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While participants on a lower dose of Wave Life Sciences’ RNA therapy lost 5.3% total fat at the six-month mark, those receiving the higher dose saw a less than 1% drop at three months.
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Overall, the top 16 largest pharmaceutical companies spent $159 billion on research and development in 2025, compared to $165 billion the year prior. Here’s where all that cash went at companies like Johnson & Johnson, Amgen and Pfizer.
Trace Neuroscience, a member of BioSpace’s NextGen Class of 2026, has learned from the success of Biogen’s Qalsody and aims to bring more treatment options to the ALS community.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.
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While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
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Leerink analysts hailed the deals as a sign that President Trump “is unlikely to attack the industry in 2026.”
The biologics center director reportedly became personally involved after the team reviewing the rare blood disorder filing asked for an extension to the CNPV-accelerated timeline.
Ceralasertib is part of AstraZeneca’s ambitious plan to hit $80 billion in revenue by 2030.
Jacobio discovered JAB-23E73, which is designed to treat several KRAS mutation subtypes, and is testing the therapy in multiple Phase I trials.
Stifel analysts said the label for cardiac myosin inhibitor Myqorzo is in line with their expectations and is differentiated compared with BMS’ Camzyos.
Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector.
The second half finished strong after two tumultuous years. What will 2026 bring for the biotech sector?
Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry.
A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in $600 million annually.