A preclinical study of an experimental cancer therapy has shown single-agent activity in models of glioblastoma, inducing long-term tumour eradications in a proportion of the treated animals. The results were published on 8 October, 2020 in Science Translational Medicine. The developer is Philogen SpA, a clinical-stage biotechnology company with headquarters in Italy.
A Phase 1/2a study of a candidate factor replacement therapy BIVV001 in adults with severe haemophilia A has shown significant levels of factor activity. The results were published on 10 September in The New England Journal of Medicine. The developer is Swedish Orphan Biovitrum AB which is collaborating with Sanofi SA on the programme.
Haemophilia A is an inherited bleeding disorder in which the blood does not clot properly. It is caused by a lack, or a decrease, of clotting Factor 8.
Switzerland-based Santhera Pharmaceuticals AG has discontinued development of Puldysa (idebenone), its candidate therapy for Duchenne muscular dystrophy after an independent review of an ongoing Phase 3 trial concluded that the trial was unlikely to meet its primary endpoint. The Data and Safety Monitoring Board based its conclusion on an interim analysis of the study, SIDEROS, which was being conducted in Duchenne muscular dystrophy patients using concomitant glucocorticoids.
This year’s Nobel Prize in Medicine or Physiology has been awarded to a team of scientists who discovered the hepatitis C virus. Harvey J Alter, Michael Houghton and Charles M Rice have made a “decisive contribution to the fight against blood-borne hepatitis,” according to the prize committee.
The virus is a major global health problem that causes cirrhosis and liver cancer. An estimated 71 million people have chronic hepatitis C virus infection, with almost 400,000 people dying from the disease in 2016, according to the World Health Organization.
The European Medicines Agency has started a rolling review of a prospective vaccine for Covid-19 being developed by Oxford University and AstraZeneca Plc on the basis of preliminary data showing that it triggered the production of antibodies and T cells that target the virus. The agency announced the review on 1 October.
A case study of a 45-year old woman with severe Covid-19 has shown that administration of APN01, an experimental biologic, enabled the generation of neutralising antibodies against SARS-CoV-2 and a rapid drop in virus load. The results of the study were reported online in The Lancet Respiratory Medicine on 24 September 2020.
APN01 is being developed by the Austrian company Apeiron Biologics AG and has been shown to be safe in Phase 1 studies of healthy volunteers. It is currently being investigated in Phase 2 trials in Austria, Germany, Denmark, the UK and Russia.
The gene and cell therapy industry has significant potential. But this will only be realised if manufacturers share information about their technologies, enabling the advanced therapies to be produced at scale and marketed at affordable prices.
Semorinemab, an antibody directed against the tau protein, has failed to have an impact on patients with early Alzheimer’s disease despite earlier promise, the developer AC Immune SA announced on 23 September. Semorinemab is being developed with Genentech, a member of the Roche Group.
AstraZeneca Plc and Oxford University have resumed clinical trials in the UK for their candidate Covid-19 vaccine following a pause earlier this month triggered by an adverse event. The event led to a voluntary stoppage of vaccination across all global trials to allow for a review of safety data by independent committees and international regulators.
On 12 September, AstraZeneca and Oxford announced that the UK regulator had ruled that it was safe to continue trials in the UK. Further details about the case were not disclosed.
Pfizer Inc and BioNTech SE are seeking regulatory approval to expand the enrolment of their Phase 3 Covid-19 vaccine trial to include 44,000 participants, up from 30,000 at present. An application to amend the trial protocol has been submitted to the US Food and Drug Administration, the companies announced on 12 September.
The prospective vaccine, BNT162b2, is a single nucleoside-modified messenger RNA that encodes an optimised SARS-CoV-2 full length spike glycoprotein which is the target of virus neutralising antibodies.