Shares of San Diego-based Capricor Therapeutics Inc. (NASDAQ: CAPR) were trading at $26.63, up $20.27, or 318%, on top-line data from the pivotal phase III Hope-3 trial testing cell therapy deramiocel in Duchenne muscular dystrophy (DMD). The 106-patient study met the primary endpoint of Performance of the Upper Limb version 2.0 and the key secondary cardiac endpoint of left ventricle ejection fraction, both achieving statistical significance (p=0.03 and p=0.04, respectively). In September, after a type A meeting between Capricor and the U.S. FDA – prompted by a complete response letter in July – the parties agreed on regulatory steps with deramiocel. The drug offers immunomodulatory, anti-inflammatory, pro-angiogenic, and anti-fibrotic properties by way of the secretion of exosomes from cardiosphere-derived cells.

Data-based concerns reveal consequence of hep B backsliding

“Do not take us backwards,” many doctors and other stakeholders implored the CDC’s Advisory Committee for Immunization Practices (ACIP) ahead of its December meeting that starts tomorrow with a discussion and votes on whether the current recommended birth dose of the hepatitis B vaccine should be delayed. Nearly 4,000 comments have been submitted to the public docket for the meeting. At the same time, researchers are driving home the cost of delaying those vaccines by even a month or two – a recommendation discussed at ACIP’s last meeting. “Even short delays in vaccination lead to substantially more infections, severe long-term health complications and sharply increased healthcare spending,” according to data released this week by Hepvu, the Hepatitis B Foundation and the National Viral Hepatitis Roundtable.

Pharvaris’ oral deucrictibant hits endpoints in acute HAE trial

Pharvaris NV looks to start filing marketing applications in the first half of 2026 on the back of positive phase III data showing oral bradykinin B2 receptor antagonist deucrictibant hit all primary and secondary endpoints as an on-demand treatment for hereditary angioedema (HAE) attacks. If approved, deucrictibant would be the second oral on-demand therapy, following Kalvista Pharmaceuticals Inc.’s Ekterly (sebetralstat), approved in July 2025, though Pharvaris executives noted in a call with investors that the drug’s rapid onset and well-established mechanism of action could distinguish deucrictibant’s position in a market still in need of new treatment options.

CTAD 2025: The challenges of combination therapies for dementia

At the Clinical Trials on Alzheimer’s Disease (CTAD) 2025 meeting, a panel of experts discussed the need for developing combination therapies for the complex diseases that result in dementia. At the same time, the people on the roundtable discussion pointed out the complex issues that combination therapies introduce. From deciding when to initiate each therapy to figuring out which biomarkers to use to measure outcomes of the combinations accurately, investigators face a challenging road ahead.

UK decline in commercial clinical trials continues

Despite a raft of government policies to turn the ship around, the latest figures show a further decline in setting up and staging commercial clinical trials in the U.K., with a 25% fall in the number of patients recruited over the past two years. According to the Association of the British Pharmaceutical Industry’s 2025 annual clinical trials report, a key barometer of the U.K.’s competitive position in attracting industry-sponsored studies, only 3.4% of all the participants recruited in 2024 took part in commercial trials being run in the National Health Service. This 3.4% – a total of 19,092 participants – was the lowest number of patients recruited to industry trials in the U.K. since 2017.

Romidepsin offers hope for high-risk neuroblastoma

Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy. Published in Science Advances on Nov. 28, 2025, the study led by David Croucher’s lab at the Garvan Institute of Medical Research in Sydney, Australia, shows how the researchers discovered that romidepsin, an approved drug for lymphoma, can trigger neuroblastoma cell death through alternative pathways when the usual routes become blocked.

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