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.
The German biotech Probiodrug AG and a US-based cooperative have received a $15 million grant from the US National Institutes of Health (NIH) to test Probiodrug’s small molecule drug in patients with mild cognitive impairment or mild dementia due to Alzheimer’s disease.
The grant will help finance a Phase 2b study of the drug PQ912 which inhibits glutaminyl cyclase, an enzyme that triggers the production of a peptide which is a constituent of Alzheimer’s disease plaques. The grant is expected to be dispersed over four years.
MolMed SpA and Genenta Science SRL, both of Italy, have renewed and extended an oncology collaboration with a view to starting trials of a candidate drug that could treat both haematologic malignancies and solid tumours. Genenta has developed an ex-vivo immune-gene transfer technology to enable the targeting of immunomodulatory molecules to tumour-infiltrating monocytes and macrophages. The goal is to deliver an immunomodulatory molecule into the tumour microenvironment triggering an immune response against multiple tumour antigens.