The amount of data captured by pharma companies today is fast outpacing best use for it. The ever-evolving scope of the field also means that many senior-level professionals do not fully understand the importance of getting data right in their business, or missing a potential opportunity that their rivals seize. Stemming from Front Line Genomics’ […]
The FDA has cleared the most expensive drug yet, Novartis’ gene therapy drug Zolgensma, for introduction to the market. Zolgensma, created to combat spinal muscular atrophy, has been priced at $2.125 million, or $425,000 annually over five years.
In potentially the first large-scale systematic analysis of thousands of cancer gene fusions, UK scientists have announced that one of the fusions could be a novel drug target for a number of cancers. CRISR editing was used to determine the most important gene fusions for cancer cell survival, before anticancer compounds were tested on them to see which might be repurposed to specifically target the fusions.
Cambridge University scientists have created the first living organism with fully synthetic DNA radically altered from its original state. The strain of E coli was given a smaller set of genetic instructions than its counterparts, proving life can continue with such a restricted code.
Takeda Pharmaceutical has announced the sizeable first sale in its proposed £10 billion-worth of assets to offset the debt it took on during its acquisition of Shire. The company announced it was divesting eye drug Xiidra to Novartis for $5.3bn total, and its TachoSil Fibrin Sealant Patch to Ethicon for $400 million in case.
Two companies with access to data from the 100,000 Genomes Project have identified patients with previously undiscovered life-threatening kidney and neurological diseases. The companies, Alexion and BioMarin, were part of Genomic England’s Discovery Forum, which grants certain industry research proposals access to data from the project after vetting.
Two separate studies have uncovered insights into why checkpoint-inhibiting immune-oncology (IO) drugs only work for a minority of patients, even when combined with other treatments. The first study uncovered a resistance mechanism within the gut microbiome, while the other relates to cancer cell-produced vesicles.
An FDA-approved drug to treat severe asthma also drastically improves the health of those individuals with hypereosinophilic syndromes (HES), rare types of chronic immune disorders. A larger placebo-controlled trial of the drug, benralizumab, will now be undertaken to confirm the results.
AstraZeneca has signed a $6.9 billion deal with Daiichi Sankyo for a single antibody-drug conjugate (ADC), trastuzumab deruxtecan.
Pharma giant Biogen and its Japanese partner Eisai have made the decision to halt two phase 3 trials of aducanumab, a drug created to slow Alzheimer’s by targeting brain-destroying beta-amyloid fragments. An independent monitoring committee decided that the drug was unlikely to benefit patients compared with a placebo.
Biogen has announced it will acquire Nightstar Therapeutics for around $800m, providing the pharma giant with a pipeline of gene therapy candidates for ophthalmology.
Roche is set to pay $4.3bn for the acquisition of Spark Therapeutics, a gene therapy company that will put the pharma giant ahead in one of the biggest growing markets around.
A combination of immunotherapy drug avelumab and targeted agent axitinib has been seen to increases progression-free survival in patients with metastatic kidney cancer, scientists from the Dana-Farber Institute have reported following a phase 3 clinical trial.
D4: Pharma is a highly-select gathering of 80-100 very senior and experienced pharma professionals responsible for making important decisions, as well as turning innovation and new ideas into measurable progress.
Esketamine, a Johnson & Johnson antidepressant based on the party drug ketamine, has received backing from an FDA independent advisory group.