Bluebird bio Inc, which is developing gene therapies for cancer and rare diseases, is partnering with Regeneron Pharmaceuticals Inc to discover, develop and commercialise new therapies for cancer. The deal includes a $100 million investment by Regeneron in bluebird stock.
Oxford BioMedica Plc has teamed up with two UK partners to produce a gene therapy for cystic fibrosis that could be administered to patients through repeat dosing. Boehringer Ingelheim GmbH has an option to commercialise the product.
A candidate treatment for Huntington’s disease has been selected by the European Medicines Agency for PRIME, a programme of regulatory support for developers of medicines that meet a major public health need. The drug, RG6042, is an antisense oligonucleotide.
It is being developed jointly by Roche and Ionis Pharmaceuticals Inc to slow or stop the progress of Huntington’s disease, a rare genetic disorder that causes nerve cells in the brain to break down. Over time, people with the disease can develop personality changes and have difficulty walking and swallowing.
Darzalex (daratumumab) is being proposed as a first-line treatment for newly diagnosed multiple myeloma in combination with three other cancer drugs for patients who are ineligible for an autologous stem cell transplant.
MorphoSys AG has raised its financial guidance for 2018 following a new cash-generating agreement with Novartis which gives the Swiss company exclusive rights to a monoclonal antibody for atopic dermatitis that MorphoSys is developing jointly with Galapagos NV. The antibody, MOR106, targets interleukin-17C, a cytokine that plays an important role in inflammatory skin disorders.
With Brexit looming, the European Medicines Agency now believes that it could lose up to 30% of its staff as the agency prepares to move from its current location in London to new premises in Amsterdam. The move is scheduled to take place in March 2019.
As an agency of the European Union, the EMA must be located in an EU member state. London will no longer be eligible after Britain leaves the EU which is set for 23.00 BST on 29 March 2019.
UK-based ReViral Ltd has raised $55 million in a Series B financing round to advance a small molecule drug against respiratory syncytial virus (RSV) infection into a Phase 2a trial in children. The drug, RV521, has been successfully tested in a Phase2a challenge trial in healthy adults.
RSV is a pathogen that infects the human respiratory tract, potentially leading to bronchiolitis and pneumonia. While healthy adults usually recover from infection, the virus poses a risk to premature infants, the elderly and individuals with certain pulmonary conditions.
Sanofi SA continued its transition to a company focused on specialty care in the first half year, but sales from new products were not sufficient to offset declines from the company’s traditional franchises. Net sales for the second quarter were €8.2 billion, down by 5.7% from a year earlier. For the first half they were €16.1 billion, down by 7.2%. Operating profit for the quarter was €961 million, down by 30%, and for the first-half it was €2.2 billion, down by 30%. Both sets of figures conform with International Financial Reporting Standards (IFRS).
The small molecule drug, laquinimod, has failed to meet the primary endpoint in a Phase 2 study of patients with Huntington’s disease, a rare neurodegenerative disease. In May 2017 the same drug failed a Phase 3 trial in multiple sclerosis.
The drug is being developed by Teva Pharmaceutical Industries Ltd under licence from Active Biotech AB of Sweden. It is an immunomodulatory therapy that aims to prevent neurodegeneration and inflammation in the central nervous system.
An RNA therapeutic developed by Alnylam Pharmaceuticals Inc is the newest drug to be recommended for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a life-threatening disease caused by mutations in the transthyretin (TTR) gene.