizing in a multi-objective manner, reducing cycle times and changing the research culture.

Current preclinical models poorly predict the potential of a new drug candidate to cause drug-induced liver injury (DILI) in humans. Here, Park and colleagues discuss current understanding of the mechanisms mediating DILI and, through an academic–industry collaboration, propose a roadmap for the development of predictive preclinical models of human DILI.