News & Comment

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.

This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.

Vertex’s goal over much of the past decade has been on treating cystic fibrosis, targeting the dysfunctional CFTR chloride channel at the root of the disease. Following the speedy approval in October of its triple combination therapy Trikafta, nearly 90% of people with cystic fibrosis now stand to benefit from the firm’s CFTR potentiators and correctors. With this landmark success in hand — and the lucrative market it has opened up — the company is now in the process of broadening its horizons. In April 2020, current CMO Reshma Kewalramani will step up to the CEO role to oversee the transition. She spoke with Asher Mullard about the lessons the company has learned from cystic fibrosis drug development and its future R&D plans.