Belgium-based argenx SE has won US Food and Drug Administration approval for its first product, Vyvgart (efgartigimod), for the treatment of generalised myasthenia gravis, a neuromuscular disease that causes weakness in the skeletal muscles. Vyvgart is an antibody fragment that binds to the neonatal Fc receptor, preventing the receptor from recycling immunoglobulin G (IgG) back into blood. This leads to a reduction in IgG levels, including reductions in abnormal anti-acetylcholine receptor (AChR) antibodies which are present in myasthenia gravis.
A new treatment for sickle cell disease, Oxbryta (voxelotor), received a positive opinion from the European Medicines Agency on 17 December for patients 12 years of age and older. This is the same drug and patient group that received an accelerated approval from the US Food and Drug Administration in 2019. Concurrent with the EMA opinion, the FDA expanded the label of Oxbryta to include children from the age of four up to 11 years.
The European Medicines Agency has issued a negative opinion for the Alzheimer’s disease treatment Aduhelm (aducanumab) saying that studies supporting the drug’s application were conflicting and did not show overall effectiveness in treating adults with early stage disease. Under the EU’s regulatory procedures, the sponsor, Biogen, Inc, has 15 days to appeal against the opinion. Aduhelm is an immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against amyloid-beta levels in the brain.
Venture capitalists, led by Ysios Capital and Forbion Growth, have assembled $80 million for NorthSea Therapeutics BV, enabling the Netherlands-based company to progress its lead product for non-alcoholic steatohepatitus (NASH) into Phase 3. The Series C financing was also supported by Novo Seeds and Sofinnova, among others.
Genenta Science SpA, an Italian gene therapy company, began trading on the US Nasdaq market on 15 December following an initial public offering that yielded gross proceeds to the company of about $36 million. On the second day of trading, the company had a market capitalisation of $196 million. Genenta is one of many European company to list on Nasdaq in order to broaden its investor base and establish a commercial presencez in the US.
Vaccitech Plc, co-inventor of the Oxford-AstraZeneca Covid-19 vaccine, is to acquire a US company with a complementary technology platform, positioning it for a further geographical expansion. The target company, Avidea Technologies Inc, has a similar research history to Vaccitech, but a different discovery platform. Vaccitech is paying $40 million upfront for Avidea of which $12.5 million is cash and $27.5 million is in shares. There is also a possibility of milestone payments. Vaccitech is listed on the US Nasdaq market.
The US Food and Drug Administration has approved the first drug for the prevention of acute graft versus host disease (GvHD), a condition that occurs when donor bone marrow or stem cells attack healthy tissues in a patient undergoing a treatment for certain cancers, or an organ transplant. The drug, Orencia (abatacept), is already on the market having been approved for rheumatoid arthritis in 2005. The manufacturer is Bristol Myers Squibb Co.
Bristol Myers Squibb Co has launched its second preclinical collaboration with Immatics NV in order to explore the potential of an antibody-like bispecific molecule as a treatment for solid tumours. The newest agreement, announced on 14 December, gives BMS exclusive rights to an engineered bispecific molecule consisting of a receptor that binds to a cancer target and a region that recruits T cells to the tumour site to become activated. The upfront payment is $150 million.
Depixus SAS has raised €30.6 million in Series A financing to support development of technology that can extract information from DNA, RNA and proteins, thereby generating new insights into the function of the human genome. The round was co-led by Lansdowne Partners and the PSIM Fund which is operated by Bpifrance on behalf of the French state. The new financing brings the company’s total equity and grant funding to more than €41 million.
Switzerland-based Vifor Pharma Group is to be acquired by CSL Ltd of Australia for CHF 10.9 billion ($11.7 billion) creating one of the largest companies in nephrology and cardio-renal therapies. The merger comes less than a month after Vifor itself purchased two smaller companies.