Denmark-based Bavarian Nordic A/S has won approval for Jynneos, a vaccine to prevent smallpox and monkeypox diseases for people at high risk of infection or in cases of a public health emergency. The development of Jynneos is the result of a 15-year partnership between Bavarian Nordic and the US government. The vaccine will become part of the US strategic national stockpile, which is the country’s largest supply of potentially life-saving medicines.
Regulatory agencies in the US, Canada and Australia have jointly approved a new treatment for endometrial cancer, the most common cancer of the female genital tract. The approval is the first of a new collaboration under which the Food and Drug Administration, Health Canada and the Australian Therapeutic Goods Administration review drug applications concurrently and issue a simultaneous decision afterwards.
The European Medicines Agency and the US Food Administration issued separate warnings on 13 September about ranitidine medicines, one of which is Zantac, which are widely used to treat heartburn and stomach ulcers. This follows tests that showed some products contained an impurity called N-nitrosodimethylamine (NDMA), classified as a probable carcinogen.
A new US company, Trucode Gene Repair Inc, has been launched with $34 million in financing from Kleiner Perkins and GV, the venture capital arm of Alphabet Inc. The company is advancing an in vivo gene editing technology designed to correct mutations that cause genetic disorders such as sickle cell disease and cystic fibrosis.
Evotec SE is to conduct small molecule drug development across multiple therapeutic areas for Takeda Pharmaceutical Company Ltd under a new multi-year collaboration designed to deliver clinical drug candidates. The upfront fee hasn’t been disclosed, but preclinical, clinical and commercial milestones could amount to more than $170 million per programme. The agreement covers at least five drug discovery programmes.
The French cell therapy start-up, TreeFrog Therapeutics SAS, has raised a further €3 million in funding for its stem cell production technology, only months after a €7.1 million Series A financing round. Based in Bordeaux, TreeFrog has a platform for mass producing stem cells, reportedly at a significantly lower cost than existing methods.
The platform uses a three-dimensional cell culture system to produce stem cells in bioreactors with short lead times. The new funding includes €450,000 from the French government and €2.4 million from the EU’s Horizon 2020 programme.
Emily Whitehead celebrated her 14th birthday in May, which was also the seventh year she has been free of cancer. The complete remission of her cancer - acute lymphoblastic leukaemia – is the first of many successes of the immunotherapy, chimeric antigen receptor (CAR) T cells, which are experiencing an expositive growth around the world.
The European Medicines Agency has issued a positive opinion for the personalised therapy Xospata (gilteritinib) to treat acute myeloid leukaemia (AML) following approvals for the same drug in both the US and Japan. The developer is Tokyo-based Astellas Pharma Inc.
Xospata is being recommended as a monotherapy for the treatment of adult patients with relapsed or refractory AML with a FLT3 gene mutation. The positive opinion will need to be adopted by the European Commission before Xospata can be marketed.
H. Lundbeck A/S is to acquire the US biopharmaceutical company Alder BioPharmaceuticals Inc in order to add migraine to its disease coverage and build expertise in monoclonal antibody technology. The transaction is valued at up to $1.95 billion net of cash.
Storm Therapeutics Ltd has disclosed data from an animal study of a new small molecule inhibitor of an RNA modifying enzyme which showed activity against cancer. The study results were announced on 18 September at a meeting in Cambridge, UK attended by scientists from Europe and the US who are at the forefront of the new science of RNA epigenetics.
RNA epigenetics describes the chemical markers on messenger RNA that carry genetic information from DNA to a cell’s protein-manufacturing factories. When this process is modified by enzymes it can lead to disease.