A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy. Read More
LONDON – The U.K. government has accepted the recommendation of medical experts and will begin the rollout of COVID-19 boosters from next week, using mRNA vaccines only. That follows data from the U.K. Cov-Boost trial, looking at combinations of initial and booster doses. The results showed Pfizer Inc./Biontech SE’s and Moderna Inc.’s products generated the best immune responses, regardless of which vaccine was administered initially. Read More
In a couple of deals worth more than $500 million, Everest Medicines Ltd. is picking up Asian rights to Providence Therapeutics Holdings Inc.’s mRNA candidates, including rights to a mRNA COVID-19 vaccine candidate currently in phase II testing. Read More
South Korea’s Ministry of Health and Welfare (MHW) is currently discussing the implementation of a preferential drug pricing system and plans to initiate an expert survey in 2022. The preferential drug pricing system is part of a three-prong strategy that South Korea plans to adopt to promote the growth of eight leading companies in the pharmaceutical, medical device and cosmetic industries by 2030. Read More
While privately held Calcimedica Inc. posted positive top-line results from a phase II study of its lead candidate in treating patients with severe COVID-19 pneumonia, Redhill Biopharma Inc. took a tumble as preliminary top-line data from a phase II/III study of opaganib in treating hospitalized COVID-19 patients showed the endpoints failed to achieve statistical significance. Read More
Given the ethics and feasibility of placebo-controlled COVID-19 vaccine trials, Access Consortium members are recognizing appropriately designed immunobridging studies as an acceptable approach for authorizing the vaccines, including those being developed to protect against SARS-CoV-2 variants. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alivamab, Celonic, Celyn, Chemdiv, Compass, Convergent, Cresset, Curevac, Defence, Eleva, Gwynant, IO, Jupiter, Merck, Mihkal, Molsoft, Novartis, Point, Rentschler, Teva, Wacker. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: ADC, Alnylam, Beigene, Chiesi, Hummingbird, Hutchmed, Instil, Mirum, Monopar, Redhill, Relief, Scynexis, Tonix, Y-Mabs. Read More
