A gene therapy being developed by Regeneron Pharmaceuticals Inc has enabled a young child, suffering from a congenital hearing loss, to overcome deafness. The child, dosed at 11 months of age, is one of two to have responded to the experimental therapy which uses gene replacement to address mutations in the OTOF gene – the cause of the disease. A second child, who received the therapy at the age of four, showed initial improvements after dosing.
BioVersys AG, a Swiss antibiotic developer, has expanded a collaboration with GSK Plc to accelerate development of a candidate product for tuberculosis. The product, alpibectir, has reached Phase 2a and shown an ability to increase the efficacy of ethionamide, an existing antibiotic. GSK will take an equity stake in a Series C financing extension for the company, raising the total proceeds from the round to CHF 44.9 million ($49.4 million). The size of the equity stake wasn’t disclosed. The new agreement was announced on 7 May.
A new gene therapy company with plans to start clinical development of its first product has raised $54 million in a Series A financing round. Latus Bio Inc is a spin-out of the Children’s Hospital of Philadelphia, US, with a new technology for producing adeno-associated viral vectors, one of the most common vectors in use for gene therapies. Announced on 2 May, the financing round was led by 8VC of Texas and DCVC Bio of California.
Novartis is to deepen its presence in radiopharmaceuticals with the acquisition of Mariana Oncology, a preclinical biotechnology company based in the US. Announced on 2 May, the deal involves an upfront cash payment of $1 billion and potential outlays of up to $750 million depending on the achievement of certain undisclosed milestones.
GSK Plc has raised its revenue forecast for 2024 on the back of a strong first quarter for its shingles and respiratory syncytial virus (RSV) vaccines and a recently launched treatment for myelofibrosis patients with anaemia. Together with high demand for the respiratory medicine Trelegy, group turnover is now expected to rise towards the upper end of the 5% to 7% range. At a press briefing on 1 May, Emma Walmsley, the chief executive, spoke confidently about the company’s continued pipeline progress.
AstraZeneca Plc achieved double digit growth in both revenue and operating profit for the first quarter, driven by gains from its oncology and rare disease portfolios. During the same period, the company completed two acquisitions and announced agreements for two more. The value of the four deals is up $5.75 billion. Looking ahead to 2030, Pascal Soriot, the chief executive, said AstraZeneca aims to be a “long-term growth company.” He made his remarks on 25 April with the release of the quarterly results.
SynOx Therapeutics Ltd, a biotech company co-located in the UK and Ireland, has raised $75 million in a Series B financing round to advance a treatment for tenosynovial giant cell tumour into Phase 3. The financing was co-led by Forbion, HealthCap and new investor Bioqube Ventures of Belgium. It will be used to generate data for a monoclonal antibody treatment called emactuzumab which has already shown a substantial effect on the tumours, which are noncancerous growths that can be chronically debilitating.
A new biotech company was launched in Scotland, UK, on 18 April with the goal of treating complex diseases by stabilising certain proteins in the cell. The company, Outrun Therapeutics Ltd, went public following the receipt of $10 million in seed funding. The investors are M Ventures, the corporate venture capital arm of Merck KGaA of Germany, and MP Healthcare Venture Management of Boston, US.
Faron Pharmaceuticals Ltd has appointed new leadership as the company prepares to advance clinical development of bexmarilimab, its wholly-owned immunotherapy for both haematological and solid tumours. The company’s founder and chief executive officer, Markku Jalkanen, is to retire and be replaced by his son, Juho Jalkanen, currently the chief operating officer. The new CEO will take up his position on 1 May.
Pathios Therapeutics Ltd of the UK has secured financial support from Bristol Myers Squibb Co as part of a Series B venture round that will advance its small molecule drug candidate for cancer into the clinic. The first close of the B round raised $25 million and will support development of PTT-4256, a small molecule inhibitor of a G protein-coupled receptor called GPR65. In addition to BMS, funding was secured from the existing investors Canaan Partners and Brandon Capital Partners.