Emicizumab is a bispecific monoclonal antibody, which brings together proteins required for natural coagulation cascade activation and thus restore blood clotting capabilities in patients with haemophilia A. The promising drug is now being included in the early access to medicines scheme in the UK for routine prophylaxis of bleeding episodes in patients with haemophilia A with factor VIII inhibitors. This decision by the MHRA is based largely on Phase III data from the HAVEN programme which showed that a tolerable safety profile and that prophylactic use of emicizumab in inhibitor patients reduced the number of treated bleeds by 87% after 24 weeks compared to standard of care bypassing agents. Please click here to read further in an article from PharmaTimes.
Shire’s experimental drug, Maribavir, to treat cytomegalovirus (CMV) infection has been awarded a Breakthrough Therapy Designation by the US FDA. CMV is a beta herpes virus that can cause complications, which are sometimes fatal, in patients with compromised immunities including those receiving organ or stem cell transplants. The drug inhibits the CMV UL97 protein kinase which potentially effects several critical processes within the CMV replication mechanism. Phase II studies with Maribavir showed that 67% of patients had no detectable signs of the virus in their blood plasma by week six. This designation will allow a potentially faster development and regulatory review meaning it could make it to patients faster. Please click here to read more from a PharmaTimes article.
Pfizer and Arvinas have signed a research collaboration and license agreement for protein degradation drug candidate’s discovery and development. This agreement is focused on the use of Arvinas’ PROTAC (Proteolysis Targeting Chimeras) platform, a technology that can be used to create small molecule drugs that degrade several key disease-causing proteins in multiple therapeutic areas. This method can offer potential advantages over traditional small molecule inhibitors as it uses the cells own system to degrade disease-causing proteins rather than just inhibit them. The deal could be potentially worth up to $830 million in upfront payments as well as milestone payments and royalties from Pfizer. If you would like to read more, please click here for an article from PharmaTimes.