A new, worldwide coalition of plasma companies seeking to develop and deliver a hyperimmune immunoglobulin therapy for fighting COVID-19 takes the view that many hands make light work.
LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications.
LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications. As an indicator of the potential therapeutic power of exosome delivery, the lead program in the collaboration rests on the ability of exosomes to cross the blood-brain barrier and deliver a correct copy of the NPC1 gene, to treat the inherited neurodegenerative disorder, Niemann-Pick type C.
The Rare Disease Day, which takes place at the end of February each year, is designed to focus global attention on the need for therapies to treat patients suffering from devastating rare diseases. The most recent event represented the 13th year it has been held. Over that period, research and development in the area has come a long way, and there are now 420 companies around the world that are active in developing regenerative medicines and advanced therapies for the treatment of rare diseases, according to a new report released by the Alliance for Regenerative Medicine (ARM).
For biopharma, 2019 can be described as a terrific year – with a few asterisks. The financial markets were flourishing, with venture capital dollars, in particular, flowing to the sector, while dealmaking reached historic proportions. Meanwhile, scientific breakthroughs led the way as cell and gene therapies gained ground, the first signs of success emerged with new technologies like CRISPR and the long-awaited promise of genomics found its way to the front lines of health care.
LONDON – Dutch startup Enzyre BV is teaming up with Takeda Pharmaceutical Co. Ltd. to complete development of a home diagnostic device that aims to make it as easy for hemophiliacs to self-test their coagulation status as it is for diabetics to monitor their blood sugar levels.
SINGAPORE – Takeda Pharmaceutical Co. Ltd. presented new findings from its phase III ALTA-1L trial evaluating its Alunbrig (brigatinib) vs. crizotinib in adults with advanced anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer (NSCLC) who had not received a prior ALK inhibitor, showing Alunbrig reduced the risk of disease progression or death by 76% after more than two years of follow-up.
SINGAPORE – Takeda Pharmaceutical Co. Ltd. presented new findings from its phase III ALTA-1L trial evaluating its Alunbrig (brigatinib) vs. crizotinib in adults with advanced anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer (NSCLC) who had not received a prior ALK inhibitor, showing Alunbrig reduced the risk of disease progression or death by 76% after more than two years of follow-up.
A late-stage test of Takeda Pharmaceutical Co. Ltd.'s oral suspension of the steroid budesonide for the treatment of eosinophilic esophagitis (EoE) achieved both its co-primary endpoints, improving histologic and dysphagia symptom responses vs. placebo during the first pivotal phase III study of an EoE treatment ever to be reported in the U.S. The study, part of the largest EoE clinical program globally to date, also met secondary endpoints, including improvements from baseline to final treatment as measured by an established patient-reported outcome measurement.
CEO Mark McKenna told BioWorld MedTech that San Diego-based Prometheus Biosciences Inc. has "cracked the code" in inflammatory bowel disease (IBD) – progress underscored by the firm's deal with Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, which brings an undisclosed up-front payment and as much as $420 million more if development, regulatory and commercial milestones are reached in three programs.
