The Belgian cell therapy company Promethera Biosciences SA has reported a positive safety profile and early efficacy signals from a Phase 2a study of its lead therapy for liver disease, HepaStem. HepaStem is an allogeneic therapy derived from the stem cells of healthy donors.
The stem cells are administered by infusion into the bloodstream of patients where they circulate until reaching the liver, settle and support regeneration.
Gilead Sciences Inc and Novo Nordisk A/S of Denmark have decided to pool their resources to test a new combination therapy for the treatment of nonalcoholic steatohepatitis, a chronic and progressive liver disease characterised by the accumulation of fat and inflammation in the liver.
Alnylam Pharmaceuticals Inc has reported positive Phase 3 data for a trial of its ribonucleic acid interference (RNAi) drug, givosiran, in patients with acute hepatic porphyria (AHP). AHP refers to a family of rare, genetic diseases characterised by potentially life-threatening attacks and for some patients, chronic debilitating symptoms that negative affect their quality of life.
UK-based Destiny Pharma Plc is stepping up the pace of development for its lead antibacterial XF-73 with the start of a 200-patient Phase 2b study in April. The topical treatment is being investigated for the prevention of post-surgical staphylococcal infections including methicillin resistant Staphylococcus aureus (MRSA), a major public health threat. XF-73 is administered as a nasal gel where it has been shown to reduce the nasal carriage of the S. aureus bacteria.
An early stage chimeric antigen receptor (CAR) T cell therapy that targets an antigen expressed on myeloma cells has been singled out for a fast-track review by the European Medicines Agency. The cell-based gene therapy, JNJ-4528, is being co-developed by Nanjing Legend Biotech Co of China and Janssen Research & Development LLC.
Galapagos NV and Gilead Sciences Inc have announced positive data from two ongoing Phase 3 trials of the Janus kinase-1 (JAK1) inhibitor filgotinib in rheumatoid arthritis (RA). The trials, FINCH 1 and FINCH 3, met the primary and key secondary endpoints with favourable safety data at the 24 week cut-off date.
A prospective therapy for birch allergy failed to achieve the primary endpoint in a Phase 3 multi-center trial, according to the developer Allergy Therapeutics Plc. The therapy, B301, is a short-course treatment for birch-pollen induced seasonal allergic rhinitis.
It failed to show statistical significance relative to placebo. The safety and tolerability profile of the therapy were positive.
Record sums were invested in biotech companies in 2018 – especially in those at the early stage of development. According to the US industry association BIO, some $12.4 billion in venture capital was raised in the US for biotech, double the amount generated in 2017, while $600 million was raised in Europe.
Two Phase 3 trials of an experimental drug for Alzheimer’s disease were stopped for futility by Biogen Inc and Eisai Co Ltd on 21 March. However a day later, Eisai announced the start of a new Phase 3 programme for another Alzheimer’s treatment which is being developing with Biogen. In both cases the drugs are monoclonal antibodies designed to treat patients with mild cognitive impairment due to Alzheimer’s disease. They apparently differ in the way they target amyloid-beta which are protein fragments in the brain, thought to contribute to neurodegeneration.
Merck KGaA and its partner Pfizer Inc have stopped a Phase 3 trial of a new drug combination for treatment-naïve patients with locally advanced or metastatic ovarian cancer. The trial was evaluating the immunotherapy avelumab in combination with chemotherapy, followed by maintenance therapy consisting of avelumab in combination with talazoparib, a poly ADP-ribose-polymerase (PARP) inhibitor.