Regenerative Medicine

The Dutch cell therapy company Kiadis Pharma NV has taken steps to strengthen its presence in the haematopoietic stem cell transplant sector with the agreed takeover of CytoSen Therapeutics Inc. The all-share transaction includes an upfront payment in stock as well as milestone payments contingent on the achievement of six clinical development and regulatory goals. CytoSen’s lead asset is a natural killer (NK) cell product designed to enable patients to receive a stem cell transplant from haploidentical donors. It is due to enter clinical development in 2020.

The ReNeuron Group Plc has entered into an exclusive licensing agreement with a subsidiary of the Shanghai Fosun Pharmaceutical (Group) Co Ltd of China for the development, manufacture and commercialisation of its two allogeneic cell lines which underpin the UK company’s candidate cell therapies for neurological disorders and diseases of the retina.

The UK gene therapy company Autolus Therapeutics Plc has raised $100.8 million in a US public share offering to support its portfolio of programmed T cell therapies for the treatment of cancer. This follows the issue of 4.2 million American Depositary Shares (ADSs) representing 4.2 million ordinary shares which were priced at $24 per ADS. In addition, Autolus has granted the underwriters a 30-day option to purchase up to an additional 630,000 ADSs at the public offering price.

An early stage chimeric antigen receptor (CAR) T cell therapy that targets an antigen expressed on myeloma cells has been singled out for a fast-track review by the European Medicines Agency. The cell-based gene therapy, JNJ-4528, is being co-developed by Nanjing Legend Biotech Co of China and Janssen Research & Development LLC.

A new gene therapy has been given a favourable scientific review in Europe paving the way for its use in patients with beta-thalassaemia as early as the second quarter. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March.

Pfizer Inc has entered into an option agreement to acquire Vivet Therapeutics SAS, a privately-held French company with a gene therapy for Wilson disease, a rare genetic disorder that prevents the body from regulating copper. The disease can lead to liver and brain damage.

Pfizer will initially take a 15% equity interest in Vivet with an exclusive option to acquire the remaining shares in the future. The exercise of the option is linked to the performance of the therapy in a Phase 1/2 trial.

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.

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.