The US Food and Drug Administration approved Libtayo (cemiplimab-rwlc) on 28 September for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) who are not eligible for curative surgery or radiation. The approval is the FDA’s first authorisation of a drug specifically for an advanced form of this disease.
The field of RNA therapeutics received a boost on 10 October when a group of investors provided Series A funding to Gotham Therapeutics Inc, a company founded by Versant Ventures to develop drugs that target RNA-modifying proteins. The new company will be exploiting discoveries in the field of epitranscriptomics, a discipline which describes the biochemical modifications of RNA within a cell. These modifications help determine the degree to which genes are translated into proteins.
GW Pharmaceuticals Plc has raised $300 million in a secondary offering on the US Nasdaq, providing it with capital to commercialise its recently approved cannabinoid medicine Epidiolex for the treatment of seizures associated with the childhood epilepsies, Lennox-Gastaut syndrome and Dravet syndrome.
Epidiolex was approved by the US Food and Drug Administration on 25 June. The medicine is currently under review at the European Medicines Agency with a decision expected in the first quarter of 2019.
Forbion Capital Partners of the Netherlands has closed its previously-announced Forbion IV biopharma fund at €360 million, significantly above the original target of €250 million. Forbion IV, like its predecessor fund Forbion III, will primarily invest in life science opportunities in the EU, including the UK, with the remainder targeting North America.
An enzyme therapy developed by Hansa Medical AB has successfully inactivated donor-specific antibodies in patients with kidney disease enabling them to receive kidney transplants. The result comes from follow-up data from two Phase 2 trials of highly sensitised patients who otherwise would not have been eligible for transplantation.
Novartis has announced plans to reduce its workforce in Switzerland and the UK by a net 2,100 over four years as it shifts away from the production of high volume pharmaceutical products to personalised medicines such as gene therapies. The shift represents a focus on higher-value specialised products.
Among the activities that will be curtailed is the production of active pharmaceutical ingredients at a plant in the UK. Novartis plans to divest or close the manufacturing site in Grimsby which currently employs just under 400 people.
The checkpoint protein on immune cells may have another role to play in treating disease besides fighting cancer. This is the proposition being put forward by Altheia Science Srl, a new Italian biotech which is building a pipeline of agents to modulate programmed cell death-1 (PD-1) and its ligand (PD-L1) for the treatment of Type 1 diabetes and multiple sclerosis.
Nightstar Therapeutics Plc has reported positive Phase 1/2 data from a trial of its gene therapy for X-linked retinitis pigmentosa, a rare inherited retinal disorder primarily affecting males. The 18-patient study showed preliminary evidence of improved visual function.
The European Medicines Agency has delivered a positive opinion recommending use of a new gene therapy, Luxturna (voretigene neparvovec), to treat adults and children suffering from an inherited retinal dystrophy caused by mutations in the RPE65 gene.
The disease causes vision loss that usually leads to blindness. There is currently no treatment for the disorder. Support to patients is limited to measures allowing disease management such as wearing a medical identification bracelet.
Abzena Plc is to apply its antibody technology to chimeric antigen receptor (CAR) T cell development under a new agreement with Tmunity Therapeutics Inc, a company co-founded by Carl June, head of the scientific team that discovered Kymriah.