Drug Development
Zanvastro, an antisense oligonucleotide therapy, represents the first independent neurology launch for Ionis Pharmaceuticals, which intends to leverage the foothold for more approvals in rare neurodegenerative diseases, including Angelman syndrome.
FEATURED STORIES
Almost as many biotechs have opted for a reverse merger to reach the public markets as those executing more traditional initial public offerings this year.
Well-known for its oncology efforts and making recent headlines for a rumored mega merger with AstraZeneca, Bristol Myers Squibb is also dedicating considerable resources to neurological and psychiatric diseases, with a “continuum of care” mindset.
The U.S. is scrambling to compete with a strategically positioned China on the biotech stage as the Asian country surpasses America in biomedical research and clinical trials. So far, China is winning.
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Novartis is planning to file for remibrutinib’s expanded approval in relapsing multiple sclerosis by next year, putting it just behind Roche’s own BTK inhibitor fenebrutinib.
Novartis hits pause on several CAR T trials after three patient deaths, followed by Bristol Myers Squibb after separate safety signals; Revolution Medicines scores early FDA approval for groundbreaking pancreatic cancer drug; BioNTech suspends a mid-stage trial of its mRNA cancer immunotherapy after a futility analysis; and nine mid-sized biopharma companies join their big pharma peers in President Donald Trump’s Most Favored Nation drug pricing scheme.
Roche has tapped Simcere Zaiming for a new T cell engager that could have broad application in B cell–mediated diseases including lymphoma and autoimmune conditions.
GSK’s vaccine—which targets two primary flu antigens—elicited higher immune responses in both younger and older adults in a mid-stage study. The Phase 3 launch, planned for September, follows the FDA’s recent approval for Moderna’s mRNA flu vaccine.
Novartis suspended multiple studies of a CAR T candidate after three patients who received the therapy suffered fatal cases of immune effector cell-associated hemophagocytic syndrome. BMS, meanwhile, paused some CAR T trials after separately observing “transient and reversible inflammatory events” with its therapy.
Safety and dosing are likely to be “key differentiators” for Arrowhead Pharmaceuticals as it looks to challenge Ionis Pharmaceuticals’ lead in the severe hypertriglyceridemia market.
Shares of Germany-based BioNTech were down as much as 8% Friday after announcing a Phase 2 trial of autogene cevumeran would be discontinued for futility. The news follows Merck and Moderna’s Phase 3 win last week for their cancer vaccine plus Keytruda in melanoma.
AstraZeneca and Ionis Pharmaceuticals’ data drop divulging more details from its highly watched Phase 3 transthyretin silencer trial “further confuses the picture” for Wainua when combined with the standard of care for ATTR-CM, analysts say.
Manufacturers are being pushed beyond mAb‑era platforms into a new design model built around molecular engineering, complex analytics and amplified upstream and downstream processing.
Thanks in part to the revolution cell therapy brought to treating blood cancers, the modality is now showing promise in some autoimmune applications. This expansion is poised to yield even wider benefits.