Astellas Pharma Inc has reported the death of a participant in a clinical trial of a gene therapy for the treatment X-linked myotubular myopathy, a rare neuromuscular disease caused by mutations in the MTM1 gene. The participant, who developed an adverse event during the trial, passed away on 9 September. “The cause of death is still pending,” the company announced on 14 September.
The US Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial of a gene therapy for adults with phenylketonuria, a genetic disorder caused by the deficiency of an enzyme needed to break down an amino acid in certain foods. The clinical hold was based on findings from a preclinical study showing that six out of seven mice receiving the highest dose of the therapy developed liver tumours. One of the tumours was cancerous. BioMarin Pharmaceutical Inc, the developer, announced the clinical hold on 6 September.
AbbVie Inc has acquired rights to a gene therapy in development by Regenxbio Inc for wet age-related macular degeneration (AMD) which the companies say has potential as a one-time treatment for the disease. The therapy’s viral vector encodes an antibody fragment which is expressed inside the body to inhibit vascular endothelial growth factor (VEGF).
Netherlands-based AM-Pharma BV has reached an agreement with Kyowa Kirin Co Ltd giving that company exclusive development and commercialisation rights in Japan to its lead product, ilofotase alfa, for sepsis-associated acute kidney injury. The deal, announced on 8 September, is valued at up to €245 million including potential milestone payments.
Eli Lilly and Company is to make an equity investment in a small Dutch company that has a proprietary RNA editing technology currently directed at eye diseases but with potential for treating other disorders. ProQR Therapeutics NV of the Netherlands is to receive an equity investment of $30 million as well as a $20 million upfront payment, to support the research and development of potential new medicines for genetic disorders in the liver and nervous system.
A Phase 3 trial evaluating the Bruton’s tyrosine kinase (BTK) inhibitor rilzabrutinib in pemphigus, a rare autoimmune skin condition, did not meet its primary or key secondary endpoints, the developer Sanofi SA announced on 9 September. No new safety signals were identified.
Sanofi SA is to acquire Kadmon Holdings Inc of the US for $1.9 billion, adding a recently Food and Drug Administration-approved treatment for chronic graft-versus-host disease (cGVHD) to its transplant portfolio. The acquisition, announced on 8 September, has been unanimously approved by the boards of both companies.
Sanofi’s transplant portfolio currently consists of Thymoglobulin (anti-thymocyte globulin), an antibody preparation that acts as a broad immunosuppressive and immunomodulating agent, and Mozobil (plerixafor), an haematopoietic stem cell mobiliser.
Adaptimmune Therapeutics Plc has expanded its presence in the field of allogeneic cell therapy under a collaboration with Genentech aimed at developing new medicines for cancer. Originally founded to research T cell receptor (TCR) technology for autologous applications, Adaptimmune has recently been branching out into allogeneic, or ‘off-the-shelf’ therapies.
Two Phase 3 trials of bumetanide, which was being investigated in children and adolescents with autism spectrum disorder, have been discontinued due to a lack of efficacy, the sponsors Les Laboratoires Servier SAS and Neurochlore SAS announced on 7 September.
Owlstone Medical Ltd of the UK has raised $58 million in a Series D financing round to advance its diagnostics services for cancer and inflammatory and infectious diseases. The core technology is based on a sensor that can be programmed into software to detect biomarkers of disease in human breath.