The checkpoint antibody Tecentriq (atezolizumab) received its fifth approval from the US Food and Drug Administration on 8 March – this time for triple-negative breast cancer. The approval is for the use of Tecentriq in combination with chemotherapy for patients with breast cancer whose tumours express the PD-L1 protein. Patients will be identified using a new FDA-approved assay.
A young Danish company, SNIPR BIOME, that is using CRISPR/Cas technology to fight bacteria has raised $50 million in a Series A financing round co-led by Life Sciences Partners (LSP) and existing investor Lundbeckfonden Emerge. The North-East Family Office of Copenhagen, Denmark and Wellington Partners of Munich, Germany also participated in the round.
The US Centers for Medicare and Medicaid Services (CMS) has proposed to reimburse cancer treatments with chimeric antigen receptor (CAR) T cell therapies under a policy called ‘Coverage with Evidence Development.” Currently there is no national Medicare policy for covering CAR T cell therapy so local Medicare contractors will have discretion over whether to pay for it. The proposed coverage would require Medicare to cover the therapy when it is offered in a CMS-approved registry or clinical study in which patients are monitored for at least two years after treatment.
Genmab A/S ended 2018 with a cash balance of DKK 6.1 billion ($920 million), up by 13% from a year earlier as royalty revenue from its lead cancer product Darzalex (daratumumab), developed with Janssen Biotech Inc, continued to roll in. Darzalex is a monoclonal antibody that binds to the protein CD38 on multiple myeloma cells for the treatment of multiple myeloma. It is now approved for six indications in the US including, most recently, as a split dose regimen.
With sales of its lead product booming – up by 51% in 2018 – the Pharming Group NV produced its first annual net profit since its founding in 1988.
Revenue from product sales rose to €134.3 million in 2018 from a restated €88.7 million in 2017 while net profit came to €24.9 million compared with a loss of €76.2 million in 2017.
Making this happen was a boom in sales of Ruconest, the company’s product for the treatment of hereditary angioedema (HAE), a condition that is estimated to affect up to 10,000 people in the US alone.
MolMed SpA and Genenta Science SRL, both of Italy, have renewed and extended an oncology collaboration with a view to starting trials of a candidate drug that could treat both haematologic malignancies and solid tumours. Genenta has developed an ex-vivo immune-gene transfer technology to enable the targeting of immunomodulatory molecules to tumour-infiltrating monocytes and macrophages. The goal is to deliver an immunomodulatory molecule into the tumour microenvironment triggering an immune response against multiple tumour antigens.
Scott Gottlieb, commissioner of the US Food and Drug Administration, has resigned his position reportedly in order to spend more time with his family in Connecticut. The FDA job involved a weekly commute to Washington DC, a distance of about 277 miles.
Announced on 5 March, the resignation is expected to take effect in about a month. It was confirmed in a tweet by President Trump who praised Dr Gottlieb for his work leading the drug regulatory authority, a job that he has held since May 2017.
A male patient who was diagnosed with HIV infection in 2003 has experienced a remission from his disease after a haematopoietic stem cell transplant. The stem cell transplant was undertaken in order to treat a cancer. At the time the patient was on also on antiretroviral therapy to treat his HIV. The patient remained on antiretroviral drug therapy for 16 months after the transplant at which point the physicians stopped the drug therapy. Remarkably, the patient remained free of HIV.
Biogen Inc is to acquire Nightstar Therapeutics Plc, a gene therapy company spun out from the University of Oxford and financed by Syncona, which has a product in Phase 3 for the treatment of choroideremia, a rare retinal disorder. The US biotech company will pay $25.50 in cash for each share of Nightstar, valuing the company at approximately $800 million.
Sangamo Therapeutics Inc is readying a second generation of its zinc finger nuclease technology to use in an in vivo genome editing trial later this year. This is expected to provide further support for the company’s strategy of using engineered nucleases to correct DNA in patients with Hunter syndrome and other genetic diseases. Hunter syndrome is a rare genetic disorder that can lead to tissue and organ damage.