Phase 3
While Acadia Pharmaceuticals’ candidate has missed the main goal of a Phase 2 trial, the company is still moving forward with the anti-psychosis candidate in Alzheimer’s disease as well as Lewy body dementia.
Analysts were keen to see a new modality compete with Roche’s approved drug Lucentis in the late-stage trial but new safety issues could complicate the future for Merck’s candidate.
Kyverna Therapeutics’ miv-cel showed improved mobility and a favorable safety profile at one year in patients with stiff person syndrome, further derisking an upcoming FDA application, William Blair said on Thursday. The biotech plans to file for priority review this year.
Amgen is awaiting another Phase 3 readout for the investigational Sjögren’s disease therapy, set for later this year. William Blair anticipates that at least two trials will be needed to support an FDA application.
A late-stage test of Roche and Ionis Pharmaceuticals’ sefaxersen showed that the drug reduced protein in the urine, which suggests the RNA-targeting therapy could improve kidney function and delay transplant.
Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic ALS subtype in a Phase 3 trial. The partners plan to approach the FDA with the results to determine potential pathways for approval.
As Xenon Pharmaceuticals announced that azetukalner has reached the FDA for focal seizures, the company also disclosed the emergence of psychosis adverse events in psychiatric trials of the drug, triggering a temporary pause.
Analysts believe Cullinan Therapeutics and Taiho Oncology can overcome the safety risks as zipalertinib “sets [a] new bar” for first-line treatment of a type of lung cancer. The candidate is already under review for the the second-line setting, with a decision expected by early next year.
BioNTech, Summit Therapeutics, GSK and Roche have made a splash at the ongoing World Conference on Lung Cancer taking place in South Korea. These companies and their partners have all shared new findings for their closely watched programs, highlighting overall survival benefits in various types of lung cancer.
Positive topline results from a second straight study in generalized anxiety disorder shift the narrative for Definium Therapeutics’ DT120 to commercialization, Jefferies analysts said Monday, while Stifel wrote that the asset is moving towards an FDA submission.
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