The US Food and Drug Administration has awarded a rare paediatric disease designation to a subsidiary of Nippon Shinyaku Co Ltd for an experimental treatment for Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive weakness and the loss of skeletal, cardiac and respiratory muscles. It occurs primarily in males.
Leqembi (lecanemab), a monoclonal antibody that binds to beta-amyloid protofibrils, was approved by the US Food and Drug Administration for Alzheimer’s disease on 6 July, the first drug to show clinical benefit in patients with a mild stage of the disease. The full approval comes six months after Leqembi was given an accelerated authorisation on 6 January which was based on a surrogate endpoint – reducing amyloid plaques in the brain.
AAVantgarde Bio Srl of Italy has raised €61 million in a Series A financing to develop genetic medicines for inherited retinal diseases. The financing was co-led by Atlas Venture of the US and Netherlands-based Forbion.
MoonLake Immunotherapeutics AG of Switzerland has completed an upsized public share offering which is expected to yield an estimated $400 million, before commissions and underwriting discounts. The shares were priced on 27 June ahead of the close of the offering which is expected to be on or about 30 June.
A Netherlands-based company with new technology for antibody-drug conjugates (ADC) announced the completion of a $65 million Series A financing on 22 June. Tagworks Pharmaceuticals BV was founded in 2011 and currently has one preclinical ADC in development and four in discovery. The financing will enable it to advance the lead programme, TGW101, and test a new concept in oncology drug development.
Eli Lilly and Co is to pay $2.4 billion to acquire DICE Therapeutics Inc of South San Francisco, US, thereby bolstering its pipeline of drugs for immunological disorders. The transaction, which was announced on 20 June, is expected to conclude in the 2023 third quarter. DICE is building a pipeline of small molecule drugs directed against diseases many of which are currently treated by biologics. The company says that its technology has the potential to modulate protein-to-protein interactions as effectively as systemic biologics.
The US Food and Drug Administration approved the first gene therapy for the treatment of paediatric Duchenne muscular dystrophy on 22 June, saying that the drug, Elevidys, addresses an urgent unmet medical need. About one in every 3,300 boys in the US are affected by DMD which is caused by a defective gene that results in the absence of dystrophin, a protein that helps keep the body’s muscle cells intact. As the disease progresses, life-threatening heart and respiratory problems may occur.
Two marketed drugs for adults with Type 2 diabetes have now been approved by the US Food and Drug Administration for children 10 years and older in order to help them improve control over abnormally high levels of glucose in their blood. The drugs, Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride), are both approved oral therapies intended to supplement diet and exercise. Both medicines contain empagliflozin, a sodium-glucose co-transporter which works by increasing the excretion of glucose in the urine.
Mendus AB of Sweden announced on 19 June that it has received a certificate from the European Medicines Agency confirming that the manufacturing preparations and preclinical data for its lead dendritic cell vaccine candidate meet the current regulatory standard. The EMA’s advanced therapy medicinal product certificates may be issued to cell and gene therapy developers at the start of product development to verify quality controls. They are not required for a future regulatory submission.
Syncona Ltd, an evergreen investment group, has confirmed its target for growth over the next 10 years while reporting a decline in the value of its net assets for the 12 months ended 31 March. The UK-based group ended the year with net assets of £1,254.7 million, compared with £1,309.8 million the previous year. Gains were reported from currency movements and from Beacon Therapeutics, a recently launched gene therapy company targeting retinal diseases. However these were offset by a decline in the value of the company’s listed life science holdings.