Though Pfizer Inc.’s work on a PD-1-targeting antibody has trailed far behind that of its fellow big pharmas, the company could be the first to market in treatment-naïve, high-risk non-muscle invasive bladder cancer (NMIBC) on the back of solid phase III data showing subcutaneously administered sasanlimab hit its endpoint of event-free survival. Pfizer anticipates meeting with regulatory agencies to discuss potential filings for what could be the first new treatment for that NMIBC population in decades.
Dyne seeks accelerated path on positive dystrophy data; shares down
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several function endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026. Dyne also said it wants to start a registrational expansion cohort of the study. The company’s stock (NASDAQ:DYN) had lost nearly a third of its value at midday as shares were down 30% to $15.92 each.
Adcom mulls risk-benefit of Ferring leg-pain prospect condoliase
The U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee is deliberating the merits of SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia. In development by Saint Prex, Switzerland-based Ferring Pharmaceuticals Inc., the compound degrades glycosaminoglycans within the nucleus pulposus of the intervertebral disc, which is meant to shrink the herniated disc by lowering its internal pressure, thereby relieving pain. In the summer of 2016, privately held Ferring was granted the exclusive worldwide rights excluding Japan to SI-6603 from Seikagaku Corp., of Tokyo.
Cancer data drive significant biopharma stock gains in a volatile 2024
Biopharma stocks saw a year of ups and downs in 2024, with the BioWorld Stock Index rising as much as 21% before dipping below 2% and ultimately closing the year up 5.16%. This performance follows a 33.59% gain in 2023 and a sharp 32.75% decline in 2022. Among the 509 stocks analyzed in the index, 169 companies (33%) ended the year with gains, while 340 (67%) saw their shares decline.
Proteomics study explores how protein changes influence disease
Fourteen global pharma companies are getting together to conduct the largest proteomics study to date, analyzing 600,000 blood samples held in the UK Biobank (UKB) to assess the levels of 5,400 different proteins. The samples include 500,000 taken when participants – then in middle age – were recruited to UKB between 2006 and 2010, and second samples taken from 100,000 of the volunteers at follow-up assessments, up to 15 years later. UKB is the only resource in the world to have samples that can be analyzed for changes over time on such a large scale. All participants in UKB have had their genomes sequenced, and once the proteomic analysis is complete it will be possible to simultaneously compare protein levels and genetic data from half a million people.
Biopharma deals hit $230B in 2024, breaking historical records
Biopharma deal activity closed out the year strong in December, reaching a total value of $29.02 billion – the second highest monthly figure of 2024, just shy of November's $29.34 billion. For the full year, deals amounted to $230.37 billion, marking a nearly 6% increase from $217.69 billion in 2023 and making 2024 the highest value year in in BioWorld’s records.
Human, mouse PD-1 differ inside and out
The PD-1 receptor, a major immune checkpoint inhibitor whose signaling is the target of multiple blockbuster anticancer drugs, differs functionally between rodents and humans in previously unknown ways. Researchers from the University of California, San Diego, and co-authors at the Chinese Academy of Sciences and the National Cancer Institute reported these findings in the Jan. 3, 2025, online issue of Science Immunology. PD-1 sets off inhibitory signaling in T cells. Blocking the interaction between PD-1 and its ligand PD-L1 can unleash the immune system. Along with CTLA-4 blockers and CAR T cells, PD-1 blockers are the first wave of tumor immunotherapy.
The BioWorld Insider podcast: a year of interviews and insights
The BioWorld Insider podcast is a monthly dive into the business and science of drug development. Conversations with CEOs and BioWorld analysts in the past 12 months have included a wide range of chats with experts who are shaping the future. Subjects included quarterly reviews for M&As and financings, the slow but steady growth of funding and partnering for therapies to treat women, the impact of artificial intelligence and quantum computing on drug development, rare disease drug development, the costs of delays in clinical trials, and an overview of the one of the year’s biggest conferences, the annual American Society of Clinical Oncology gathering in Chicago.
2024 BioWorld Year in Review
BioWorld’s staff recaps the therapeutic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as biopharma forges ahead in 2025.
2024 BioWorld MedTech Year in Review
BioWorld MedTech’s staff recaps the med-tech and diagnostic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as med tech forges ahead in 2025.
2024 BioWorld Science Year in Review
Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health.