Investigational New Drug

An investigational PCSK9 inhibitor with a different mechanism from existing drugs in this class has been shown to safely and effectively lower cholesterol levels.

Researchers are exploring strategies to alter the gut microbiome to improve patients' responses to immunotherapy. New research provide encouraging, but very early, data.

Quotable quotes heard by MedPage Today's reporters

Early data support the concept that targeting pituitary adenylate cyclase–activating polypeptide signaling could be an effective new approach for migraine prevention.

The results of a phase 2 trial led by Roswell Park's Dr. Brian Betts answer a longstanding question in stem cell transplantation. While adding the JAK...

At highest dose, novel cendakimab hits primary endpoint in phase II study

The United States Food and Drug Administration issued a formal warning against Human Microbes, a California company that describes itself as "the world's largest, highest quality stool donor bank." They sell human poop by mail and lack a license to "lawfully market" these biological products.

Zodasiran to join plozasiran in cardiovascular outcomes trial

Two assessments suggest more investment is needed

The DEA's plan to reschedule marijuana from a Schedule I controlled substance to Schedule III was expected. But what does it mean for clinicians and patients?

Three-drug combo over 4 months was comparable to standard treatment over 6 months

The manufacturer of the drug, zolbetuximab, says it is working closely with the FDA to resolve the issues.

Numerical advantages in OS, PFS fall short of statistical significance

/PRNewswire-PRWeb/ -- FreeOx Biotech SL, a late-stage clinical biopharmaceutical company pioneering the development of a novel drug class for Acute Ischemic...

For 1 in 4 patients with primary biliary cholangitis (PBC) who received the investigational PPAR-delta agonist, alkaline phosphatase levels normalized.

Results look good, but more data needed

Oral muvalaplin lowered lipoprotein(a) in a phase 1 study, and injectable olpasiran did so in an extension of a phase 2 study, both with no adverse safety signals, researchers report.

Scientists have pinned down a fleeting molecular quirk that's responsible for a certain type of nerve damage, ending a decades-long quest.

The first clinical trial of a three-month TB treatment regimen is closing enrollment because of a high rate of unfavorable outcomes with the investigational course of treatment.

Sarah Holstein leads a roundtable discussion on updates in the class