The Roche Group has received a ‘breakthrough therapy designation’ from the US Food and Drug Administration for a new breast cancer treatment intended for patients with a PIK3CA mutation. The PIK3CA gene makes an enzyme which is involved in many important functions in a cell. Mutations can cause the enzyme to become overactive and cancer cells to grow.
The World Health Organization has designated the European Medicines Regulatory Network, a group of institutions that include the European Medicines Agency, as a WHO Listed Authority. The list, which was launched in March 2022, essentially gives the WHO access to the expertise of qualified regulators across the globe to help it improve the availability of safe and effective vaccines and medicines to people across geographies. This includes conducting evidence-based medicine assessments in situations where this expertise might not otherwise be available.
A new bispecific antibody drug has been approved for extensive-stage small cell lung cancer, marking an advance in the treatment of a disease with high relapse rates and very few therapy options. The drug, Imdelltra (tarlatamab), was given an accelerated approval by the US Food and Drug Administration on 16 May following a Phase 2 trial in patients with small cell lung cancer who had failed two or more prior lines of treatment. The developer is Amgen Inc.
Small and medium-sized enterprises (SMEs) saw a 33% increase in the success rate for their marketing authorisation applications in 2023, according to a new report by the European Medicines Agency. SMEs are broadly defined as companies with a staff headcount of between 10 and 250 people and a turnover ranging from less than €2 million to up to €50 million. Despite their small size, they represent the backbone of the European life science industry with activity in nine distinctive sectors. There were 1,925 registered SMEs on 31 December 2023.
France-based SparingVision SA, which is developing gene therapies for retinal diseases, has presented data from an ongoing natural history study of rod-cone dystrophy showing the structural features of the disease and identifying a subset of patients with a higher rate of disease progression. This is to inform the company’s development of an ocular gene therapy for the disease which is in a separate Phase 1/2 trial in patients for severe rod-cone dystrophy.
The following article gives the 2024 financial outlook for a selection of European biopharma companies ranging in size from multinational groups to smaller companies
The US Food and Drug Administration has approved a new treatment for uncomplicated urinary tract (UTI) infections in women. Approximately one-half of all women experience at least one type of infection at least once in their lifetime. The drug, Pivya (pivmecillinam) is a narrow spectrum antibiotic which has been shown to be effective against infections caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.
Commit Biologics ApS was launched in Denmark on 9 May with seed funding of €16 million to use a proprietary platform to develop new drugs for cancer and autoimmune diseases. The seed funding was provided by Bioqube Ventures and Novo Holdings. Commit is a spin-out from Aarhus University in Denmark and was initially incubated by the country’s BioInnovation Institute.
UK based Pheon Therapeutics Ltd has secured $120 million in a Series B financing round to support the development of antibody-drug conjugates (ADCs). These are drugs that use an antibody to deliver a toxic payload directly to a cancer cell and kill the cancer in situ. The financing was led by TCGX of the US and other new investors including BVF Partners, Lightspeed and Perceptive Advisors. Four existing investors also participated in the round. They were Atlas Venture, Brandon Capital, Forbion, and Research Corporation Technologies.
ADC Therapeutics SA of Switzerland reached agreement with investors on 6 May to sell shares and warrants valued at $105 million to support its portfolio of antibody-drug conjugates for cancer. The new financing will lengthen the company’s cash runway enabling it to advance new indications for Zynlonta (loncastuximab tesirine), a marketed drug, and clinical-stage assets in its portfolio. ADC is developing antibody-drug conjugates for both haematological cancers and solid tumours. On 31 March it had cash and cash equivalents of $234.3 million compared with $278.6 million on 31 December 2023.