Drug Development
BridgeBio Pharma plans to file for approval in the third quarter. If granted, the oral drug could present a “highly differentiated” option over current achondroplasia therapies, according to Jefferies.
FEATURED STORIES
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
Speaking to media on Tuesday, BIO CEO John Crowley complimented China’s rise as a biotech powerhouse but said U.S. policy needs to protect and maintain America’s lead.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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Encouraging data for combination regimens of Revolution Medicines’ zoldonrasib “reinforce the path to leadership in PDAC” for the biotech, according to Truist Securities.
Recent BioSpace industry conference conversations point to sponsors pushing earlier on safety, quality and performance data, leading to demand for nitrosamine analysis, IVRT/IVPT and microbiome database capabilities.
After 27 deaths were reported in a group receiving its blood cancer drug, ADC Therapeutics is downsizing, implementing layoffs for about 30 workers.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
Novartis and Antares Therapeutics are shooting for the stars, launching a joint mission to develop small molecule oncology therapies.
AbbVie scooped up immunology player Apogee Therapeutics for nearly $11 billion in one of the year’s top deals to-date, while Sanofi made a big play to survive its upcoming Dupixent patent cliff; FDA uncertainty continues as the agency changes direction on gene therapies by uniQure and REGENXBIO; and Jef Akst and Annalee Armstrong report back from San Diego.
As Sangamo runs out of cash, Eli Lilly and Astellas have emerged as stalking horse bidders for key assets, including a Fabry gene therapy currently being submitted for potential FDA approval.
Given its intravenous route of administration, Merck’s tulisokibart will likely “need to be meaningfully improved” over Roche’s afimkibart, which can be given subcutaneously, BMO Capital Markets analysts said. Both assets are being tested for ulcerative colitis.
While existing antibody-drug conjugates have been incredible advancements in cancer care, patients are ready for a better experience. At the BIO International Convention in San Diego on Thursday, BioSpace will lead a discussion on the next generation of ADCs.
Despite an overall survival miss, Leerink Partners said Pfizer’s antibody-drug conjugate showed “promising” signals of efficacy in a subgroup of patients who had undergone only one prior line of treatment.