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William Blair analyst Myles Minter in a Monday note to investors said that Vertex’s povetacicept “has maintained its potential to be a best-in-class asset” in the IgA nephropathy space and could become a “multibillion-dollar pipeline-in-a-drug product” for autoimmune disorders, while “outstanding questions” remain for Biogen’s felzartamab before moving into pivotal studies.
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.
Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could address the damage underlying alpha-1 antitrypsin deficiency and make today’s treatments a thing of the past.
Pfizer CEO Albert Bourla is in a tough spot as activist investor Starboard Value continues to call for a change in the company’s leadership. However, analysts are supportive of the embattled executive.
The French drugmaker’s newly launched respiratory syncytial virus antibody Beyfortus far exceeded analyst expectations, bringing in almost $700 million in the third quarter. Leerink Partners analyst David Risinger in a Friday note to investors said Sanofi expects Q4 Beyfortus sales similar to Q3.
The CDC’s vaccine advisors on Thursday maintained that respiratory syncytial virus immunization is only recommended for adults aged 75 and older, and in seniors 60 to 74 years who are at risk of severe disease.
Bank of America analysts said prior to Thursday’s readout that Tyra Biosciences’ TYRA-300 could rival Johnson & Johnson’s kinase inhibitor Balversa, which has suffered from safety concerns and poor tolerability.
Jefferies analyst Maury Raycroft said in a note to investors that Thursday’s mid-stage readout pointed to the “unprecedented” complete response rate of Intellia’s investigational in vivo gene editing therapy in the disorder.
Researchers have linked Novo Nordisk’s semaglutide to a 40% to 70% reduction in the risk of Alzheimer’s disease diagnosis in a study of medical records from 1 million patients.
With an upfront payment of $50 million from Roche, the partnership will leverage Dyno Therapeutics’ in vivo gene therapy delivery technology, which synthesizes virus capsids with better functionality and manufacturability.
In this episode of Denatured, Lori and guests from Teva Pharmaceuticals and TOWER Capital review the investment landscape this year and the policies influencing investment heading into 2025.