Molnupiravir, the take-at-home pill from Merck & Co. Inc. hailed as a potential game-changer against COVID-19, is not as effective as previously thought, according to newly released trial data from the company.
Another drug from the emerging menin inhibitor drug class has hit safety issues, after the FDA put an early stage acute myeloid leukemia trial of Kura Oncology Inc.’s KO-539 on partial clinical hold because of a patient death. The news from Kura follows safety issues that emerged from Syndax Pharmaceuticals Inc.’s rival SNDX-5613, where an issue with QT prolongation emerged in at least 5% of patients in otherwise encouraging phase I/II trial results announced in April.
Mereo Biopharma plc is heading into phase III with a bone-building drug to treat the rare condition osteogenesis imperfecta – and CEO Denise Scots-Knight reckons the company has the financial backing to get setrusumab marketed thanks to a partnership with Ultragenyx Pharmaceutical Inc.
Novo Nordisk A/S has taken the plunge and bought its RNA interference (RNAi) technology development partner Dicerna Pharmaceuticals Inc. for around $3.3 billion. Bagsværd, Denmark-based Novo Nordisk has been working with Dicerna, of Lexington, Mass., since 2019 on therapies that selectively silence genes that cause or contribute to disease. RNAi is triggered by double stranded RNA, which causes degradation of a specific RNA target that codes for a rogue disease-causing protein.
NHS England has struck new pricing agreements that expands access in the U.K. to blood thinning direct oral anticoagulants (DOACs) to tackle strokes in patients with atrial fibrillation. Though still available to NHS clinicians, Boehringer Ingelheim GmbH’s blockbuster DOAC Pradaxa (dabigatran) is notably not involved.
The FDA has approved Pharmaessentia Corp.'s Besremi (ropeginterferon alfa-2b) for the rare blood disorder polycythemia vera, the interferon offering an alternative to JAK inhibitor therapy. PV is a rare, chronic and life-threatening blood cancer caused by a mutation in stem cells in the bone marrow, resulting in overproduction of blood cells. Besremi, which is already approved in Taiwan, Europe and South Korea, is a monopegylated, long-acting interferon, which counteracts the effects of the mutated bone marrow cells and is initially taken fortnightly.
It’s been a decade since the first checkpoint inhibitors were approved, but the problem of low response rates remains in many forms of cancer. Highlight Therapeutics SL, of Valencia, Spain, hopes to change all that with an approach that primes the body to become more responsive to immunotherapies.
European regulators have put off a decision on Biogen Inc and Eisai, Co. Ltd.’s controversial Alzheimer’s drug, aducanumab, for likely another month, after the companies announced further supportive data from a follow-up under review by the FDA.
The University of Oxford has begun recruiting for a phase I Ebola vaccine trial, with the first vaccinations based around the same technology as the university’s COVID-19 vaccine.
After signing a potential $1.2 billion cell therapy deal with Vertex Pharmaceuticals Inc. in August, Arbor Biotechnologies Inc. has raised $215 million in series B financing to advance next-generation precision gene editing therapeutics.
