More than four years after a phase II/III trial with Sanofi SA’s oral glucosylceramide synthase inhibitor venglustat was stopped for lack of benefit in autosomal dominant polycystic kidney disease, the company rolled out mixed phase III data from two more efforts.
An AI-based software developed by Screenpoint Medical BV could help detect breast cancers earlier and reduce the number of cancers that appear between routine screenings. In a trial of over 100,000 women, the use of the company’s Transpara Detection system resulted in a 12% reduction in the rate of interval cancers, compared to standard mammography screening. The results from the Mammography Screening with Artificial Intelligence study was published in the Lancet.
BioWorld tracked 215 clinical trial readouts spanning phases I through III in December 2025, a sizeable increase from 170 updates in November, but below the 252 reported in October.
Quince Therapeutics Inc. is scrapping work with dexamethasone sodium phosphate encapsulated in autologous erythrocytes (eDSP) for patients with ataxia-telangiectasia (A-T), and Boral analyst Jason Kolbert said “the core value driver has been effectively removed, and the equity appears fully impaired at this stage.”
With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
Heeding the recommendation of an independent data monitoring committee, Calcimedica Inc. said it is discontinuing the phase II study testing calcium release-activated calcium channel inhibitor Auxora in patients with acute kidney injury with associated acute hypoxemic respiratory failure.
Suzhou Alphamab Co. Ltd.’s HER2 bispecific antibody, anbenitamab (KN-026), combined with chemotherapy has the potential to become a new standard of care for patients with HER2-positive gastric cancer who failed at least one prior line of therapy, according to newly released phase III results that show the combo cut disease progression risk by 75%.
Cardiff Oncology Inc. welcomed Mani Mohindru as interim CEO and simultaneously provided what the company dubbed a “positive” but stock-denting clinical update. The firm rolled out data from the 113-patient phase II trial called CRDF-004, designed to test the oral Polo-like kinase 1 (PLK1) inhibitor onvansertib in first-line, RAS-mutated metastatic colorectal cancer (mCRC). Shares (NASDAQ:CRDF) closed Jan. 27 at $2, down 94 cents, or 32%, as Wall Street took in the results.
After selling off amyotrophic lateral sclerosis therapeutics last month, Tanabe Pharma America Inc. emerged a forerunner in another rare disease space with positive top-line phase III data of dersimelagon (MT-7117) for erythropoietic protoporphyria/X-linked protoporphyria.
After a roller coaster of a year for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics Inc. looks to focus on the efficacy narrative in 2026, starting with newly unveiled three-year data showing durable efficacy across all key motor function assessments for treated DMD patients vs. external controls.
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