News

Philip Morris offer for Vectura unconditional

Country
United Kingdom

A bid by Philip Morris International Inc for Vectura Group Plc became unconditional on 15 September following acceptance by investors representing 74.77% of Vectura’s shares, the two companies announced. In August, the Vectura board of directors accepted the offer and this has now been approved by shareholders.

Oxitope Pharma launched in the Netherlands

Country
Netherlands

A new Dutch biotech company was launched on 16 September to discover and develop antibody-based drugs for diseases caused by oxidative stress. Oxitope Pharma BV was founded and seed funded by Forbion Capital Partners and uses technology in-licensed from the University of California San Diego, US.

Series A financing for non-viral gene therapies

Country
Switzerland

Anjarium Biosciences AG, a Swiss company with technology for delivering gene therapies to patients without the use of a viral vector, has raised CHF 55.5 million (€50.96 million) from a group of investors co-led by Gimv and Abingworth LLP. The money will be used to expand the company’s staff and advance several of its programmes towards the clinic.

Coave Therapeutics out-licenses gene therapy

Country
France

France-based Coave Therapeutics (formerly Horama SAS) has entered into a licensing deal, giving Théa Open Innovation, an ophthalmology company, rights to co-develop and commercialise a gene therapy for retinitis pigmentosa.

Astellas reports death of gene therapy trial participant

Country
Japan

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.

Clinical hold on gene therapy trial

Country
United States

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 acquires rights to gene therapy

Country
United States

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).

AM-Pharma secures deal with Kyowa Kirin

Country
Netherlands

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.

Lilly invests in RNA editing platform

Country
Netherlands

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.

Rilzabrutinib study in pemphigus fails to meet endpoint

Country
France

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.