Companies that develop drugs for rare diseases typically expect they will be treating small patient populations. Strategically therefore they might put a premium price on a rare disease medicine in order recover their investment quickly. Or they might sustain a loss on the first drug, hoping to build a lucrative franchise in the future. In both cases, the goal is to deliver a life-saving medicine to patients without alternative treatments.
Negotiations between Johnson & Johnson Inc and Actelion Ltd about a potential transaction have ended without an agreement, the two companies announced. Separately, the Wall Street Journal reported that Sanofi SA is reportedly in talks with Actelion.
UK-based Oxford Nanopore Technologies Ltd has raised £100 million in a private share placement to support the commercial development of its new hand-held device for sequencing whole human genomes at a fraction of the cost of earlier technologies.
Five pharmaceutical companies have been cited by a unit of the ABPI, the UK pharmaceutical industry association, for violations of its code of practice. The companies are Grünenthal, Boehringer Ingelheim, Eli Lilly, AstraZeneca and Janssen.
AstraZeneca Plc said that its recently approved lung cancer drug Tagrisso (osimertinib) has achieved another milestone, showing superiority to chemotherapy in a Phase 3 trial of patients with mutated metastatic non-small cell lung cancer.
Two Phase 3 trials which were seeking to show the superiority of a new combination treatment for patients with age-related macular degeneration (AMD) compared with the use of the licenced drug Lucentis alone, have failed to show an added benefit.
While most efforts at treating Parkinson’s disease have involved brain research, scientists from the California Institute of Technology (Caltech) have now shown in experiments with mice that bacteria in the gut may be releasing chemicals that over-activate parts of the brain leading to the disease.
ReNeuron Plc has reported positive data from a Phase 2 trial of its allogeneic cell therapy for patients disabled by a stroke and plans to apply to regulatory authorities in the US and Europe to begin a pivotal study that could lead to possible registration.
The cell-therapy company TiGenix NV has announced plans to make an initial public offering of its shares on the Nasdaq exchange in the US in order to raise money for the clinical development of its portfolio. The company is already listed on Euronext Brussels.
Spark Therapeutics Inc has agreed to make a $10 million cash payment plus an equity investment in Selecta Biosciences Inc for access to a nanoparticle technology that could be used to modulate the immune system and allow repeat dosing of a gene therapy.